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160412 innoboot 2016 hans schikan the rollercoaster

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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.

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Entrepreneurship in Life Sciences:
The Rollercoaster
Innovation Bootcamp
Nijmegen, April 12, 2016
Hans Schikan, former CEO Prosensa
Entrepreneurship in Life Sciences:
The Rollercoaster
Innovation Bootcamp
Nijmegen, April 12, 2016
Hans Schikan, former CEO Prosensa
 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
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)
Healthy control
Duchenne patient
Cause: absence of dystrophin in muscles
Duchenne Muscular Dystrophy
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
Ad

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160412 innoboot 2016 hans schikan the rollercoaster

  • 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
  • 10. Landmark Publication 2 N Engl J Med 2011; 364:1513 - 1522
  • 11. the biotech dilemma to partner or not to partner
  • 14. 2009
  • 17. Glaxo’s $679 Million Accord Signals Foray Into Rare Diseases Share | Email | Print | A A A 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
  • 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
  • 31. Title
  • 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”
  • 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