Alzheimer's Disease (Preclinical & Research) · ALRT 4204 The Michael J. Fox Foundation for...
Transcript of Alzheimer's Disease (Preclinical & Research) · ALRT 4204 The Michael J. Fox Foundation for...
Indication Phase Route Country Name Role Country Type OwnershipAGN 194204 Prostate cancer Phase II PO USA Allergan Originator USA Large Pharma, Pharmaceutical Public
AGN-194204 Alzheimer's disease Preclinical PO USA Io Therapeutics Owner World Biopharmaceutical Private
AGN194204 Parkinson's disease Preclinical PO USA Mount Sinai School of Medicine Collaborator USA University N/A
ALRT 4204 The Michael J. Fox Foundation for Parkinsons Research Collaborator USA Institution N/A
ALRT-4204
ALRT4204
IRX-4204
IRX4204
NRX 194204
NRX 4204
NRX-194204
NRX-4204
NRX194204
NRX4204
VTP 194204
VTP-194204
VTP194204
Adipose stem cell therapies - RNL BIO Facial hemiatrophy Phase II Parenteral South Korea Asan Medical Center Collaborator South Korea Hospital N/A
Adult-derived stem cell therapies - RNL BIO Osteoarthritis Phase II Intra articular South Korea RNL BIO Originator South Korea Biotechnology
Astrostem Thromboangiitis obliterans Phase II Parenteral South Korea RNL BIO Owner South Korea Biotechnology
Autologous adipose stem cells - RNL BIO Intervertebral disc degeneration I/II Parenteral South Korea RST Biomedikal Sanayi Licensee Turkey Biotechnology
Cancerstem Osteonecrosis I/II Parenteral South Korea University of Newcastle upon Tyne Collaborator United Kingdom University N/A
Fat-derived stem cell therapies - RNL BIO Spinal cord injuries I/II Intraspinal South Korea
Lipostem-DM Spinal cord injuries I/II Intrathecal South Korea
Lipostem-Femoran Spinal cord injuries I/II IV South Korea
Mesenchymal stem cell therapies - RNL BIO Liver disorders Clinical IV Asia
RNL-Astrostem™ Alzheimer's disease Preclinical IV South Korea
RNL-JointStem® Cancer Preclinical Parenteral South Korea
Vascostem™ Cerebral infarction Preclinical Parenteral South Korea
Diabetes mellitus Preclinical Parenteral South Korea
Fracture Preclinical Parenteral South Korea
Ischaemic heart disorders Preclinical Intraventricular South Korea
hCNS-SC Age-related macular degeneration I/II Intraocular USA Oregon Health & Science University Collaborator USA University N/A
HuCNS-SC Spinal cord injuries I/II Intraosseous Switzerland ReNeuron Technology Provider United Kingdom Biopharmaceutical Public
HuCNS-SC cells Pelizaeus-Merzbacher disease Phase I Intracerebral USA StemCells Originator USA Biotechnology Public
HuCNS-SC® Alzheimer's disease Preclinical Parenteral USA StemCells Owner USA Biotechnology Public
Neurodegenerative disorders stem cell therapy -
StemCells
Stroke Preclinical Parenteral USA University of California at Irvine Collaborator USA University N/A
Neuronal ceroid lipofuscinosis Discontinued(I) Intracerebral USA
Haemophilia B I/II IV USA ARIAD Pharmaceuticals Collaborator USA Pharmaceutical Public
Neuronal ceroid lipofuscinosis Phase I Intracranial USA ARIAD Pharmaceuticals Technology Provider USA Pharmaceutical Public
Age-related macular degeneration Preclinical Ophthalmic USA ReGenX Biosciences Originator USA Biopharmaceutical
Alzheimer's disease Preclinical Parenteral USA ReGenX Biosciences Owner USA Biopharmaceutical
Haemophilia A Preclinical Parenteral USA St. Jude Childrens Research Hospital Collaborator USA Hospital N/A
Hypercholesterolaemia Preclinical Parenteral USA University of Pennsylvania Medical Center Collaborator USA University N/A
Leber congenital amaurosis Preclinical Ophthalmic USA Weill Cornell Medical College Collaborator USA University N/A
Metachromatic leukodystrophy Preclinical Intracranial USA
Mucopolysaccharidosis I Preclinical Parenteral USA
Mucopolysaccharidosis III Preclinical Intracranial USA
Mucopolysaccharidosis VI Preclinical Parenteral USA
Retinitis pigmentosa Preclinical Ophthalmic USA
RNS-60 G protein-coupled receptor modulators Asthma (Combination therapy) I/II Inhalation USA Revalesio Originator USA Biotechnology Private
RNS60 Ion channel modulators Asthma Phase I IV USA Revalesio Owner USA Biotechnology Private
Myocardial infarction (In volunteers) Phase I IV USA Rush University Collaborator USA University N/A
Alzheimer's disease Preclinical Unknown route USA
Multiple sclerosis Preclinical IV USA
Parkinson's disease Preclinical Unknown route USA
AEN 100 Antioxidants Amyotrophic lateral sclerosis I/II PO USA Adeona Pharmaceuticals Originator USA Biotechnology Public
AEN-100 Free radical scavengers Alzheimer's disease Preclinical PO USA Synthetic Biologics Owner USA Biotechnology Public
AEN-100/AEN-200 Glutathione peroxidase stimulants
reaZin™
Zinc cysteine - Adeona
Adult neural stem cells - Neuralstem Amyotrophic lateral sclerosis Phase I Intraspinal USA China Medical University Hospital Collaborator Taiwan Hospital N/A
HNSC Alzheimer's disease Preclinical Unknown route USA CJ Cheiljedang Corp. Collaborator South Korea Biopharmaceutical
HSSC Amyotrophic lateral sclerosis Preclinical Intraspinal Taiwan Emory University Collaborator USA University N/A
NSI 566 Brain injuries Preclinical Unknown route USA Neuralstem Originator Usa Company
NSI 566RSC Cerebral palsy Preclinical Unknown route USA Neuralstem Owner Usa Company
NSI-566 Diabetic neuropathies Preclinical Unknown route USA University of California, San Diego Technology Provider USA University N/A
NSI-566RSC Huntington's disease Preclinical Unknown route Germany University of Freiburg Collaborator Germany University N/A
NSI566 Lysosomal storage diseases Preclinical Unknown route USA
Multiple sclerosis Preclinical Unknown route USA
Optic neuritis Preclinical Unknown route USA
Paraplegia Preclinical Unknown route USA
Parkinson's disease Preclinical Unknown route USA
Spinal cord injuries Preclinical Intraspinal South Korea, USA
Stroke Preclinical Intracerebral China, USA
NSI-189 Major depressive disorder Phase I PO USA Neuralstem Originator Usa Company
NSI-189 phosphate Alzheimer's disease Preclinical PO USA Neuralstem Owner Usa Company
NSI189 Brain injuries Preclinical Unknown route USA Summit Pharmaceuticals International Corporation Licensee Japan Company
Cognition disorders Preclinical PO USA
Neurodegenerative disorders Preclinical PO USA
Post-traumatic stress disorders Preclinical PO USA
Stroke Preclinical Unknown route USA
CNS disorders (In volunteers) Phase I IV Canada, USA Northwestern University Originator USA University N/A
Alzheimer's disease Preclinical PO Canada Northwestern University Owner USA University N/A
Rheumatoid arthritis Preclinical PO Canada Transition Therapeutics Licensee Canada Biopharmaceutical Public
9
TT 301 Transition Therapeutics is developing TT 301, a
small molecule compound that inhibits the
overproduction of inflammatory cytokines, such
as interleukin-1 beta and tumour necrosis factor
alpha, for the treatment of central nervous
system indications such as intracerebral
haemorrhage and traumatic brain injury. [CONT.]
TT301 Cytokine inhibitors
8
NSI 189 Neuralstem is developing NSI 189, an orally
active, first-in-class, small molecule neurogenic
therapeutic for the treatment of major depressive
disorder. The compound can cross the blood-
brain barrier and appears to have the effect of
stimulating neurogenesis in the hippocampus by
"recruiting" the patient's own neural stem cells to
repair or protect against CNS damage. [CONT.]
Neurogenesis stimulants
7
Neurological disorder stem cell
therapies - Neuralstem
Neuralstem is developing neural stem cells from
the human brain and spinal cord for the
treatment of a range of neurological disorders,
including amyotrophic lateral sclerosis (ALS),
Huntington's disease, ischaemic spastic
paraplegia, stroke and traumatic spinal cord
injuries. The company's patent-protected
technology is used to control the differentiation
of the stem cells into mature physiologically
relevant human neurons and glia, and to produce
commercial quantities of these cells [CONT.]
Cell replacements
6
Zinc monocysteine Synthetic Biologics (formerly Adeona
Pharmaceuticals) is developing zinc-based
therapeutics, including a proprietary formulation
of orally available zinc monocysteine, for the
treatment of Alzheimer's disease and
amyotrophic lateral sclerosis (ALS). The product
was originally developed as a prescription
medical food; however, following positive results
from a 42 patient clinical study, the company
now plans to develop the product as a
prescription pharmaceutical [CONT.]
5
RNS 60 RNS 60 is a charge-stabilised nanostructure
(CSN) product that is in development with the
biotechnology company Revalesio for the
treatment of asthma and other neurological
inflammatory disorders. CSNs are aqueous-based
nano-bubble cores surrounded by a stable
molecular scaffolding that could alter whole cell
conductance through effects on voltage-gated ion
channels and other voltage-sensing proteins, and
thereby modulating the expression of G-protein
coupled receptors and secretion of cytokines,
chemokines and adhesion molecules [CONT.]
4
Gene therapies - ReGenX Biosciences ReGenX Biosciences is developing personalised
gene therapies for eye, neurodegenerative,
haematological and metabolic disorders, using its
NAV™ gene delivery technology. NAV™ includes
a range of proprietary recombinant adeno-
associated viral (rAAV) vectors, including rAAV7,
rAAV8, rAAV9 and rAAVrh10. [CONT.]
NAV™ therapeutics - ReGenX Biosciences Gene transference
3
Adult neural stem cell therapy -
StemCells
StemCells is developing an adult neural stem cell
therapy, known as HuCNS-SC®, for the
treatment of disorders in all three regions of the
CNS (brain, spinal cord, eyes). Researchers at
StemCells have succeeded in purifying stem cells
away from other cells in brain tissue by using
specific stem cell markers combined with state-of-
the-art cell sorting. [CONT.]
Cell replacements
2
Stem cell therapies - RNL BIO RNL BIO (South Korea) is researching and
developing stem cell therapies derived from adult
sources, such as cord blood, placenta, hair
follicle and adipose (fat) tissue. The company is
focussing on the development of mesenchymal
stem cell therapies derived from adipose fat.
Clinical development is underway in
osteonecrosis, interverterbral disc degeneration,
osteoarthritis, thromboangiitis obliterans, facial
hemiatrophy, liver disorders, and spinal cord
injury [CONT.]
Cell replacements
Drug Development Phase Organisations
1
IRX 4204 IRX 4204 is an orally bioavailable, first in class,
retinoid X receptor (RXR; rexinoid) agonist being
developed by Io Therapeutics. The initial focus is
for the treatment of castration-resistant prostate
cancer, but may also be developed for non-small
cell lung cancer. IRX 4204 demonstrated activity
against a variety of human cancers in vitro and
in animal models, including prostate, breast,
pancreatic, and lung [CONT.]
Retinoid X receptor agonists
Alzheimer's Disease (Preclinical & Research)
Drug Introduction Synonyms Mechanism Of Action
Neuropathic pain (In volunteers) Phase I PO USA Virobay Originator USA Pharmaceutical Private
Alzheimer's disease Research PO USA Virobay Owner USA Pharmaceutical Private
VBY 106 Cathepsin B inhibitors Autoimmune disorders 0 PO USA Virobay Originator USA Pharmaceutical Private
VBY 285 Cathepsin inhibitors Bone cancer 0 Unknown route USA Virobay Owner USA Pharmaceutical Private
VBY 825 Cathepsin S inhibitors Neuropathic pain 0 PO USA
VBY-106 Hepatitis C protease inhibitors Alzheimer's disease Research PO USA
VBY-285 Cancer pain Discontinued(Preclinical) PO USA
VBY-825 Hepatitis C Discontinued(Preclinical) PO USA
AZD-3102 Alzheimer's disease Preclinical Parenteral Europe, USA AstraZeneca Licensee World Large Pharma, Pharmaceutical Public
AZD3102 Dyax Originator USA Biopharmaceutical Public
Dyax Owner USA Biopharmaceutical Public
AZD-6319 Alzheimer's disease Preclinical IM USA AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
AZD6319 AstraZeneca Owner United Kingdom Large Pharma, Pharmaceutical Public
ReXceptor is developing bexarotene as a
treatment for Alzheimer's disease (AD).
Preclinical development is underway in the US.
Peroxisome proliferator-activated receptor
agonists
Alzheimer's disease Preclinical PO USA Alzheimers Drug Discovery Foundation Funder USA Company
The drug was originated by Ligand
Pharmaceuticals and is available in both oral and
topical formulations for the treatment of
cutaneous T-cell lymphoma in the US, Europe,
Bolivia and Chile [see separate profiles]. [CONT.]
Protein synthesis inhibitors BrightFocus Foundation Funder USA Investor
Retinoic acid receptor agonists Case Western Reserve University Technology Provider USA University N/A
Retinoid X receptor agonists Ligand Pharmaceuticals Originator USA Biotechnology Public
ReXceptor Owner USA Biopharmaceutical
Alzheimer's disease Preclinical PO Italy Lay Line Genomics Market Licensee World Biopharmaceutical Private
University of Bologna Originator Italy University N/A
University of Bologna Owner Italy University N/A
Alzheimer's disease (Adjunctive treatment:Adjunct to
donepezil)
Preclinical PO USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
Merck & Co Owner USA Large Pharma, Pharmaceutical Public
NC-1900 Alzheimer's disease Preclinical Injection Japan Nippon Chemiphar Originator Japan Pharmaceutical Public
NC1900 Alzheimer's disease Preclinical Inhalation USA Nippon Chemiphar Owner Japan Pharmaceutical Public
Schizophrenia Preclinical Injection Japan
NXD 1191, a spheron targeting agent, is
undergoing preclinical investigation with Nymox
Pharmaceutical Corporation in Canada for its
potential to inhibit the formation of senile plaques
from spherons and as a therapeutic for patients
with Alzheimer's disease (AD).
NXD-1191 Alzheimer's disease Preclinical Unknown route Canada Nymox Pharmaceutical Corporation Originator Canada Biopharmaceutical Public
Spherons, a brain entity discovered by Nymox
researchers, are tiny balls of densely packed
protein found in brain cells. [CONT.]
Spheron targeting drug Nymox Pharmaceutical Corporation Owner Canada Biopharmaceutical Public
NXD 3109, a spheron targeting agent, is in
preclinical development with Nymox
Pharmaceutical Corporation in Canada for its
potential to inhibit the formation of senile plaques
from spherons and as a potential therapeutic for
Alzheimer's disease (AD).
NXD-3109 Alzheimer's disease Preclinical Unknown route Canada Nymox Pharmaceutical Corporation Originator Canada Biopharmaceutical Public
Spherons, a brain entity discovered by Nymox
researchers, are tiny balls of densely packed
protein found in brain cells. [CONT.]
Spheron targeting agents Nymox Pharmaceutical Corporation Owner Canada Biopharmaceutical Public
NXD-9062 Alzheimer's disease Preclinical Unknown route Canada Nymox Pharmaceutical Corporation Originator Canada Biopharmaceutical Public
NXD9062 Nymox Pharmaceutical Corporation Owner Canada Biopharmaceutical Public
DWJ301 Acetylcholinesterase inhibitors Alzheimer's disease Preclinical PO South Korea Cenomed BioSciences Sub-licensee World Biopharmaceutical Private
Phenserine tartrate Amyloid beta-protein precursor inhibitors Poisoning Preclinical PO USA Daewoong Pharmaceutical Sub-licensee South Korea Pharmaceutical Public
Alzheimer's disease Discontinued(Clinical) PO USA National Institute on Aging Originator USA Institution N/A
Cognition disorders Discontinued PO USA National Institutes of Health (USA) Originator USA Institution N/A
QR Pharma Sub-licensee World Biotechnology
Raptor Pharmaceutical Corp Licensee World Pharmaceutical Public
Alzheimer's disease Preclinical Unknown route South Korea Medifron DBT Originator South Korea Technology provider Public
21
Phenserine Phenserine is a phenylcarbamate analogue of
physostigmine and a selective
acetylcholinesterase inhibitor that is being
developed for the teatment of poisoning following
exposure to organophosphorous nerve agents
and Alzheimer's disease (AD). The compound
was discovered by the National Institute on Aging
(NIA) of the US National Institutes of Health
(NIH). [CONT.]
22
Research programme: advanced
glycosylation end product receptor
antagonists - Medifron
Medifron DBT is developing advanced
glycosylation end-product receptor antagonists
for the treatment of Alzheimer's disease. The
efficacy of lead candidates have been proven in a
transgenic mice model, in preclinical
development, underway in South Korea.
Advanced glycosylation end-product receptors
are established as active transporters of beta
amyloid from plasma to the brain. [CONT.]
Advanced glycosylation end-product receptor
antagonists
19
NXD 3109
20
NXD 9062 Nymox Pharmaceutical Corporation is
preclinically developing NXD 9062 as a potential
treatment for Alzheimer's disease (AD) in
Canada. NXD 9062 works by inhibiting cell
damage similar to that found in the AD brain. The
agent is undergoing further preclinical
development in Canada.
Undefined mechanism
17
NC 1900 Japan-based pharmaceutical company Nippon-
Chemiphar is developing the active-fragment
analogue of arginine-vasopressin, NC 1900, for
the potential treatment of Alzheimer's disease.
The drug has also been investigated in a
preclinical model of schizophrenia. [CONT.]
Undefined mechanism
18
NXD 1191
Undefined mechanism
16
MK 7622 Merck is developing MK 7622, a small-molecule
quinazolinone compound, as a potential therapy
for Alzheimer's disease (AD). A phase IIb trial
(MK-7622-012 AM1) is planned in the US to
assess the efficacy and tolerability of different
doses of MK 7622 capsules (5mg, 15mg or
45mg once daily) as an adjunct to donepezil in
patients with mild-to-moderate AD
(NCT01852110). [CONT.]
MK-7622
14
Bexarotene - ReXceptor
15
Memoquin Lay Line Genomics is developing memoquin, a
low molecular weight molecule based on a
chemical scaffold able to support multiple
pharmacophores, as a potential therapeutic for
Alzheimer's disease. It has been developed with
the aim of obtaining a multifunctional molecule
effective on different biochemical cascades,
relevant for Alzheimer's-like pathology. [CONT.]
12
AZD 3102 AZD 3102 is a human monoclonal antibody with
high affinity for an undisclosed Alzheimer's
disease (AD) target. The antibody is being
developed by AstraZeneca in collaboration with
Dyax, and could have utility in the modification of
AD progression. Preclinical development is
underway in the US and Europe.
Undefined mechanism
13
AZD 6319 AstraZeneca is developing AZD 6319, a selective
alpha7 nicotinic receptor (NNR) agonist for the
treatment of Alzheimer's disease (AD). Studies
have shown that selective acute alpha7 NNR
stimulation has delayed effects on the neural
systems engaged in spatial working memory;
delayed, persistent improvements in cognition
may indicate a long-term alteration in synaptic
efficacy and/or receptor signalling in working
memory circuitry [CONT.]
Nicotinic receptor agonists
11
Research programme: protease
inhibitors - Virobay
Virobay in the US has a research programme
developing small-molecule protease inhibitors for
the treatment of Alzheimer's disease,
autoimmune diseases, bone cancer, hepatic
fibrosis, neuropathic pain and cancer. The
company has developed HCV protease inhibitors
and cathepsin inhibitors. Development has
reached phase 0 development in autoimmune
disorders, bone cancer and neuropathic pain, and
early research is being conducted in Alzheimer's
disease [CONT.]
10
VBY 036 Virobay is developing a selective small-molecule
cathepsin S inhibitor, VBY 036, as an oral
treatment for neuropathic pain and Alzheimer's
disease. ViroBay proposes that the drug is first-in-
class with its therapeutic approach, and that the
drug is suitable for once-daily dosing. Early
research is underway for Alzheimer's disease and
phase I clinical development is underway for
neuropathic pain in the US. [CONT.]
VBY-036 Cathepsin S inhibitors
Medifron DBT Owner South Korea Technology provider Public
A2A PAM Adenosine A2 receptor modulators Alcoholism Preclinical PO Switzerland Addex Pharmaceuticals Originator Switzerland Pharmaceutical Public
ADX 68692 Follicle stimulating hormone modulators Alzheimer's disease Preclinical PO Switzerland Addex Therapeutics Owner Switzerland Pharmaceutical Public
ADX 71441 GABA B receptor agonists Charcot-Marie-Tooth disease Preclinical PO Germany, Netherlands Viva Biotech Collaborator China CRO Private
ADX 71743 Interleukin 2 receptor modulators Endometriosis Preclinical PO Switzerland
ADX 71943 Interleukin-1 receptor modulators Gastro-oesophageal reflux Preclinical PO Switzerland
ADX 92639 Metabotropic glutamate receptor 2 modulators Major depressive disorder Preclinical PO Switzerland
ADX-71743 Metabotropic glutamate receptor 7 modulators Muscle spasticity Preclinical PO Switzerland
ADX-71943 Metabotropic glutamate receptor modulators Overactive bladder Preclinical PO Switzerland
ADX-92639 Orexin receptor type 2 modulators Pain Preclinical PO Switzerland
ADX68692 Ovarian follicle modulators Prostate cancer Preclinical PO Switzerland
ADX71441 TrkB receptor agonists Urinary incontinence Preclinical PO Switzerland
ADX71743 TrkB receptor modulators [CONT.] Anxiety disorders Research PO Switzerland
ADX71943 Gout Research PO Switzerland
ADX92639 Huntington's disease Research PO Switzerland
Cpd 23 Inflammatory bowel diseases Research PO Switzerland
FSHR/LHR NAM for sex hormone dependent
tumours and reproductive system disorders -
Addex Therapeutics
Osteoarthritis Research PO Switzerland
GABA-BR PAM for overactive bladder - Addex Parkinson's disease Research PO Switzerland
IL1R1 NAM (CD121a) Post-traumatic stress disorders Research PO Switzerland
mGluR2 NAM Psoriasis Research PO Switzerland
mGluR7 NAM Rheumatoid arthritis Research PO Switzerland
Orexin 2R NAM Sleep disorders Research PO Switzerland
TNFR1 NAM (CD120a) Type 2 diabetes mellitus Research PO Switzerland
TNFR1 NAM (TNF receptor superfamily) [CONT.] Contraception Discontinued(Preclinical) PO Switzerland
Osteoporosis Discontinued(Preclinical) PO Switzerland
EVP 4473 Alzheimer's disease Preclinical PO USA Bayer HealthCare Originator Germany Pharmaceutical Public
EVP-4473 Schizophrenia Preclinical PO USA Bayer HealthCare Pharmaceuticals Owner Germany Pharmaceutical Public
EnVivo Pharmaceuticals Licensee World Biopharmaceutical Private
alpha7 nAChR agonists - Memory
Pharmaceuticals/Roche
Alpha7 nicotinic acetylcholine receptor agonists Alzheimer's disease Preclinical PO USA Memory Pharmaceuticals Originator USA Biopharmaceutical Public
Nicotinic alpha-7 receptor agonists - Memory
Pharmaceuticals/Roche
Nicotinic receptor agonists Schizophrenia Preclinical PO USA Memory Pharmaceuticals Owner USA Biopharmaceutical Public
BNC 1881 Alzheimer's disease Preclinical Unknown route Australia Bionomics Originator Australia Biotechnology Public
BNC 375 Cognition disorders Preclinical Unknown route Australia Bionomics Owner Australia Biotechnology Public
BNC1881
BNC375
Alzheimer's disease (Diagnosis) Preclinical Unknown route Singapore Bayer HealthCare Pharmaceuticals Owner Germany Pharmaceutical Public
Parkinson's disease (Diagnosis) Preclinical Unknown route Singapore Bayer Schering Pharma Originator Germany Pharmaceutical Public
WisTa Laboratories Originator Singapore Pharmaceutical Private
WisTa Laboratories Owner Singapore Pharmaceutical Private
INVENT Pharmaceuticals is developing small
molecule therapeutics for the treatment of
Alzheimer's disease and age-related cognitive
and memory impairment. The company utilises
its proprietary site-specific drug design and
discovery platform for creation of new
candidates.
Alzheimer's disease Preclinical Unknown route USA INVENT Pharmaceuticals Originator USA Biotechnology Private
INVENT is actively seeking partners for potential
drug discovery, and drug development and
commercialisation collaborations.
