Prosensa – 2012 Fierce 15

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Hans Schikan, CEO of Prosensa

Based: Leiden, The Netherlands
Founded: 2002
CEO: Hans Schikan
Clinical Focus: Duchenne muscular dystrophy
Website: www.prosensa.eu

The Scoop: After 10 years of work, Prosensa is coming down to the pivotal wire on a new treatment for Duchenne muscular dystrophy. Its lead drug--PRO051/GSK2402968, an exon-51 skipping RNA drug designed to increase dystrophin expression--has significantly improved walking distance among boys in a small study. Among 10 boys evaluated in a clinical study for 12 weeks, the mean increase in walking distance was 35 meters compared to the baseline, with a range of minus 6 to plus 69 meters. 

"After two years we saw a mean increase of plus-11 meters," says CEO Hans Schikan, a Genzyme vet with a long track record in the hot rare-disease field. "You would expect boys would have declined over a two-year interval," he added, with losses likely to range from a minus 50 to 200 meters.

What Makes it Fierce?

DMD is a terrible inherited affliction that destroys the muscles and the lives of the boys it affects. Prosensa's exon-skipping technology corrects the genetic mutations that interfere with dystrophin expression, restoring an essential function that can delay and significantly correct the debilitating disease. And Prosensa has more than one program to consider; there are 6 that target various DMD populations.

The lead compound, 051, is being developed by GlaxoSmithKline ($GSK), which has been making a major investment in rare diseases. GSK licensed 051 three years ago for $25 million upfront and another $655 million in milestones. And the pharma giant has been keeping up its end of the deal.

Only a few weeks ago investigators stopped enrolling patients for the Phase III study, which is likely to read out in the second half of next year. And with 300 patients enrolled in several studies, including 180 in the pivotal study, there are several new key milestones to look forward to by the end of 2013.

GSK also has an option on the exclusive global rights on PRO044, the next most advanced program now in Phase I/II along with a follow-up program. And the biotech is keeping PRO045 and PRO053 "exclusively" for Prosensa.

For the first few years of its existence, patient groups like Charley's Fund largely sustained the company. Then the venture players stepped up. At the end of last year, Prosensa had €47 million in the bank after raising a total of €55 million in venture cash. The last €23 million--about $30 million--included a contribution from New Enterprise Associates, which picked the Dutch biotech as its first European play. And Prosensa's experience in fundraising over the years has helped create the game plan for other startups in the rare disease field.

Prosensa now has enough cash in the bank to fund operations through early 2014. So Schikan has options: Raise more venture funds, go public perhaps, or see how well GlaxoSmithKline's milestone payoffs can fund the company, which has 80 staffers.

Steady progress has placed Prosensa at the crossroads. Success in the next year will make this company a biotech to remember.

Investors: Include Abingworth, New Enterprise Associates, Life Sciences Partners, GIMV, AGF and MedSciences Capital, Charley's Fund

-- John Carroll (Twitter | LinkedIn)