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Under the terms of the agreement, PTC will commercialize PTC124 in theUnited States and Canada, and Genzyme will commercialize the treatment in allother countries. Genzyme will make an up-front payment of $100 million toPTC, plus potential milestone and royalty payments. PTC will be financiallyresponsible for one ongoing and three additional clinical trials of PTC124,which is potentially applicable to hundreds of genetic diseases.
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PTC124 is currently being evaluated in a phase 2b trial for Duchennemuscular dystrophy (DMD), and a phase 2b trial in cystic fibrosis (CF) isexpected to begin by the end of this year. In its collaboration with PTC,Genzyme will draw on its expertise in genetic disorders and its strongregulatory, manufacturing and marketing infrastructure outside of the UnitedStates. Genzyme has extensive experience with cystic fibrosis, havingconducted more than six clinical trials among CF patients. In the field ofDMD, Genzyme's experience with Myozyme(R) (alglucosidase alfa), for thetreatment of the genetic disorder Pompe disease, will be directly applicableas patients with both diseases are treated by the same specialist physicians.
"Over the past two decades, Genzyme has successfully developed fourtherapies for patients with severe genetic diseases. PTC124 is a powerful newapproach that holds great potential to help CF and DMD patients, and manyothers with a variety of devastating diseases," stated Henri A. Termeer,Genzyme's chairman and chief executive officer. "This collaboration is anexcellent strategic fit for Genzyme and will be managed within the company'sstated financial guidance."
"One of PTC's earliest scientific insights was that targeting nonsensemutations represented a novel approach to treating a large number of geneticdisorders. The translation of that insight through the discovery and rapiddevelopment of PTC124 has been very gratifying," commented Stuart W. Peltz,Ph.D., PTC's president and chief executive officer. "This collaborationsupports PTC's business strategy of establishing a fully integratedbiopharmaceutical company by retaining commercial rights in the United Statesand Canada while engaging an experienced and capable partner to swiftlyaddress additional markets."
PTC initiated the clinical development of PTC124 in 2004. Based on phase2a clinical proof of concept in both DMD and CF, further development in eachof these indications is being pursued in international, multicenter trials. Aphase 2b trial of PTC124 in DMD is currently enrolling, and is expected toinclude 165 patients. A phase 2b trial of PTC124 in CF is planned to begin bythe end of this year. With demonstration of clinical benefit, these twotrials are expected to serve as the basis for registration of PTC124 in theseindications. Further development of PTC124 will include clinical trials inmultiple additional genetic disorders.
In the United States, there are an estimated 10,000 DMD patients,approximately 13 percent of which have nonsense mutations. Of the more than30,000 U.S. patients with CF, about 10 percent have nonsense mutations. Thereis a significant unmet medical need for new treatments for these diseases. Inaddition to DMD and CF, the companies plan to explore PTC124's potential tomake a difference for patients with other types of severe and debilitatinggenetic diseases.
"We are impressed by the quality of the preliminary PTC124 data, whichsuggest broad applicability to a large number of genetic disorders," saidGeoff McDonough, M.D., Genzyme's senior vice president and gene