Cognition disorders Preclinical Unknown route USA INVENT Pharmaceuticals Owner USA Biotechnology Private
Memory disorders Preclinical Unknown route USA
Alzheimer's disease (Diagnosis) Preclinical Unknown route USA Avid Radiopharmaceuticals Originator USA Technology provider Public
Avid Radiopharmaceuticals Owner USA Technology provider Public
Siemens Medical Solutions Originator USA Technology provider Public
Alzheimer's disease (Diagnosis) Preclinical Unknown route USA Blanchette Rockefeller Neurosciences Institute Originator USA Institution N/A
Alzheimer's disease Preclinical Unknown route USA Neurotrope BioScience Originator USA Biotechnology
Anti-Abeta oligomers - LLG Amyloid beta-protein inhibitors Alzheimer's disease (Diagnosis) Preclinical Unknown route Italy Lay Line Genomics Originator Italy Biopharmaceutical Private
Anti-beta-amyloid oligomers - LLG Nerve growth factor stimulants Alzheimer's disease Preclinical Unknown route Italy Lay Line Genomics Owner Italy Biopharmaceutical Private
Anti-proNerve Growth Factor - LLG
Anti-proNGF - LLG
Diagnostic antibody IM13 - LLG
NGF-based therapy - LLG
Alzheimer's disease Preclinical Unknown route USA Nuron Biotech Licensee World Biotechnology Private
University of Texas Southwestern Medical Center Originator USA University N/A
VITRUVIAN BioMedical Owner USA Biotechnology
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route Israel NasVax Originator Israel Biopharmaceutical Public
33
Research programme: Alzheimer's
disease immunotherapeutics -
NasVax
NasVax in Israel is developing
immunotherapeutic products for Alzheimer's
disease. The company is developing two products
based on BBS technology. The two products are
a monoclonal antibody (MAb) and a vaccine that
is designed to elicit antibodies with similar
specificity to the MAb. Development is at the
preclinical stage. [CONT.]
32
Research programme: Alzheimer's
disease DNA vaccine - VITRUVIAN
BioMedical/Nuron Biotech
VITRUVIAN BioMedical is developing a DNA
amyloid beta 42 (Abeta42) vaccine for treatment
of Alzheimer's disease (AD). Unlike a traditional
vaccine for AD, this vaccine does not contain
Abeta itself, but rather it contains a piece of the
gene encoding the Abeta protein. Once in the
body, its is expected that the vaccine will
stimulate an immune response in which
antibodies that bind to and significantly reduce
Abeta are produced [CONT.]
Amyloid beta 42 DNA vaccine for Alzheimer's
disease - Nuron Biotech
Immunostimulants
30
Research programme: Alzheimer's
disease diagnostics and therapeutics -
Blanchette Rockefeller
Neurosciences Institute/Neurotrope
BioScience
The Blanchette Rockefeller Neurosciences
Institute (BRNI) and Neurotrope BioScience are
collaborating to develop and commercialise
diagnostic and therapeutic agents for Alzheimer's
disease. Development is at the preclinical stage
in the US.
31
Research programme: Alzheimer's
disease diagnostics and therapies -
Lay Line Genomics
Lay Line Genomics is researching several
products for use as diagnostics and/or therapies
for Alzheimer's disease. Preclinical development
is underway in Italy.
28
Research programme: Alzheimer's
disease and cognition disorder
therapeutics - INVENT
Pharmaceuticals
Undefined mechanism
29
Research programme: Alzheimer's
disease diagnostics - Avid
Radiopharmaceuticals
Avid Radiopharmaceuticals, a molecular imaging
subsidiary of Eli Lilly, is developing positron
emission tomography (PET) tracers for the
diagnosis of Alzheimers disease (AD). AD
hallmarks such as cognitive impairment and
neuronal circuit degeneration can be correlated
with the severity of tau abnormalities and
neurofibrillary tangles (NFT) burden. [CONT.]
Tau tangle diagnostic program Positron-emission tomography enhancers
27
Research programme: Alzheimer's
and Parkinson's disease diagnostic
agents - Bayer HealthCare
Pharmaceuticals/TauRx
WisTa Laboratories Ltd., a wholly owned
subsidiary of TauRx Pharmaceuticals, is
collaborating with Bayer HealthCare
Pharmaceuticals (formerly Bayer Schering
Pharma) in the development of specific markers
that will aid in the diagnosis and management of
Tau-protein related Alzheimer's disease (AD),
using brain-imaging technology. The programme
may also have potential in the development of
diagnostics for Parkinson's disease [CONT.]
26
Research programme: alpha7
nicotinic acetylcholine receptor
modulator therapeutics - Bionomics
Bionomics is developing positive allosteric
modulators (PAMs) of the alpha7 nicotinic
acetylcholine receptor (nAChR) for the treatment
of Alzheimer's disease and cognitive impairment
related to conditions such as attention deficit
hyperactivity disorder (ADHD), mood and anxiety
disorders, multiple sclerosis, Parkinson's disease,
and schizophrenia. [CONT.]
Alpha7 nicotinic acetylcholine receptor
modulators
25
Research programme: alpha-7
nicotinic acetylcholine receptor
agonists - Roche
Roche are developing orally active, nicotinic
alpha-7 (alpha7) receptor agonists for the
treatment of various central nervous system
disorders (CNS), particularly Alzheimer's disease
(AD) and cognitive impairment associated with
schizophrenia (CIAS). The nicotinic alpha7
receptor acts to promote neuronal function,
acting on synaptic plasticity and cytoprotection.
[CONT.]
24
Research programme: alpha-7
nicotinic acetylcholine receptor
agonists - EnVivo Pharmaceuticals
EnVivo Pharmaceuticals is developing selective,
orally active alpha-7 nicotinic acetylcholine
receptor (alpha7-nAChR) agonists for the
treatment of cognitive decline in patients with
Alzheimer's disease (AD) and schizophrenia. The
rationale behind selective activation of alpha7-
nAChRs is to enhance cognition without causing
the adverse events associated with
overactivation of other nAChRs or muscarinic
acetylcholine receptors [CONT.]
Alpha7 nicotinic acetylcholine receptor agonists
23
Research programme: allosteric
modulators - Addex Therapeutics
Positive and negative allosteric modulators are
being developed by Addex Therapeutics (formerly
Addex Pharmaceuticals) for the treatment of a
variety of diseases. The compounds are orally
available small molecules that have potential use
in overactive bladder, gastro-oesophageal reflux
disease (GERD), endometriosis, pain, urinary
incontinence, glaucoma, depression, anxiety,
Alzheimer's disease, type [CONT.]
22
Research programme: advanced
glycosylation end product receptor
antagonists - Medifron
Medifron DBT is developing advanced
glycosylation end-product receptor antagonists
for the treatment of Alzheimer's disease. The
efficacy of lead candidates have been proven in a
transgenic mice model, in preclinical
development, underway in South Korea.
Advanced glycosylation end-product receptors
are established as active transporters of beta
amyloid from plasma to the brain. [CONT.]
Advanced glycosylation end-product receptor
antagonists
Immunostimulants NasVax Owner Israel Biopharmaceutical Public
anti-ADDL antibodies Alzheimer's disease Preclinical Injection USA Acumen Pharmaceuticals Licensee USA Biotechnology
HuC 091 Northwestern University Originator USA University N/A
HuC091 Northwestern University Owner USA University N/A
Alzheimer's disease Preclinical Parenteral Australia Prana Biotechnology Originator Australia Biotechnology Public
Prana Biotechnology Owner Australia Biotechnology Public
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route Finland Medeia Therapeutics Originator Finland Pharmaceutical Private
Cell replacements Medeia Therapeutics Owner Finland Pharmaceutical Private
Alzheimer's disease Preclinical PO Japan Institute of Medicinal Molecular Design Originator Japan Pharmaceutical Private
Institute of Medicinal Molecular Design Owner Japan Pharmaceutical Private
ACI-518 Alzheimer's disease Preclinical Unknown route Switzerland AC Immune Originator Switzerland Biotechnology Private
ACI-636 AC Immune Owner Switzerland Biotechnology Private
Alzheimer's disease Preclinical Unknown route USA AgeneBio Originator USA Pharmaceutical Private
Mild cognitive impairment Preclinical Unknown route USA AgeneBio Owner USA Pharmaceutical Private
Amgen Inc is developing orally available small
molecule BACE (beta-site APP-cleaving enzyme)
inhibitors for the treatment of Alzheimer's
disease. Preclinical development is ongoing in the
US.
AMG-0683 Alzheimer's disease Preclinical PO USA Amgen Originator USA Large Pharma, Biotechnology Public
Cerebral deposition of amyloid beta peptide
(Abeta) is an early and critical feature of
Alzheimer's disease. Abeta generation depends
on the proteolytic cleavage of the amyloid
precursor protein (APP) by two proteases, beta-
secretase and gamma-secretase [CONT.]
AMG0683 Amgen Owner USA Large Pharma, Biotechnology Public
CMP 015
Alzheimer's disease Preclinical Unknown route USA Applied NeuroSolutions Originator USA Biopharmaceutical Public
Applied NeuroSolutions Owner USA Biopharmaceutical Public
Eli Lilly Licensee World Large Pharma, Pharmaceutical Public
ARC 050 Amyloid precursor protein secretase inhibitors Alzheimer's disease Preclinical Unknown route USA Archer Pharmaceuticals Originator USA Pharmaceutical Private
ARC 069 Calcium channel antagonists Archer Pharmaceuticals Owner USA Pharmaceutical Private
ARC050
ARC069
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route Switzerland Merck Serono Originator Switzerland Biopharmaceutical Public
Tau protein inhibitors Merck Serono Owner Switzerland Biopharmaceutical Public
Alzheimer's disease Preclinical Unknown route Austria, Germany Boehringer Ingelheim Licensee Germany Large Pharma, Pharmaceutical Private
Evotec AG Originator Germany Pharmaceutical Public
Evotec AG Owner Germany Pharmaceutical Public
Research Institute of Molecular Pathology Collaborator Austria Institution N/A
Abeta oligomerisation inhibitors Alzheimer's disease Preclinical Intracerebro ventricular United Kingdom BTG Originator United Kingdom Pharmaceutical Public
Amyloid-beta oligomerisation inhibitors BTG Owner United Kingdom Pharmaceutical Public
BGC 200406 Senexis Licensee World Company
44
Research programme: Alzheimer's
disease therapeutics - Boehringer
Ingleheim/Evotec AG
Evotech and Boehringer Ingleheim are
collaboratively investigating potential points of
intervention in the treatment of Alzheimer's
disease. Evotec utilises a Fragment Based Drug
Discovery (FBDD) and screening platform to
identify small molecules in a biologically relevant
environment. A library of 20,000 fragments and
protein-ligand X-ray crystallography allow
Evotech to progress fragment programmes to
identify lead structures and preclinical
development candidates [CONT.]
BACE1 protein inhibitors
45
Research programme: Alzheimer's
disease therapeutics - BTG/Senexis
Senexis is investigating small molecule, peptide-
based inhibitors of amyloid-beta (Abeta)-induced
toxicity as well as neuroinflammation for the
treatment of Alzheimer's disease (AD) and other
amyloid-associated diseases. Toxic oligomers are
created by Abeta aggregation, which is
considered key to the onset of AD. Senexis has in-
licensed three small molecule candidates
comprising BGC 201178 [RS 1178], BGC
200406 [RS 0406] and BGC 200466 [RS 0466]
from BTG [CONT.]
Amyloid beta-protein inhibitors
42
Research programme: Alzheimer's
disease therapeutics - Archer
Pharmaceuticals
Archer Pharmaceuticals, a spin-off company of
The Roskamp Institute, is developing compounds
for the treatment of Alzheimer's disease (AD).
The Roskamp Institute is devoted to
understanding causes and finding cures for
neuropsychiatric and neurodegenerative
disorders and addictions. Archer Pharmaceuticals
aims to find existing drugs that can be
repositioned into AD therapies and to use these
drugs as templates for the development of new,
more effective drugs. [CONT.]
43
Research programme: Alzheimer's
disease therapeutics - Asceneuron
Asceneuron (a Merck Serono spin-off company)
is developing small-molecule therapeutics for the
potential treatment of Alzheimer's disease and
other tau protein-related neurological disorders.
The research will initially focus on three drug
discovery programmes that were in preclinical
development with Merck Serono. Compounds
identified through these programmes will target
Tau protein and amyloid-beta peptides, and are
expected to improve cognitive function.
Preclinical development is underway in
Switzerland. [CONT.]
40
Research programme: Alzheimer's
disease therapeutics - Amgen
BACE1 protein inhibitors
41
Research programme: Alzheimer's
disease therapeutics - Applied
NeuroSolutions/Eli Lilly
Applied NeuroSolutions (formerly Molecular
Geriatrics Corporation), in collaboration with Eli
Lilly, is using in vitro screens in a combinatorial
chemistry programme that aims to discover
novel therapeutics for the treatment of
Alzheimer's disease (AD). Development is at the
research stage in the US. [CONT.]
Undefined mechanism
Plasminogen activator inhibitor-1 inhibitors
38
Research programme: Alzheimer's
disease therapeutics - AC Immune
AC Immune has a research programme
investigating therapeutics for the treatment of
Alzheimer's disease. The programme is
investigating Morphomers™ which are non-
covalent complexes of small molecules. The
Morphomers™ are designed to specifically bind to
misfolded proteins and alter the conformation of
the protein to a benign state. [CONT.]
39
Research programme: Alzheimer's
disease therapeutics - AgeneBio
AgeneBio is developing small-molecule
therapeutics for the treatment of amnestic mild
cognitive impairment, which is a borderline
condition between normal ageing and Alzheimer's
disease. Progression to Alzheimer's disease
occurs in approximately 15% of patients with
amnestic mild cognitive impairment. AgeneBio is
designing drug candidates to potentially stabilise
neural activity, which has been shown to be
excessive in cognitive disorders such as amnestic
mild cognitive impairment [CONT.]
GABA modulators
36
Research programme: Alzheimer's
disease stem cell therapy - Medeia
Therapeutics
Medeia Therapeutics is developing MDA 208C, a
stem cell therapy for the treatment of
Alzheimer's disease (AD). The company is using
its MDA 200C cell-based therapy platform
involving stem cells derived from bone marrow.
These cells may be recruited into the central
nervous system (CNS) and stimulated using its
proprietary technology and pharmacological
agents as a disease-modifying approach for the
clearance of beta-amyloid (Abeta) peptide
deposits/accumulation [CONT.]
MDA-208C
37
Research programme: Alzheimer's
disease therapeutic - Institute of
Medicinal Molecular Design
The Institute of Medicinal Molecular Design, Inc.
(IMMD) is developing IMD 4690, an orally
administered inhibitor of PAI-1 (plasminogen
activator inhibitor-1), for the treatment of
Alzheimer's disease. IMD 4690 is undergoing
preclinical development in Japan.
IMD-4690
Amyloid beta-protein inhibitors
35
Research programme: Alzheimer's
disease immunotherapy - Prana
Biotechnology
Prana Biotechnology is developing an
immunotherapy for Alzheimer's disease. The
research is assessing the effectiveness of the
technology to enhance the production of
antibodies as an effective Alzheimer's disease
treatment. Prana have developed monoclonal
antibodies against a unique form (a
conformational epitope) of beta-amyloid which is
produced as a result of the oxidative processes
central to the toxic pathway in Alzheimer's
disease [CONT.]
4,00E+10 Immunostimulants
33
Research programme: Alzheimer's
disease immunotherapeutics -
NasVax
NasVax in Israel is developing
immunotherapeutic products for Alzheimer's
disease. The company is developing two products
based on BBS technology. The two products are
a monoclonal antibody (MAb) and a vaccine that
is designed to elicit antibodies with similar
specificity to the MAb. Development is at the
preclinical stage. [CONT.]
34
Research programme: Alzheimer's
disease immunotherapy -
Acumen/Merck
Acumen Pharmaceuticals is developing
immunotherapies against amyloid beta-derived
diffusible ligand (ADDL). ADDLs are highly
ordered aggregates of amyloid beta1-42
peptides. The company is developing antibodies
and vaccines against ADDLs to delay the
progression of Alzheimer's disease, prevent its
onset, and potentially restore cognitive function
in patients with memory-related disorders.
[CONT.]
BGC 200466
BGC 201178
RS 0406
RS 0466
RS 1178
SEN 1269
SEN-1269
SEN1269
haw AD14 Alzheimer's disease Preclinical PO USA CoPlex Therapeutics Originator USA Pharmaceutical Private
hawAD14 CoPlex Therapeutics Owner USA Pharmaceutical Private
Hawthorn Pharmaceuticals Licensee World Pharmaceutical Private
DWJ 501 Alzheimer's disease Preclinical PO South Korea Daewoong Pharmaceutical Originator South Korea Pharmaceutical Public
DWJ501 Daewoong Pharmaceutical Owner South Korea Pharmaceutical Public
FGL-S Alzheimer's disease Preclinical Parenteral Denmark ENKAM Pharmaceuticals A/S Originator Denmark Biotechnology Private
FGLS Cognition disorders Preclinical Parenteral Denmark ENKAM Pharmaceuticals A/S Owner Denmark Biotechnology Private
Forenap Collaborator France CRO Private
Lundbeck A/S Collaborator Denmark Large Pharma, Pharmaceutical Public
Prague Psychiatric Center Collaborator Czech Republic Institution N/A
Qualissima Collaborator France CRO Private
University Hospital of Cologne Collaborator Germany Hospital N/A
University of Copenhagen Collaborator Denmark University N/A
KaloBios Pharmaceuticals is investigating an
antibody to granulocyte macrophage colony-
stimulating factor (GM-CSF) as a potential
therapy for Alzheimer's disease. Preclinical
studies are in progress in the US.
Alzheimer's disease Preclinical Parenteral USA KaloBios Pharmaceuticals Originator USA Biopharmaceutical Private
Analysis of brain tissue from patients with
Alzheimer's disease has revealed the presence of
inflammation caused by proliferation of microglia
and astrocytes. [CONT.]
KaloBios Pharmaceuticals Owner USA Biopharmaceutical Private
Alzheimer's disease Preclinical Unknown route Switzerland Kareus Therapeutics Originator Switzerland Pharmaceutical Private
Kareus Therapeutics Owner Switzerland Pharmaceutical Private
Alzheimer's disease Preclinical Unknown route USA Medisyn Technologies Originator USA Biotechnology
Mount Sinai School of Medicine Originator USA University N/A
Alzheimer's disease Preclinical Unknown route Germany, Israel Merz Pharmaceuticals GmbH Originator Germany Pharmaceutical Private
Merz Pharmaceuticals GmbH Owner Germany Pharmaceutical Private
Ramot at Tel Aviv University Technology Transfer Israel Technology Transfer Private
Tel Aviv University Collaborator Israel University N/A
Tel Aviv University Technology Provider Israel University N/A
Alzheimer's disease Preclinical Unknown route USA Mithridion Licensee World Biopharmaceutical
University of Wisconsin System Originator USA University N/A
University of Wisconsin System Owner USA University N/A
Wisconsin Alumni Research Foundation Technology Transfer World Technology Transfer Private
Neu AZ1 Alzheimer's disease Preclinical Unknown route Israel Neurim Pharmaceuticals Originator Israel Pharmaceutical Private
Neu-AZ1 Neurim Pharmaceuticals Owner Israel Pharmaceutical Private
NeuAZ1
2 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route USA Neuro-Hitech Originator USA Pharmaceutical Public
3 Tau protein inhibitors Neuro-Hitech Owner USA Pharmaceutical Public
4-Trihydroxybenzophenone
4-Amino 1
1'-azobenzene-3
4'-disulfonic acid sodium salt
Pain Therapeutics is developing orally available,
small molecule compounds for the treatment of
Alzheimer's disease. A lead candidate from this
programme, known as PTI 125, is in preclinical
development in the US.
PTI 125 Alzheimer's disease Preclinical PO USA Pain Therapeutics Originator USA Biopharmaceutical Public
PTI 125 binds with high affinity to filamin A
(FLNA), a microfilament scaffolding protein which
has been shown to play an important role in
amyloid-beta42 signalling. [CONT.]
PTI-125 Pain Therapeutics Owner USA Biopharmaceutical Public
Roche is developing inhibitors of beta-secretase
(BACE-1), for the treatment of Alzheimer's
disease (AD). The programme is undergoing
preclinical development in Switzerland.
R 641 Alzheimer's disease Preclinical Unknown route Switzerland Roche Originator Switzerland Large Pharma, Pharmaceutical Public
The accumulation of amyloid beta-peptide
(Abeta) is the primary cause for AD
pathogenesis. Sequential processing of the
amyloid precursor protein (APP) by BACE-1 and
gamma-secretase results in the formation of
Abeta. [CONT.]
R-641 Roche Owner Switzerland Large Pharma, Pharmaceutical Public
56
Research programme: Alzheimer's
disease therapeutics - Pain
Therapeutics
Microfilament protein inhibitors
57
Research programme: Alzheimer's
disease therapeutics - Roche
BACE1 protein inhibitors
54
Research programme: Alzheimer's
disease therapeutics - Neurim
Pharmaceuticals
Neurim Pharmaceuticals is developing
compounds for the treatment of Alzheimer's
disease. The mechanism of action is undisclosed.
The company has identified Neu AZ1 as a lead
candidate, and is in preclinical development,
presumably in Israel.
Undefined mechanism
55
Research programme: Alzheimer's
disease therapeutics - Neuro-Hitech
Neuro-Hitech (formerly Q-RNA) is developing
therapeutics for the treatment of Alzheimer's
disease (AD) that target as well as inhibit
amyloid-beta (Abeta) protein and tau protein
oligomer formation. The extraneuronal deposition
of Abeta plaques and the intraneuronal
accumulation of hyperphosphorylated tau protein
as neurofibrillary tangles are implicated in the
pathogenesis of AD. Development is at the
preclinical stage in the US. [CONT.]
52
Research programme: Alzheimer's
disease therapeutics - Merz
Pharmaceuticals/Tel Aviv University
Merz Pharmaceuticals and Tel Aviv University are
collaborating in a research programme to
develop and commercialise therapeutics for
Alzheimer's disease. Both parties will jointly
evaluate candidate compounds identified using
the University's proprietary drug discovery
technology. The programme is being conducted
in Germany and Israel.
Undefined mechanism
53
Research programme: Alzheimer's
disease therapeutics - Mithridion
Mithridion is undertaking a research programme
to develop next-generation, disease-modifying
compounds for the treatment of Alzheimer's
disease (AD). Such small molecule drugs are
intended to delay or halt AD progression,
particularly the degeneration and death of
neurons via inhibition of amyloid-beta (Abeta).
This approach is expected to preserve memory,
cognitive and other brain functions. [CONT.]
Amyloid beta-protein inhibitors
51
Research programme: Alzheimer's
disease therapeutics - Medisyn
Medisyn and the Mount Sinai School of Medicine
are developing therapeutics for the treatment of
Alzheimer's disease. Using Medisyn's Forward
Engineering™ technology, eight lead compounds
have been discovered with significant in vitro
activity, two have also been tested in vivo with
promising results. [CONT.]
Amyloid beta-protein inhibitors
49
Research programme: Alzheimer's
disease therapeutics - KaloBios
Pharmaceuticals
Granulocyte macrophage colony stimulating
factor antagonists
50
Research programme: Alzheimer's
disease therapeutics - Kareus
Therapeutics
Kareus Therapeutics is developing compounds for
the treatment of Alzheimer's disease. The
company aims to create therapeutics that
intervene in the disease causing mechanism, to
slow down or block its progression. Kareus is
developing a second generation therapeutic,
dimethyl acetylenedicarboxylate (DMAD),
utilising the company's Karlect chemistry
platform and its Bend biology platform. [CONT.]
DMAD Amyloidogenic protein inhibitors
47
Research programme: Alzheimer's
disease therapeutics - Daewoong
Pharmaceutical
Daewoong Pharmaceutical is developing oral
therapeutics for the treatment of Alzheimer's
disease. DWJ 501, a lead candidate from the
programme, is a herbal extract. Daewoong is
also conducting research to identify new chemical
entities that may have efficacy in treating the
disease. The programme is in preclinical
development in South Korea. [CONT.]
Undefined mechanism
48
Research programme: Alzheimer's
disease therapeutics - ENKAM
ENKAM Pharmaceuticals is developing a peptide
compound, FGLS, for the treatment of
Alzheimer's disease and potentially other
conditions that feature impaired cognition, such
as Parkinson's disease and stroke. FGLS is one of
a family of compounds known as FGL (or FGL
peptides) that function as allosteric fibroblast
growth factor receptor modulators. [CONT.]
Fibroblast growth factor receptor modulators
46
Research programme: Alzheimer's
disease therapeutics - CoPlex
Therapeutics/Hawthorn
Pharmaceuticals
Hawthorn is developing haw AD14, an orally-
administered small molecule originated by CoPlex
Therapeutics, which may have potential in the
treatment of Alzheimer's disease. The compound
has been shown to significantly decrease levels
of amyloid beta (Abeta), as well as tau
phosphorylation activity in preclinical studies.
These represent key components of the amyloid
plaques and paired helical filaments which
comprise the neurofibrillary tangles in
Alzheimer's disease [CONT.]
45
Research programme: Alzheimer's
disease therapeutics - BTG/Senexis
Senexis is investigating small molecule, peptide-
based inhibitors of amyloid-beta (Abeta)-induced
toxicity as well as neuroinflammation for the
treatment of Alzheimer's disease (AD) and other
amyloid-associated diseases. Toxic oligomers are
created by Abeta aggregation, which is
considered key to the onset of AD. Senexis has in-
licensed three small molecule candidates
comprising BGC 201178 [RS 1178], BGC
200406 [RS 0406] and BGC 200466 [RS 0466]
from BTG [CONT.]
