Innoboot 2016 event by SMB Life Sciences, Radboud University and Novio Tech Campus on April 12, 2016. Inspiring presentation by Hans Schikan (former CEO Prosensa) on the rollercoaster of entrepreneurship in Life Sciences.

1. Entrepreneurship in Life Sciences:
The Rollercoaster
Innovation Bootcamp
Nijmegen, April 12, 2016
Hans Schikan, former CEO Prosensa

2. Entrepreneurship in Life Sciences:
The Rollercoaster
Innovation Bootcamp
Nijmegen, April 12, 2016
Hans Schikan, former CEO Prosensa

3.  Founded in 2002, In Leiden
 Focus on rare diseases
 Partner with patients
 RNA modulation platform
 IPO-ed on NASDAQ (ticker symbol RNA)
 Pioneer in Duchenne Muscular Dystrophy (DMD)
 4 compounds in clinical development; lead asset in registration phase
 Recently acquired by BioMarin
Corporate Highlights

4. 0 5 10 15 20 25 30
death
ventilation 24h
ventilation at night
very limited use of arms
wheelchair - skeletal deformity
walking problems
Age
Severely debilitating and invariably fatal progressive neuromuscular disease
Duchenne Muscular Dystrophy (DMD)

5. Healthy control
Duchenne patient
Cause: absence of dystrophin in muscles
Duchenne Muscular Dystrophy

6. Indication Compound Discovery Pre-Clinical Phase I/II Phase III
Drisapersen
PRO044
PRO045
PRO053
PRO052
PRO055
PROSPECT
PRO135
PRO289
Duchenne
Muscular
Dystrophy
Myotonic
Dystrophy
Huntington’s
Disease
R&D Pipeline
13% of DMD patients
6% of DMD patients
8% of DMD patients
8% of DMD patients
4% of DMD patients
2% of DMD patients

7. Four DMD patients
Single intramuscular 0.8 mg dose
Biopsy performed after 28 days
Effective in specifically inducing exon
51 skipping and dystrophin
restoration
Safe and well-tolerated
First Human Study

8. Immunohistochemical Analysis
First Human Study

9. Landmark Publication 1

10. Landmark Publication 2
N Engl J Med 2011; 364:1513 - 1522

11. the biotech dilemma
to partner or
not to partner

13. COME
COME
A FULLY
INTEGRATED
COMPANY

14. 2009

15. Strategic Partnership with GSK

17. Glaxo’s $679 Million Accord Signals Foray Into Rare Diseases
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By Trista Kelley
Oct. 13 (Bloomberg) -- GlaxoSmithKline Plc agreed to pay as much as 460 million euros ($679 million) to develop drugs from closely
held Dutch biotechnology company Prosensa aiming to treat a rare disease called Duchenne muscular dystrophy.
The London-based company will get rights to develop and sell Prosensa’s most advanced experimental treatment, called PRO051,
and three others as part of the research collaboration the companies announced today in an e-mailed statement.
Glaxo joins Genzyme Corp. of the U.S. and Switzerland’s Santhera Pharmaceuticals AG in developing therapies for Duchenne, a
rare neuromuscular disease with no known cure that affects one in 3,500 newborn boys. PRO051, scheduled to enter the final stage
of human tests next year, may get orphan-drug designation, a benefit extended to medicines that address rare diseases,
spokeswoman Claire Brough said. The status would allow Glaxo to charge more and give the product a seven-year monopoly in the
U.S.
“This is a move to have a much broader portfolio base,” WestLB analyst Simon Mather said in an interview. “Orphan drugs and
specialty pharma is attractive because you can charge a higher price and you don’t need to do much marketing.”
Chief Executive Officer Andrew Witty has struck about a dozen acquisitions or development partnerships since taking over in May
2008 in an effort to replace revenue that will be lost when generic treatments rival Glaxo’s best-sellers.
Most of Witty’s deals so far have focused on expansion in emerging markets and consumer products.
Defending Margins
“The problem with the strategy until now is that they’ve been furthering the consumer and emerging-market credentials, which tend
to be low-margin,” Charles Stanley & Co. analyst Jeremy Batstone-Carr said in an interview. “So perhaps with this deal, Glaxo is
cognizant that it has to defend its margins.”
Under the terms of the agreement, Glaxo will pay Prosensa 16 million pounds ($25 million) upfront, as much as 412 million pounds
for reaching development targets, and “double-digit” royalties on potential sales.
Moncef Slaoui, Glaxo’s head of research and development, last month told Bloomberg that the company was preparing a move into
so-called orphan drugs. Brough declined to say whether the company would further expand into the area.
Duchenne muscular dystrophy is characterized by progressive muscle weakening that eventually affects the heart and breathing,
and survival is rare beyond age 30, according to the Muscular Dystrophy Association.
Bloomberg news

19. Prosensa’s Partnerships

20. Investors: Abingworth; LSP; MedSciences Capital
Investors: Abingworth; GIMV; Idinvest Partners; LSP; MedSciences Capital
Investors: Abingworth; GIMV; Idinvest Partners; LSP; MedSciences Capital; NEA
Jan 2012 EUR 23M
Dec 2008 EUR 18M
Jan 2007 EUR 13.5M

29. Runners Action Duchenne
May 12th 2013

30. Ticker Symbol

31. Title

35. RNA
-70%

39. An angry investor
“This company is based in Netherlands, where drugs and
prostitution is legalized. RNA should focus on pimping
business instead of biotech...what bunch of morons”

40. Prosensa still hopeful
Prosensa will continue

41. January 13, 2014
JP Morgan
Healthcare Conference
San Francisco

42. Press Release Jan 13, 2014
…Prosensa has regained all rights from GSK to
drisapersen and will retain rights to all other
programs for the treatment of Duchenne Muscular
Dystrophy…
...Prosensa will now have the full, unencumbered
rights to continue the development of drisapersen
as well as each of its DMD programs...

44. FDA meeting January 24, Washington DC

47. Status Update