Pharma R&D Annual Review 2018...Pharma R&D Annual Review 2018 Ian Lloyd Senior Director...

Pharma R&D Annual Review

2018 Ian Lloyd

Senior Director Pharmaprojects & Data Integration

Alexandra Shimmings

Executive Editor Scrip, Pink Sheet and In Vivo

Wednesday May 16th 2018

Pharma intelligence | informa 2

Agenda •  The total drug R&D pipeline size in 2018

•  New active substance launches 2017

•  The drug pipeline in 2018, and how it is changing

•  What to expect in the year ahead

•  Q&A


Total pipeline size in 2018


Total pipeline size has grown by 2.7%

2.7%increase

Source:Pharmaprojects


Key Facts – Total pipeline size


3,807 drugs

entered

pipeline during

the year, but

net increase

of just 395

The 2018

growth rate of

2.7% well

down on the

2017 rate of

8.4%

Growth has

picked up

again for 2018

in some

indications

Therefore,

3,412 left

development

during the year

– a churn rate

of over 20%

Continuing

growth is a

cost, so only a

good thing if

it's matched

by new drug

launches


New active substance launches 2017

Pharma intelligence | informa 7 Source:Pharmaprojects

2017 was a good year for New Active Substance launches


Key Facts – NAS launches in 2017


32% increase

on the 41 NAS

launches in

2016

54 New Active

Substances

were launched

Pushes mean

up to 46 for

this decade

Second-

highest tally

since 2000

Increase in

first-in-class

products

launched


Selected New Drug Launches 2017 – A Year of Firsts

First 2 CAR-T cell therapies launched

•  Novartis’ Kymriah (tisagenleucel-t) •  Gilead’s Yescarta (axicabtagene ciloleucel)

•  Spark Therapeutics’ Luxturna (voretigene neparovec)

•  Sanofi/Regeneron’s Dupixent (dupilumab)

First US approval for a gene therapy

First systemic therapy for atopic dermatitis

More success in immuno-oncology

Poised to become a blockbuster

•  AstraZeneca’s Imfinzi (durvalumab) •  Merck KGaA/Pfizer’s Bavencio (avelumab)

•  GSK’s shingles vaccine Shingrix

Biggest first year sales in recent years

•  Roche’s Ocrevus (ocrelizumab) for MS, generating ~US$1 billion in nine months



Top Companies By NAS Launches 2017



NAS Launches 2017 By Therapeutic Group



NAS Launches 2017 By Region



Other First-In-Class Products In 2017 – Part 1

CELL AND GENE THERAPIES

•  Holostem and Chiesi Farmaceutici’s Holoclar – an autologous corneal epithelial cell transplant therapy

•  TissueGene and Kolon Life Science’s Invossa for osteoarthritis – the first TGF-ß1 agonist

•  Puma Biotechnology’s Nerlynx (neratinib) •  BioCryst Pharmaceuticals/Mundipharma’s

Fodosine (forodesine)

CANCER

ANTI-INFECTIVES •  AiCuris/Merck & Co.’s Prevymis (letermovir) •  Maruhi Co.’s Amenalief (amanamevir) •  Merck & Co.’s Zinplava (bezlotoxumab) in

Clostridium difficile infections



Other First-In-Class Products In 2017 – Part 2

RESPIRATORY •  AstraZeneca’s Fasenra (benralizumab) for severe asthma – an IL-5 receptor antagonist

•  Roche’s Hemlibra (emicizumab) – a bispecific antibody to Factors IXa and X

HEMOPHILIA

ENZYME REPLACEMENT

THERAPIES

•  BioMarin Pharmaceuticals’ Brineura (cerliponase alfa)

•  Ultragenyx Pharmaceuticals’ Mepsevii (vestronidase alfa)



The drug R&D pipeline in 2018, and

how it is changing


Breakdown of pipeline by phase



Trends in clinical phase drug R&D



Pipeline quality assessment of late stage drugs

Source:Biomedtracker


Top 10 pharma companies by pipeline size Posi:on

2018(2017)Company

NoofDrugsinPipeline2018(2017)

NoofOriginatedDrugs2018

1(1) Novar's 223(251) 138

2(5) Johnson&Johnson 216(214) 116

3(6) AstraZeneca 205(213) 117

4(3) Pfizer 192(232) 126

5(7) Roche 191(206) 114

6(2) GlaxoSmithKline 191(250) 111

7(4) Merck&Co. 191(229) 109

8(8) Sanofi 179(193) 78

9(10) Takeda 164(141) 96

10(9) Bristol-MyersSquibb 134(144) 96Source:Pharmaprojects


Top 10 pharma companies – are they delivering new drugs? Posi:on

2018(2017)Company

NoofDrugsinPipeline2018(2017)

NoofNASLaunches2017(2016)