Amyloid beta-protein inhibitors
Alzheimer's disease Preclinical Unknown route Switzerland Roche Originator Switzerland Large Pharma, Pharmaceutical Public
Neurodegenerative disorders Preclinical Unknown route Italy Siena Biotech Originator Italy Company
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route USA Senex Biotechnology Originator USA Biotechnology Private
Amyloid beta-protein precursor inhibitors Senex Biotechnology Owner USA Biotechnology Private
BACE1 protein inhibitors
Signal transduction pathway inhibitors
Transglutaminase inhibitors
SEN1176 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route USA Senexis Originator United Kingdom Company
SEN1500 Macrophage inhibitors Senexis Owner United Kingdom Company
SEN1576
SEN304
SEN606
M007 LRPIV Alzheimer's disease Preclinical Unknown route USA Socratech Originator USA Biotechnology Private
M007-LRPIV Socratech Owner USA Biotechnology Private
Alzheimer's disease Preclinical Unknown route USA Sonexa Therapeutics Licensee World Biopharmaceutical Private
Unknown Originator USA Company
Unknown Owner USA Company
ANA-1 Amyloid beta-protein inhibitors Alzheimer's disease (Diagnosis) Preclinical PO Australia Alzhyme Originator Australia Biopharmaceutical Private
ANA-5 Superoxide dismutase inhibitors Alzheimer's disease Preclinical PO Australia Alzhyme Owner Australia Biopharmaceutical Private
ANSTO Radiopharmaceuticals and Industrials Collaborator Australia Company
CSIRO Collaborator Australia Agency
Alzheimer's disease Preclinical PO United Kingdom Galapagos NV Originator Belgium Pharmaceutical Public
Senexis Originator United Kingdom Company
Biogen Idec is developing therapies for
Alzheimer's disease (AD). In its Form 10-K (filed
4 February 2011), the company reported that a
product candidate had entered preclinical trials:
a gamma-secretase modulator (or amyloid
precursor protein secretase modulator).
Alzheimer's disease Preclinical Unknown route Unknown Biogen Idec Originator USA Large Pharma, Biotechnology Public
The neurodegeneration observed in AD is thought
to be a result of a pathological cascade initiated
by formation of insoluble plaques caused by the
accumulation of amyloid beta (Abeta) peptides
[CONT.]
Biogen Idec Owner USA Large Pharma, Biotechnology Public
Amyloid precursor protein secretase modulators Alzheimer's disease Preclinical PO USA Cellzome Originator Germany Pharmaceutical Private
Tau protein modulators Johnson & Johnson Pharmaceutical Research &
Development
Originator USA Pharmaceutical Public
Mazal Plant Pharmaceuticals, a majority owned
subsidiary of Adavanced Plant Pharmaceuticals,
is developing MAALZ [LMH 123] for the
treatment of leukaemia. MAALZ is a plant based
composition. Preclinical trials with the compound
are ongoing in the US.
LMH-123 Alzheimer's disease Preclinical Unknown route USA Mazal Plant Pharmaceuticals Originator USA Company
MAALZ was originally developed by Advanced
Plant Pharmaceuticals Inc (APPI). [CONT.]
MAALZ Mazal Plant Pharmaceuticals Owner USA Company
SEN 12333 Alzheimer's disease Preclinical Unknown route Italy Siena Biotech Originator Italy Company
SEN 34625 Siena Biotech Owner Italy Company
SEN34625
WAY-317538
WYE 103914
WYE-103914
Alzheimer's disease therapy research programme
- Satori Pharmaceuticals
Alzheimer's disease Preclinical Unknown route USA Satori Pharmaceuticals Originator USA Company
69
Research programme: Alzheimer's
disease therapy - Satori
Pharmaceuticals
Satori Pharmaceuticals is developing gamma-
secretase modulators as a disease modifying
therapy for Alzheimer's disease (AD). AD is
characterized by the formation of amyloid beta
(Abeta) plaques in brain. There are two main
forms of Abeta: Abeta40 and Abeta42. Satori's
programme aims to selectively reduce Abeta42,
which may overcome the adverse effects
associated with reducing total Abeta levels.
[CONT.]
Amyloid precursor protein secretase modulators
67
Research programme: Alzheimer's
disease therapies - Mazal Plant
Pharmaceuticals
Undefined mechanism
68
Research programme: Alzheimer's
disease therapies - Siena Biotech
Siena Biotech is developing compounds for the
treatment of neurodegenerative disorders,
particularly Alzheimer's disease. One aspect of
the research focus involves investigations of
nicotinic acetylcholine receptors of the alpha-7
type (alpha-7 nAChR) as potential therapeutic
targets. These receptors are expressed in brain
regions associated with cognitive function,
regulate cholinergic neurotransmission and have
been shown to be down-regulated in Alzheimer's
disease. Development is at the preclinical stage.
[CONT.]
Alpha7 nicotinic acetylcholine receptor agonists
65
Research programme: Alzheimer's
disease therapies - Biogen Idec
Gamma secretase modulator - Biogen Idec Amyloid precursor protein secretase modulators
66
Research programme: Alzheimer's
disease therapies - Cellzome/Ortho-
McNeil Pharmaceutical
Cellzome Inc., and Johnson & Johnson
Pharmaceutical Research & Development in the
US are collaborating in a research programme
focused on identifying orally administered,
amyloid precursor protein secretase (gamma-
secretase) modulators and tau protein
modulators for the treatment of Alzheimer's
disease. Cellzome will provide access to its
existing Alzheimer's disease programme
technology, including insights into its amyloid
precursor protein processing pathway map and
new drug targets [CONT.]
63
Research programme: Alzheimer's
disease therapies - Alzhyme
Alzhyme, an Australian biotechnology company,
is developing orally available, peptide analogues
for the treatment of Alzheimer's disease. The
company has identified two peptides, ANA-1 and
ANA-5, which bind to human beta-amyloid
(Abeta), the protein found in the plaques which
characterise Alzheimer's disease. In preclinical
trials, the peptides were able to prevent beta-
amyloid-induced toxicity by neutralising the beta-
amyloid catalytic production of hydrogen
peroxide [CONT.]
64
Research programme: Alzheimer's
disease therapies - BioFocus/Senexis
Galapagos (formerly BioFocus) and Senexis
Limited in the UK are collaborating in the
discovery of small molecule beta sheet (beta-
sheet) breakers, and novel inhibitors of amyloid-
induced toxicity and neuroinflammation, for the
potential treatment of Alzheimer's disease.
BioFocus' medicinal chemistry capabilities will be
used to accelerate the lead discovery of Senexis'
compounds.
beta-sheet breakers Amyloid inhibitors
61
Research programme: Alzheimer's
disease therapeutics - Socratech LLC
Socratech LLC is developing therapeutics
targeting the vascular system for the treatment
of Alzheimer's disease. The vascular system
controls cerebral blood flow, transport of
molecules across the blood-brain barrier
including removal of critical toxins, and the
formation of new blood vessels during aging and
neurodegenerative disorders. The vascular
system plays a key role in initiating the pathology
of brain cells and therefore represents a primary
therapeutic target. [CONT.]
Transcription factor modulators
62
Research programme: Alzheimer's
disease therapeutics - Sonexa
Therapeutics
Sonexa Therapeutics is undertaking a research
programme to develop an undisclosed drug for
the treatment of Alzheimer's disease (AD). The
company has licensed exclusive rights worldwide
(except for Japan and certain Asian countries) to
the compound from an undisclosed Japanese
pharmaceutical company. Development is at the
preclinical stage.
Undefined mechanism
60
Research programme: Alzheimer's
disease therapeutics - Senexis
Senexis is investigating peptide- and non-peptide-
based inhibitors of amyloid-beta (Abeta)-induced
toxicity and neuroinflammation for the treatment
of Alzheimer's disease (AD) and other amyloid-
associated diseases. Toxic oligomers are created
by Abeta aggregation, which is considered key to
the onset of AD. Development of peptide-based
inhibitors of amyloid aggregation is at the
preclinical stage in the UK. [CONT.]
58
Research programme: Alzheimer's
disease therapeutics - Roche/Siena
Biotech
Roche and Siena Biotech are collaborating on the
development of compounds for the treatment of
neurodegenerative disorders, including
Alzheimer's disease. Preclinical development is
underway in Italy and Switzerland.
Undefined mechanism
59
Research programme: Alzheimer's
disease therapeutics - Senex
Biotechnology
Senex Biotechnology is undertaking a drug
discovery programme to develop compounds for
the treatment of major age-related diseases,
particularly Alzheimer's disease (AD). The
company is developing drugs targeting signalling
pathways activated in the process of cell
senescence or aging. Studies on functional
genomics have revealed that aging cells
overproduce proteins causing AD and other age-
related diseases [CONT.]
SPI 014 Satori Pharmaceuticals Owner USA Company
SPI 1865
SPI-014
SPI-1865
Alzheimer's disease Preclinical Unknown route USA Thuris Corporation Originator USA Pharmaceutical Private
Thuris Corporation Owner USA Pharmaceutical Private
EB 101 Alzheimer's disease (Prevention) Preclinical Parenteral USA Atlas Pharmaceuticals Originator USA Pharmaceutical
EB101 Alzheimer's disease Preclinical Parenteral USA Atlas Pharmaceuticals Owner USA Pharmaceutical
Alzheimer's disease Preclinical Transmucosal Japan DNAVEC Corporation Originator Japan Technology provider Private
Eisai Co Ltd Originator Japan Large Pharma, Pharmaceutical Public
DNAVEC is co-developing a nasal spray vaccine
against Alzheimer's disease with the National
Institute for Longevity Sciences of the National
Center for Geriatrics and Gerontology in Japan.
Alzheimer's disease Preclinical Intranasal Japan DNAVEC Corporation Originator Japan Technology provider Private
The vaccine is being developed using Sendai
Virus Vectors (SeV), DNAVEC's vector that
develops amyloid-beta. The aim of the SeV-
based vaccine is to help reduce senile plaques in
the brains of patients with Alzheimer's disease.
[CONT.]
National Institute for Longevity Sciences Originator Japan Institution N/A
Alzheimer's disease Preclinical Parenteral Switzerland AC Immune Originator Switzerland Biotechnology Private
AC Immune Owner Switzerland Biotechnology Private
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Parenteral Austria AFFiRiS Originator Austria Biotechnology Private
Immunostimulants AFFiRiS Owner Austria Biotechnology Private
GlaxoSmithKline Licensee World Large Pharma, Pharmaceutical Public
RV 01 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Parenteral USA Intellect Neurosciences Originator USA Biopharmaceutical Public
RV 02 Immunostimulants Intellect Neurosciences Owner USA Biopharmaceutical Public
RV 03 T lymphocyte stimulants University of California at Irvine Collaborator USA University N/A
RV01 Tau protein inhibitors
RV02
RV03
Alzheimer's disease Preclinical Intraperitoneal USA Mapp Biopharmaceutical Originator USA Biopharmaceutical
Mapp Biopharmaceutical Owner USA Biopharmaceutical
Alzheimer's disease Preclinical Unknown route USA Pfizer Originator USA Large Pharma, Pharmaceutical Public
Asthma Preclinical Unknown route USA Pfizer Owner USA Large Pharma, Pharmaceutical Public
Cancer Preclinical Unknown route USA Washington University School of Medicine Collaborator USA University N/A
Diabetes mellitus Preclinical Unknown route USA
Alzheimer's disease Preclinical Unknown route USA Star Scientific Originator USA Pharmaceutical Public
Neurological disorders Preclinical Unknown route USA Star Scientific Owner USA Pharmaceutical Public
ADDL therapeutics Alzheimer's disease Preclinical Unknown route USA Acumen Pharmaceuticals Originator USA Biotechnology
anti-ADDL therapeutics Acumen Pharmaceuticals Owner USA Biotechnology
A 887755 Alzheimer's disease Preclinical Parenteral Germany, USA Abbott GmbH & Co. KG Originator Germany Pharmaceutical Public
A-887755 Abbott GmbH & Co. KG Owner Germany Pharmaceutical Public
CT 0093 Alzheimer's disease Preclinical Unknown route USA Cognition Therapeutics Originator USA Pharmaceutical Private
82
Research programme: amyloid beta-
protein inhibitors - Cognition
Therapeutics
Cognition Therapeutics is developing small
molecule, amyloid beta-protein inhibitors, as a
disease-modifying treatment for Alzheimer's
disease. The lead candidates inhibit the activity
of the oligomeric form of the amyloid beta-
protein that interferes with learning and memory.
Memory deficits caused by the oligomeric protein
are among the earliest changes seen in
Alzheimer's disease. [CONT.]
Amyloid beta-protein inhibitors
80
Research programme: amyloid beta-
derived diffusible ligand inhibitors -
Acumen
Acumen is developing small molecule
therapeutics for the treatment of Alzheimer's
disease that target amyloid beta-derived
diffusable ligands (ADDLs). ADDLs are highly
ordered aggregates of amyloid beta1-42
peptides. Soluble ADDLs trigger malfunctions in
nerve cell signaling, causing early and reversible
memory dysfunction followed by synaptic loss
and nerve cell death. [CONT.]
Amyloid beta-protein inhibitors
81
Research programme: amyloid beta-
protein inhibitors - Abbott
Abbott GmbH is developing amyloid-beta (Abeta)
globulomer-selective monoclonal antibodies for
the treatment of Alzheimer's disease. Recent
research has implicated the role of the Abeta
peptide found in amyloid plaques as a causal
factor of the disease. However, new studies
showed that memory loss and cognitive function
were already impaired in patients with
Alzheimer's disease before the plaques started
forming, providing evidence of the poor
correlation between amyloid plaques and
dementia in such patients [CONT.]
Amyloid beta-protein inhibitors
79
Research programme: amyloid beta
protein inhibitors - Star Scientific
Star Scientific, in collaboration with SRQ Bio
(Roskamp Institute), are developing an amyloid
beta protein inhibitor for the treatment of
neurological disorders, including Alzheimer's
disease. Preclinical investigation has produced
positive results finding that the compound also
has potential to encourage new neuronal cell
growth. Preclinical development is ongoing in the
US.
Amyloid beta-protein inhibitors
77
Research programme: Alzheimer's
disease vaccines - MAPP
Biopharmaceutical
MAPP Biopharmaceutical is developing
monoclonal antibodies against amyloid beta-
protein (13-28) for the treatment of cognitive
deficits associated with Alzheimer's disease (AD).
Development is at preclinical stage in the US.
Amyloid beta-protein inhibitors
78
Research programme: Alzheimer's
disease, cancer, metabolic disorders
and respiratory disease therapeutics -
Pfizer/Washington University
Pfizer and Washington University School of
Medicine at St. Louis have entered into a
collaboration for the discovery of new indications
for a number of Pfizer's existing compounds that
have previously undergone preclinical or clinical
testing. The identificiation of new uses for
existing compounds is known as indications
discovery. [CONT.]
76
Research programme: Alzheimer's
disease vaccines - Intellect
Neurosciences
Intellect Neurosciences is developing therapeutic
approaches, including vaccines and antibodies,
for the treatment and prevention of Alzheimer's
disease (AD). The company believes that such
approaches will potentially overcome the
incidence of autoimmune reactions or other
serious adverse events, which have hampered
progress in the field of AD therapy. Development
is at the preclinical stage in the US. [CONT.]
75
Research programme: Alzheimer's
disease vaccines -
AFFiRiS/GlaxoSmithKline
AFFiRiS and GlaxoSmithKline (GSK) are
collaborating in the development of vaccine
candidates for the treatment of Alzheimer's
disease. The vaccines will be customised using
AFFiRiS' Affitope platform technology (Affitom®)
and are designed to induce cross-reactive
antibodies against a native antigen, without
producing a systemic immune response. [CONT.]
73
Research programme: Alzheimer's
disease vaccine intranasal - DNAVEC
Alzheimer's disease vaccine intranasal research
programme - DNAVEC
Immunostimulants
74
Research programme: Alzheimer's
disease vaccines - AC Immune
AC Immune is developing new therapies for the
treatment of Alzheimer's disease, using
vaccination or passive immunisation with
antibodies. The company has two programmes
investigating immunotherapies targeting two
different proteins believed to be involved in
Alzheimer's disease. Both therapies use AC
Immune's antigen technology (SupraAntigen™)
which induces changes in the conformation of
misfolded proteins, shifting the equilibrium in
favour of benign conformations [CONT.]
Alzheimer's disease vaccine research programme
- AC Immune
Immunostimulants
71
Research programme: Alzheimer's
disease vaccine - Atlas
Pharmaceuticals
Atlas Pharmaceuticals is developing an amyloid
beta vaccine formulated into a liposomal
adjuvant for the treatment and prevention of
Alzheimer's disease. This vaccine, known as EB
101, utilises a novel amyloid beta1-42 core and
acts to evoke a Th2 T-cell reaction. Preclinical
development is ongoing in the US.
Immunostimulants
72
Research programme: Alzheimer's
disease vaccine - DNAVEC/Eisai
DNAVEC and Eisai have entered into a drug
discovery research agreement to develop a
vaccine treatment for Alzheimer's disease. Using
DNAVEC's Sendai virus vector, the two
companies plan to develop a gene vaccine able to
selectively induce production of antibodies
against the beta amyloid protein. Under the
terms of the agreement, Eisai has rights to
develop, manufacture and sell any compounds
identified in the collaboration [CONT.]
Immunostimulants
70
Research programme: Alzheimer's
disease therapy - Thuris/D-Pharm
Thuris Corporation is investigating compounds
for the treatment of Alzheimer's disease. Thuris
is using its RapidAgingTM technology to identify
and develop potential lead candidates.
RapidAgingTM is an assay that creates the
hallmarks of Alzheimer's disease, in fewer than 6
days, for using as a preclinical screen for
potential treatments.
Alzheimer's disease therapy research programme
- Thuris/D-Pharm
Undefined mechanism
69
Research programme: Alzheimer's
disease therapy - Satori
Pharmaceuticals
Satori Pharmaceuticals is developing gamma-
secretase modulators as a disease modifying
therapy for Alzheimer's disease (AD). AD is
characterized by the formation of amyloid beta
(Abeta) plaques in brain. There are two main
forms of Abeta: Abeta40 and Abeta42. Satori's
programme aims to selectively reduce Abeta42,
which may overcome the adverse effects
associated with reducing total Abeta levels.
[CONT.]
Amyloid precursor protein secretase modulators
CT 0109 Cognition Therapeutics Owner USA Pharmaceutical Private
CT0093
CT0109
Amyloid oligomer small molecule Alzheimer's disease Preclinical Parenteral USA Kinexis Market Licensee World Company
Amyloid oligomer vaccine Neurodegenerative disorders Preclinical Parenteral USA University of California at Irvine Originator USA University N/A
Anti-amyloid oligomer MAb Parkinson's disease Preclinical Parenteral USA University of California at Irvine Owner USA University N/A
Anti-amyloid oligomer monoclonal antibody
AZ 12066871 Alzheimer's disease Preclinical Unknown route United Kingdom Astex Pharmaceuticals Originator USA Pharmaceutical Public
AZ-12066871 AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
AZ12066871
BACE1 inhibitors - Astex
Pharmaceuticals/AstraZenecaBeta-secretase inhibitors - Astex
Pharmaceuticals/AstraZenecaMemapsin-2 inhibitors - Astex
Pharmaceuticals/AstraZenecaBristol-Myers Squibb is developing gamma
secretase (gamma-secretase) inhibitors for the
treatment of Alzheimer's disease (AD). Lead
compounds from this programme include BMS
289948, BMS 299897 and BMS 433796.
BMS 289948 Alzheimer's disease Preclinical Unknown route USA Bristol-Myers Squibb Originator USA Large Pharma, Biopharmaceutical Public
The neurodegeneration observed in AD is thought
to be a result of a pathological cascade initiated
by formation of insoluble plaques caused by the
accumulation of amyloid beta (Abeta) peptides
[CONT.]
BMS 299897 Bristol-Myers Squibb Owner USA Large Pharma, Biopharmaceutical Public
BMS 433796
BMS-289948
BMS-299897
BMS-433796
EVP 14936 Alzheimer's disease Preclinical PO USA Amorfix Life Sciences Collaborator Canada Biopharmaceutical Public
EVP 15962 EnVivo Pharmaceuticals Originator USA Biopharmaceutical Private
EVP-14936 EnVivo Pharmaceuticals Owner USA Biopharmaceutical Private
EVP-15962
Merck & Co. is developing small molecule
inhibitors of amyloid precursor protein secretase
(beta-secretase and gamma-secretase) for the
treatment of Alzheimer's disease (AD). Preclinical
development of a number of compounds is
underway in the US.
beta-secretase-1 inhibitors - Merck & Co Alzheimer's disease Preclinical PO USA Merck & Co Owner USA Large Pharma, Pharmaceutical Public
beta-secretase is a proteolytic enzyme involved
in the cleavage of amyloid precursor protein
(APP) to produce toxic beta-amyloid (Abeta)
peptides 40 and 42 [CONT.]
gamma-secretase inhibitors - Merck & Co Schering-Plough Originator USA Large Pharma, Pharmaceutical Public
BACE programme - Merck & Co
SCH 1375975
SCH 697466
SCH 723909
SCH 741216
SCH 745966
SCH 747123
SCH-1375975
SCH-745966
NGP 555 Alzheimer's disease Preclinical PO USA NeuroGenetic Pharmaceuticals Owner USA Pharmaceutical
NGP-555 TorreyPines Therapeutics Inc Originator USA Biopharmaceutical Public
NGP555
NGX 555
NGX555
anti-Abeta antibodies - Takeda Alzheimer's disease Preclinical Injection Japan Takeda Originator Japan Large Pharma, Pharmaceutical Public
BA 27 Takeda Owner Japan Large Pharma, Pharmaceutical Public
BA27
BC 05
BC05
Alzheimer's disease Preclinical Parenteral USA Centocor Originator USA Biopharmaceutical Public
Johnson & Johnson Pharmaceutical Research &
Development
Originator USA Pharmaceutical Public
AstraZeneca is developing compounds for the
treatment of anxiety and neurological disorders,
including affective disorders and Alzheimer's
disease. Preclinical development is ongoing in the
UK, US and Sweden.
5-HT1A receptor antagonists - AstraZeneca Opioid delta receptor agonists Alzheimer's disease Preclinical Unknown route Sweden AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
AstraZeneca and Columbis University Medical
Center are also collaborating to examine
neurogenesis as a potential pathway for
treament of depression and anxiety. [CONT.]
AZ 12320927 Serotonin 1A receptor antagonists Anxiety disorders Preclinical Unknown route United Kingdom AstraZeneca Owner United Kingdom Large Pharma, Pharmaceutical Public
AZ12320927 Serotonin 1B receptor antagonists Major depressive disorder Preclinical Unknown route USA Columbia University Collaborator USA University N/A
Karolinska Institute Collaborator Sweden University N/A
University of Texas Southwestern Medical Center Collaborator USA University N/A
Eli Lilly is developing lentiviral vectors expressing
apolipoprotein E (apoE) isoforms for treatment of
Alzheimer's disease (AD). Development is at the
preclinical stage in the US.
Alzheimer's disease Preclinical Unknown route USA Eli Lilly Originator USA Large Pharma, Pharmaceutical Public
Studies have indicated that direct gene delivery
of apoE2 via intracerebral administration may
prevent or reduce brain amyloid-beta (Abeta)
burden and the subsequent development of
neuritic plaques. [CONT.]
Eli Lilly Owner USA Large Pharma, Pharmaceutical Public92
Research programme: apolipoprotein
E gene therapy - Eli Lilly
ApoE2 gene therapy - Eli Lilly Apolipoprotein E agonists
90
Research programme: anti-beta-
amyloid antibodies - Centocor/
Johnson & Johnson
Centocor and Johnson & Johnson Pharmaceutical
Research and Development are developing
monoclonal antibodies specific to a truncated
form of beta-amyloid (Abeta), for the treatment
of Alzheimer's disease. The truncated form of
Abeta, Abeta11-40/42, results from further
cleavage of the Abeta by the BACE1 enzyme at
position 11, and is increased in biological
samples from Alzheimer's patients with the
Swedish mutation in APP [CONT.]
JRF/hAb11/1 Amyloid beta-protein inhibitors
91
Research programme: anxiety and
neurological disorder therapeutics -
AstraZeneca
89
Research programme: anti-beta
amyloid antibodies - Takeda
Takeda is developing passive immunotherapy for
the treatment of Alzheimer's disease. Previous
work showed vaccination was an effective
treatment for Alzheimer's disease but the risk of
adverse events associated with the therapy,
including meningoencephalitis, prompted Takeda
to develop antibodies against beta amyloid
(Abeta) as a potentially safer option.
Development is at the preclinical stage in Japan.
[CONT.]
Amyloid beta-protein inhibitors
88
Research programme: amyloid
precursor protein secretase
modulators - NeuroGenetic
Pharmaceuticals
NeuroGenetic Pharmaceuticals is developing
small molecule modulators of human amyloid
precursor protein secretase (gamma-secretase)
for the treatment of Alzheimer's disease (AD).
The company has identified a series of orally
available 2-aminothiazole derivatives that
lowered pathogenic beta-amyloid (Abeta)
Abeta42 levels and shifted the cleavage towards
shorter Abeta peptides (such as Abeta37 ,
Abeta38) [CONT.]
