ThromboGenics NV: from an Academic Research Lab to a Biopharmaceutical Company. Désiré Collen, Founder & Chairman of ThromboGenics NV

Transcription

1 ThromboGenics NV: from an Academic Research Lab to a Biopharmaceutical Company Désiré Collen, Founder & Chairman of ThromboGenics NV 1

2 Important assets for Biopharmaceutical developments. 1. Intellectual Property 2. A business environment with adequate incentives by the authorities 3. Smooth arms length interaction with academia 4. Focussed and flexible programs for translational research 5. Money 2

3 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 3

4 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA Agreement wit LRD VZW (1976) Research on the regulation of fibrinolysis Patent application t PA (1980) Agreement with Genentech ( ) First patient treated with melanoma t PA in 1981 Royalty return ( ) Litigation jury verdict II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 4

5 5

6 Activator t PA upa SK Sak Plasminogen Plasmin α 2 Antiplasmin Fibrin FDP 6

7 Regulation of fibrinolysis: plasminogen activator as a thrombolytic agent D. Collen Pathobiology of the Endothelial Cell pag

8 8

9 9

10 April 15 thrombotic mass in the iliofemoral vein, protruding in the vena cava and occluding renal vein May 8 After tpa therapy: normal patency of renal vein and normal contrast dilution in open iliofemoral vein 10

11 ROYALTY PAYMENTS Year Amount 1988 $ 1,284, $ 4,963, $ 6,690, $ 7,343, $ 7,192, $ 7,051, $ 8,849, $ 10,747, $ 11,941, $ 11,654, $ 10,904, $ 9,187, $ 9,843, $ 7,602, $ 4,618, $ 6,214, $ 6,856, $ 7,203, $ 3,509, Donations to DCRF (± 50%) Sale (675 MBF)/Donation (600 MBF) to Collen Trust/Biggar Total $ 143,662,

12 12

13 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research Constitution of DCRF (1988) Co financing 9 th floor GHB (1992) Expansion of CMVB Development of VIB programs (1994) III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 13

14 14

15 15

16 9th floor Katholieke Universiteit Leuven, Belgium Campus Gasthuisberg KU LEUVEN Center for Molecular and Vascular Biology Director H.R. Lijnen, Ph.D. Vesalius Research Center Director P. Carmeliet, M.D., Ph.D. ThromboGenics ThromboGenics N.V. Chairman D. Collen, M.D., Ph.D. 16

17 ThromboGenics Relationship With KULeuven, the VIB, and the IWT KUL, VIB ThromboGenics R&D ThromboGenics Vesalius Research Center, VIB and Center for Molecular and Vascular Biology, KULeuven 200 Researchers 20 million annual budget Provides Basic Scientific Research Licenses and Develops Key Proprietary Technologies Develops programs through (early pilot) clinical trials Speed/flexibility, cost control Commercialises Cardiovascular and Ophtalmic Products Conducts Clinical Trials Raises Funds Experienced Management Team Total 130 people 17

18 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development Constitution of TG Ltd Financing of TG Ltd Development of project portfolio V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 18

19 ROYALTY PAYMENTS Year Amount 1988 $ 1,284, $ 4,963, $ 6,690, $ 7,343, $ 7,192, $ 7,051, $ 8,849, $ 10,747, $ 11,941, $ 11,654, $ 10,904, $ 9,187, $ 9,843, $ 7,602, $ 4,618, $ 6,214, $ 6,856, $ 7,203, $ 3,509, Donations to DCRF (± 50%) Sale (675 MBF)/Donation (600 MBF) to Collen Trust/Biggar Total $ 143,662,

20 Development of Capital of ThromboGenics Ltd 20

21 ThromboGenics Ltd portfolio microplasmin microplasmin Staphylokinase Anti FVIII Anti PlGF 21

22 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke Stroke infarct size in gene targeted mice Patent for compounds that reduce α 2 antiplasmin Microplasmin Patent for recombinant microplasmin Phase II study in stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 22

