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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 16 th 2018 Agenda The total drug R&D pipeline size in


  1. 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 16 th 2018

  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 2 Pharma intelligence | informa

  3. Total pipeline size in 2018 3 Pharma intelligence | informa

  4. Total pipeline size has grown by 2.7% 2.7% increase Source: Pharmaprojects 4 Pharma intelligence | informa

  5. Key Facts – Total pipeline size The 2018 3,807 drugs Therefore, Growth has Continuing growth rate of entered 3,412 left picked up growth is a 2.7% well pipeline during development again for 2018 cost, so only a down on the the year, but during the year in some good thing if 2017 rate of net increase – a churn rate indications it's matched 8.4% of just 395 of over 20% by new drug launches Source: Pharmaprojects 5 Pharma intelligence | informa

  6. New active substance launches 2017 6 Pharma intelligence | informa

  7. 2017 was a good year for New Active Substance launches Source: Pharmaprojects 7 Pharma intelligence | informa

  8. Key Facts – NAS launches in 2017 54 New Active 32% increase Second- Pushes mean Increase in Substances on the 41 NAS highest tally up to 46 for first-in-class were launched launches in since 2000 this decade products 2016 launched Source: Pharmaprojects 8 Pharma intelligence | informa

  9. Selected New Drug Launches 2017 – A Year of Firsts • Novartis’ Kymriah (tisagenleucel-t) First 2 CAR-T cell therapies launched • Gilead’s Yescarta (axicabtagene ciloleucel) First US approval for a • Spark Therapeutics’ Luxturna (voretigene neparovec) gene therapy First systemic therapy for • Sanofi/Regeneron’s Dupixent (dupilumab) atopic dermatitis • AstraZeneca’s Imfinzi (durvalumab) More success in • Merck KGaA/Pfizer’s Bavencio (avelumab) immuno-oncology Poised to become a • GSK’s shingles vaccine Shingrix blockbuster Biggest first year sales • Roche’s Ocrevus (ocrelizumab) for MS, generating ~US$1 billion in nine months in recent years Source: Pharmaprojects 9 Pharma intelligence | informa

  10. Top Companies By NAS Launches 2017 Source: Pharmaprojects 10 Pharma intelligence | informa

  11. NAS Launches 2017 By Therapeutic Group Source: Pharmaprojects 11 Pharma intelligence | informa

  12. NAS Launches 2017 By Region Source: Pharmaprojects 12 Pharma intelligence | informa

  13. Other First-In-Class Products In 2017 – Part 1 • Holostem and Chiesi Farmaceutici’s Holoclar CELL AND GENE – an autologous corneal epithelial cell THERAPIES transplant therapy • TissueGene and Kolon Life Science’s Invossa for osteoarthritis – the first TGF-ß1 agonist • Puma Biotechnology’s Nerlynx (neratinib) CANCER • BioCryst Pharmaceuticals/Mundipharma’s Fodosine (forodesine) • AiCuris/Merck & Co.’s Prevymis (letermovir) ANTI-INFECTIVES • Maruhi Co.’s Amenalief (amanamevir) • Merck & Co.’s Zinplava (bezlotoxumab) in Clostridium difficile infections Source: Pharmaprojects 13 Pharma intelligence | informa

  14. Other First-In-Class Products In 2017 – Part 2 • AstraZeneca’s Fasenra (benralizumab) for RESPIRATORY severe asthma – an IL-5 receptor antagonist • Roche’s Hemlibra (emicizumab) – a HEMOPHILIA bispecific antibody to Factors IXa and X ENZYME • BioMarin Pharmaceuticals’ Brineura (cerliponase alfa) REPLACEMENT • Ultragenyx Pharmaceuticals’ Mepsevii THERAPIES (vestronidase alfa) Source: Pharmaprojects 14 Pharma intelligence | informa

  15. The drug R&D pipeline in 2018, and how it is changing 15 Pharma intelligence | informa

  16. Breakdown of pipeline by phase Source: Pharmaprojects 16 Pharma intelligence | informa

  17. Trends in clinical phase drug R&D Source: Pharmaprojects 17 Pharma intelligence | informa

  18. Pipeline quality assessment of late stage drugs Source: Biomedtracker 18 Pharma intelligence | informa

  19. Top 10 pharma companies by pipeline size Posi:on No of Drugs in Pipeline No of Originated 2018 (2017) Company 2018 (2017) Drugs 2018 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-Myers Squibb 134 (144) 96 Source: Pharmaprojects 19 Pharma intelligence | informa

  20. Top 10 pharma companies – are they delivering new drugs? Posi:on No of Drugs in Pipeline No of NAS Launches 2018 (2017) Company 2018 (2017) 2017 (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-Myers Squibb 134 (144) 1 (0) Source: Pharmaprojects 20 Pharma intelligence | informa

