Prosensa Corporate Presentation. Biotech-Middag. November Management Board Prosensa

Transcription

1 Prosensa Corporate Presentation Biotech-Middag November 2012 Management Board Prosensa

2 Our Mission To develop innovative, RNA-based therapeutics to fill unmet medical needs for patients with rare genetic diseases 1

3 Corporate Highlights Rare disease company Lead compound in Phase III for Duchenne Muscular Dystrophy Encouraging efficacy data from long-term extension study Key collaboration with GSK Deal value up to USD 680M for selected part of DMD franchise Significant retained assets in DMD for own commercialization RNA modulation platform, applicable to rare and common diseases Management experience in rare diseases & strong investor basis 2

4 Duchenne Muscular Dystrophy Rapid progression of muscle degeneration, due to the absence of dystrophin 75,000 patients in the developed countries Big impact on patients and families / caregivers Age Rare, severely debilitating progressive disease walking problems wheel chair - skeletal deformity very limited use of arms ventilation at night ventilation 24h death Clinical symptoms DMD 3

5 Duchenne Muscular Dystrophy Cause: no dystrophin protein in muscles Picture 1. Healthy control Picture 2. Duchenne patient 4

6 The Dystrophin Gene PRO044 PRO051 PRO Aartsma-Rus & van Deutekom, Antisense Elements (Genetics) Research Focus, 2007 Nova Science Publishers Exon skipping can restore the reading frame 5

7 R&D Pipeline Indication Compound Discovery Pre-clinical Phase I/II Phase III Duchenne PRO051 13% drisapersen PRO044 6% PRO045 8% PRO053 8% PRO052 3% PRO055 3% PRO000 40% Myotonic Dystrophy PRO135 Huntington s Disease PRO289 6

8 Strategic Partnership with GSK Exclusive worldwide collaboration for the development and commercialization of RNA based therapeutics for DMD Worldwide license to develop and commercialize PRO051 Three additional compounds under option (including exon 44) Prosensa retains commercial participatory rights Prosensa has an option to expand its commercial rights in certain European countries GBP 16M (USD 25M) upfront GBP 412M (USD 655M) in milestones If all four compounds successfully developed Double-digit royalties on product sales 7

9 Drisapersen Systemic Clinical Study Design 12 patients / 4 groups 5 weekly s.c. injections Escalating dose ( mg/kg) Endpoints Pharmacokinetic profile Safety parameters Muscle biopsies at two time-points: RNA and protein effects CK levels Muscle strength/performance 8

10 Drisapersen Systemic Clinical Study Stable dystrophin in all treated samples Dose response observed Safe and well tolerated by all patients Subcutaneous administration easy and effective No neutralizing antibodies detected in any patients Pre-treatment Dose * *Revertant fiber 9

11 Drisapersen Landmark Publications N Engl J Med 2011; 364:

12 Drisapersen Extension Study > 3 Years May Week mg/kg 2 mg/kg July/August 2009 Extension Study Ongoing October mg/kg 6 mg/kg 5-week treatment 13-week follow-up 6 mg/kg/wk (n=12) Week Muscle biopsy 3/2011 NEJM 4/2011 AAN 6-minute walk tests Longest running treatment study in Duchenne muscular dystrophy All 12 patients still on treatment since July/August

13 6-Minute Walk Distance, m Functional Outcome 6MWT Conceptual representation of walking distance performance by DMD patients and healthy controls Age 12

14 Natural History - 6MWT +13 m -57 m McDonald et al, Muscle & Nerve; Volume 42, Issue 6, pages , December

15 Drisapersen - 48 Weeks Results Change in 6MWT from extension baseline Subjects able to complete the 6MWT at extension baseline n Mean m SD m Median m Range m Week to +69 Week to +115 Week to +127 N=10 (subjects who completed all 6-minute walk test [6MWT] assessments). One subject stopped test early and one subject was non-ambulant at baseline. 14

16 More than 300 Patients in Clinical Program Completed Enrolled PRO051-CLIN-02 Repeat dose escalation (5wks) 12 Subjects, mg/kg/week 12 Subjects; 6mg/kg/week Extension study ongoing (more than 3 years) DMD Dose-escalation, placebo controlled (3:1) 18 Non-ambulant subjects -> 12mg/kg single dose PK/safety DEMAND II/DMD DEMAND III/DMD Dose regime comparison, placebo-controlled (2:2:1:1) 53 Subjects; 6mg/kg, 24 wks efficacy/48 wks safety Pivotal study, placebo-controlled (2:1) 186 Subjects; 6mg/kg/week, 48 weeks DEMAND IV/DMD & 117 subjects Extension study DEMAND V/DMD Dose comparison, placebo-controlled (2:2:1:1) 54 Subjects; 3 or 6mg/kg/week, 48 weeks DEMAND VI Natural History study 250 subjects (aged 3 18 years), 3 year follow-up 15

17 Intellectual Property FTO analysis performed in DMD Fully enabled to commercialize exon-skipping DMD drugs Good level of IP protection in both Europe and US: Issued claims in Europe (DMD exon claims) and US Successful outcome opposition procedure in EU in Nov 2011, IP position lead product further strengthened Successful outcome re-examination procedure in US in Feb 2012 Pending claims in both Europe and US Broad set of patent families Orphan Drug designations granted in Europe and the US 16

18 News Flow EUR 23M financing, led by New Enterprise Associates (Q1-2012) Follow-on exons milestone of EUR 12M achieved (Q2-2012) Recognition as Fierce 15 company (Q4-2012) All regulatory studies with drisapersen fully recruited (Q4-2012) Presentation of 141 week results in extension study (Q4-2012) Orphan Drug designation for PRO052 and PRO055 (Q4-2012) First Prosensa retained DMD compound enters clinic (H1-2012) Top-line results pivotal study (Q4-2013) 17

19 Leadership Management Board CEO - Hans Schikan (Genzyme) CFO - Berndt Modig (Jerini) CMO - Giles Campion (GE-Healthcare) CBO - Luc Dochez (Tigenix) Supervisory Board Daan Ellens (Chair) Stephen Bunting (Abingworth) Rémi Droller (Idinvest Partners) Peter Goodfellow Jim Van Heusden (Gimv) Martijn Kleijwegt (LSP) David Mott (NEA) 18

20 Corporate Highlights Rare disease company Lead compound in Phase III for Duchenne Muscular Dystrophy Encouraging efficacy data from long-term extension study Key collaboration with GSK Deal value up to USD 680M for selected part of DMD franchise Significant retained assets in DMD for own commercialization RNA modulation platform, applicable to rare and common diseases Management experience in rare diseases & strong investor basis 19

21 Inspiration Just wanted to acknowledge your great success with GSK and thank you for working so hard to possibly save this generation of boys! To think that this could really become a reality and that I might not have to watch my only grandson die before me just sends chills up my spine! 20

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