23 23

24 Microplasmin for treatment of stroke Clinical Phase IIa Objectives - Safety and efficacy - > 4 to 12 hours after event - 1 application - Dose ranging Protocol - Multi-center - Double masked - Placebo controlled Results Presented at World Stroke Congress (Sep 2008) -Well tolerated Future - Promising preliminary findings on reperfusion and secondary markers of neurovascular protection ThromboGenics will make no further investment in microplasmin for stroke indication without a partner 24

25 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma Letter from Dr. M. de Smet, 25 MAY 2001 Prospectus IPO 2006: Clinical development outlined VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 25

26 26

27 THROMBOGENICS NV Prospectus for IPO June 2006 page

28 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments Portfolio Financing rounds Clinical trials with ocriplasmin in svma ThromboGenics highlights VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 28

29 Focus R&D activities to maximize investment (2006) PRECLINICAL CLINICAL DRUG CANDIDATE INDICATION Discovery Development Phase I Phase II Phase III 29 Microplasmin Microplasmin Microplasmin Microplasmin Staphylokinase Anti-factor VIII (TB-402) Eye disease - Vitrectomy Diabetic retinopathy Stroke Intravenous / Intra-arterial Peripheral vascular disease (DVT/PAOD) Acute myocardial infarction Deep vein thrombosis Atrial fibrillation Anti-PIGF (TB-403) Cancer PIGF Anti-GPIb (6B4) Age related macular degeneration Coronary artery disease, Peripheral arterial occlusive disease Acute coronary syndrome, Thrombotic thrombocytopenic purpura Anti-VPAC Thrombocytopenia Visual disorders Cardiovascular disease 29

30 To Date over 800 Patients were Treated with Ocriplasmin in 8 Clinical Trials Ongoing trials Completed trials 30

31 Out of the microplasmin treated patients 44% achieved Macular Hole closure at one month FTMH closure (%) All Patients *** Patients without protocol violations *** (n=32) (n=57) 0 (n=26) (n=46) ***P Placebo Microplasmin 31

32 Guarantee Financial Resources to Achieve Corporate Goals since IPO raised a total of > 340M Pre IPO capital rounds (71 mio from DCRF/DC) and 14 mio from Easthill IPO 2006 ( 35M) Out licensing TB 403 to Roche 2008: upfront payment of 30M Private placement 2007 ( 23M) Private placement 2009 ( 43M) Private placement 2010 ( 56 M) Private placement 2012 ( 78 M) Partnership with Alcon 2012: upfront payment of 75M 32

33 ThromboGenics Highlights Lead product Ocriplasmin Positive Phase III clinical trials Submission to the EMA and FDA Anticipated approval and commercial launch end of 2012/begin 2013 Market capitalization > 1 B 33

34 Management adopting a Common Sense Approach to ensure a Successful Launch of Ocriplasmin Stepwise expansion of organizational struture Profiles with big pharma experience and a good sense of entrepreneurship Matrix structure /flat structure Empowerment of the employees 34

35 Board of Directors of ThromboGenics NV,

36 36

37 ThromboGenics NV: Development Stages I on Research at CMVB KUL leading to t PA II on D. Collen Research Foundation, VZW for academic research III on Thromb X NV to develop Staphylokinase for AMI IV on ThromboGenics Ltd (Ireland) to optimize financing of clinical development V on Thromb X NV and microplasmin for stroke VI on Thromb X NV and microplasmin for svma VII ThromboGenics NV: IPO for financing of phase II/phase III developments VIII ThromboGenics NV progressive prioritization of microplasmin (ocriplasmin) for svma IX. Conclusions 37

38 Important assets for Biopharmaceutical developments. 1. Intellectual Property Of vital importance but 2. A business environment with adequate incentives by the authorities Major efforts by the (Flemish) government of essential importance 3. Smooth arms length interaction with academia Via VIB and tech transfer depts of universities 4. Focussed and flexible programs for translational research Pragmatic changes of orientation 5. Money t-pa royalties as seed money 38