PTC Therapeutics

Fierce 15 Winner

Based: South Plainfield, NJ
Founded: 1998
Website: www.ptcbio.com

The Scoop: "PTC has combined clear data supporting the broad applicability of PTC124 with a genius for raising money through partnerships and grants in a way that has provided the company ample funding and a bright future."

What makes it Fierce: Earlier this year, PTC Therapeutics announced Phase II results for an experimental therapy that normally wouldn't have excited tremendous interest. Its lead candidate, PTC124, was effective in treating a particular group of patients with Duchenne muscular dystrophy triggered by nonsense mutations. Those nonsense mutations are responsible for about 13 percent of all cases of DMD. But the study also highlighted that PTC124 could be the key to dealing with the nonsense mutations involving an array of more than 1,800 genetic disorders like cystic fibrosis (10 percent of cases) and Hurler's disease (70 percent of cases).

"I always knew this was a very important drug," says Stuart Peltz, Ph.D., the CEO, co-founder and a scientist who's keenly aware of the potential of a therapy like PTC124. "It has tremendous applicability."

"We've always been attached to our flexibility," adds Peltz. In fact, Peltz was so attached to flexibility that the company recently decided to abandon its pursuit of an IPO, comfortable that it had the financing to keep a substantial staff of 130 on track to mid-2009, when even more efficacy data would likely propel the value of the company to a significantly higher level.

Peltz learned at Rutgers how to gain grants, and he's employed that knowledge to great effect at PTC Therapeutics, garnering tens of millions in grants as well as government contracts. The company has also earned significant operating capital through partnerships. PTC has inked deals that have delivered $42 million in up-front payments alone. In January, Pfizer put up $10 million to gain access to PTC's GEMS technology with a potential $121 million in milestones for each of up to 10 identified targets. GEMS tech focuses on post-transcriptional control (PTC) mechanisms, the regulatory events that take place after a messenger RNA molecule is copied from DNA. Other deals have been signed with CV Therapeutics and Schering-Plough.

What to look for: Peltz says he expects to sign one or two more partnerships revolving around the use of their technology. On the discovery side, he plans to keep breaking new scientific ground and adding to the value of the company, which can be translated into cash value during a later IPO.

PTC Therapeutics
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