  21. Total companies involved in pharma R&D Source: Pharmaprojects 21 Pharma intelligence | informa

  22. Big pharma companies vs small – how the balance is shifting Source: Pharmaprojects 22 Pharma intelligence | informa

  23. Locations of pharma companies worldwide Source: Pharmaprojects 23 Pharma intelligence | informa

  24. The R&D pipeline by Therapeutic Area Source: Pharmaprojects 24 Pharma intelligence | informa

  25. Cancer taking an ever bigger slice of the R&D pie Source: Pharmaprojects 25 Pharma intelligence | informa

  26. Ongoing clinical trials by Therapeutic Area Source: Trialtrove 26 Pharma intelligence | informa

  27. Top 10 diseases/indications Posi:on No. of Ac:ve drugs 2018 (2017) Disease Trend 2018 (2017) 1 (1) Cancer, breast 727 (654) ↑ 2 (2) Cancer, lung, non-small cell 544 (477) ↑ 3 (3) Cancer, colorectal 503 (476) ↑ 4 (6) Cancer, ovarian 434 (386) ↑ 5 (4) Cancer, pancrea'c 430 (416) ↑ 6 (5) Diabetes, Type 2 407 (415) ↔ 7 (9) Cancer, prostate 381 (362) ↑ 8 (7) Alzheimer's disease 381 (376) ↔ 9 (10) Cancer, brain 361 (322) ↑ 10 (8) Arthri's, rheumatoid 352 (372) ↓ Source: Pharmaprojects 27 Pharma intelligence | informa

  28. Top diseases/indications – 11-25 Posi:on No. of Ac:ve drugs 2018 (2017) Disease Trend 2018 (2017) 11 (11) Cancer, melanoma 346 (312) ↑ 12 (12) Cancer, leukaemia, acute myelogenous 326 (285) ↑ 13 (15) Cancer, liver 272 (264) ↔ 14 (14) Pain, nocicep've, general 262 (268) ↔ 15 (19) Cancer, head and neck 258 (227) ↑ 16 (13) Psoriasis 256 (283) ↓ 17 (17) Cancer, myeloma 254 (246) ↔ 18 (18) Parkinson's disease 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) Chronic obstruc've pulmonary disease 179 (192) ↔ 24 (25) Pain, neuropathic, general 178 (169) ↔ 25 (24) Infec'on, HIV/AIDS 177 (183) ↔ Source: Pharmaprojects 28 Pharma intelligence | informa

  29. Top 10 Mechanisms of Action (pharmacologies) No. of Ac:ve % of Posi:on Compounds 2018 Compounds 2018 (2017) Mechanism of Ac:on (Pharmacology) (2017) PR/R/L 1 (1) Immunos'mulant 1501 (1736) 10.0 2 (2) An'cancer immunotherapy 1332 (889) 1.6 3 (-) Immune checkpoint inhibitor 211 (-) 3.3 4 (3) Immunosuppressant 208 (215) 26.4 5 (4) Angiogenesis inhibitor 169 (179) 17.8 Vascular endothelial growth factor 6 (5) 138 (123) 14.5 (VEGF)receptor antagonist 7 (7) Apoptosis s'mulant 116 (115) 14.7 8 (6) DNA inhibitor 111 (120) 20.7 9 (9) Opioid mu receptor agonist 110 (114) 40.9 10 (-) Radiopharmaceu'cal 108 (-) 10.2 Source: Pharmaprojects 29 Pharma intelligence | informa

  30. Top 10 Drug protein targets No. of Ac:ve Posi:on compounds 2018 (2017) Target Trend 2018 (2017) 1 (1) opioid receptor, mu 1 140 (143) ↔ 2 (4) erb-b2 receptor tyrosine kinase 2 [Her-2] 120 (113) ↔ 3 (6) vascular endothelial growth factor A 119 (105) ↑ nuclear receptor subfamily 3, group C, member 1 4 (2) 113 (123) ↓ (glucocor'coid receptor) 5 (3) tumour necrosis factor 113 (123) ↓ prostaglandin-endoperoxide synthase 2 (prostaglandin 6 (5) 107 (106) ↔ G/H synthase and cyclooxygenase) [COX-2] 7 (7) epidermal growth factor receptor 107 (104) ↔ 8 (8) insulin receptor 99 (94) ↔ 9 (9) opioid receptor, kappa 1 88 (87) ↔ 10 (10) glucagon-like pep'de 1 receptor 80 (82) ↔ Source: Pharmaprojects 30 Pharma intelligence | informa

  31. New drug protein targets identified by year Source: Pharmaprojects 31 Pharma intelligence | informa

  32. Biologicals advance towards 40% of R&D Source: Pharmaprojects 32 Pharma intelligence | informa

  33. Orphan drug designations and expedited reviews granted by year Source: Pharmaprojects 33 Pharma intelligence | informa

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