Neuronano update august 2013

29
neuronano pharma Company and operational activities -9 o UL\YV © 2013 Neuro10-9 Pharma, Inc. All rights reserved.

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Transcript of Neuronano update august 2013

Page 1: Neuronano update august 2013

neuronano pharmaCompany and operational activities

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

Page 2: Neuronano update august 2013

Company overview and business model

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

>L�HYL�H�MVY�WYVÄ[�WOHYTHJL\[PJHS�JVTWHU`�KL]LSVWPUN�HUK�JVTTLYJPHSPaPUN�UHUVTLKPJPULZ�MVY�[OL�[YLH[TLU[�VM�[OL�Z\I�VW[PTHSS`�HKKYLZZLK�VY�WYLZLU[S`�PUJ\YHISL�diseases.

ACADEMIA INDUSTRY

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7YLJSPUPJHS�9LZLHYJO�

7YLJSPUPJHS�+L]LSVWTLU[�

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

ACADEMIA INDUSTRY

commerciability

inno

vatio

n

MAR

KET

idea

platform concept

platform technology

lead compounds

drug candidatedrug

-9oneuro

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

Co-founder &

lead inventor

Kabanov

Co-founder &

acting CEO

Gaymalov

7YPUJPWHS�ZJPLU[PZ[Z�(for

each plafrom

technology)

Yi BatrakovaManickam

Board of KPYLJ[VYZ

Kabanov Polinsky RomarMalkov

Board of advisors

Banks FarrellMcCall Zalipsky

Alakhova

=PJL�7YLZPKLU[�for BD &

Licensing

De Ridder

Bloom

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6\Y�VWLYH[PVUZ

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

4+ delivery platforms20+ lead compounds10+ indications

Academia

Industry key activities

ReCoPE: reverse concept.

product engineeriing

IP generation

& management

business development -

partnering & licensing

securing grants

& equity investment

translational research key outcomes

Spin-off portfolio

companies

Partnerships

Out-licensing deals

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Lean :OHYLK�HUK�SV^LYLK�YPZR *HWP[HSPaPUN�VU�LZ[HISPZOLK�HUK�LTLYNPUN�YLZV\YJLZ�PU�5*

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pluroMAC™Platform Delivery Technology

Binary:\WLYHJ[P]LPLD™Platform Delivery Technology

nanoZYME™Platform Delivery Technology

*LSS�4LKPH[LK�DeliveryTechnology

Macromolecules

Small molecules

Macromolecules

Small molecules

Antibodies Genes

����WYVK\J[�JHUKPKH[LZ 12+ disease models ����WH[LU[Z�HWWSPJH[PVUZ

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LUa`TLZ! SOD *H[HSHZL OPH BCHEHRP

3LW[PU

7HJSP[H_LS�

Doxoru-IPJPU�

BDNF

OVYTVULZ!

J`[VRPULZ!

���WOHYTHJL\[PJHS�SLHK�JVTWV\UKZ!

���IPVWOHYTHJL\[PJHS�SLHK�JVTWV\UKZ!

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NNP-bdnfNZ Rett syndrome

UveitisNNP-sodNZ

NNP-sodNZ Amyotrophic

lateral sclerosis

leptiPOL-LM Obesity

Discovery CandidateSelection

Pre-clinicalResearch

Pre-clinical Development

Clinical Phase 1,2Product Indications

CancerNNP-D773c 505(b)(2)

leptiPOL-HM Alzheimer's disease

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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.

pluroMAC™Platform Delivery Technology

Binary:\WLYHJ[P]LPLD™Platform Delivery Technology

nanoZYME™Platform Delivery Technology

*LSS�4LKPH[LK�DeliveryTechnology

Macromolecules

Small molecules

Macromolecules

Small molecules

Antibodies Genes

����WYVK\J[�JHUKPKH[LZ 12+ disease models ����WH[LU[Z�HWWSPJH[PVUZ

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June 18, 2013;OL�(TLYPJHU�4LKPJHS�(ZZVJPH[PVU�OHZ�VMÄJPHS-ly recognized obesity as a disease, a move

that could induce WO`ZPJPHUZ� [V� WH`�TVYL�H[[LU[PVU� and ZW\Y� TVYL� PUZ\YLYZ� [V� WH`�MVY�[YLH[TLU[Z.

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6ILZP[`���KPZLHZL�VY�JVUKP[PVU&�

6ILZP[`���VUL�VM�[OL�IPNNLZ[�WV[LU[PHS�NSVIHS�THYRL[Z�

US EU

17%32%

6ILZP[`�[YLH[TLU[�JOHSSLUNL

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In obese patients, the transport of

gut hormones to the brain is impaired

(gut hormone desensitization)

Its possible to treat obesity by de-

livering gut hormones directly to the

brain and restore natural sensitivity

to the hormone observed in health

people.

,MMLJ[P]L�HUK�ZHML�KLSP]LY`�VM�N\[�OVYTVULZ�HJYVZZ�ISVVK�IYHPU�IHYYPLY

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group of hormones secreted by enteroendocrine cells in the stomach,

pancreas, and small intestine that play a role of neurotransmitters

and neuromodulators in the central and peripheral nervous systems.