1(1) Novar's 223(251) 4(0)

2(5) Johnson&Johnson 216(214) 1(0)

3(6) AstraZeneca 205(213) 3(1)

4(3) Pfizer 192(232) 3(0)

5(7) Roche 191(206) 2(2)

6(2) GlaxoSmithKline 191(250) 1(1)

7(4) Merck&Co. 191(229) 3(2)

8(8) Sanofi 179(193) 2(0)

9(10) Takeda 164(141) 1(0)

10(9) Bristol-MyersSquibb 134(144) 1(0)Source:Pharmaprojects


Total companies involved in pharma R&D



Big pharma companies vs small – how the balance is shifting



Locations of pharma companies worldwide



The R&D pipeline by Therapeutic Area



Cancer taking an ever bigger slice of the R&D pie



Ongoing clinical trials by Therapeutic Area

Source:Trialtrove


Top 10 diseases/indications Posi:on

2018(2017)Disease

No.ofAc:vedrugs2018(2017)

Trend

1(1) Cancer,breast 727(654) ↑

2(2) Cancer,lung,non-smallcell 544(477) ↑

3(3) Cancer,colorectal 503(476) ↑

4(6) Cancer,ovarian 434(386) ↑

5(4) Cancer,pancrea'c 430(416) ↑

6(5) Diabetes,Type2 407(415) ↔

7(9) Cancer,prostate 381(362) ↑

8(7) Alzheimer'sdisease 381(376) ↔

9(10) Cancer,brain 361(322) ↑

10(8) Arthri's,rheumatoid 352(372) ↓Source:Pharmaprojects


Top diseases/indications – 11-25 Posi:on

2018(2017)Disease

No.ofAc:vedrugs2018(2017)

Trend

11(11) Cancer,melanoma 346(312) ↑12(12) Cancer,leukaemia,acutemyelogenous 326(285) ↑13(15) Cancer,liver 272(264) ↔14(14) Pain,nocicep've,general 262(268) ↔15(19) Cancer,headandneck 258(227) ↑16(13) Psoriasis 256(283) ↓17(17) Cancer,myeloma 254(246) ↔18(18) Parkinson'sdisease 252(246) ↔19(21) Cancer,gastrointes'nal,stomach 242(213) ↑20(16) Asthma 224(254) ↓21(22) Cancer,renal 218(197) ↑22(20) Cancer,lymphoma,non-Hodgkin's 215(217) ↔23(23) Chronicobstruc'vepulmonarydisease 179(192) ↔24(25) Pain,neuropathic,general 178(169) ↔25(24) Infec'on,HIV/AIDS 177(183) ↔



Top 10 Mechanisms of Action (pharmacologies) Posi:on

2018(2017)MechanismofAc:on(Pharmacology)

No.ofAc:veCompounds2018

(2017)

%ofCompoundsPR/R/L

1(1) Immunos'mulant 1501(1736) 10.0

2(2) An'cancerimmunotherapy 1332(889) 1.6

3(-) Immunecheckpointinhibitor 211(-) 3.3

4(3) Immunosuppressant 208(215) 26.4

5(4) Angiogenesisinhibitor 169(179) 17.8

6(5)Vascularendothelialgrowthfactor(VEGF)receptorantagonist

138(123) 14.5

7(7) Apoptosiss'mulant 116(115) 14.7

8(6) DNAinhibitor 111(120) 20.7

9(9) Opioidmureceptoragonist 110(114) 40.9

10(-) Radiopharmaceu'cal 108(-) 10.2Source:Pharmaprojects


Top 10 Drug protein targets Posi:on

2018(2017)Target

No.ofAc:vecompounds2018(2017)

Trend

1(1) opioidreceptor,mu1 140(143) ↔

2(4) erb-b2receptortyrosinekinase2[Her-2] 120(113) ↔

3(6) vascularendothelialgrowthfactorA 119(105) ↑

4(2)nuclearreceptorsubfamily3,groupC,member1(glucocor'coidreceptor)

113(123) ↓

5(3) tumournecrosisfactor 113(123) ↓

6(5)prostaglandin-endoperoxidesynthase2(prostaglandinG/Hsynthaseandcyclooxygenase)[COX-2]

