Genzyme is walking away from a troubled program at PTC Therapeutics after spending $100 million back in 2008 to gain rights in certain markets for ataluren, which is being evaluated to treat rare diseases linked to nonsense mutations.
The program fell on hard times in early 2010 when PTC flunked a Phase IIb trial for the drug against nonsense mutation Duchenne/Becker muscular dystrophy. Data from the study failed to show the molecule could provide a statistically significant change in 6-minute walk distance. Today, the collaborators announced Genzyme would be returning its rights to the compound outside of the U.S. and Canada to PTC while getting an option to commercialize the drug in indications other than Duchenne/Becker. PTC says it's continuing to develop the drug for that form of muscular dystrophy and nonsense mutation cystic fibrosis.
Genzyme, the world's largest provider of rare-disease drugs, has been a unit of French drugmaker Sanofi ($SNY) for the past 5 months and has been reviewing its R&D projects. Clearly, the PTC program didn't make the cut.
"The decision to restructure this agreement is a result of a recent portfolio assessment, and will enable us to intensify our focus on other areas of development within Genzyme where we believe we have the best ability to develop therapies to benefit the rare disease community," David Meeker, Genzyme's chief of operations, said in statement.
PTC, fortunately, has multiple collaborators. For instance, back in June, the developer inked a licensing deal with AstraZeneca ($AZN) to discover and develop treatments for cancer and other diseases.
- here's the release