It may not be the most complimentary assessment of a biotech’s initial public offering to say that if PTC Therapeutics can reach its public offering target of $125 million, then the market really is coming back for IPOs. But to be sure, the 15 year old company has had a couple of significant setbacks on the way to developing its lead drug, Ataluren– a treatment for Duchenne muscular dystrophy and cystic fibrosis that involves correcting a “nonsense”mutation.
The drug failed to meet expectations on a Phase 2b study for Duchhenne muscular dystrophy as well as a Phase 3 study for cystic fibrosis. But it it is currently in the midst of additional Phase 3 trials, taking on board what it’s learned from past experience, as the company notes in its S1 filing:
“We did not achieve the primary efficacy endpoint in either trial with the pre-specified level of statistical significance. However, we believe that the collective data from these trials, including retrospective and subgroup analyses that we have performed, provide strong support for concluding that Ataluren was active and showed clinically meaningful improvements over placebo in these trials.”
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The nonsense mutation is the cause of Duchenne muscular dystrophy in about 13 percent of patients, or 2,000 people in the United States and 2,500 in the European Union. The company also plans to treat nonsense mutations in other genetic disorders.
The South Plainfield, New Jersey company began trading on the NASDAQ today under the symbol PTCT. After opening at $15.89, at the high end of its $13 to $16 range, It had jumped 11 percent and as of 11:15 am it was $16.65.
Although the days of IPOs as an exit for biotech companies are past, it still is a useful financing vehicle and quite a few companies are taking advantage of a receptive market.
This week Iroko Pharmaceuticals, a drug developer with a novel nonsteroidal anti-inflammatory drug to treat mild to chronic pain, which recently opened an office at the Navy Yard in Philadelphia near GlaxoSmithKline’s offices, filed a registration statement for an IPO this week. Onconova Therapeutics in Newtown, Pennsylvania indicated last week it will also go public.
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[Photo credit: Scrum from BigStock Photos]