107(106) ↔

7(7) epidermalgrowthfactorreceptor 107(104) ↔

8(8) insulinreceptor 99(94) ↔

9(9) opioidreceptor,kappa1 88(87) ↔

10(10) glucagon-likepep'de1receptor 80(82) ↔Source:Pharmaprojects


New drug protein targets identified by year



Biologicals advance towards 40% of R&D



Orphan drug designations and expedited reviews granted by year



Key Trends – The drug R&D pipeline in 2018 – Part 1


2017 was a

good year for

new drug

launches, with

some notable

firsts

The R&D

pipeline is still

growing,

although the

growth rate has

slowed

Top 10 pharma

companies all

delivered new

drugs, but their

share of the

overall pipeline

is in decline

Little change in

drugs at clinical

stages – in

quantity or

quality


Key Trends – The drug R&D pipeline in 2018 – Part 2


The immuno-

oncology boom

shows no sign

of ending

Cancer now

taking over a

third of all

pipeline drugs,

and all of the

top 5 indications

Companies still

focusing on rare

diseases,

despite

stumbling

innovation

levels

Biologicals

advance

towards 40% of

the R&D

portfolio


What to expect in the year ahead


Selected drugs approved in 2017 due for early 2018 launch


Spark Therapeutics’ Luxturna (voretigene neparvovec) for biallelic RPE65 mutation-associated retinal dystrophy

Pfizer’s Steglatro (ertugliflozin) for Type 2 diabetes

Novo Nordisk’s Ozempic (semaglutide) for Type 2 diabetes

Aerie Pharmaceuticals’ Rhopressa (netarsudil) for glaucoma

Launched Q1

Launched April

Launched February

Launched January


Important approvals so far in 2018

•  Gilead’s Biktarvy (bictegravir in FDC with emtricitabine/TAF) [February]

•  Theratechnologies/TaiMed Biologics’ Trogarzo (ibalizumab) [March]

•  Rigel’s Tavalisse (fostamatinib) [April]

Further anti-HIV drugs

Immune thrombocytopenic

purpura Non-metastatic

castration-resistant prostate cancer

Moderate-to-severe plaque psoriasis

•  J&J’s Erleada (apalutamide) [April]

•  Merck & Co & Sun’s tildrakizumab [March]

Source:Scrip


Important approvals due later in 2018

•  Amgen/Novartis’s Aimovig (erenumab) [17 May]

•  Teva’s fremanezumab [15 June] •  Lilly’s galcanezumab [October]

•  AbbVie’s elagolix [was due in May but now Q3]

Anti-CGRP monoclonals for migraine

Endometriosis and uterine

fibroids

Hairy cell leukemia

Moderate-to-severe rheumatoid arthritis

•  AstraZeneca's moxetumomab [PDUFA September]

•  Eli Lilly/Incyte's baricitinib [mid-year]

Source:Scrip


Phase III Results to look out for in 2018

PD-1/PD-L1 inhibitors and CTLA-4 inhibitors in indications beyond melanoma

•  AstraZeneca’s study of Imfinzi (durvalumab) with tremelimumab in NSCLC

•  Bristol-Myers Squibb’s study of Opdivo (nivolumab) with Yervoy (ipilimumab), also in NSCLC

•  Phase III ECHO 301 trial of Merck & Co’s Keytruda (pembrolizumab) in combination with IDO inhibitor epacadostat

Vascular Biogenics’ gene therapy for GBM, ofranergene obadenovec

Oncology

Neuroscience

Cardiometabolic

Further Phase III trial read-outs in Alzheimer’s •  Roche’s amyloid-targeting crenezumab and gantenerumab in AD •  vTv Therapeutics’ RAGE inhibitor azeliragon in AD – STEADFAST trial

J&J’s esketamine for treatment-resistant depression in Phase III

Esperion’s bempedoic acid in elevated LDL-cholesterol as add-on to ezetimibe

Source:Scrip


What to look out for in 2018

Generic competition

•  Lifting barriers to generic drug competition will be a priority of the US government and FDA

New pricing mechanisms for combination therapies •  New formulae for reimbursing combinations where elements come from

different manufacturers

Brexit •  Continued uncertainty on harmonization and ex-UK recruitment into UK

pharmas

Source:Scrip

Pricing and policy



Personalized Medicine

•  Improvements in cost-effectiveness and sensitivity of genomic analysis meaning a wide range of tests are beginning to emerge

•  PM will improve response rates dramatically and make treatments much more effective and efficient

The rise of Artificial Intelligence •  Companies will significantly scale their use of AI via applications such as

predictive customer engagement

Tech companies become industry disruptors? •  Amazon and Google could enter the pharma fray

Technology

Source:Scrip



•  Takeda acquiring Shire and TiGenix

•  Sanofi outbid Novo Nordisk to buy Ablynx

•  Bioverativ also to be acquired by Sanofi

•  Celgene buying cancer drug developer Impact Biomedicines

•  Biocryst and Idera set to merge

Source:Scrip

Selected M&A activity already announced in 2018

Thank you for your attention

Questions? [email protected]

Pharma R&D Annual Review 2018...Pharma R&D Annual Review 2018 Ian Lloyd Senior Director...

Documents

Transcript of Pharma R&D Annual Review 2018...Pharma R&D Annual Review 2018 Ian Lloyd Senior Director...