(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[Z

+LSP]LY`�WSH[MVYT�[LJOUVSVN`���WS\YV4(* 7YVK\J[�7YV[V[`WL���SLW[P763�34�

Macro-molecules

;LJOUVSVN`� PU]VS]LZ� Z\YMHJL� TVKPÄJH[PVU� VM� [OLYHWL\[PJ� WYV[LPUZ�with precisely crafted poloxamers and allows active delivery of these

WYV[LPUZ�[V�[OL�KLÄULK�IYHPU�YLNPVUZ�

SLW[P763�34��HK]HU[HNLZ!�� Rapid brain delivery

�� Targeted hypothalamus delivery

�� Immediate effectiveness

�� Independence of diet and exercise

�� Convenience of intranasal delivery

�� Safety

SLW[P763�34�!�Poloxamer conjugated leptin hormone for intranasal

and intravenous hypothalamus delivery

SLW[PU GLP-1 amylin OXM

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50/�MVY�:;;9�7OHZL�0�00;YLH[PUN�6ILZP[`�^P[O�0U[YHUHZHS�3LW[PU�(UHSVNZ�����$1,150,000 7LUKPUN

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�� Tong et al. (2012). Mol Pharm. 10:360-77

�� Tong et al. (2010). Mol Pharm 7:984-92

�� Price et al. (2010). 1�7OHYTHJVS�,_W�;OLY�333:253-63

�� Batrakova et al. (2008). )PVJVUQ\N�*OLT 19:1071-7

�� Batrakova et al (2005). )PVJVUQ\N�*OLT 16:793-802

UNC - NIH RO17VS`WLW[PKL�4VKPÄJH[PVU�MVY�,UOHUJLK�)YHPU�+LSP]LY`������$1,920,000 (J[P]L

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0UK\Z[Y`�JVSSHIVYH[PVUZ!�� Dr. Catherine Farrell, Ph.D. 0UKLWLUKLU[�JVUZ\S[PUN (Amgen)

�� Dr. John 6NLa, Ph.D. 7LHR�[V�7LHR�)PV+L]LSVWTLU[ (Amgen)

�� Dr. David Essayan, M.D. 6UJVYK��0UJ (Amgen)

�� Dr. Samuel AHSPWZR`, Ph.D. SJZ (ALZA)

�� Dr. John McCall, Ph.D. 7OHYTHJ��33*��7ÄaLY�

1 issued, ��WLUKPUNWH[LU[Z

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.SVIHS�PUJPKLUJL�KLH[OZ�]Z�UL^�JHZLZ��PU�TPSSPVUZ���

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5 yearsurvival

45%

V]HYPHU�JHUJLY

0

5 yearsurvival

5 yearsurvival

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IV

2008

2030

7.6 12.7

13.2 22.2

5 yearsurvival

5 yearsurvival

68% 68%

T\S[PWSL�T`LSVTH RHWVZP�ZHYJVTH

;YLH[TLU[�HK]HU[HNLZ�� rapid API internalization

�� low immunogenicity

�� improved solubility

�� enhanced biocompatibility

�� increased bioavailability

�� superior API protection

(70�YLSLHZL�MYVT�SPWVZVTLZ�\WVU�HYYP]HS�PU[V�[OL�[\TVY

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This group of possible APIs includes emulsions, micellar solutions,

liposomes, lipid nanoparticles, and drug-lipid conjugates.

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(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[Z

+LSP]LY`�WSH[MVYT�[LJOUVSVN`���IPUHY`�ZH73? 7YVK\J[�7YV[V[`WL���557�+���J�MVY�73+

liposomal chemotherapies adjuvation with poloxamers

liposomal chemotherapies

:[LW����3PWVZVTHS�(70�JOLTV[OLYHW` :[LW����4L[H�HKQ\]H[PVU�^P[O�557�73+��

7YVSVUNLK�(70�OHSM�SPML

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Low serum levels of free API

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Tumor

Liposomal drug at

the tumor site

Drug release from liposomes

and penetration into tumor site

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UNC Carolina CCNE$55,000(J[P]L

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�� Dr. William Zamboni, PhD. UNC Chapel Hill�� Dr. Victoria Bae-Jump. M.D., PhD., UNC Chapel Hill�� Dr. David Darr, PhD., UNC Chapel Hill�� Dr. Charles Perou, PhD., UNC Chapel Hill�� Dr. Gabriel 3VWLa, M.D., PhD., 4+�(UKLYZVU�*HUJLY�*LU[LY�� Dr. Bulent 6aWVSH[, M.D., PhD., 4+�(UKLYZVU�*HUJLY�*LU[LY

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UNC Carolina CNPP$20,000(J[P]L

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<]LP[PZ�PZ�Z^LSSPUN�HUK�PYYP[H[PVU�VM�[OL�\]LH��[OL�TPKKSL�SH`LY�VM�[OL�L`L��Uveitis requires an urgent referral and thorough

examination by an ophthalmologist or optometrist—HUK�\YNLU[�[YLH[TLU[�[V�JVU[YVS�[OL�PUÅHTTH[PVU�

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BlindnesJH\ZLZ

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7YV�PUÅHTTH[VY`�JLSSZ�PUÄS[YH[PUN�Z[PT\SH[LK�ZP[LZ��PUK\JPUN�[OL�96:�production and accumulation. This leads to the oxidative stress, cell

death, and ultimately blindness.