Amyloid precursor protein secretase modulators
86
Research programme: amyloid
precursor protein secretase
inhibitors - EnVivo
EnVivo Pharmaceuticals is developing small-
molecule, oral amyloid precursor protein
secretase (gamma-secretase) inhibitors for the
treatment of Alzheimer's disease (AD). The
company believes that its compounds are brain-
permeable and have disease-modifying potential.
Lead compounds identified under the programme
include EVP 14936 which has entered into
preclinical development in the US. [CONT.]
Amyloid precursor protein secretase inhibitors
87
Research programme: amyloid
precursor protein secretase
inhibitors - Merck & Co
Amyloid precursor protein secretase inhibitors
85
Research programme: amyloid
precursor protein secretase
inhibitors - Bristol-Myers Squibb
Amyloid precursor protein secretase inhibitors
84
Research programme: amyloid
precursor protein secretase
inhibitors - Astex
Pharmaceuticals/AstraZeneca
Astex Pharmaceuticals and AstraZeneca are
collaborating on the development of a series of
pyrimidine compounds targeting the amyloid
precursor protein secretase, beta-secretase, a
key protein implicated in Alzheimer's disease.
Astex Pharmaceuticals, a structure-based drug
discovery company, is providing an initial series
of small-molecule, high affinity, beta-secretase
inhibitors. [CONT.]
Amyloid precursor protein secretase inhibitors
83
Research programme: amyloid
oligomer therapeutics - Kinexis
Kinexis Inc., a life science R&D company, is
developing a variety of amyloid oligomer
therapeutics for the treatment of
neurodegenerative diseases. The company
exclusively licensed the intellectual property and
know-how for this programme from the
University of California, Irvine. [CONT.]
82
Research programme: amyloid beta-
protein inhibitors - Cognition
Therapeutics
Cognition Therapeutics is developing small
molecule, amyloid beta-protein inhibitors, as a
disease-modifying treatment for Alzheimer's
disease. The lead candidates inhibit the activity
of the oligomeric form of the amyloid beta-
protein that interferes with learning and memory.
Memory deficits caused by the oligomeric protein
are among the earliest changes seen in
Alzheimer's disease. [CONT.]
Amyloid beta-protein inhibitors
Alzheimer's disease Preclinical Unknown route USA Merck & Co Market Licensee World Large Pharma, Pharmaceutical Public
The J. David Gladstone Institutes Originator USA Institution N/A
The J. David Gladstone Institutes Owner USA Institution N/A
COG 112 Alzheimer's disease Preclinical Unknown route USA Cognosci Owner USA Pharmaceutical Private
COG 133 Brain injuries Preclinical Unknown route USA Duke University Originator USA University N/A
COG 1410 Multiple sclerosis Preclinical Unknown route USA
COG 241 Subarachnoid haemorrhage Preclinical Unknown route USA
COG 68 Inflammatory bowel diseases Discontinued(Preclinical) Unknown route USA
COG112 Chronic lymphocytic leukaemia No development reported(Preclinical) Unknown route USA
COG133 Rheumatoid arthritis No development reported(Preclinical) Unknown route USA
COG1410 Parkinson's disease No development reported(Research) Unknown route USA
COG241
COG68
Alzheimer's disease Preclinical Unknown route USA Madera BioSciences Originator USA Biotechnology Private
Madera BioSciences Owner USA Biotechnology Private
BACE inhibitors - CoMentis Alzheimer's disease Preclinical PO USA Astellas Pharma Licensee World Large Pharma, Pharmaceutical Public
Disease modifying Alzheimer's disease therapy -
CoMentis
CoMentis Originator USA Biopharmaceutical Private
OM 991 CoMentis Owner USA Biopharmaceutical Private
OM 992 Oklahoma Medical Research Foundation Technology Provider USA Institution N/A
University of Illinois at Chicago Technology Provider USA University N/A
Alzheimer's disease Preclinical Unknown route USA Bristol-Myers Squibb Originator USA Large Pharma, Biopharmaceutical Public
Bristol-Myers Squibb Owner USA Large Pharma, Biopharmaceutical Public
Alzheimer's disease Preclinical Unknown route USA Johnson & Johnson Pharmaceutical Research &
Development
Originator USA Pharmaceutical Public
Johnson & Johnson Pharmaceutical Research &
Development
Owner USA Pharmaceutical Public
Merck & Co. in the US is developing brain-
penetrating small-molecule inhibitors of human
BACE1 (beta-amyloid converting enzyme, or beta-
secretase) as potential treatments for
Alzheimer's disease. Several series' of
compounds are being investigated and preclinical
development is ongoing in the US.
SCH 1359113 Alzheimer's disease Preclinical PO USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
It is believed that the amyloid cascade plays a
significant role in the cause of Alzheimer's
disease. [CONT.]
SCH 1682496 Sunesis Pharmaceuticals Originator USA Biopharmaceutical Public
SCH 785532
SCH-1359113
SCH-1682496
SCH-785532
Alzheimer's disease Preclinical IV Switzerland Novartis Originator Switzerland Large Pharma, Pharmaceutical Public
Novartis Owner Switzerland Large Pharma, Pharmaceutical Public
WAC-547415 Alzheimer's disease Preclinical PO USA Pfizer Owner USA Large Pharma, Pharmaceutical Public
WAY-258131 Wyeth Originator USA Large Pharma, Pharmaceutical Public
WY-24454
BACE1 inhibitors - Vitae Alzheimer's disease Preclinical PO USA Boehringer Ingelheim Licensee World Large Pharma, Pharmaceutical Private
Beta secretase inhibitors - Vitae Vitae Pharmaceuticals Originator USA Pharmaceutical Private
Vitae Pharmaceuticals Owner USA Pharmaceutical Private
PD 0118057 Alzheimer's disease Preclinical Unknown route Japan, USA Astellas Pharma Originator Japan Large Pharma, Pharmaceutical Public
PD 0202091 Pfizer Originator USA Large Pharma, Pharmaceutical Public
Alzheimer's disease Preclinical Unknown route Switzerland Actelion Pharmaceuticals Originator Switzerland Large Pharma, Biopharmaceutical Public
104
Research programme: beta secretase
inhibitors - Actelion
Researchers at Actelion Pharmaceuticals are
investigating aspartyl protease inhibitors of beta
secretase (BACE, BACE1, beta amyloid precursor
protein-cleaving enzyme) as potential treatments
for Alzheimer's disease. BACE is an enzyme that
mediates the deposition of amyloid plaque in the
brain.
Research programme: beta-secretase inhibitor Amyloid beta-protein precursor inhibitors
102
Research programme: BACE1 protein
inhibitors - Vitae Pharmaceuticals
Vitae Pharmaceuticals, and Boehringer
Ingelheim, are developing oral BACE1 (bate site
APP cleaving enzyme 1) protein inhibitors for the
treatment of Alzheimer's disease (AD). The
inhibitors have demonstrated the ability to cross
the blood-brain barrier and to lower brain
amyloid beta (Abeta) peptide following a single
oral dose in preclinical, in vivo models of AD.
[CONT.]
BACE1 protein inhibitors
103
Research programme: beta amyloid
aggregation inhibitors - Astellas
Pharma/Pfizer
Pfizer and Astellas Pharma are developing
amyloid beta peptide (Abeta) aggregation
inhibitors for the treatment of Alzheimer's
disease. Formation of insoluble plaques caused
by aggregation of Abeta is thought to initiate a
pathological cascade that results in Alzheimer's-
related neurodegeneration. Inhibition of protein
aggregation with small molecule inhibitors may
be a potential way to slow or stop disease
progression [CONT.]
Amyloid beta-protein inhibitors
101
Research programme: BACE1 protein
inhibitors - Pfizer
Pfizer is developing orally active, small molecule
inhibitors of beta-secretase (BACE1) for the
treatment of Alzheimer's disease. beta-secretase
is a human brain protease that catalyzes the first
step of the two-step amyloid precursor protein
(APP) cleavage process, which in turn leads to
the formation of toxic beta-amyloid
(Abeta)40/42 peptides. Preclinical development
is underway in the US. [CONT.]
BACE1 protein inhibitors
100
Research programme: BACE1 protein
inhibitors - Novartis
Novartis is developing cyclic sulfoxide
hydroxyethylamine-based BACE-1 inhibitors for
the treatment of Alzheimer's disease. BACE-1 is
an aspartyl protease which cleaves amyloid
precursor protein to form that N-terminus of
Abeta, the major constituent of amyloid plaques.
To identify compounds, Novartis has developed a
design approach involving X-rays of co-
crystallisation of macrocyclic ethanolamine
inhibitors with BACE-1 [CONT.]
BACE1 protein inhibitors
98
Research programme: BACE1 protein
inhibitors - Johnson & Johnson
Johnson & Johnson Pharmaceutical R&D is
developing a series of BACE1 (beta-amyloid site
cleaving enzyme) inhibitors for the treatment of
Alzheimer's disease (AD). The amyloid
hypothesis is one of the mechanisms through
which the etiology of AD may be explained; beta-
secretase (BACE) and gamma-secretase cleave
amyloid precursor protein (APP) at the N- and C-
terminus, respectively, producing the beta-
amyloid peptides, Abeta1-40 and Abeta1-42,
which subsequently aggregate into neurotoxic
oligomers and fibrils [CONT.]
BACE1 protein inhibitors
99
Research programme: BACE1 protein
inhibitors - Merck and Co
BACE1 protein inhibitors
96
Research programme: BACE1 protein
inhibitors - Astellas
Pharma/CoMentis
CoMentis (formerly Zapaq) and Astellas Pharma
are co-developing orally available, small
molecule inhibitors of BACE1 (beta-amyloid
converting enzyme 1 or memapsin 2) as disease-
modifying agents for Alzheimer's disease (AD).
beta-secretase is a human brain aspartic
protease that catalyses the rate-limiting step in
amyloid-beta (Abeta) peptide production, a key
event in the progression of AD. [CONT.]
BACE1 protein inhibitors
97
Research programme: BACE1 protein
inhibitors - Bristol-Myers Squibb
Bristol-Myers Squibb is developing BACE1 (beta-
secretase) inhibitors for the treatment of
Alzheimer's disease. BACE is a human brain
protease that catalyzes the first step of the two-
step amyloid precursor protein (APP) cleavage
process, which in turn leads to the formation of
toxic beta-amyloid (Abeta)40/42 peptides.
[CONT.]
BMS 782450 BACE1 protein inhibitors
95
Research programme: apolipoprotein
E-targeted Alzheimer's disease
therapeutics - Madera Biosciences
Madera Biosciences is developing small molecule
compounds that increase the expression of
apolipoprotein E (apoE) for the treatment of
Alzheimer's disease (AD) in the US. Through the
increase expression of ApoE, the molecules
mediate beta-amyloid clearance from the brain,
preventing its accumulation into the plaques that
are characteristic of AD. [CONT.]
Apolipoprotein E modulators
93
Research programme: apolipoprotein
E therapies - Gladstone
Institutes/Merck
Merck & Co., Inc. is collaborating with the J.
David Gladstone Institutes in the development of
a treatment for neurodegenerative diseases
linked to apolipoprotein E (apoE)-regulated
pathways, such as Alzheimer's disease (AD).
Three forms of apoE, (aopE2, E3 and E4), are
involved in the regulation of lipid homeostasis
and the maintenance of neuronal integrity and
function. [CONT.]
Undefined mechanism
94
Research programme: apolipoprotein
E-mimetic peptide therapeutics -
Cognosci Inc.
Cognosci Inc. of Research Triangle Park (North
Carolina, US) is developing small molecule
apolipoprotein E (apoE)-mimetic peptides of ten
to seventeen amino acids for the treatment of
multiple sclerosis (MS), traumatic brain injury,
subarachnoid haemorrhage and Alzheimer's
disease. These compounds may also have
potential in the treatment of rheumatoid arthritis
and Parkinson's disease. [CONT.]
Apolipoprotein E agonists
Actelion Pharmaceuticals Owner Switzerland Large Pharma, Biopharmaceutical Public
Abeta inhibitors - Prana Biotechnology Amyloid beta-protein inhibitors Alzheimer's disease Preclinical PO Australia Prana Biotechnology Originator Australia Biotechnology Public
Metallocomplex programme - Prana
Biotechnology
Chelating agents Alzheimer's disease (Diagnosis) Research PO Australia Prana Biotechnology Owner Australia Biotechnology Public
VK-11 Diagnostic imaging enhancers
VK-12
Abeta ligands - Affibody Alzheimer's disease Preclinical Unknown route Sweden Affibody Originator Sweden Biotechnology Private
Affibody® molecules Affibody Owner Sweden Biotechnology Private
Beta-amyloid ligands - Affibody
beta-amyloid secretion inhibitors Alzheimer's disease Preclinical Unknown route Italy Chiesi Originator Italy Pharmaceutical Private
CHF 4801 Chiesi Owner Italy Pharmaceutical Private
CHF 5022
CHF-5077
CHF4801
CHF5022
CHF5077
CHF5096
CHF5105
Alzheimer's disease Preclinical PO Japan, USA Janssen Pharmaceuticals Licensee USA Pharmaceutical Public
Shionogi Originator Japan Large Pharma, Pharmaceutical Public
Shionogi Owner Japan Large Pharma, Pharmaceutical Public
A 705253 Alzheimer's disease Preclinical Unknown route USA Abbott Laboratories Originator USA Large Pharma, Pharmaceutical Public
A-705253 AbbVie Owner USA Biopharmaceutical Public
APH 0802 Alzheimer's disease Preclinical PO USA Aphios Corporation Originator USA Biopharmaceutical Private
APH-0802 Cachexia Preclinical PO USA Aphios Corporation Owner USA Biopharmaceutical Private
Cannabinoid therapeutics research programme -
Aphios Corporation
Cancer pain Preclinical PO USA
Delta-9-tetrahydrocannabinol Drug abuse Preclinical PO USA
Delta-9-tetrahydrocannabinolic acid Nausea and vomiting Preclinical PO USA
Delta-9-THC
Delta-9-THC nanotech formulation
Delta-9-THCA
Cell mobility activators - Pharmaxon Cell movement activators Spinal cord injuries Preclinical Unknown route France CNRS Originator France Institution N/A
Polysialyl neural cell adhesion molecule
mimotope
Polysialyl neural cell-adhesion-molecule-agonists Alzheimer's disease Research Unknown route France Pharmaxon Licensee World Biopharmaceutical Private
PR-21C Mild cognitive impairment Research Unknown route France Schafer-N Originator Denmark Biotechnology Private
PR-21S Universite de la Mediterranee Originator France University N/A
PSA-NCAM mimotope University Medical Center Hamburg-Eppendorf Originator Germany University N/A
AGT 110 Acetylcholinesterase inhibitors Alzheimer's disease (Diagnosis) Preclinical IV USA ArmaGen Technologies Originator USA Biotechnology Private
AGT 115 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical IV USA ArmaGen Technologies Owner USA Biotechnology Private
AGT 120 Erythropoietin receptor modulators Parkinson's disease Preclinical IV USA
AGT 160 Glial cell line-derived neurotrophic factor
modulators
Poisoning Preclinical Unknown route USA
AGT 185 Tumour necrosis factor alpha inhibitors Stroke Preclinical IV USA
AGT 190 Tumour necrosis factor decoy receptor
modulators
Brain cancer No development reported(Preclinical) IV USA
AGT 3 Tumour necrosis factor inhibitors Brain disorders No development reported(Preclinical) IV USA
AGT 3100 Major depressive disorder No development reported(Preclinical) IV USA
AGT-110 Spinal cord injuries No development reported(Preclinical) IV USA
AGT-115
AGT-120
AGT-160
AGT-185
AGT-190
AGT-3
AGT-3100
PA 1637 Alzheimer's disease Preclinical Unknown route France PALUMED Originator France Biotechnology Private
PA1637 PALUMED Owner France Biotechnology Private
ST 1859 Alzheimer's disease Preclinical Unknown route Italy sigma-tau SpA Originator Italy Pharmaceutical Private
ST 1936 Depressive disorders Preclinical Unknown route Italy sigma-tau SpA Owner Italy Pharmaceutical Private
ST 2472 Schizophrenia Preclinical Unknown route Italy
ST1936
ST2472
ANAVEX 1 41 Calcium channel antagonists Alzheimer's disease Preclinical IV Europe Anavex Life Sciences Originator Switzerland Biopharmaceutical Public
ANAVEX 1-41 Chloride channel modulators Alzheimer's disease Preclinical PO Europe Anavex Life Sciences Owner Switzerland Biopharmaceutical Public
ANAVEX 19-144 Muscarinic M1 receptor agonists Epilepsy Preclinical Unknown route Europe
Muscarinic M2 receptor antagonists Major depressive disorder Preclinical Unknown route Europe
Muscarinic M3 receptor antagonists Stroke Preclinical Unknown route Germany
NMDA receptor antagonists
Serotonin modulators
Sigma-1 receptor agonists
Sodium channel antagonists
Sodium channel modulators
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical PO USA Intra-Cellular Therapies Originator USA Company
Dopamine D1 receptor agonists Intra-Cellular Therapies Owner USA Company
Dopamine receptor agonists Takeda Licensee World Large Pharma, Pharmaceutical Public
116
Research programme: CNS disorders
therapeutics - Intra-Cellular
Therapies
Intra-Cellular Therapies is developing orally-
available, small molecule therapeutics for the
treatment of various central nervous system
(CNS) disorders, including Alzheimer's disease
(AD). The company is using its CNSProfile™
technology platform to select and optimise its
internally-derived and proprietary drug
candidates as well as to evaluate potential in-
licensed candidates. [CONT.]
ITI-009
115
Research programme: CNS disorders
therapeutics - Anavex Life Sciences
Anavex Life Sciences is developing compounds
targeted at sigma receptors for the treatment of
the central nervous system (CNS) disorders
Alzheimer's disease, epilepsy, depression and
stroke. Pharmacological research has indicated
that sigma-1 and sigma-2 receptors act as
intracellular amplifiers, creating a supersensitive
state of signal transduction in biochemical
pathways, which might contribute to these
disorders [CONT.]
114
Research programme: CNS disorder
therapies - sigma-tau
sigma-tau has a research programme directed at
disorders of the central nervous system (CNS)
and peripheral nervous system (PNS). Lead
compounds from this programme include ST
2472 and ST 1859 for the treatment of
schizophrenia and Alzheimer's disease,
respectively. Another lead candidate, ST 1936, is
also in development as a treatment for unipolar
depressive disorders. [CONT.]
Undefined mechanism
113
Research programme: chelating
agents - PALUMED
PALUMED SA, in France, is researching copper
chelating agents for the treatment of Alzheimer's
disease (AD) including lead candidate PA 1637.
Preclinical development of PA 1637 is ongoing
and PALUMED is preparing it to enter clinical
trials. [CONT.]
Chelating agents
112
Research programme: central
nervous system therapeutics -
ArmaGen
ArmaGen is developing therapies for the
treatment of central nervous system (CNS)
disorders utilising their trojan horse delivery
system. This delivery system involves binding a
substrate capable of crossing the blood brain
barrier (BBB) to a therapeutic agent. [CONT.]
111
Research programme: cell movement
activators - Pharmaxon
Pharmaxon is developing compounds that
specifically stimulate neural cell mobility and
neuroplasticity for the treatment of spinal cord
injuries as well as mild cognitive impairment and
Alzheimer's disease. A lead candidate generated
from this research is a polysialyl neural cell
adhesion molecule (PSA-NCAM) mimotope (a
cyclic 12-amino acid peptide that can
conformationally mimic antigenic specificity)
[CONT.]
109
Research programme: calpain
inhibitors - AbbVie
AbbVie (formerly Abbott Laboratories) is
developing calpain inhibitors for potential use in
the treatment of Alzheimer's disease (AD). The
over activation of calpain, a calcium-dependent
cysteine protease, is believed to play a role in the
pathophysiology of several neurodegenerative
disorders, including AD. [CONT.]
Calpain inhibitors
110
Research programme: cannabinoid
therapeutics - Aphios Corporation
The US biopharmaceutical company Aphios
Corporation is developing natural cannabinoid
products, including lead candidate APH 0802,
derived from the marijuana plant Cannabis
sativa, for the treatment of a marijuana
addiction, cancer pain, cachexia in AIDS patients,
nausea and vomiting, and relief to Alzheimer's
patients while increasing their appetite. [CONT.]
Cannabinoid receptor agonists
108
Research programme: beta-secretase
inhibitors - Janssen/Shionogi
Janssen Pharmaceuticals and Shionogi are
collaborating to discover, develop and
commercialise small molecule oral beta-
secretase (BACE) (amyloid precursor protein
secretase) inhibitors for the treatment of
Alzheimer's disease. Development is at the
preclinical stage in Japan and the US. [CONT.]
Amyloid precursor protein secretase inhibitors
107
Research programme: beta-amyloid
secretion inhibitors - Chiesi
Chiesi in Italy is developing flurbiprofen
analogues for the potential treatment of patients
with Alzheimer's disease. The chronic use of a
subset of non-steroidal anti-inflammatory drugs
(flurbiprofen, ibuprofen and indomethacin) has
been reported to selectively inhibit the production
of beta amyloid1-42 (Abeta42) in vitro and limit
the progression of Abeta plaque pathology in
mice over expressing human amyloid precursor
protein [CONT.]
Amyloid beta-protein inhibitors
106
Research programme: beta-amyloid
ligands - Affibody
Affibody AB is developing Affibody® molecules as
small protein ligands that target beta-amyloid
(Abeta) for the treatment of Alzheimer's disease
(AD). Such ligands stabilise the Abeta peptide
and inhibits the formation of toxic Abeta fibrils
and plaques in the brain. Development of lead
candidates is at the preclinical stage in Sweden.
[CONT.]
Amyloid beta-protein inhibitors
104
Research programme: beta secretase
inhibitors - Actelion
Researchers at Actelion Pharmaceuticals are
investigating aspartyl protease inhibitors of beta
secretase (BACE, BACE1, beta amyloid precursor
protein-cleaving enzyme) as potential treatments
for Alzheimer's disease. BACE is an enzyme that
mediates the deposition of amyloid plaque in the
brain.
Research programme: beta-secretase inhibitor Amyloid beta-protein precursor inhibitors
105
Research programme: beta-amyloid
inhibitors - Prana Biotechnology
Prana Biotechnology is developing a compound
library comprising a next-generation class of
orally available, platinum-based complexes
('metallocomplexes') with 1,10-phenanthroline-
derived ligands as beta-amyloid (Abeta)
inhibitors for the treatment of neurodegenerative
disorders, particularly Alzheimer's disease (AD).
Such compounds bind and block the actual metal-
binding site on Abeta peptide in the brain
[CONT.]
Type 1 cyclic nucleotide phosphodiesterase
inhibitors3-Methoxy-pregnenolone Microtubule-associated protein stimulants Alzheimer's disease Preclinical Parenteral France Mapreg Originator France Biotechnology Private
Pregnenolone analogues Tubulin polymerisation promoters Brain injuries Preclinical Parenteral France Mapreg Owner France Biotechnology Private
Pregnenolone derivatives Depressive disorders Preclinical Parenteral France
Schizophrenia Preclinical Parenteral France
Spinal cord injuries Preclinical Parenteral France
Stroke Preclinical Parenteral France
Alzheimer's disease Preclinical Unknown route Canada MedGenesis Therapeutix Originator Canada Biopharmaceutical Private
Glioblastoma Preclinical Unknown route Canada MedGenesis Therapeutix Owner Canada Biopharmaceutical Private
Parkinson's disease Preclinical Unknown route Canada
CB 2433 Alzheimer's disease Preclinical Unknown route USA Cenomed BioSciences Licensee World Biopharmaceutical Private
CB-2 Mild cognitive impairment Preclinical Unknown route USA Medical College of Georgia Originator USA Institution N/A
433 Medical College of Georgia Owner USA Institution N/A
CB-2433
CB2433
CM 2433
CM 2450
CM-2
433
CM-2
450
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical PO United Kingdom Nonindustrial source Originator Unknown Institution N/A
Antioxidants Cancer Research Unknown route United Kingdom Nonindustrial source Owner Unknown Institution N/A
Apoptosis inhibitors ReGen Therapeutics Market Licensee United Kingdom Company
Cell differentiation stimulants
Minozac Alzheimer's disease Preclinical PO Canada, USA Chinese Academy of Sciences Collaborator China Institution N/A
MW 015188WH Rheumatoid arthritis Preclinical PO Canada, USA InnovatePharma Collaborator USA Pharmaceutical Private
MW01-5-188WH Alzheimer's disease Discontinued(Preclinical) PO China Northwestern University Originator USA University N/A
TT302 Brain injuries Discontinued(Preclinical) PO Canada, China Northwestern University Owner USA University N/A
Multiple sclerosis Discontinued(Preclinical) PO Canada, China, USA Transition Therapeutics Licensee World Biopharmaceutical Public
Parkinson's disease Discontinued(Preclinical) PO Canada, USA University of California at Los Angeles Collaborator USA University N/A
Cholinesterase inhibitors Alzheimer's disease Preclinical Unknown route USA ChemDiv Originator USA CRO Private
G protein-coupled receptor modulators Cognition disorders Preclinical Unknown route USA ChemDiv Owner USA CRO Private
NMDA receptor antagonists
Serotonin 6 receptor antagonists
Alzheimer's disease Preclinical Unknown route Italy Siena Biotech Originator Italy Company
Siena Biotech Owner Italy Company
Alzheimer's disease Preclinical Unknown route USA Diaxonhit Originator France Pharmaceutical
Down syndrome Preclinical Unknown route USA Diaxonhit Owner France Pharmaceutical
acetyl-L-leucyl-L-valyl-L-lysinal-aldehyde Amyloid precursor protein secretase modulators Alzheimer's disease Preclinical Unknown route USA American Life Science Pharmaceuticals Originator USA Pharmaceutical
CA 074Me Cathepsin B inhibitors American Life Science Pharmaceuticals Owner USA Pharmaceutical
CA074Me Endopeptidase inhibitors
E 64d
E64d
Alzheimer's disease Preclinical Unknown route USA Chicago Labs Originator USA Company
Pain Preclinical Unknown route USA Chicago Labs Owner USA Company
Stroke Preclinical Unknown route USA
ESN-282 G protein-coupled receptor modulators Alzheimer's disease Preclinical Unknown route Belgium Euroscreen Originator Belgium Company
ESN-401 G-protein coupled receptor 43 modulators Amyotrophic lateral sclerosis Preclinical Unknown route Belgium Euroscreen Owner Belgium Company
ESN-502 Brain injuries Preclinical Unknown route Belgium
ESN-603 Parkinson's disease Preclinical Unknown route Belgium
Ulcerative colitis Preclinical Unknown route Belgium
Obesity Research Unknown route Belgium
Type 2 diabetes mellitus Research Unknown route Belgium
Inflammation No development reported(Research) Unknown route Belgium
SPH 1285 Alzheimer's disease Preclinical Unknown route Austria Sanochemia Pharmazeutika Originator Austria Pharmaceutical Public
SPH 1286 Dementia Preclinical Unknown route Austria, United Kingdom Sanochemia Pharmazeutika Owner Austria Pharmaceutical Public
SPH 1371 Neurological disorders Preclinical Unknown route Austria, United Kingdom
128
Research programme: galantamine
derivatives - Sanochemia
Pharmazeutika
Sanochemia Pharmazeutika is investigating
galantamine derivatives as potential treatments
for various neurodegenerative disorders,
including dementia, stroke, brain injury,
Parkinson's disease, Alzheimer's disease and
neuropathic pain. The company's most developed
molecule [SPH 1285] has a partially novel
mechansim of action and is a candidate for
phase I clinical trials. [CONT.]
