バイオベンチャーの現状と課題 - meti.go.jp

1 - - - 2 10 I II II II III III 8PJ I II III VC 3 27 471 M&A 4 DATA BOOK 2017 30 6 30 17 PMDA PMDA Phase3 5 6 / DNA DNA/RNA ~50 ~10

2 ~10 ~1000 ~500 ~10 ~10 1899 1982 2001 Kymriah/Yescarta 2017 Luxturna 2018 Exondys51 2016 1983 /Phase1 Sarepta Kite Gilead Spark 1900 7 Active IngredientApproval DateApproval Year Nusinersen12/23/20162016 IonisPharmaceuticals Rucaparib12/19/20162016 Pfizer Crisaborole12/14/20162016 AnacorPharmaceuticals Bezlotoxumab10/21/20162016 Bristol-Myers Squibb Olaratumab10/19/20162016 ImClone Systems Eteplirsen9/19/20162016 SareptaTherapeutics Lixisenatide7/27/20162016 Zealand Pharma lifitegrastophthalmic solution7/11/20162016 Sunesis Pharmaceuticals sofosbuvirand velpatasvir6/28/20162016 Gilead Sciences obeticholicacid5/27/20162016C2N Diagnostics Daclizumab5/27/20162016 PDL BioPharma Atezolizumab5/18/20162016 Genentech Pimavanserin4/29/20162016 ACADIA Pharmaceuticals Venetoclax4/11/20162016 Abbott Laboratories/Roche defibrotide sodium3/30/30162016 Crinos Reslizumab3/23/20162016 Cel-Sci Ixekizumab3/22/20162016 Eli Lilly Obiltoxaximab3/18/20162016 EluSysTherapeutics Brivaracetam2/18/20162016 UCB elbasvirand grazoprevir1/28/20162016 Merck FDA 2016 2016 FDA 20 5 15

3 4 10 4 6 2017 7 2017 10 8 9 * 1 2010 2011 5~20% / 1311712763725321361913365827825206021025 00100200300400500600700800900 Phase 1 Phase 2 Phase 3 * * SM Paul et al.

4 , How to improve R&D productivity: the pharmaceutical industry s grand challenge , Nature Review Drug Discovery 9, 203-214 (2010) 363 833 1,894 2,178 8,753 22,422 29,734 3,081 4,485 2,596 6,245 4,197 6,902 6,222 05,00010,00015,00020,00025,00030,0002009 201020112012201320142015[$M]IPO M&A 10 M&A IPO NASDAQ IPO M&A IPO M&A M&A 1 IPO Industry Eye 15 , (2015) 2009201020112012201320142015 IPO 4111321367053M&A 103117941121031268511 71632834351784757 755281187 7386 Phase1 Phase3 Phase2 51%49%28%72% 2008 2014 / 76 6638374453513051 (104 ) (81 ) 4422 1819 12 5 (11 -16 ) FDA +402% 2016 13 (2006-16 )

5 +584%+505% BiogenCelgeneBioMarin16%37%82%100% 1405,000,00010,000,00015,000,00020,000,0 0025,000,000 [$M] Vistide AIDS CMV Tamiflu Roche Hepsera B Emtriva HIV Viread Tamiflu 1B Truvada Pharmasset Sovaldi Sovaldi/Harvoni Kite CAR-T Truvada HIV Sovaldi/Harvoni C Gilead Sciences M&A GileadSciences, Inc. 05,000,00010,000,00015,000,00020,000,000 AmgenCelgeneGil eadBiogenRegeneronShireVertexAlexionIncy teIllumina(Genentech) ( Genentech)

6 15 NBI Top10 3 20 - - - 16 17 3 2 30 15 18 J-TEC 3D HD HMT DNA PSS / / UMN DWTI JCR 30 2015 6 19 2007200820092010201120122013201420152016 2017

7 14974 20,982-21520,27856018,91342619,32555318, 7371,09719,5781,54720,5641,69123,9051,95 425,9692,30229,7292,66729,3753,20224587 ----------269-186781608182012,4741,3914, 3282,5484,8952,49034565 740-6,651709-6,270152-3,667919-1,854716- 1,876862-1,9621,9588532,0697563,6711,043 8,1511,07518,90112,38944592 --------------204-5843,2292,2481,174-1,1 25949-1,93257774J-TEC 103-973111-1,071114-1,102211-1,067351-1, 145473-1,107563-1,1031,008-1,0251,321-68 81,430-7222,13531264564 804-1,3491,973-3853,327275,2573185,36122 36,2231,1843,277-1,1131,017-3,785769-1,9 62266-2,980286-3,00474571 103-717262-452353-531117-49284-519346-33 3373-526472-1,123675-1,108243-2,082218-2 ,71287777 --------158-4821,10735432-999107-1,51899 -1,903141-1,814615-1,24092160 274-1,285204-94981-442272-26990-457161-4 64183-785474-3781,016-6331,306-276--1045 85 --------1,000-1,075108-2,07293-4,4211,10 8-3,942202-3,20771-3,564--114596 ------------------870-4,121--124583 ------463-231633-7324-413434-708186-8202 80-1,269252-1,042--132191 269425461099742331,1451441,322711,544221 1,539231,865-2931,909-6011,801-621--1423 70 1,606-8231,838-6672,9002933,2023342,674- 3032,190-7112,110-8771,843-1,4071,674-1, 7411,909-1,681--154576 --------20-351112-25780-20880-19661-2901 68-319--164591 --------------151-4144796121-53293-78517 4875 ( ----------803-10,9456.)

8 003-4,0210-9,2230-8,8220-10,881--184563 1,720-2,040951-2,685585-2,610286-2,01024 3-2,100444-1,785491-1,363909-2,273430-4, 171514-4,763--194572 --500-296687-344571-398592-393510-457771 -300611-6341,569472811-423--204582 ------1,449-6121,882-2,0661,955-1,7001,5 32-1,6801,955-1,3031,933-2,5512,368-2,12 7--214586 --------741-43487-56768-60426-1,00337-99 922-1,342--224584 ----------207-31660-358301-512321-8241,1 60-8201,089-1,184234579 ------1,186-1,345684-1,91628-2,636228-2, 137153-2,183145-1,864705-759--244575 ----161-245148-4800-1,4200-1,1030-6510-4 8360-283105-399109-406257776 ----87-78566-1,20486-1,41875-846105-5348 6-601193-568100-1,413--264588 ------------1-66528-827121-951178-861--2 74552 JCR 8,544-16311,87128112,08254614,3872,00714 ,4571,40712,8451,08914,0991,15015,7051,5 4516,8552,01417,4382,15218,0852,36228459 3 --------------279-56898-1,06077-3,507--2 94594 ----------------821-492822-988529-1,1133 04597 ---------------------- 10 / 60%15%24%30%3%66%12%2%85%77%14%8%26%2%69 %18%0%6%75%55%26%18%21%76%37%2%61%10%89% 20 60%15%24%NBIG rowthINDEXV alue/YIELD/GARPO thers / 4,000 2,000 156 (Top4 )

9 AgiosPharmaImpaxLaboratories 81 Swedish OrphanEvotec 42 Medy-Tox 30 21 GrowthGrowth 1-Year Revenue Growth Aggressive Growth 40 Growth P/E P/E 40 Growth Aggressive Growth 1-Year Revenue Growth 75 Aggressive Growth P/E P/E 50 Aggressive Growth IndexIndex / ValueValue P/B Yield Deep Value P/B 70 Value P/E P/E 70 Value Deep Value Price-to-Book (P/B) Yield P/B 90 Deep Value Price-to-Earnings (P/E) P/E