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ROS

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ROS scavenger enzymes are aimed to reduces the hypoxic stress

HUK�[V_PJP[`�K\YPUN�OHYTM\S�JOYVUPJ�PUÅHTTH[PVU

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(SaOLPTLY»Z�+PZLHZL

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In AD patients, the transport of leptin

to the brain is impaired.

Its possible to slow down the degen-

erative process of AD by delivering

SLW[PU�KPYLJ[S`�[V�[OL�IYHPU��ZWLJPÄJHS-ly to hippocampus.

,MMLJ[P]L�HUK�ZHML�KLSP]LY`�VM�[OL�SLW[PU�OVYTVUL�HJYVZZ�ISVVK�IYHPU�IHYYPLY�[V�[OL�OPWWVJHTW\Z

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Leptin is a 16-kDa protein

hormone that plays a key role

in regulating energy intake

and expenditure, including

appetite and hunger,

metabolism, and behavior.

(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[

+LSP]LY`�WSH[MVYT�[LJOUVSVN`���WS\YV4(* 7YVK\J[�7YV[V[`WL���SLW[P763�/4��

Macromolecules

;LJOUVSVN`� PU]VS]LZ� Z\YMHJL� TVKPÄJH[PVU� VM� [OLYHWL\[PJ� WYV[LPUZ�with precisely crafted poloxamers and allows active delivery of these

WYV[LPUZ�[V�[OL�KLÄULK�IYHPU�YLNPVUZ�

SLW[P763�/4��HK]HU[HNLZ!�� Rapid brain delivery

�� Targeted hippocampus delivery

�� Immediate effectiveness

�� Convenience of intranasal delivery

�� Safety

SLW[P763�/4�!�Poloxamer conjugated leptin hormone for�PU[YHUHZHS�OPWWVJHTW\Z��KLSP]LY`

SLW[PU

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�� In preapration

(JHKLTPJ�JVSSHIVYH[PVUZ!

0UK\Z[Y`�JVSSHIVYH[PVUZ!�� Dr. Catherine Farrell, Ph.D. 0UKLWLUKLU[�JVUZ\S[PUN (Amgen)

�� Dr. John 6NLa, Ph.D. 7LHR�[V�7LHR�)PV+L]LSVWTLU[ (Amgen)

�� Dr. David Essayan, M.D. 6UJVYK��0UJ (Amgen)

�� Dr. Samuel AHSPWZR`, Ph.D. SJZ (ALZA)

�� Dr. John McCall, Ph.D. 7OHYTHJ��33*��7ÄaLY�

��WH[LU[�WLUKPUN

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(3:�PZ�H�WYVNYLZZP]L�UL\YVKLNLULYH[P]L�KPZLHZL�[OH[�HMMLJ[Z�TV[VY�UL\YVUZ��[OL�ULY]LZ�^OPJO�JVTT\UPJH[L�^P[O�T\ZJSLZ�[OYV\NOV\[�[OL�IVK �̀�HYL�WYVNYLZZP]LS`�KLZ[YV`LK�

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[OLYL�HYL40,000 ALS JHZLZ�PU�<:

[OLYL�HYL200,000 ALS

JHZLZ�PU�YLZ[�VM�[OL�^VYSK

The standard of care therapy, Riluzole is modestly-effective and

does not cure ALS. It decreases glutamate-induced excitotoxicity

and extends patient survival by only 3-6 months while they need to

be continuously monitored for possible liver toxicity. There is an \YNLU[�ULLK�[V�KL]LSVW�H�ZHML�HUK�LMMLJ[P]L�[OLYHW`�MVY�(3:��

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SOD

557�ZVK5A!� SOD enzyme encaged in nanoZYME with extended

half-life for�LUOHUJLK�PU[YHUHZHS�KLSP]LY`�[V�[OL�IYHPU

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1:R917-28

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Effective and safe delivery of BDNF

and other pleotrophic factors to the

brain will ameliorate Rett Syndrome

symptoms. However BDNF has L_[YLTLS`�ZOVY[�OHSM�SPML�HUK�WVVY�WLUL[YH[PVU�[OYV\NO�)))��

Rett syndrome (RTT) is an X-linked postnatal neurodevelopmental

disorder, which is primarily caused by mutations in the gene

encoding methyl-CpG binding protein 2 (MeCP2). A number

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a secreted protein (cytokine)

that, in humans, is encoded

by the BDNF gene.

(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[

7YVK\J[�7YV[V[`WL557�IKUM5A!�BDNF cytokine encaged in nanoZYME with extended

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Brain-derived UL\YV[YVWOPJ�MHJ[VY

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Macromolecules

Small molecules

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*VU[HJ[�<Z�Zagit Gaymalov, Ph.D.

919.923.7401

[email protected]