Acetylcholinesterase inhibitors
127
Research programme: G protein-
coupled receptor modulators -
Euroscreen
Euroscreen in Belgium has a broad research
programme focussed on discovering small
molecule therapeutics that target G protein-
coupled receptors (GPCRs). Lead compounds
under investigation include ESN 282, a GPR43
modulator in development for ulcerative collitis,
and ESN 502, a GPR modulator investigated for
neurodegenerative diseases such as Alzheimer's,
Parkinson's, amyotropic lateral sclerosis (ALS)
and traumatic brain injury [CONT.]
126
Research programme: endothelin
receptor antagonists - Chicago Labs
Chicago Labs is developing endothelin (ET)
receptor antagonists for the treatment of pain,
stroke, and Alzheimer's disease. Endothelin
receptors are prevalent throughout the
vasculature and are responsible for vasodilation
and vasoconstriction. Development is at the
preclinical stage in the US.
Endothelin receptor antagonists
124
Research programme: DYRK
inhibitors - Diaxonhit
Diaxonhit is developing small molecule, selective
DYRK1a/1b (dual specificity tyrosine-
phosphorylation-regulated kinase) inhibitors for
the treatment of Alzheimer's disease and Down
syndrome. DYRK1a/1b kinases are involved in
several pathways associated with CNS disorders
or cell proliferation. Diaxonhit believes that
DYRK1a kinase inhibitors have the potential to be
disease modifying agents for tauopathies and
Tau-mediated amyloid beta toxicity, including
Alzheimer's disease and Down syndrome [CONT.]
DYRK kinase inhibitors
125
Research programme: endopeptidase
inhibitors - American Life Science
Pharmaceuticals
American Life Science Pharmaceuticals is
developing small molecule inhibitors of
proprietary endopeptidase targets for the
treatment of Alzheimer's disease. The company
is focusing on the development of secretase and
cathepsin B endopeptidase targets. Secretases
cleave amyloid precursor protein (APP) into beta-
amyloid (Abeta) peptides which are thought to be
responsible for the pathology of Alzheimer's
disease [CONT.]
123
Research programme: DKK1
antagonists - Siena Biotech
Siena Biotech is developing small molecule
selective antagonists of the Dickkopf-1 (DKK1)
protein as a treatment for Alzheimers disease
(AD). It has been demonstrated that DKK1
causes the inhibition of the Wnt signalling
pathway, which when impaired causes an
increase in the expression of amyliod-beta
peptide in neuronal cultures and in patients
suffering from AD. [CONT.]
Wnt protein modulators
121
Research programme: cytokine
inhibitors - Transition Therapeutics
Transition Therapeutics is developing small
molecule cytokine inhibitors for the potential
treatment of neurological and inflammatory
disorders. A lead candidate, TT 302, is in
preclinical development in the US and Canada for
the potential treatment of Alzheimer's disease
and rheumatoid arthritis. Development for the
latter indication is being conducted in conjunction
with the University of California at Los Angeles
(California, USA) [CONT.]
Cytokine inhibitors
122
Research programme: dimebolin
analogues - ChemDiv
ChemDiv is undertaking a research programme
to develop a series of small molecule, water-
soluble, dimebolin analogues for the treatment of
cognition disorders and Alzheimer's disease
(AD). This series of dimebolin analogues has
been found to penetrate the blood-brain barrier
and possess promising cognition-enhancing
properties. Optimised lead compounds are
undergoing preclinical testing in the US. [CONT.]
120
Research programme: colostrinin
constituent peptides - ReGen
Therapeutics
ReGen Therapeutics Plc is characterising of the
biologically active peptide constituents of
Colostinin™ in order to identify properties that
might lend them towards development as
pharmaceutical agents for CNS
neurodegenerative diseases and cancer. The
company hopes to identify smaller molecular
weight therapeutics that have similar or greater
activity than Colostrinin™. [CONT.]
Colostinin™
119
Research programme: cognitive
disorders therapeutics - Cenomed
BioSciences
Cenomed BioSciences, in collaboration with the
Medical College of Georgia Research Institute, is
developing small molecules that target
components of acetylcholine neurons and thereby
enhance cholinergic function. These agents are
being developed to treat cognitive disorders such
as mild cognitive impairment and Alzheimer's
disease, while avoiding cholerinergic side effects.
[CONT.]
Cholinergic receptor modulators
118
Research programme: CNS disorders
therapeutics - MedGenesis
Therapeutix
MedGenesis Therapeutix Inc is developing
compounds to treat diseases of the central
nervous system (CNS) including Alzheimer's
disease, Parkinson's disease and brain cancers.
The company is using its Convection Enhanced
Delivery (CED) methods to target and transport
therapeutic agents to the relevant area in the
CNS. [CONT.]
Undefined mechanism
116
Research programme: CNS disorders
therapeutics - Intra-Cellular
Therapies
Intra-Cellular Therapies is developing orally-
available, small molecule therapeutics for the
treatment of various central nervous system
(CNS) disorders, including Alzheimer's disease
(AD). The company is using its CNSProfile™
technology platform to select and optimise its
internally-derived and proprietary drug
candidates as well as to evaluate potential in-
licensed candidates. [CONT.]
ITI-009
117
Research programme: CNS disorders
therapeutics - Mapreg
Mapreg SAS of France is developing a series of
synthetic neurosteroids derived from
pregnenolone for the treatment of various central
nervous system (CNS) disorders, including spinal
cord injuries, traumatic brain injuries and stroke
along with depressive disorders, schizophrenia
and Alzheimers's disease. These pregnenolone
derivatives do not exhibit metabolic interactions
with other steroid hormones and are designed for
administration by injection with the ability to
cross the blood-brain barrier. Development is at
the preclinical stage in France. [CONT.]
SPH 1373 Neuropathic pain Preclinical Unknown route Austria
SPH 1375
SPH-1285
SPH-1286
SPH-1371
SPH-1373
SPH-1375
SPH1285
Galantamine pro-drug - Galantos Pharma Acetylcholinesterase inhibitors Alzheimer's disease Preclinical Intranasal Germany Galantos Pharma Originator Germany Pharmaceutical Private
GLN 1062 Nicotinic receptor modulators Alzheimer's disease Preclinical IV Germany Galantos Pharma Owner Germany Pharmaceutical Private
GLN-1062
GLN1062
Memogain®
Pro-galantamine - Galantos Pharma
MRK 003 Amyloid precursor protein secretase inhibitors Alzheimer's disease Preclinical PO USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
MRK 560 Notch signalling pathway inhibitors Cancer Preclinical PO USA Merck & Co Owner USA Large Pharma, Pharmaceutical Public
MRK-003
MRK-560
2B3 201 Immunosuppressants Inflammation Preclinical Unknown route Netherlands to-BBB technologies Originator Netherlands Biotechnology Private
2B3-201 Steroid receptor agonists Multiple sclerosis Preclinical Unknown route Netherlands VIB Originator Belgium Institution N/A
Brain-targeted liposomal methylprednisolone Undefined mechanism Alzheimer's disease Research Unknown route Netherlands
Glutathione pegylated liposomal
methylprednisolone
Brain cancer Research Unknown route Netherlands
CNS disorders Research Unknown route Netherlands
Alzheimer's disease Preclinical Unknown route USA ALS Biopharma Originator USA Biotechnology Private
Amyotrophic lateral sclerosis Preclinical Unknown route USA ALS Biopharma Owner USA Biotechnology Private
Vybion Technology Provider USA Company
Alzheimer's disease Preclinical Unknown route USA Ligand Pharmaceuticals Originator USA Biotechnology Public
Attention-deficit hyperactivity disorder Preclinical Unknown route USA Ligand Pharmaceuticals Owner USA Biotechnology Public
Narcolepsy Preclinical Unknown route USA
Schizophrenia Preclinical Unknown route USA
EVX 001688 Alzheimer's disease Preclinical PO Canada, USA CHDI Collaborator USA Biotechnology Private
EVX001688 Huntington's disease Preclinical PO Canada, USA EnVivo Pharmaceuticals Licensee World Biopharmaceutical Private
HDAC inhibitors - EnVivo Neurodegenerative disorders Preclinical PO Canada, USA MethylGene Originator Canada Biopharmaceutical Public
HDACi - EnVivo Parkinson's disease Preclinical PO Canada, USA Mirati Therapeutics Owner USA Biopharmaceutical Public
Isotope-selective HDAC inhibitors (Series B) -
EnVivoCNS 101 Alzheimer's disease Preclinical Unknown route USA Coyote Pharmaceuticals Originator USA Biopharmaceutical Private
CNS 102 Amyotrophic lateral sclerosis Preclinical Unknown route USA Coyote Pharmaceuticals Owner USA Biopharmaceutical Private
CNS 101
CNS 102
CNS-101
CNS-102
CNS101
CNS102
NPT-007 Alpha-synuclein modulators Alzheimer's disease Preclinical Unknown route USA NeuroPhage Pharmaceuticals Originator USA Biotechnology
NPT-014 Amyloid beta-protein modulators Parkinson's disease Preclinical Unknown route USA NeuroPhage Pharmaceuticals Owner USA Biotechnology
NPT-088 Tau protein modulators
NPT007
NPT014
NPT088
MNLP462a Glucose modulators Alzheimer's disease Preclinical PO United Kingdom MNLpharma Originator United Kingdom Pharmaceutical Private
SMT 14400 Haemoglobin modulators Cancer No development reported(Preclinical) PO United Kingdom Summit plc Owner World Biotechnology Public
SMT C2100 Hepatitis C virus NS3 protein modulators Diabetes mellitus No development reported(Preclinical) PO United Kingdom
SMT-14224 Hexosaminidase C inhibitors Viral infections No development reported(Preclinical) PO Europe
SMT-14400 Immunomodulators Hepatitis C No development reported(Research) PO United Kingdom
SMT-C2100 Immunostimulants
SMT14224 Triglyceride modulators
SMT14400
VOX 14400
VOX-14400
VOX14400
AD 0802 Alpha-synuclein inhibitors Alzheimer's disease Preclinical Parenteral Sweden BioArctic Neuroscience Originator Sweden Biotechnology Private
AD-0802 Amyloid beta-protein inhibitors Parkinson's disease Preclinical Parenteral Sweden BioArctic Neuroscience Owner Sweden Biotechnology Private
AD0802 Immunomodulators
Alzheimer's vaccine - BioArctic Neuroscience
BAN 2203
BAN-2203
BAN2203
Monoclonal antibodies - BioArctic Neuroscience
PD 0805
PD-0805
PD0805
Omni-Raf inhibitors - Plexxikon Fms-like tyrosine kinase 3 inhibitors Alzheimer's disease Preclinical PO USA Plexxikon Originator USA Pharmaceutical Private
PLX FK1 Proto-oncogene protein b-raf modulators Cancer Preclinical PO USA Plexxikon Owner USA Pharmaceutical Private
PLX 7486 Raf kinase inhibitors Inflammation Preclinical PO USA139
Research programme: kinase
inhibitors - Plexxikon
Plexxikon (a Daiichi Sankyo subsidiary) is
developing a portfolio of orally-administered
small-molecule kinase inhibitors. One focus of
this programme appears to be inhibitors of Raf
kinase (proto-oncogene protein b-raf-inhibitors)
as potential therapies for cancer. The programme
is also investigating compounds that target Fms
kinase for the treatment of cancer, Alzheimer's
disease, and inflammatory diseases such as
multiple sclerosis and lupus [CONT.]
138
Research programme:
Immunotherapeutics - BioArctic
Neuroscience
BioArctic Neuroscience in Sweden is developing
immunotherapeutics, including monoclonal
antibodies and vaccines, for the treatment of
Alzheimer's disease (AD) and Parkinson's disease
(PD). The company's proprietary technology
platform is used to isolate proteins implicated in
neurological disorders, such as beta-amyloid and
alpha-synuclein, study their aggregation
properties, prepare and stabilise other
protofibrils, and develop protofibril specific
monoclonal antibodies [CONT.]
137
Research programme: imino sugar
therapeutics - Summit
Summit Corporation plc (VASTox) is developing
orally available, small molecule imino sugar
compounds for the treatment of Alzheimer's
disease. The compounds were also being
developed for the treatment of hepatitis C,
diabetes mellitus and cancer, but no recent
development has been reported in these
indications. [CONT.]
136
Research programme: Ig fusion
GAIM dimers - NeuroPhage
Pharmaceuticals
Neurophage Pharmaceuticals is developing
therapies targeted at protein misfolding. The
proprietary phage technology used results in
products with a novel mechanism of action based
on a general amyloid interaction motif (GAIM).
The motif is characteristic of distinctive, recurring
components of protein structures and interaction,
including those of pathological protein
aggregates that are hallmarks of both
Alzheimer's and Parkinson's diseases [CONT.]
135
Research programme: homeostasis
modulators - Coyote Pharmaceuticals
Coyote Pharmaceuticals is developing therapies
that modulate homeostasis for the treatment of
diseases with large unmet medical needs in
several therapeutic areas, including
neurodegenerative, cardiovascular, oncology,
and inflammatory areas. The way that these
therapies modulate homeostasis may include
interactions with molecular chaperones, heat
shock proteins, ubiquitin proteasomes, and DNA-
RNA transcriptional regulators of homeostasis
network components [CONT.]
Heat-shock protein stimulants
134
Research programme: histone
deacetylase inhibitors - EnVivo
Pharmaceuticals
EnVivo Pharmaceuticals is undertaking a
research programme to develop a series of small
molecule, orally available, histone deacetylase
(HDAC) inhibitors for the treatment of
neurodegenerative disorders, including
Parkinson's disease (PD), Huntington's disease
(HD), dementia and Alzheimer's disease (AD).
These isotype-selective, brain-permeable HDAC
inhibitors have been exclusively in-licensed from
MethylGene which were generated using the
company's HDAC technology [CONT.]
Histone deacetylase inhibitors
133
Research programme: histamine H3
receptor antagonists - Ligand
Ligand Pharmaceuticals is developing histamine
H3 antagonists for the treatment of sleep
disorders (narcolepsy), attention deficit
hyperactivity disorder (ADHD) and cognitive
disorders (schizophrenia and Alzheimer's
disease). The H3 receptor modulates histamine
release in the brain, controlling the release of
excitatory neurotransmitters such as glutamate
and acetylcholine by stimulating H1 receptors
[CONT.]
Histamine H3 receptor antagonists
132
Research programme: heat shock
protein 70 inhibitors - ALS
Biopharma
ALS Biopharma is developing brain-penetrant
inducers of heat shock protein 70 (Hsp70), for
the treatment of neurodegenerative disorders
such as Alzheimer's disease and amytrophic
lateral sclerosis (ALS). Hsp70 clears two
proteins, the toxic form of tau protein and beta-
amyloid, involved in disease progression.
Development is at the preclinical stage in the US.
HSP70 heat shock protein inhibitors
131
Research programme: glutathione-
pegylated liposome therapeutics - to-
BBB
to-BBB is developing therapeutics formulated
using its proprietary glutathione-pegylated
liposomes for the treatment of Alzheimer's
disease, brain cancer and CNS disorders.
Traditionally, targeting these indications has
been difficult due to the existence of the blood-
brain barrier which impedes the access of many
therapeutic agents to the brain. [CONT.]
130
Research programme: gamma
secretase inhibitors - Merck
Merck & Co. is developing small molecule
inhibitors of human amyloid precursor protein
secretase (gamma-secretase) for the treatment
of Alzheimer's disease (AD) and cancer. Various
classes of potent, functionally active gamma-
secretase inhibitors have been discovered and
characterised including benzodiazepine
derivatives, aryl sulfones and [4.2.1]-bicyclo-
based sulfonamides. [CONT.]
129
Research programme: galantamine
prodrug - Galantos Pharma
Galantos Pharma GmbH is developing an inactive
prodrug of galantamine, GLN 1062, for the
treatment of Alzheimer's disease (AD). The
prodrug is proposed to be an improved version of
the acetylcholinesterase inhibitor and nicotinic
receptor modulator galantamine, which is
conventionally used for the treatment of mild-to-
moderate AD [see separate profile for
galantamine]. [CONT.]
128
Research programme: galantamine
derivatives - Sanochemia
Pharmazeutika
Sanochemia Pharmazeutika is investigating
galantamine derivatives as potential treatments
for various neurodegenerative disorders,
including dementia, stroke, brain injury,
Parkinson's disease, Alzheimer's disease and
neuropathic pain. The company's most developed
molecule [SPH 1285] has a partially novel
mechansim of action and is a candidate for
phase I clinical trials. [CONT.]
Acetylcholinesterase inhibitors
PLX-FK1 TrkA receptor antagonists Multiple sclerosis Preclinical PO USA
PLX4720 TrkB receptor antagonists Polycystic kidney disease Preclinical PO USA
PLX7486 TrkC receptor antagonists Solid tumours Preclinical PO USA
PLX7486-TsOH Vascular endothelial growth factor receptor-1
antagonistsAMP activated protein kinase modulators Alzheimer's disease Preclinical Parenteral USA National Institutes of Health (USA) Funder USA Institution N/A
Amyloid beta-protein inhibitors Neurotez Originator USA Biotechnology Private
Neurotez Owner USA Biotechnology Private
VTP 4 Alzheimer's disease Preclinical PO USA Vitae Pharmaceuticals Originator USA Pharmaceutical Private
VTP-4 Atherosclerosis Preclinical PO USA Vitae Pharmaceuticals Owner USA Pharmaceutical Private
Alzheimer's disease Preclinical PO USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
Merck & Co Owner USA Large Pharma, Pharmaceutical Public
BT 2111 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Parenteral Canada biOasis Technologies Originator USA Biotechnology Private
BT 2211 Biological transport stimulants Brain metastases Preclinical Parenteral Canada biOasis Technologies Owner Canada Biotechnology Private
BT-2111 Enzyme replacements Lysosomal storage diseases Preclinical IV Canada Texas Tech University Health Sciences Center Collaborator USA University N/A
BT-2211 Transferrin receptor modulators University of British Columbia Originator Canada University N/A
BT2111
BT2211
Herceptin®-BT2111
D-Pharm in Israel is developing small molecule,
lipophilic, membrane activated, transition-metal
ion chelators for the treatment of Alzheimer's
disease.
DP 460 Alzheimer's disease Preclinical PO Israel D-Pharm Originator Israel Pharmaceutical Private
The company has selected a lead compound,
called DP 460. DP 460, a derivative of the
calcium chelator BAPTA, showed selective
membrane-dependent modulation of copper and
zinc homeostasis. [CONT.]
DP-460 Amyotrophic lateral sclerosis Discontinued(Preclinical) PO Israel D-Pharm Owner Israel Pharmaceutical Private
ADX 47273 Alzheimer's disease Preclinical PO Switzerland Addex Pharmaceuticals Originator Switzerland Pharmaceutical Public
ADX 50938 Cognition disorders Preclinical PO Switzerland Addex Therapeutics Owner Switzerland Pharmaceutical Public
ADX 63365 Schizophrenia Preclinical PO Switzerland
ADX47273
ADX50938
ADX63365 (mGluR5 PAMb)
ADX63365 (mGluR5 positive allosteric modulator
[PAM])CDPPB
Metabotropic glutamate receptor-5 modulators -
AddexmGluR5 modulators - Addex
MPAC - Prana Biotechnologies Alpha-synuclein inhibitors Age-related macular degeneration Preclinical Unknown route Australia Prana Biotechnology Originator Australia Biotechnology Public
PBT 434 Chelating agents Alzheimer's disease Preclinical Unknown route Australia Prana Biotechnology Owner Australia Biotechnology Public
PBT-434 Iron chelating agents Parkinson's disease Preclinical PO Australia The Michael J. Fox Foundation for Parkinsons Research Funder USA Institution N/A
PBT3 series - Prana Biotechnologies
PBT4 series - Prana Biotechnologies
PBT434
1A10 Amyloid beta-protein inhibitors Age-related macular degeneration Preclinical Parenteral USA Immuno-Biological Laboratories Originator Japan Company
8,20E+02 Amyloid inhibitors Alzheimer's disease Preclinical IV USA Intellect Neurosciences Licensee USA Biopharmaceutical Public
Anti-Abeta monoclonal antibodies Antioxidants Alzheimer's disease Preclinical Parenteral USA MRC Technology Technology Provider United Kingdom Company
IN N01 Protein aggregation inhibitors Glaucoma Preclinical Parenteral USA Northwestern University Originator USA University N/A
IN-N01 Tau protein inhibitors
IN-N01-OX2
N01-OX2
T01-OX2
tauC3
TOC 1
TOC-1
CDD 0097 Muscarinic M1 receptor agonists Alzheimer's disease Preclinical Unknown route USA Mithridion Licensee USA Biopharmaceutical
CDD 0098J Muscarinic receptor agonists Schizophrenia Preclinical Unknown route USA University of Toledo Originator USA University N/A
CDD 0199J University of Toledo Owner USA University N/A
CDD 0304
CDD 0312
CDD 0313
CDD 0314
CDD 0316
CDD 0317
CDD 0322
CDD-0097
CDD-0098J
CDD-0199J
CDD-0304
CDD-0312
CDD-0313
CDD-0314
CDD-0316
CDD-0317
CDD-0322
MI 10 022
MI-10-022
Alzheimer's disease Preclinical Parenteral Belgium, Germany Ablynx Originator Belgium Biopharmaceutical Private
Autoimmune disorders Preclinical Parenteral Belgium, Germany Boehringer Ingelheim Originator Germany Large Pharma, Pharmaceutical Private
Cancer Preclinical Parenteral Belgium, Germany
Respiratory tract disorders Preclinical Parenteral Belgium, Germany
NNI 362 Alzheimer's disease Preclinical PO USA Neuronascent Originator USA Biotechnology Private
NNI 370 Parkinson's disease Research PO USA Neuronascent Owner USA Biotechnology Private
NNI-362
150
Research programme:
neurodegenerative disease
therapeutics - Neuronascent
Neuronascent is developing a series of chemically
diverse neurogenic compounds as disease-
modifying drugs for the treatment of
neurodegenerative diseases including Alzheimer's
disease (AD) and Parkinson's disease. The
neurogenic agents are designed to enable
replacement of damaged neurons in key areas of
the brain through stimulation of neurogenesis
(i.e. nerve cell growth) and enhancement of
neuroprotection [CONT.]
149
Research programme: Nanobodies -
Ablynx/Boehringer Ingelheim
Boehringer Ingelheim and Ablynx are
collaborating in the development of
Nanobodies®, a class of therapeutic proteins, for
potential use in the treatment of Alzheimer's
disease (AD), autoimmune disorders, cancer, and
respiratory disorders. Nanobodies® are antibody-
derived proteins developed by Ablynx which
contain novel structural and functional properties
of naturally-occurring heavy-chain antibodies
[CONT.]
Undefined mechanism
148
Research programme: muscarinic
receptor agonists - Mithridion
Mithridion (formerly Cognitive Pharmaceuticals)
in the US is developing small molecule,
muscarinic receptor agonists for the treatment of
various central nervous system (CNS) disorders,
particularly schizophrenia and associated
cognitive deficits. The muscarinic programme
was originated by the University of Toledo, which
licensed it to Cognitive Pharmaceuticals (later
Mithridion). [CONT.]
