No-nonsense drug discovery

Genzyme and PTC Therapeutics to develop small molecule for genetic diseases

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CAMBRIDGE, Mass.—Biotechnology company Genzyme Corp. and PTC Therapeutics, a developer of small molecule drugs that target post-transcriptional control processes, have joined forces to develop and commercialize PTC124, PTC's novel oral therapy in late-stage development for the treatment of genetic disorders due to nonsense mutations.

According to the agreement, announced July 17, PTC will commercialize PTC124 in the United States and Canada, while Genzyme will commercialize the treatment in all other countries. Genzyme will make a $100 million upfront payment to PTC Therapeutics, which will conduct and bear the expense for the Phase IIb trial of PTC124 in Duchenne muscular dystrophy (DMD), the Phase IIb trial in cystic fibrosis (CF) and two proof-of-concept studies in other indications to be determined. Once the four studies are completed, the companies will share research, development and commercialization costs equally.

PTC is also eligible to receive up to $165 million in development and approval milestones, the majority of which are to be paid upon approvals in Genzyme territories; and up to $172 million in sales milestones, contingent upon the achievement of specific sales levels, for a total potential of $337 million. The sales milestone payments begin when annual net revenue reaches $300 million, and increase in increments up to $2.4 billion.

PTC124, initiated in 2004, has already been granted orphan drug status for the treatment of DMD and CF due to nonsense mutations by the FDA and the European Commission.
Genzyme Chairman and CEO Henri A. Termeer says the collaboration is a strategic fit for Genzyme, which has extensive experience with cystic fibrosis and has developed Myozyme, a treatment for the genetic disorder Pompe disease, which is often treated by physicians who also treat DMD.

"Over the past two decades, Genzyme has successfully developed four therapies for patients with severe genetic diseases. PTC124 is a powerful new approach that holds great potential to help CF and DMD patients, and many others with a variety of devastating diseases," Termeer said in a statement released by Genzyme.

Dr. Stuart W. Peltz, PTC's president and CEO, says the collaboration supports PTC's goal of establishing a fully integrated biopharmaceutical company by retaining commercial rights in the United States and Canada, while engaging an experienced partner to address additional markets. The collaboration will also fill a significant unmet medical need, he adds.

"This partnership will fulfill our dream of not just developing drugs, but also commercializing them," Peltz says. "PTC124 is exciting because it brings to fruition the concept of personalized medicine. PTC124 is a investigational drug that helps treat the underlying disease due to a type of mutation. A subset of patients in hundreds, if not thousands, of different genetic diseases may be helped by this drug. I think this will have a significant impact on how we think about making drugs, where you not only need to know about the disease, but also the genotype of the disease, down to the nucleotide level." DDN

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