147
Research programme: monoclonal
antibodies and antibody-drug
conjugates - Intellect Neurosciences
Intellect Neurosciences is using its proprietary
Antisenilin® passive immunisation technology to
develop monoclonal antibodies that target beta-
amyloid (Abeta) protein for the treatment of
Alzheimer's disease (AD). The company licensed
two Abeta-specific monoclonal antibodies, 82E1
(IN N01) and 1A10, from Immuno-Biological
Laboratories (IBL). [CONT.]
146
Research programme: metal-protein-
attenuating compounds - Prana
Biotechnology
Prana Biotechnology is undertaking a research
programme to develop disease-modifying agents
derived from its proprietary metal-protein-
attenuating compound (MPAC) library for the
treatment of Alzheimer's disease, Parkinson's
disease (PD), and possibly age-related macular
degeneration (AMD). [CONT.]
144
Research programme: membrane
active metal ion chelators - D-Pharm
Chelating agents
145
Research programme: metabotropic
glutamate receptor 5 modulators -
Addex Therapeutics
Addex Therapeutics (formerly Addex
Pharmaceuticals) is evaluating positive and
negative allosteric metabotropic glutamate
receptor-5 (mGluR5) modulators for potential
application in schizophrenia, Alzheimer's disease
and other cognitive disorders. Allosteric
modulators are a novel class of small-molecule,
oral drug candidates with a chemical structure
unrelated to that of competitive agonist or
antagonist drugs [CONT.]
Metabotropic glutamate receptor 5 modulators
143
Research programme:
melanotransferrin conjugated
therapeutics - biOasis Technologies
biOasis Technologies is developing
melanotransferrin (p97; CD228) as a carrier of
therapeutic and imaging agents across the blood-
brain barrier (BBB) for use in various indications,
including brain metastases and Alzheimer's
disease. A melanotransferrin-trastuzumab
(Herceptin®) conjugate, known as BT 2111, is
undergoing preclinical studies for the treatment
of HER2+ brain cancer that has metastasised
from primary breast cancer, in Canada [CONT.]
Liver X receptor modulators
142
Research programme: M1 muscarinic
receptor modulators - Merck and Co.
Merck & Co. is developing selective M1
muscarinic positive allosteric receptor
modulators for the treatment of Alzheimer's
disease. Development of the programme is at the
preclinical stage in the US.
Muscarinic M1 receptor modulators
140
Research programme: leptin -
Neurotez
Neurotez is developing a hormone product
candidate leptin for the treatment of Alzheimer's
disease (AD) and other cognitive disorders.
Leptin is a protein that is derived from adipocyte
hormone and has been linked in pathological
pathways relevant to AD. In particular, it controls
AMP-dependent kinase which regulates glycogen
synthase kinase-3, which then modulates tau
phosphorylation. [CONT.]
Leptin - Neurotez
141
Research programme: liver X
receptor modulators - Vitae
Pharmaceuticals
Vitae Pharmaceuticals is developing small
molecule liver X receptor (LXR) modulators for
the treatment of atherosclerosis and Alzheimer's
disease. Discovery is based on Vitae's
CONTOUR® technology, a structure-based drug
design platform which allows design and
optimisation of compounds within the binding site
of a target protein. [CONT.]
139
Research programme: kinase
inhibitors - Plexxikon
Plexxikon (a Daiichi Sankyo subsidiary) is
developing a portfolio of orally-administered
small-molecule kinase inhibitors. One focus of
this programme appears to be inhibitors of Raf
kinase (proto-oncogene protein b-raf-inhibitors)
as potential therapies for cancer. The programme
is also investigating compounds that target Fms
kinase for the treatment of cancer, Alzheimer's
disease, and inflammatory diseases such as
multiple sclerosis and lupus [CONT.]
NNI-370
PYM 60086 Alzheimer's disease Preclinical PO United Kingdom Phytopharm Originator England Pharmaceutical Public
PYM60086 Cognition disorders Preclinical PO United Kingdom Phytopharm Owner England Pharmaceutical Public
Dementia Preclinical PO United Kingdom
Memory disorders Preclinical PO China
DP 68 Alpha-synuclein inhibitors Alzheimer's disease Preclinical Intranasal USA ProteoTech Originator USA Biotechnology Private
DP 74 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical PO USA ProteoTech Owner USA Biotechnology Private
DP-68 Parkinson's disease Preclinical PO USA
DP-74
PeptiClere™
PTI 777
PTI-777
Synuclere™
TTT 3002 LRRK2 protein inhibitors Alzheimer's disease Preclinical PO USA TauTaTis Originator USA Pharmaceutical Private
TTT-3002 Protein kinase inhibitors Cancer Preclinical PO USA TauTaTis Owner USA Pharmaceutical Private
Tau protein modulators Parkinson's disease Preclinical PO USA
Alzheimer's disease Preclinical Unknown route Sweden BioChromix Pharma Originator Sweden Pharmaceutical Private
BioChromix Pharma Owner Sweden Pharmaceutical Private
Alzheimer's disease Preclinical Unknown route USA Bristol-Myers Squibb Originator USA Large Pharma, Biopharmaceutical Public
Neurodegenerative disorders Preclinical Unknown route USA KineMed Originator USA Biotechnology Private
ORY 2001 Lysine specific demethylase 1 modulators Huntington's disease Preclinical Unknown route Spain Oryzon Originator Spain Biotechnology Private
ORY-2001 Monoamine oxidase B inhibitors Alzheimer's disease Research Unknown route Spain Oryzon Owner Spain Biotechnology Private
Dementia Research Unknown route Spain
Parkinson's disease Research Unknown route Spain
PharmaNeuroBoost N.V. of Belgium is
undertaking a research programme to develop
compounds for the treatment of
neurodegenerative disorders, particularly
Alzheimer's disease (AD) and Parkinson's disease
(PD). Lead candidates are undergoing preclinical
development for AD and PD.
PNB 03 Alzheimer's disease Preclinical Unknown route Belgium PharmaNeuroBoost Originator Belgium Pharmaceutical Private
Two lead candidates to emerge is PNB 03 and
PNB 04, which are undergoing IND-enabling
studies for PD and AD, respectively.
PNB 04 Parkinson's disease Preclinical Unknown route Belgium PharmaNeuroBoost Owner Belgium Pharmaceutical Private
PNB03
PNB04
Caprospinol Acetylcholinesterase inhibitors Alzheimer's disease Preclinical PO Canada Advinus Therapeutics Collaborator India CRO Private
SP 04 Amyloid beta-protein inhibitors Huntington's disease Preclinical PO USA CHDI Collaborator USA Biotechnology Private
SP 04m Sigma-1 receptor antagonists Georgetown University Originator USA University N/A
SP 08 McGill University Originator Canada University N/A
SP 233 Samaritan Pharmaceuticals Originator USA Biopharmaceutical Public
SP-004
SP-008
SP-04
SP-04m
SP-08
SP-233
sNN 0126 Neuron stimulants Alzheimer's disease Preclinical Unknown route Sweden Genentech Technology Provider USA Large Pharma, Biotechnology Public
sNN 0465 Neuropilin-1 inhibitors Amyotrophic lateral sclerosis Preclinical Intracerebro ventricular Sweden NeuroNova AB Originator Sweden Biopharmaceutical Private
sNN0126 Platelet derived growth factor BB modulators Huntington's disease Preclinical Unknown route Sweden NeuroNova AB Owner Sweden Biopharmaceutical Private
sNN0465 Stem cell stimulants Major depressive disorder Preclinical Unknown route Sweden
Neurological disorders Preclinical Intracerebro ventricular Sweden
Neurological disorders Preclinical PO Sweden
Parkinson's disease Preclinical Intracerebro ventricular Sweden
AF 243 Cell differentiation stimulants Hearing loss Preclinical Otic France Affichem Originator France Biotechnology Private
AF 250s Neuron modulators Parkinson's disease Preclinical Unknown route France Affichem Owner France Biotechnology Private
AF243 Alzheimer's disease Research Unknown route France
AF250s Brain injuries Research Unknown route France
Retinal disorders Research Ophthalmic France
Alzheimer's disease Preclinical Unknown route Australia Neurosciences Victoria Originator Australia Technology Transfer Private
Amyotrophic lateral sclerosis Preclinical Unknown route Australia Neurosciences Victoria Owner Australia Technology Transfer Private
Epilepsy Preclinical Unknown route Australia
Multiple sclerosis Preclinical Unknown route Australia
Neurological disorders Preclinical Unknown route Australia
Parkinson's disease Preclinical Unknown route Australia
Stroke Preclinical Unknown route Australia
ORS 1006 DYRK kinase inhibitors Alzheimer's disease Preclinical PO USA Arrien Pharmaceuticals Collaborator USA Pharmaceutical Private
ORS 1104 LRRK2 protein inhibitors Down syndrome Preclinical PO USA Oros Pharmaceuticals Originator India Manufacturer
ORS-1006 Parkinson's disease Preclinical PO USA Oros Pharmaceuticals Owner India Manufacturer
ORS-1104
162
Research programme: neurological
disorders kinase inhibitors - Arrien
Pharmaceuticals
Arrien Pharmaceuticals is developing orally
administered, small molecule inhibitors of protein
kinases for the treatment of Alzheimer's disease
(AD), Parkinson's disease (PD) and Down
syndrome. These compounds were originated by
Oros Pharmaceuticals, and are being developed
with Arrien's proprietary Fragment-Field Drug
Design (FFDD)™ technology platform. [CONT.]
161
Research programme: neurological
disorder therapies - Neurosciences
Victoria
Australia-based Neurosciences Victoria is
conducting research into several neurological
disorders, with the aim of discovering potential
therapeutics. The company's areas of research
include epilepsy, stroke, neurodegenerative
disorders, primarily Alzheimer's disease,
Parkinson's disease and amyotrophic lateral
sclerosis, and glial cell disorders, particularly
multiple sclerosis (MS). [CONT.]
Undefined mechanism
160
Research programme: neurological
disorder therapeutics - Affichem
Affichem is developing therapeutics for disorders
with neurological involvement including; hearing
loss, Parkinson's disease, Alzheimer's disease,
retinal disorders (retinopathy) and medullary
lesions. A lead candidate, AF 243, is undergoing
preclinical development. Early research is also
being conducted on another candidate known as
AF 250s. [CONT.]
159
Research programme: neurogenesis
therapeutics - NeuroNova AB
NeuroNova AB is engaged in the discovery and
development of small molecule drugs that
promote neurogenesis (i.e. the formation of new
neurons) via activity on neural stem and
progenitor cells for the treatment of CNS
disorders, including Parkinson's disease (PD),
Alzheimer's disease (AD), Huntington's disease
(HD), amyotrophic lateral sclerosis (ALS) and
depression. [CONT.]
158
Research programme:
neurodegenerative disorders
therapeutics - Samaritan/McGill
University
The US company Samaritan Pharmaceuticals is
collaborating with McGill University in Canada in
the discovery and development of therapeutics
for the treatment of neurodegenerative disorders
such as Alzheimer's disease (AD) and
Huntington's disease (HD). Certain compounds in
this programme (such as caprospinol) inhibit
beta-amyloid and others (such as SP 04) inhibit
acetylcholinesterase and the sigma-1 receptor
[CONT.]
156
Research programme:
neurodegenerative disorders
therapeutics - Oryzon
Oryzon is developing small molecule therapeutics
for the treatment of neurodegenerative disorders,
such as Alzheimer's disease (AD), Parkinson's
disease (PD), dementia, and Huntington's
disease (HD). The research programme focuses
on targeting lysine specific demethylase 1
(LSD1), which plays a role in regulating the
expression of crucial genes that support neuron
viability. [CONT.]
157
Research programme:
neurodegenerative disorders
therapeutics - PharmaNeuroBoost
154
Research programme:
neurodegenerative disorders
therapeutics - BioChromix Pharma
BioChromix Pharma has a research programme
investigating therapeutics for the treatment of
neurodegenerative disorders. The company's
initial focus is on Alzheimer's disease. Lead
compounds based on proprietary polymer
technology are in preclinical investigation.
155
Research programme:
neurodegenerative disorders
therapeutics - KineMed/Bristol-
Myers Squibb
KineMed and Bristol-Myers Squibb are developing
therapeutics for the treatment of Alzheimer's
disease and other neurodegenerative disorders.
KineMed will utilise their stable isotope
technology to aid in drug discovery. Preclinical
development is ongoing in the US.
Undefined mechanism
153
Research programme:
neurodegenerative disorder
therapeutics - TauTaTis
TauTaTis is undertaking a research programme
to discover and develop a series of proprietary
and orally bioavailable, small molecules as multi-
targeted kinase inhibitors for the treatment of
Alzheimer's disease (AD), Parkinson's disease
(PD) and cancer. Such compounds uniquely
affect various targets, which are relevant for
several disease processes, thereby, offer a
disease-modifying strategy versus one solely for
symptomatic relief [CONT.]
152
Research programme:
neurodegenerative disease
therapeutics - ProteoTech
ProteoTech is developing proprietary, small
molecules for the potential use in
neurodegenerative disorders such as Alzheimer's
disease (AD) and Parkinson's disease (PD). Drug
classes that have emerged from this programme
include small molecules and peptides that reduce
and/or trigger clearance of amyloid-beta (Abeta)
for AD and molecules targeting the formation and
persistence of alpha-synuclein in PD [CONT.]
151
Research programme:
neurodegenerative disease
therapeutics - Phytopharm
UK-based pharmaceutical company Phytopharm
is developing botanical-based pharmaceuticals
for the treatment of dementia including
Alzheimer's disease (programme P58),
Parkinson's disease (programme P63), vascular
dementia and age-related memory impairment.
Additional indications include diabetic neuropathy
and neuropsychiatric disorders, such as
schizophrenia, depression, anxiety and epilepsy.
[CONT.]
Nerve growth factor stimulants
150
Research programme:
neurodegenerative disease
therapeutics - Neuronascent
Neuronascent is developing a series of chemically
diverse neurogenic compounds as disease-
modifying drugs for the treatment of
neurodegenerative diseases including Alzheimer's
disease (AD) and Parkinson's disease. The
neurogenic agents are designed to enable
replacement of damaged neurons in key areas of
the brain through stimulation of neurogenesis
(i.e. nerve cell growth) and enhancement of
neuroprotection [CONT.]
SP sc4 Alzheimer's disease Preclinical Unknown route Canada CHDI Licensee World Biotechnology Private
SP sc7 Huntington's disease Preclinical Unknown route USA Georgetown University Originator USA University N/A
SP-sc4 Neurodegenerative disorders Preclinical Unknown route Canada McGill University Originator Canada University N/A
SP-sc7 Samaritan Pharmaceuticals Originator USA Biopharmaceutical Public
BN 201 TrkA receptor agonists Glaucoma Preclinical Ophthalmic Spain Bionure Originator Spain Biotechnology Private
BN-201 TrkB receptor agonists Multiple sclerosis Preclinical IV Spain Bionure Owner Spain Biotechnology Private
BN201 Multiple sclerosis Preclinical PO Spain
G 79 Optic neuritis Preclinical IV Spain
G-79 Alzheimer's disease Research IV Spain
G79 Amyotrophic lateral sclerosis Research IV Spain
Parkinson's disease Research IV Spain
ADNF-9 Neuron stimulants Alzheimer's disease Preclinical Unknown route Canada Allon Therapeutics Originator Canada Biotechnology Public
AL 209 Tubulin modulators Fetal alcohol syndrome Preclinical Unknown route Canada Paladin Labs Owner Canada Pharmaceutical Public
AL 309 Neuroprotection Preclinical PO USA
AL 408 Peripheral nervous system diseases Preclinical PO Canada
AL 508 Peripheral nervous system diseases Preclinical SC Canada
AL-209 Peripheral nervous system diseases (Chemotherapy-
induced:oxaliplatin induced peripheral neuropathy)
Preclinical PO France
AL-309 Eye disorders No development reported(Preclinical) Unknown route Germany, Israel
AL-408
AL-508
SAL
SALLRSIPA
Alzheimer's disease Preclinical Intranasal Italy Lay Line Genomics Originator Italy Biopharmaceutical Private
Lay Line Genomics Owner Italy Biopharmaceutical Private
NRX 1050 Alzheimer's disease Preclinical PO USA Naurex Originator USA Biopharmaceutical Private
NRX 1051 Autistic disorder Preclinical PO USA Naurex Owner USA Biopharmaceutical Private
NRX 1059 Major depressive disorder Preclinical IV USA
NRX 105x Major depressive disorder Preclinical PO USA
NRX 1060 Neuropathic pain Preclinical PO USA
NRX 2085 Schizophrenia Preclinical IV USA
NRX 20xx Schizophrenia Preclinical PO USA
NRX-1050
NRX-1051
NRX-1059
NRX-105x
NRX-1060
NRX-2085
NRX-20xx
AD Inov collaborative programme Cytoplasmic and nuclear receptor modulators Alzheimer's disease Preclinical Unknown route France Genfit Originator France Biopharmaceutical Public
G-15750 Nuclear receptor subfamily 1 group F member 3
antagonists
Cardiovascular disorders Preclinical Unknown route France Genfit Owner France Biopharmaceutical Public
G15750 Peroxisome proliferator-activated receptor
modulators
Metabolic disorders Preclinical Unknown route France
TGFTX1 Autoimmune disorders Research Unknown route France
TGFTX3
RAP 103 CCR2 receptor antagonists Alzheimer's disease Preclinical PO USA McGill University Collaborator Canada University N/A
RAP 160 CCR3 receptor antagonists Hepatitis B Preclinical PO Germany, USA RAPID Pharmaceuticals Originator Switzerland Biotechnology Private
RAP 163 CCR5 receptor antagonists Neuropathic pain Preclinical PO Canada, USA RAPID Pharmaceuticals Owner Switzerland Biotechnology Private
RAP 164 Chemokine receptor antagonists Prostate cancer Preclinical PO USA University of Duisburg-Essen Collaborator Germany University N/A
RAP 310 Chemokine receptor modulators Psoriasis Preclinical PO USA University of Michigan Collaborator USA University N/A
RAP 701 Immunostimulants Ovarian cancer Research PO USA
RAP103 Stem cell inhibitors
RAP160
RAP163
RAP164
RAP310
RAP701
[18F]AppCHFppA Atherosclerosis (Diagnosis) Preclinical Parenteral USA FluoroPharma Licensee USA Biopharmaceutical Private
AZPET Coronary disorders (Diagnosis) Preclinical Parenteral USA Harvard Medical School Originator USA University N/A
VasoPET Alzheimer's disease (Diagnosis) Research Parenteral USA Massachusetts General Hospital Originator USA Hospital N/A
GSK3787 Alzheimer's disease Preclinical PO USA GlaxoSmithKline Originator England Large Pharma, Pharmaceutical Public
GW 0742 Cancer Preclinical Unknown route USA GlaxoSmithKline Owner England Large Pharma, Pharmaceutical Public
GW0742 Diabetes mellitus Preclinical Unknown route USA
PPAR agonists research programme -
GlaxoSmithKline
Lipid metabolism disorders No development reported(Preclinical) Unknown route USA
PPARpan Metabolic syndrome No development reported(Preclinical) Unknown route USA
PPARpan™
Research programme: PPARpan agonists -
GlaxoSmithKlineND 602 Alzheimer's disease Preclinical Unknown route Canada Neurodyn Originator Canada Biotechnology
ND-602 Amyotrophic lateral sclerosis Preclinical Unknown route Canada Neurodyn Owner Canada Biotechnology
Parkinson's disease Preclinical Unknown route Canada
Spinal muscular atrophy Preclinical Unknown route Canada
NPT 001 Alpha-synuclein inhibitors Alzheimer's disease Preclinical Parenteral USA NeuroPhage Pharmaceuticals Originator USA Biotechnology
NPT 002 - NeuroPhage Amyloid beta-protein inhibitors Parkinson's disease Preclinical Unknown route USA NeuroPhage Pharmaceuticals Owner USA Biotechnology
NPT-001 Tau protein inhibitors
NPT-002 - NeuroPhage
NPT001
NPT002 - NeuroPhage
173
Research programme: protein
aggregation inhibitors - NeuroPhage
Pharmaceuticals
NeuroPhage Pharmaceuticals is developing
therapeutics for the treatment of
neurodegenerative disease, including Alzheimer's
disease and Parkinson's disease. The company is
using its proprietary protein disaggregation
technology targeting both beta-amyloid (Abeta)
and tau proteins as a disease-modifying
approach in the treatment of Alzheimer's disease.
[CONT.]
172
Research programme: progranulin
stimulants - Neurodyn
Neurodyn is developing a neuroprotectant, called
ND 602, for the treatment of Parkinson's disease
(PD), Alzheimer's disease (AD), amyotrophic
lateral sclerosis (ALS) and spinal muscular
atrophy (SMA). ND 602 is designed to target
synthesis of progranulin, a multifunction protein
expressed by neurons and microglia. Progranulin
is thought to be involved in neuronal survival and
CNS inflammatory processes [CONT.]
Nerve tissue protein stimulants
170
Research programme: PET imaging
agents - FluoroPharma
FluoroPharma is developing positron emission
tomography (PET) imaging agents for use in the
diagnosis of atherosclerosis, coronary disorders
and Alzheimer's disease. The imaging agents
under evaluation consist of molecules labelled
with the 18F radioactive isotope. One lead
preclinical candidate, VasoPET or p2,p3-
[18F]monofluoromethylene diadenosine-5',5'''-
p1,p4-tetraphosphate ([18F]AppCHFppA), is a
purine analogue that targets active adenosine
phosphate molecule receptors, which are
associated with inflammatory conditions [CONT.]
Positron-emission tomography enhancers
171
Research programme: PPAR
modulators - GlaxoSmithKline
GlaxoSmithKline is developing modulators for the
three known peroxisome proliferator-activated
receptor (PPAR) subtypes alpha, gamma and
delta, for the potential treatment of cancer, type
2 diabetes and Alzheimer's disease (AD).
Preclinical studies of a series of compounds are
underway in the US. A PPARdelta agonist, GW
0742, has been tested in a mice model of AD. A
PPAR beta/delta antagonist, GSK 3787, has also
been evaluated in the treatment of cancer.
[CONT.]
Peroxisome proliferator-activated receptor
modulators
169
Research programme: peptide-based
therapeutics - RAPID
Pharmaceuticals
RAPID Pharmaceuticals is developing small
stable peptides for the treatment of Alzheimer's
disease (RAP 310), chronic hepatitis B (RAP
164), neuropathic pain (RAP 103), ovarian
cancer (RAP 701), prostate cancer (RAP 163)
and psoriasis (RAP 160). The company has
stated that these compounds, which target
specific chemokine receptors, represent a fourth
generation of peptide development. [CONT.]
168
Research programme: nuclear
receptor-targeting therapeutics -
Genfit
Genfit is developing compounds that target
various nuclear receptors for the treatment of
Alzheimer's disease, cardiometabolic disorders
and autoimmune disorders. Drug candidates
include G 15750, a selective peroxisome
proliferator-activated receptor modulator. Genfit
has two programmes, TGFTX1 and TGFTX3,
which target nuclear receptors that are involved
in the circadian rhythm (Rev-Erbalpha and
RORalpha/RORgamma) [CONT.]
166
Research programme: NGF-based
Alzheimer's disease therapy - Lay
Line Genomics
Lay Line Genomics is developing proprietary
formulations of nerve growth factor (NGF)
coupled to a diagnostic system for the non-
invasive treatment of Alzheimer's disease. An
intranasal formulation of NGF is undergoing proof-
of-principle, preclinical studies in Italy. [CONT.]
Neuron stimulants
167
Research programme: NMDA
receptor modulators - Naurex
Naurex is developing peptides and synthetic
small molecules as modulators of the N-methyl-D-
aspartate (NMDA) receptor for the treatment of
major depressive disorder, schizophrenia,
Alzheimer's disease, neuropathic pain and
autistic disorder. In preclinical trials, NMDA
modulators have exhibited therapeutic activity
without the limiting side effects, such as
dissociative effects and sedation, commonly
observed with other compounds targeting the
NMDA receptor [CONT.]
NMDA receptor modulators
165
Research programme:
neuroprotective therapies - Allon
Therapeutics
Allon Therapeutics (a subsidiary of Paladin Labs)
has a research programme aimed at developing
neuroprotective compounds derived from its two
technology platforms, Activity-Dependent
Neurotrophic Factor (ADNF) and Activity-
Dependent Neuroprotective Protein (ADNP), for
the treatment of degenerative diseases and
injuries. [CONT.]
164
Research programme:
neuroprotectants - Bionure
Bionure is developing neuroprotectants for the
treatment of neurological and ophthalmological
indications, including multiple sclerosis,
Parkinson's disease, Alzheimer's disease,
glaucoma and acute optic neuritis. A lead
candidate from this programme is BN 201 (also
known as G 79), a first-in-class peptoid
neuroprotectant that works by binding to the
extracellular portion of TrkA and TrkB and
activating the neurotrophin pathway [CONT.]
163
Research programme: neuronal stem
cell therapeutics - Samaritan/McGill
University
Samaritan Pharmaceuticals in the US is
collaborating with McGill University in Canada in
the discovery and development of small molecule
compounds that induce neural stem cell
differentiation, as potential treatments of various
neurodegenerative disorders. [CONT.]
Stem cell stimulants
Alpha-synuclein inhibitors Alzheimer's disease Preclinical Parenteral Ireland Neotope Biosciences Originator United Kingdom Biopharmaceutical Public
Amyloid inhibitors Amyloidosis Preclinical Parenteral Ireland Prothena Corporation Owner Ireland Biotechnology Public
Protein aggregation inhibitors Neurodegenerative disorders Preclinical Parenteral Ireland
Tau protein inhibitors Parkinson's disease Preclinical Parenteral Ireland
SIG 1012 Alzheimer's disease Preclinical PO USA GlaxoSmithKline (China) Investment Co. Licensee China Pharmaceutical Public
SIG 1106 Parkinson's disease Research PO USA Princeton University Technology Provider USA University N/A
SIG1012 Type 2 diabetes mellitus Research PO USA Signum Biosciences Originator USA Biotechnology Private
SIG1033 Signum Biosciences Owner USA Biotechnology Private
SIG1106
A 992400 Alzheimer's disease Preclinical Parenteral Germany Abbott Laboratories Originator USA Large Pharma, Pharmaceutical Public
A 992401 AbbVie Owner USA Biopharmaceutical Public
A-992400 CSL Behring Originator USA Biopharmaceutical Public
A-992401 CSL Behring Owner USA Biopharmaceutical Public
EHT 0101 Amyloid beta-protein inhibitors Alzheimer's disease Preclinical Unknown route France ExonHit Therapeutics Originator France Technology provider Public
EHT 0206 GTP phosphohydrolase inhibitors Cancer Preclinical Unknown route France ExonHit Therapeutics Owner France Technology provider Public
EHT 101 Ras signal transduction pathway inhibitors Chronic myeloid leukaemia Preclinical Unknown route France
EHT 1864 Rho GTP-binding protein-inhibitors
EHT 206
Acetylcholinesterase inhibitors Alzheimer's disease Preclinical Transdermal South Korea Samyang Biopharmaceuticals Corporation Originator South Korea Pharmaceutical
Butyrylcholinesterase inhibitors Samyang Group Originator South Korea Pharmaceutical Public
Merck & Co is developing orally bioavailable S-
adenosylhomocysteine hydrolase (AHCY)
inhibitors for the treatment of Alzheimer's
disease. Development is at the preclinical stage
in the US.
L 002259713 Alzheimer's disease Preclinical PO USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
Homocysteine is produced by the conversion of S-
adenosylhomocysteine via the AHCY cytosolic
enzyme to homocysteine and adenosine. [CONT.]
L-002259713 Merck & Co Owner USA Large Pharma, Pharmaceutical Public
SUVN-F90101 Alzheimer's disease Preclinical Unknown route India Suven Life Sciences Originator India Company
SUVN-F91201 Neuropathic pain Preclinical Unknown route India Suven Life Sciences Owner India Company
Alzheimer's disease Preclinical Unknown route USA Abide Therapeutics Originator USA Biotechnology Private
Down syndrome Preclinical Unknown route USA University of California, San Diego Originator USA University N/A
SUVN D1003019 Alzheimer's disease Preclinical PO India Suven Life Sciences Originator India Company
SUVN-D1003019 Suven Life Sciences Owner India Company
A 964324 Alzheimer's disease Preclinical Unknown route USA Abbott Laboratories Originator USA Large Pharma, Pharmaceutical Public
A-964324 Schizophrenia Preclinical Unknown route USA AbbVie Owner USA Biopharmaceutical Public
A964324
SUVN-501 Alzheimer's disease Preclinical PO India Suven Life Sciences Originator India Company
SUVN-504 Cognition disorders Preclinical PO India Suven Life Sciences Owner India Company
SUVN-507 Obesity Preclinical PO India
SUVN-623 Parkinson's disease Preclinical PO India
SUVN-901 Schizophrenia Preclinical PO India
SUVN-976
321348 Alzheimer's disease Preclinical PO USA Pfizer Owner USA Large Pharma, Pharmaceutical Public
5-HT6 receptor antagonists - Wyeth Wyeth Originator USA Large Pharma, Pharmaceutical Public
WAY 101
WAY-101
185
Research programme: serotonin 6
receptor antagonists - Wyeth
Wyeth (Pfizer) is developing serotonin 6 (5-HT6)
receptor antagonists for the oral treatment of
cognitive impairment in neurodegenerative
diseases such as Alzheimer's disease. Serotonin
6 receptor antagonists may improve deficits in
learning and memory by increasing levels of the
neurotransmitters acetylcholine and glutamate. A
series of arylsulfonyl compounds has been
developed with optimised pharmacodynamic
properties [CONT.]
Serotonin 6 receptor antagonists
184
Research programme: serotonin 6
receptor antagonists - Suven Life
Sciences
Suven Life Sciences is developing orally active,
selective serotonin 6 (5-HT6) receptor
antagonists, including tetrahydro carbazole
derivatives, for the treatment of various cognition
disorders such as Alzheimer's disease and
schizophrenia, as well as obesity. SUVN 501,
SUVN 504, SUVN 507 (and its active metabolite,
M1), SUVN 901 and SUVN 976 are in preclinical
development in India. [CONT.]
Serotonin 6 receptor antagonists
183
Research programme: serotonin 6
receptor antagonists - AbbVie
AbbVie (formerly Abbott laboratories) is
researching serotonin-6 (5HT6) receptor
antagonists for their potential in the treatment of
cognitive deficits in Alzheimer's disease and
schizophrenia in the US. Lead candidate A
964324 is a highly selective serotonin-6 receptor
antagonist in in vitro and in vivo studies.
Preclinical development is ongoing in the US.
[CONT.]
Serotonin 6 receptor antagonists
181
Research programme: serine
hydrolase inhibitors - Abide
Therapeutics/University of California
Abide Therapeutics and the University of
California, San Diego are collaborating on the
development of small-molecule inhibitors of
monoacylglycerol lipase (MAGL), a serine
hydrolase, as potential therapies for Down
syndrome and Alzheimer's disease. The
compounds were created by application of Abide
Therapeutics' selective targeting technology.
Initially, the researchers will assess the agents in
an animal model of Down syndrome. [CONT.]
Hydrolase inhibitors
182
Research programme: serotonin 4
receptor agonists - Suven Life
Sciences
Suven Life Sciences is developing orally
bioavailable, selective serotonin 4 receptor
(5HT4) agonists for the treatment of Alzheimer's
disease. 5-HT4 receptor is expressed at a high
density in the limbic system including the
hippocampus and frontal cortex, suggesting a
role of this sub-type of 5-HT receptors in
conditions associated with memory and cognition
impairments. [CONT.]
Serotonin 4 receptor agonists
179
Research programme: S-
adenosylhomocysteine hydrolase
inhibitors - Merck & Co
Adenosylhomocysteinase inhibitors
180
Research programme: selective
nicotinic acetylcholine receptor
agonists - Suven Life Sciences
Suven Life Sciences is developing potent and
selective nicotinic acetylcholine alpha4beta2
receptor agonists for the treatment of
neurological disorders. SUVN F91201 has been
identified as a lead candidate for the treatment of
Alzheimer's disease, and SUVN F90101 is the
lead candidate for the treatment of neuropathic
pain. Preclinical development is underway in
India.
Nicotinic receptor agonists
178
Research programme: rivastigmine
transdermal - Samyang
A transdermal formulation of rivastigmine is
being developed by Samyang Biopharmaceuticals
Corporation (a division of Samyang Group) for
the treatment of Alzheimer's disease. The
transdermal delivery technology applied by the
company is designed to provide extended-release
of drug into the patient's system, and holds the
advantage of bypassing first pass metabolism.
[CONT.]
176
Research programme: RAGE
monoclonal antibodies: AbbVie/CSL
Behring
AbbVie and CSL Behring are developing
monoclonal antibodies (mABs) to receptor for
advanced glycation-end-products (RAGE) for the
treatment of Alzheimer's disease (AD). RAGE is
thought to be a receptor for amyloid-beta
(Abeta) and other ligands such as S100B,
potentially contributing to AD disease
progression. It is overexpressed in the brain
microvasculature of patients with AD. [CONT.]
Advanced glycosylation end-product receptor
antagonists
177
Research programme: Rho GTP-
binding protein inhibitors - ExonHit
Therapeutics
ExonHit Therapeutics is utilising its Differential
Analysis of Transcripts with Alternative Splicing
(DATAS™) technology to develop small molecule
inhibitors of rho GTP-binding proteins (GTPases)
for the treatment of cancer and Alzheimer's
disease. Rho GTP-binding proteins are involved in
regulation of actin organisation, gene expression
and cell cycle expression. Preclinical studies are
being conducted by ExonHit in France. [CONT.]
175
Research programme: protein
phosphatase 2A modulators -
GlaxoSmithKline/Signum
Biosciences
Signum Biosciences is using its proprietary
Phosphatase plaform to develop small molecule,
orally active protein phosphatase 2A (PP2A)
modulators for the treatment of Alzheimer's
disease, Parkinson's disease, and type 2 diabetes
mellitus. GlaxoSmithKline (China) is collaborating
with Signum on the research and development in
Alzheimer's disease. Development is at the
preclinical stage in the US.
Protein phosphatase 2A modulators
174
Research programme: protein
aggregation inhibitors - Prothena
Corporation
Neotope Biosciences (a wholly-owned subsidiary
of Elan Corporation) initiated the development of
monoclonal antibodies that modulate neo-epitope
amyloid related targets, alpha synuclein and tau
protein. These compounds could potentially be
used for the treatment of a range of
neurodegenerative disorders including primary
(AL) amyloidosis, Lewy body dementia,
Parkinson's disease and Alzheimer's disease
[CONT.]
NEOD002
Alzheimer's disease Preclinical Unknown route Scotland Antoxis Originator United Kingdom Biotechnology
Cancer Preclinical Unknown route Scotland Antoxis Owner United Kingdom Biotechnology
Cardiovascular disorders Preclinical Unknown route Scotland
Diabetes mellitus Preclinical Unknown route Scotland
Parkinson's disease Preclinical Unknown route Scotland
Cyclin dependent kinase 9 inhibitors Alzheimer's disease Preclinical PO Germany Evotec AG Collaborator Germany Pharmaceutical Public
Glutaminyl-peptide cyclotransferase inhibitors Inflammation Preclinical PO Germany Probiodrug Originator Germany Pharmaceutical Private
Probiodrug Owner Germany Pharmaceutical Private
TauRx Pharmaceuticals of Singapore is
undertaking a research programme to develop
orally available, second-generation inhibitors of
tau protein for the treatment of Alzheimer's
disease (AD). The programme is in preclinical
development.
Alzheimer's disease Preclinical PO Singapore TauRx Pharmaceuticals Originator Singapore Pharmaceutical
TauRx is pursuing commercialisation, licensing
and/or partnering opportunities for its
programme in neurodegenerative disorders,
particularly AD. [CONT.]
TauRx Pharmaceuticals Owner Singapore Pharmaceutical
HL 152B AMP activated protein kinase stimulants Age-related macular degeneration Preclinical Ophthalmic South Korea HanAll Biopharma Originator South Korea Pharmaceutical Private
HL 156Can Cell adhesion molecule inhibitors Autoimmune disorders Preclinical IV South Korea Korea Research Institute of Chemical Technology Originator South Korea Institution N/A
HL004 Emt protein-tyrosine kinase inhibitors Cancer Preclinical PO South Korea
HL010 Fc receptor antagonists Dry eyes Preclinical Ophthalmic South Korea
HL011 Gluconeogenesis inhibitors Hypertension Preclinical PO South Korea, USA
HL020 Immunomodulators Inflammatory bowel diseases Preclinical PO South Korea
HL021 Tumour necrosis factor alpha inhibitors Uveitis Preclinical Ophthalmic South Korea
HL022 Alzheimer's disease Research PO South Korea
HL023 Angina pectoris Research PO South Korea
HL024 Asthma Research Unknown route South Korea
HL025 Atherosclerosis Research PO South Korea
HL026 Atopic dermatitis Research Unknown route South Korea
HL027 Diabetes mellitus Research PO South Korea
HL030 Obesity Research PO South Korea
HL034 (oral TPO agent) - HanAll Parkinson's disease Research PO South Korea
HL036 (anti-TNF-alpha protein) Thrombocytopenia Research PO South Korea
HL038
HL039
HL051
HL076
HL102
HL130
HL152B
HL156
HL156Can
HL157 (oral monoclonal antibody) - HanAll
BiopharmaHL160
metformin butyrate - HanAll BioPharma
Second-generation metformin products - HanAll
BiopharmaTissue protection technology research
programme - Warren Pharmaceuticals/Lundbeck
Alzheimer's disease Preclinical Unknown route USA Asterand plc Technology Provider USA Pharmaceutical Public
Tissue-protective cytokines Neuroprotection Preclinical Unknown route USA Lundbeck A/S Collaborator World Large Pharma, Pharmaceutical Public
Warren Pharmaceuticals Originator USA Biotechnology Private
Warren Pharmaceuticals Owner USA Biotechnology Private
hUCB stem cell therapies - Saneron CCEL
Therapeutics
Amyloid beta-protein inhibitors Alzheimer's disease Preclinical IV USA Cryo-Cell International Funder USA Technology provider Public
Human umbilical cord blood stem cell therapies -
Saneron CCEL Therapeutics
Cell replacements Amyotrophic lateral sclerosis Preclinical IV USA Saneron CCEL Therapeutics Originator USA Biotechnology Private
U-CORD-CELL™ Enzyme stimulants Mucopolysaccharidosis III Preclinical IV USA Saneron CCEL Therapeutics Owner USA Biotechnology Private
Nerve growth factor stimulants Myocardial infarction Preclinical IV USA University of Minnesota Technology Provider USA University N/A
Stroke Preclinical IV USA University of South Florida Collaborator USA University N/A
Allergy vaccine - VLP Biotech Immunomodulators Hepatitis C Preclinical Unknown route USA VLP Biotech Originator USA Biotechnology Private
Alzheimer's disease vaccine - VLP Biotech Immunostimulants Influenza virus infections Preclinical Unknown route USA VLP Biotech Owner USA Biotechnology Private
Atherosclerosis vaccine - VLP Biotech Malaria Preclinical Unknown route USA
Cancer vaccine - VLP Biotech Alzheimer's disease Research Unknown route USA
Hepatitis C virus vaccine - VLP Biotech Atherosclerosis Research Unknown route USA
Hypersensitivity vaccine - VLP Biotech Cancer Research Unknown route USA
Influenza A virus vaccine - VLP Biotech Hypersensitivity Research Unknown route USA
Lipid disorder vaccine - VLP Biotech Lipid metabolism disorders Research Unknown route USA
Malaria vaccine - VLP Biotech Obesity Research Unknown route USA
Obesity vaccine - VLP Biotech
Immunostimulants Cancer Preclinical Unknown route USA Idera Pharmaceuticals Collaborator USA Biotechnology Public
Toll-like receptor 7 agonists Alzheimer's disease Research Unknown route USA Merck & Co Originator USA Large Pharma, Pharmaceutical Public
Toll-like receptor 8 agonists Bacterial infections Research Unknown route USA Merck & Co Owner USA Large Pharma, Pharmaceutical Public
Toll-like receptor 9 agonists Viral infections Research Unknown route USA
Alzheimer's disease Preclinical Unknown route United Kingdom, USA Roche Originator Switzerland Large Pharma, Pharmaceutical Public
Roche Owner Switzerland Large Pharma, Pharmaceutical Public
NK 002 Alzheimer's disease Research Unknown route Canada Neurokine Pharmaceuticals Originator Canada Pharmaceutical Public
NK-002 Neurokine Pharmaceuticals Owner Canada Pharmaceutical Public
194
RO 638695 Scientists at the University College of London are
collaborating with Roche in the development of
RO 638695 for the treatment of Alzheimer's
disease. RO 638695 inhibits the binding of a
blood protein called serum amyloid P (SAP)
component to amyloid fibrils. The compound is
currently undergoing preclinical trials.
CPHPC
195
Etanercept liposomal - Neurokine
Pharmaceuticals
Neurokine Pharmaceuticals in Canada is
developing NK 002 (liposomal encapsulated
etanercept) for the treatment of Alzheimer's
disease. The company expects to complete large
scale preparation and optimization of liposomal
etanercept and complete trials to assess blood
brain barrier penetration by August 2011.
Tumour necrosis factor inhibitors
193
Research programme: vaccines
containing toll-like receptor agonists
- Idera/Merck
Idera Pharmaceuticals and Merck are
collaborating on the development of vaccines
containing agonists of toll-like receptors (TLRs)
7, 8 and 9 for the treatment and prevention of
cancer, infectious diseases and Alzheimer's
disease. Preclinical development is underway for
cancer, while early research activities are in
progress for infectious diseases and Alzheimer's
disease, in the US. [CONT.]
191
Research programme: umbilical cord
blood stem cell therapies - Saneron
CCEL Therapeutics
Saneron CCEL Therapeutics is collaborating with
the University of South Florida to develop human
umbilical cord blood (hUCB) stem cell therapies
based on the U-CORD-CELL™ technology
platform. The programme is focused on the early
intervention and treatment of several
neurodegenerative, cardiovascular and metabolic
disorders that lack suitable treatment options.
[CONT.]
192
Research programme: vaccines - VLP
Biotech
VLP Biotech is focusing on developing a universal
vaccine against influenza A virus strains, as well
as vaccines against malaria, hepatitis C virus,
cancer, lipid disorders (atherosclerosis and
obesity), Alzheimer's disease and various
hypersensitivities. VLP Biotech's vaccine platform
is based on virus-like particles (VLP). [CONT.]
190
Research programme: tissue
protection technology -
Lundbeck/Warren Pharmaceuticals
Warren Pharmaceuticals, is using its tissue
protection technology to develop haemopoietic
cytokines which cross the blood brain barrier, as
potential therapeutics for the treatment of tissue
injury associated with conditions such as brain
and spinal cord injuries, Alzheimer's Disease and
other neurological disorders. H. Lundbeck A/S is
collaborating with Warren in the development of
therapeutics in these neurological and pyschiatric
areas [CONT.]
189
Research programme: therapeutic
agents - HanAll Biopharma
HanAll Biopharma is a Korean-based company
that is developing therapeutic agents (oral
biopharmaceuticals and/or small molecules) for
treatment of major diseases of global
importance, such as diabetes, obesity,
cardiovascular indications (hypertension,
hyperlipidaemia, atherosclerosis, angina),
opthalmic disorders, autoimmune disorders, and
cancer. [CONT.]
187
Research programme: small
molecule therapeutics - Probiodrug
Probiodrug AG is engaged in a research
programme focussed on the identification of
orally active compounds for selectively modifying
enzymatic activity, as potential therapeutics for
inflammation and Alzheimer's disease. The main
targets will be glutaminyl cyclase (QC) and cyclin-
dependent kinase 9 (CDK9). QC is a
metalloenzyme involved in peptide hormone final
maturation and inhibition of QC may provide a
strategy in the treatment of Alzheimer's disease
[CONT.]
188
Research programme: tau protein
inhibitors - TauRx Pharmaceuticals
Tau protein inhibitors
186
Research programme: small
molecule therapeutics - Antoxis
Antoxis, an Aberdeen-based biotechnology
company, is developing small molecule flavonoid
therapeutics for the treatment of
neurodegenerative disorders, such as Parkinson's
and Alzheimer's diseases, cardiovascular
disorders, diabetes, and certain cancers.
Flavonoids are polyphenolic phytochemicals that
are found in a variety of foods and beverages.
[CONT.]
Free radical modulators
Alzheimer's disease (Diagnosis) Research Unknown route USA Bristol-Myers Squibb Originator USA Large Pharma, Biopharmaceutical Public
Bristol-Myers Squibb Owner USA Large Pharma, Biopharmaceutical Public
Alzheimer's disease (Diagnosis) Research Unknown route USA GE Healthcare Originator United Kingdom Technology provider Public
Janssen Pharmaceutica Originator Belgium Pharmaceutical Public
Alzheimer's disease Research IM USA Ichor Medical Systems Originator USA Technology provider Private
Ichor Medical Systems Technology Provider USA Technology provider Private
Institute for Molecular Medicine Originator USA Institution N/A
National Institute of Neurological Disorders and Stroke Funder USA Institution N/A
University of California at Irvine Collaborator USA University N/A
Amyloid beta-protein inhibitors Alzheimer's disease Research Parenteral Canada Cangene Corporation Originator Canada Biopharmaceutical Public
Immunomodulators Cangene Corporation Owner Canada Biopharmaceutical Public
University of British Columbia Technology Provider Canada University N/A
Alzheimer's disease Research Unknown route USA ADispell Originator USA Biotechnology Private
ADispell Owner USA Biotechnology Private
Cornell University Technology Provider USA University N/A
Alzheimer's disease Research Unknown route Japan Astellas Pharma Originator Japan Large Pharma, Pharmaceutical Public
RIKEN Originator Japan Institution N/A
Alzheimer's disease Research Unknown route USA AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
Penn State College of Medicine Originator USA University N/A
Alzheimer's disease Research Unknown route USA AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
The Feinstein Institute for Medical Research Collaborator USA Institution N/A
University of British Columbia Collaborator Canada University N/A
Washington University School of Medicine Collaborator USA University N/A
Weill Cornell Medical College Originator USA University N/A
Axerion Therapeutics and MedImmune, the global
biologics arm of AstraZeneca, are collaborating
to develop small and large molecules for the
treatment of Alzheimer's disease. Development is
at the research stage in the US.
Alzheimer's disease Research Unknown route USA Axerion Therapeutics Licensee USA Biotechnology Private
Both companies will work together to develop
compounds that directly block the amyloid-beta
(Abeta) oligomer binding to cellular prion
proteins in the brain. [CONT.]
MedImmune Sub-licensee USA Biotechnology Public
Yale University Originator USA University N/A
Yale University Owner USA University N/A
Alzheimer's disease Research Unknown route Italy Alzheimers Drug Discovery Foundation Collaborator USA Company
Axxam Originator Italy Biotechnology
Axxam Owner Italy Biotechnology
Alzheimer's disease Research Parenteral Sweden MIVAC Development Originator Sweden Biopharmaceutical Private
MIVAC Development Owner Sweden Biopharmaceutical Private
Alzheimer's disease Research Unknown route USA OLIGOMERIX Originator USA Biopharmaceutical Private
OLIGOMERIX Owner USA Biopharmaceutical Private
Alzheimer's disease Research Unknown route France BioSystems International Originator France Company
INSERM Originator France Institution N/A
208
Research programme: Alzheimer's
disease therapeutics -
Pharnext/Biosystems
Pharnext, Biosystems International, the
University of Bordeaux Segalen and Inserm are
collaborating on a 6-year project to develop
therapeutics and diagnostics for Alzheimer's
disease. The project, known as DIPPAL, will be
lead by Pharnext and will involve development of
Pleodrugs™; cocktails of low-dose, off-patent
drugs which have been approved for other
diseases. [CONT.]
Undefined mechanism
206
Research programme: Alzheimer's
disease therapeutics - MIVAC
Development
MIVAC Development in Sweden is developing
monoclonal antibodies for the potential treatment
of Alzheimer's disease. The company's research
is based around its discovery of methods to
engineer preparations of stabilised, highly-toxic
oligomeric amyloid-beta (Abeta). Soluble
oligomeric aggregates of the Abeta peptide are
thought to be involved in the pathogenesis of
Alzheimer's disease. [CONT.]
Amyloid beta-protein modulators
207
Research programme: Alzheimer's
disease therapeutics - Oligomerix
Oligomerix is developing small molecules and
antibodies that target soluble aggregates of tau
protein as a disease-modifying treatment option
for Alzheimer's disease (AD). Development is
focussed on compounds that inhibit tau oligomer
formation, disrupt tau oligomers or inhibit tau-
mediated neurotoxicity. [CONT.]
Tau protein inhibitors
204
Research programme: Alzheimer's
disease therapeutics - Axerion
Therapeutics/ MedImmune
205
Research programme: Alzheimer's
disease therapeutics -
Axxam/Alzheimer's Drug Discovery
Foundation
Axxam in Italy and a US-based public charity,
the Alzheimer's Drug Discovery Foundation, are
collaborating on the development of small
molecule compounds that block the purinergic
receptor P2Z (also known as P2X7) as potential
therapies for Alzheimer's disease. Axxam is to
conduct the drug discovery and development and
ADDF is contributing financially to the
programme. Development is at the research
stage in Italy. [CONT.]
Purinoceptor P2Z antagonists
202
Research programme: Alzheimer's
disease therapeutics - AstraZeneca
AstraZeneca and the University of Pennsylvania
School of Medicine are collaborating on the
development of novel therapeutics for the
potential treatment of Alzheimer's disease (AD).
The research will focus on the tau-mediated
neurodegeneration in AD. Early research is
underway in the US.
203
Research programme: Alzheimer's
disease therapeutics -
AstraZeneca/Weill Cornell Medical
College
AstraZeneca and academic research laboratories,
led by Weill Cornell Medical College, are
collaborating to assess the role of the
apolipoprotein E4 genotype (ApoE) in the
development of Alzheimer's disease and to
discover therapeutics against this target.
Development is at the research stage in the US.
Apolipoprotein E modulators
201
Research programme: Alzheimer's
disease therapeutics - Astellas
Pharma/RIKEN
Astellas Pharma and RIKEN are collaborating on
the discovery and development of therapies for
Alzheimer's disease. The companies will first
identify candidate molecular targets for drug
therapy by analysis of animal models of
Alzheimer's disease and samples from patients
with the disease. The involvement of such
candidate targets in the pathogenic mechanism
of Alzheimer's disease will then be evaluated, and
their potential as targets for drug discovery will
be validated [CONT.]
Undefined mechanism
200
Research programme: Alzheimer's
disease therapeutics - ADispell
ADispell is developing small molecule,
neuroprotective drug candidates that target a
novel site on the nicotinic acetylcholine receptor
(nAChR) for the treatment of Alzheimer's
disease. The company licensed a proprietary
technology from Cornell University that acts on
brain neurons to treat and prevent cognition loss
caused by Alzheimer's disease and other
cognition disorders. [CONT.]
Nicotinic receptor modulators
198
Research programme: Alzheimer's
disease DNA-vaccine - Ichor Medical
Systems
Ichor Medical Systems and the Institute for
Molecular Medicine, in the US, are developing an
intramuscular DNA-vaccine for the treatment of
Alzheimer's disease. DNA vaccine encoding
proteins, which are implicated in the development
of Alzheimer's disease, will be delivered using
Ichor's proprietary TriGrid™ electroporation
technology. This vaccine is undergoing
development in the US. [CONT.]
Immunostimulants
199
Research programme: Alzheimer's
disease immunotherapy -
Cangene/University of British
Columbia
Cangene Corporation and the University of British
Columbia (UBC) are developing immune-based
therapies for the treatment of Alzheimer's
disease. Research led by an investigator at UBC
succeeded in targeting a specific epitope of
amyloid beta (Abeta) oligomers that is not
present in regular Abeta molecules. [CONT.]
196
Research programme: Alzheimer's
disease diagnostic agents - Bristol-
Myers Squibb/Meso Scale Discovery
Bristol-Myers Squibb and Meso Scale Discovery
are collaborating in the development of
diagnostic assays for Alzheimer's disease (AD).
The diagnostic assays will be developed using
Meso Scale Discovery's MULTI-ARRAY®
technology platform, and will measure
cerebrospinal fluid biomarkers for use in AD
research and drug development. [CONT.]
197
Research programme: Alzheimer's
disease diagnostics - GE
Healthcare/Janssed Pharmaceutica
GE Healthcare in collaboration with Janssen
Pharmaceutica N.V. is developing non-invasive or
minimally invasive diagnostic biosignatures for
pre-symptomatic detection of Alzheimer's
disease (AD). The collaboration will combine
Janssen's clinical, biomarker and informatics
expertise with GE Healthcare's diagnostic
capabilities to identify diagnostic biosignatures,
also known as combination of biomarkers, that
are specific to AD [CONT.]
Pharnext Originator France Biopharmaceutical
University of Bordeaux Originator France University N/A
Alzheimer's disease Research PO USA Reviva Pharmaceuticals Originator USA Pharmaceutical Private
Reviva Pharmaceuticals Owner USA Pharmaceutical Private
Alzheimer's disease Research Unknown route Sweden Umecrine Cognition Originator Sweden Pharmaceutical Private
Umecrine Cognition Owner Sweden Pharmaceutical Private
Alzheimer's disease Research Unknown route USA Yuma Therapeutics Originator USA Biopharmaceutical Private
Yuma Therapeutics Owner USA Biopharmaceutical Private
EPalpha Alzheimer's disease Research Unknown route USA Icogenex Corporation Originator USA Pharmaceutical Private
EPbeta Icogenex Corporation Owner USA Pharmaceutical Private
Alzheimer's disease Research Unknown route Japan Kyoto University Originator Japan University N/A
Shionogi Originator Japan Large Pharma, Pharmaceutical Public
Alzheimer's disease Research PO Canada, USA Alectos Therapeutics Originator Canada Biopharmaceutical Private
Merck & Co Originator USA Large Pharma, Pharmaceutical Public
Alzheimer's disease Research Unknown route Sweden AstraZeneca Originator United Kingdom Large Pharma, Pharmaceutical Public
AstraZeneca Owner United Kingdom Large Pharma, Pharmaceutical Public
Alzheimer's disease Research PO Canada Critical Outcome Technologies Originator Canada Biotechnology Private
Critical Outcome Technologies Owner Canada Biotechnology Private
Alzheimer's disease Research Unknown route USA Elan Pharmaceuticals Originator USA Biotechnology Public
Elan Pharmaceuticals Owner USA Biotechnology Public
PPD Collaborator World CRO Public
Alzheimer's disease Research Unknown route USA Biogen Idec Originator USA Large Pharma, Biotechnology Public
Multiple sclerosis Research Unknown route USA Johns Hopkins Brain Science Institute Originator USA Institution N/A
Neurodegenerative disorders Research Unknown route USA
Pain Research Unknown route USA
Medivir AB is undertaking a research programme
to develop multiple series of compounds as
inhibitors of BACE1 (beta-site amyloid precursor
protein-cleaving enzyme) for the treatment of
Alzheimer's disease (AD). Lead compounds are
undergoing optimisation in preparation for
advanced preclinical development in Sweden.
Alzheimer's disease Research Unknown route Sweden Medivir AB Originator Sweden Pharmaceutical Public
BACE1 catalyses the first step of the two-step
amyloid precursor protein (APP) cleavage
process in the brain, i [CONT.]
Medivir AB Owner Sweden Pharmaceutical Public
CATAB I - Ozgene Alzheimer's disease Research Parenteral Australia Ozgene Originator Australia Company
CATAB II - Ozgene HIV infections Research Parenteral Australia Ozgene Owner Australia Company
CATAB III - Ozgene Rheumatoid arthritis Research Parenteral Australia
CATAB IV - Ozgene
CATAB V - Ozgene
219
Research programme: BACE1 protein
inhibitors - Medivir AB
Alzheimer's disease protease inhibitors - Medivir
AB
BACE1 protein inhibitors
220
Research programme: catalytic
antibody therapeutics - Ozgene
Ozgene is developing catalytic antibodies for the
treatment of various diseases, including
rheumatoid arthritis, HIV infections and
Alzheimer's disease. Catalytic antibodies are
highly substrate specific and catalyse a number
of biological processes including the
actication/inactivation of proteins. Ozgene has
patented a catalytic antibody platform and early
research is ongoing in Australia. [CONT.]
Undefined mechanism
218
Research programme: analgesics and
neurodegenerative disorders
therapies - Biogen Idec/BSi
Biogen Idec and the Johns Hopkins Brain Science
Institute (BSi) are collaborating to discover and
develop therapies for pain and for
neurodegenerative disorders such as multiple
sclerosis and Alzheimer's disease (and possibly
also Parkinson's disease). Development is at the
early research stage in the US.
BSi-Biogen Idec Research Program Undefined mechanism
216
Research programme: amyloid
precursor protein secretase
inhibitors - Critical Outcome
Technologies
Critical Outcome Technologie (COTI) is
developing oral, small molecule inhibitors that
target amyloid precursor protein secretases (beta-
secretase and gamma-secretase) for the
treatment of Alzheimer's disease (AD). Using its
computational platform technology, CHEMSAS®,
COTI has optimised six lead candidates on three
different scaffolds that are ready for synthesis
and preclinical evaluation. [CONT.]
Amyloid precursor protein secretase inhibitors
217
Research programme: amyloid
precursor protein secretase
inhibitors - Elan
Elan Pharmaceuticals, a subsidiary of Elan
Corporation, is developing small molecule
inhibitors of amyloid precursor protein secretase
(beta-secretase and gamma-secretase) for the
treatment of Alzheimer's disease (AD).
Neurodegeneration in AD is thought to result
from the pathological cascade initiated by
formation of insoluble plaques of accumulated
beta-amyloid (Abeta) peptides. [CONT.]
ELND007 Amyloid precursor protein secretase inhibitors
214
Research programme: Alzheimer's
disease therapy - Alectos/Merck
Alectos Therapeutics in collaboration with Merck
is developing small molecule therapeutics for the
treatment of Alzheimer's disease. The
collaboration is based on Alectos' proprietary
portfolio of compounds, modulators of O-linked N-
acetylglucosaminidase (O-GlcNAcase), an
enzyme implicated in the development of
Alzheimer's disease. Development is at the
research stage in the US and Canada.
Hexosaminidase C modulators
215
Research programme: amyloid beta-
derived diffusible ligand inhibitors -
AstraZeneca
AstraZeneca is developing therapeutics that
target soluble polymers (SPs) of the amyloid
beta peptide, also known as amyloid beta-
derived diffusible ligands (ADDLs), for the
potential treatment of Alzheimer's disease.
ADDLs are highly ordered aggregates of amyloid
beta1-42 peptides which are believed to provoke
deposition of hyperphosphorylated Tau (paired
helical filaments or PHF-Tau). [CONT.]
ADDL inhibitors Amyloid beta-protein inhibitors
212
Research programme: Alzheimer's
disease therapies - Icogenex
Icogenex (formerly Icogen) Corporation is
developing therapeutics for the treatment of
Alzheimer's disease (AD). The company are using
its ICOCEPT™ platform to investigate
inappropriate proteolytic processing and to
develop small molecules with the aim of
normalising the beta-amyloid production without
disrupting other systems. Preclinical development
is ongoing in the USA for the treatment of AD.
[CONT.]
Amyloid beta-protein precursor modulators
213
Research programme: Alzheimer's
disease therapies - Kyoto
University/Shionogi
Shionogi and Kyoto University are collaborating
on a drug discovery and medical research
project, the SK Project, to develop therapies for
Alzheimer's disease and other disorders of the
central nervous system. The project is based on
identifying new drug targets relating to synapses
and the regeneration of neurological function.
Development is at the stage of early research in
Japan. [CONT.]
SK Project Central nervous system modulators
210
Research programme: Alzheimer's
disease therapeutics - Umecrine
Cognition
Umecrine Cognition is developing compounds for
the treatment of Alzheimer's disease and
dementia. These compounds selectively modulate
the effects of GABA-steroids on GABA A
receptors in the brain. A research programme to
investigate these compounds is underway in
Sweden. [CONT.]
GABA A receptor modulators
211
Research programme: Alzheimer's
disease therapeutics - Yuma
Therapeutics
Yuma Therapeutics is developing small molecule
compounds targeting tau pathway for the
treatment of Alzheimer's disease (AD). One of
the hallmarks of AD is the presence of
neurofibrillary tangles in the brains of affected
individuals. These tangles are made up of
aberrant tau protein clusters comprising paired
helical filaments (PHFs, as characterised by a
double-twisted ribbon shape), which aggregate
over time causing toxicity and neuronal cell death
[CONT.]
Tau protein inhibitors
208
Research programme: Alzheimer's
disease therapeutics -
Pharnext/Biosystems
Pharnext, Biosystems International, the
University of Bordeaux Segalen and Inserm are
collaborating on a 6-year project to develop
therapeutics and diagnostics for Alzheimer's
disease. The project, known as DIPPAL, will be
lead by Pharnext and will involve development of
Pleodrugs™; cocktails of low-dose, off-patent
drugs which have been approved for other
diseases. [CONT.]
Undefined mechanism
209
Research programme: Alzheimer's
disease therapeutics - Reviva
Pharmaceuticals
Reviva Pharmaceuticals in the US is developing
orally bioavailable therapeutics for the treatment
of Alzheimer's disease. The company's internally
discovered small molecule therapeutics are
designed to be safe, highly potent and selective
for improved patient compliance. Designated RP
4000, Reviva's programme targeting Alzheimer's
disease is at the research stage of development.
[CONT.]
RP4000 - Reviva Pharmaceuticals Undefined mechanism
Alzheimer's disease Research PO USA National Pharmaceutical Corp Originator USA
Parkinson's disease Research PO USA Spencer Pharmaceutical Originator USA Biopharmaceutical Private
Amyloid beta-protein precursor inhibitors Alzheimer's disease Research Unknown route Belgium Galapagos NV Originator Belgium Pharmaceutical Public
G protein-coupled receptor antagonists Galapagos NV Owner Belgium Pharmaceutical Public
Alzheimer's disease Research Unknown route United Kingdom Aquapharm Biodiscovery Originator United Kingdom Biotechnology Private
University of Dundee Originator United Kingdom University N/A
Nitric oxide modulators Alzheimer's disease Research Unknown route USA NeuroMolecular Pharmaceuticals Originator USA Pharmaceutical Private
NMDA receptor antagonists Neurodegenerative disorders Research Unknown route USA NeuroMolecular Pharmaceuticals Owner USA Pharmaceutical Private
Neuropathic pain Research Unknown route USA
Alpha-synuclein inhibitors Alzheimer's disease Research Parenteral Switzerland, USA Biogen Idec Licensee World Large Pharma, Biotechnology Public
Amyloid beta-protein inhibitors Neurodegenerative disorders Research Parenteral Switzerland, USA Neurimmune Therapeutics Originator Switzerland Biotechnology Private
Tau protein inhibitors Neurimmune Therapeutics Owner Switzerland Biotechnology Private
TDP-43 protein inhibitors
Aurogen is developing therapeutics for the
treatment of neurodegenerative diseases,
including diabetic neuropathy and Alzheimer's
disease. Development is at the early research
stage in the US.
Alzheimer's disease Research Parenteral USA Aurogen Originator USA Biotechnology Private
The company's proprietary technology is based
on the discovery of the neurotrophic role of
insulin-like growth factors (IGF) that are able to
cross the blood-brain-barrier and prevent the
death of neurons. [CONT.]
Diabetic neuropathies Research Parenteral USA Aurogen Owner USA Biotechnology Private
Protein aggregation modulators Alzheimer's disease Research Unknown route United Kingdom Prothena Corporation Originator Ireland Pharmaceutical Public
Protein conformation modulators Parkinson's disease Research Unknown route United Kingdom University of Cambridge Originator United Kingdom University N/A
Protein folding modulators
Alzheimer's disease Research Unknown route USA Afraxis Originator USA Biotechnology Private
CNS disorders Research Unknown route USA Afraxis Owner USA Biotechnology Private
Schizophrenia Research Unknown route USA Genentech Licensee World Large Pharma, Biotechnology Public
Alzheimer's disease Research Unknown route USA PharmatrophiX Originator USA Biotechnology Private
PharmatrophiX Owner USA Biotechnology Private
Protein folding modulators - Reata
Pharmaceuticals
Alpha-synuclein inhibitors Alzheimer's disease Research PO USA Reata Pharmaceuticals Originator USA Biopharmaceutical Private
Protein misfolding reagents for
neurodegenerative diseases - Reata
Pharmaceuticals
Amyloid beta-protein inhibitors Amyotrophic lateral sclerosis Research PO USA Reata Pharmaceuticals Owner USA Biopharmaceutical Private
RTA 801 Protein folding modulators Huntington's disease Research PO USA
SOD1 reactivators - Reata Pharmaceuticals Superoxide dismutase stimulants Parkinson's disease Research PO USA
SOD1 stabilisers - Reata Pharmaceuticals
Alpha 2c adrenergic receptor antagonists Alzheimer's disease Research Unknown route Finland Orion Originator Finland Pharmaceutical Public
Undefined mechanism Schizophrenia Research IV Finland Orion Owner Finland Pharmaceutical Public
NMDA receptor modulators Alzheimer's disease Research Unknown route USA Jubilant Pharmaceuticals Collaborator USA Pharmaceutical
232
Research programme: subtype-
selective NMDA receptor modulators -
Mnemosyne Pharmaceuticals
Mnemosyne Pharmaceuticals is developing small
molecule therapeutics that target and modulate
the specific NMDA receptor subtypes of the
receptor subunit, NR2, as potential treatment for
cognitive and neuropsychiatric disorders.
Development is at the research stage and
indications being investigated include
schizophrenia (the lead indication), Alzheimer's
disease (AD), recovery from stroke and
traumatic brain injury, and attention-deficit
hyperactivity disorder (ADHD) [CONT.]
231
Research programme: schizophrenia
and Alzheimer's disease therapeutics
- Orion Pharma
Orion Pharma in Finland is researching
compounds for the potential treatment of
schizophrenia and Alzheimer's disease. Three
lead compounds emerged from this programme,
ORM 14540, ORM 10921, and ORM 12741 [see
separate profile]. ORM 14540 completed phase 0
development in an IV formulation in healthy men
(NCT00831077), with potential for schizophrenia
and possibly also Alzheimer's disease. [CONT.]
ORM-14540
229
Research programme: p75 ligands -
PharmatrophiX
PharmatrophiX in the US is developing small
molecule p75 ligands for the treatment of
Alzheimer's disease and possibly other
neurological disorders such as Parkinson's
disease and multiple sclerosis. The compounds
imitate the function of neurotrophins, interacting
with neurons susceptible to degeneration in
Alzheimer's disease. Specifically, they have the
potential to provide protection against
degeneration caused by the beta-amyloid
protein, a possible cause of Alzheimer's disease.
[CONT.]
Amyloid beta-protein inhibitors
230
Research programme: protein folding
modulators - Reata Pharmaceuticals
Reata Pharmaceuticals is developing small
molecule, protein folding modulators for the
treatment of various genetic diseases caused by
misfolded proteins. Defects in protein folding
underlie a large number of diseases including
cancer, amyotrophic lateral sclerosis (ALS),
Alzheimer's disease (AD), Parkinson's disease
(PD), cystic fibrosis, and Huntington's disease
(HD). [CONT.]
228
Research programme:
neuropsychotherapeutics - Afraxis
Afraxis is developing inhibitors of p21-activated
kinase (PAK) as potential therapeutics for CNS
disorders such as Alzheimer's disease and mental
disorders such as schizophrenia. The company
has stated that the PAK inhibitors function as
disease-modifying agents that directly target the
cellular pathology underlying the disorders. PAK
is a protein kinase that regulates the
development and activity of dendritic spines
[CONT.]
P21 activated kinase inhibitors
226
Research programme:
neurodegenerative disorders
therapeutics - Aurogen
Insulin-like growth factor I stimulants
227
Research programme:
neurodegenerative therapeutics -
Prothena Corporation/The University
of Cambridge
Prothena Corporation (originally Elan
Corporation) and The University of Cambridge
are collaborating to develop treatments for
Alzheimer's disease and Parkinson's disease. The
goal of the collaboration is to discover novel
compounds that can alter the molecular
behaviour of proteins (i.e folding, misfolding,
aggregation and turnover) associated with these
neurodegenerative disorders. [CONT.]
225
Research programme:
neurodegenerative disease
therapeutics - Biogen
Idec/Neurimmune Therapeutics
Biogen Idec and Neurimmune Therapeutics are
collaboratively developing fully human antibodies
for the treatment of neurodegenerative diseases.
The alliance will focus on the development of
antibodies that bind to amyloid beta (Abeta),
alpha-synuclein, tau, and TDP-43. These targets
may result in antibodies for the treatment and
prevention of a wide variety of neurodegenerative
diseases, including Parkinson's disease,
Alzheimer's disease, and amyotrophic lateral
sclerosis (ALS). [CONT.]
Hexosaminidase C inhibitors
224
Research programme: memantine
derivatives - NeuroMolecular
Pharmaceuticals
NeuroMolecular Pharmaceuticals is undertaking a
research programme to develop a series of
memantine derivatives with enhanced efficacy
and improved tolerability for the treatment of
neurodegenerative disorders such as Alzheimer's
disease (AD) and neuropathic pain. Development
is at the early research stage in the US. [CONT.]
Memantine analogues - NeuroMolecular
222
Research programme: G-protein
coupled receptor 3 antagonists -
Galapagos
Galapagos NV is undertaking a research
programme focussed on the development of
small molecule drugs that target and inhibit the
orphan G-protein coupled receptor 3 (GPR3) for
the treatment of Alzheimer's disease (AD). GPR3
was identified in human cells using Galapagos'
technology platform. Development is at the early
research stage in the Belgium. [CONT.]
GPR3 inhibitors programme
223
Research programme:
hexosaminidase-C-inhibitors -
Aquapharm Biodiscovery/University
of Dundee
Aquapharm Biodiscovery and the University of
Dundee are developing hexosaminidase-C
inhibitors (O-GlcNAcase inhibitors) for the
treatment of Alzheimer's disease.
Hexosaminidase-C modulates the solubility of the
proteins in the aggregates associated with the
progressive neurodegeneration of Alzheimer's
disease. The compounds being investigated are
derived from Aquapharm's marine microbial
collection [CONT.]
221
Research programme: CNS disorders
therapeutics - National
Pharmaceutical/Spencer
Pharmaceutical
Spencer Pharmaceutical and National
Pharmaceutical are collaborating to develop
orally available prodrugs to treat central nervous
system (CNS) disorders, with particular focus on
Alzheimer's and Parkinson's disease. Using
Spencer's drug delivery technology as the
primary platform, the companies will work on bio-
oxidable prodrugs that can efficiently cross the
blood brain barrier, where they are converted to
the parent drug in the CNS via a redox-activation
process [CONT.]
Undefined mechanism
NR2B N-Methyl D-Aspartate receptor modulators Attention-deficit hyperactivity disorder Research Unknown route USA Mnemosyne Pharmaceuticals Originator USA Biotechnology Private
Brain injuries Research Unknown route USA Mnemosyne Pharmaceuticals Owner USA Biotechnology Private
Schizophrenia Research Unknown route USA
Stroke Research Unknown route USA
IMD2 (SR form) Alzheimer's disease Research Unknown route South Korea Daewoong Pharmaceutical Originator South Korea Pharmaceutical Public
IMD3 (SR form) Gastric ulcer Research Unknown route South Korea Daewoong Pharmaceutical Owner South Korea Pharmaceutical Public
IMD4 (SR form) Thrombosis Research Unknown route South Korea
Alzheimer's disease Research Unknown route USA Bristol-Myers Squibb Originator USA Large Pharma, Biopharmaceutical Public
The J. David Gladstone Institutes Originator USA Institution N/A
BLV0703 Alzheimer's disease Research Unknown route Portugal BIOALVO Originator Portugal Biopharmaceutical Private
BLV200703 Neurodegenerative disorders Research Unknown route Portugal BIOALVO Owner Portugal Biopharmaceutical Private
SDGI-T200801 Parkinson's disease Research Unknown route Portugal
TAU Programme
Alzheimer's disease Research Parenteral France Rockefeller University Originator USA University N/A
Rockefeller University Owner USA University N/A
Sanofi Licensee World Large Pharma, Pharmaceutical Public
Virobay is developing VBY 129, a selective, orally
active, small-molecule cathepsin S inhibitor, for
the treatment of Alzheimer's disease. The drug
was in-licensed by Virobay from an undisclosed
company and appears to be suitable for once-
daily dosing. Development is at the stage of early
research in the US.
Alzheimer's disease Research PO USA Unknown Originator USA Company
VBY 129 was originally being developed to treat
autoimmune disease. [CONT.]
Psoriasis Discontinued(I) PO USA Unknown Owner USA Company
Virobay Licensee USA Pharmaceutical Private
236
Research progranne: anti-beta-
amyloid antibodies - sanofi-
aventis/Rockefeller University
sanofi-aventis is developing a monoclonal
antibody, targeting specific forms of the amyloid
beta (Abeta) parenchymal plaque for the
treatment of Alzheimer's disease. sanofi-aventis
licensed a specific antibody from the Rockefeller
University to be studied. Preclinical research is
being conducted in France.
Amyloid beta-protein inhibitors
237
VBY 129 Cathepsin S inhibitors
235
Research programme: tau protein
modulators - BIOALVO
BIOALVO, located in Portugal, has a research
programme directed at tauopathies for the
treatment of neurodegenerative diseases such as
Alzheimer's disease and Frontotemporal
Dementia (FTD) with Parkinsonism. Tau protein
interacts with tubulin to stabilise microtubules
and promote tubulin assembly into microtubules.
Tau aggregation into neurofibrillary tangles (NFT)
is a major feature in some neurodegenerative
diseases [CONT.]
Tau protein modulators
233
Research programme: sustained-
release drugs - Daewoong
Pharmaceutical
Daewoong Pharmaceutical is developing
sustained-release incrementally modified drugs
(IMDs) for the treatment of thrombosis (IMD2),
Alzheimer's disease (IMD3), and gastric ulcers
(IMD4). The drugs are at the research stage of
development in South Korea.
Undefined mechanism
234
Research programme: Tau
dysfunction regulators - Bristol-
Myers Squib/Gladstone Institutes
Bristol-Myers Squib and the Gladstone Institutes
are conducting a discovery-based research
collaboration to identify and validate targets that
affect Tau dysfunction for the treatment of
Alzheimer's disease. Tau is a protein that binds
the internal skeleton of the cell and may help
regulate brain cell activity. Tau forms abnormal
deposits, termed neurofibrillary tangles, in
Alzheimer's disease. [CONT.]
Tau protein modulators
232
Research programme: subtype-
selective NMDA receptor modulators -
Mnemosyne Pharmaceuticals
Mnemosyne Pharmaceuticals is developing small
molecule therapeutics that target and modulate
the specific NMDA receptor subtypes of the
receptor subunit, NR2, as potential treatment for
cognitive and neuropsychiatric disorders.
Development is at the research stage and
indications being investigated include
schizophrenia (the lead indication), Alzheimer's
disease (AD), recovery from stroke and
traumatic brain injury, and attention-deficit
hyperactivity disorder (ADHD) [CONT.]