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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has earned a $7 million milestone from its partner Genzyme, a Sanofi company, for achieving Phase II success with patisiran (ALN-TTR02). Alnylam recently presented positive Phase II results at the IXth International Symposium on Familial Amyloidotic Polyneuropathy (ISFAP) held in Rio de Janeiro, Brazil, November 10 – 13. Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%. Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein. In addition, patisiran was found to be generally safe and well tolerated in this study. Alnylam has also recently initiated its APOLLO Phase III trial of patisiran in ATTR patients with FAP, with the study now open for enrollment.

“We believe patisiran holds considerable promise to become a breakthrough therapy for the treatment of ATTR, a progressive and debilitating orphan disease. As the lead program in our ‘Alnylam 5x15’ product strategy, this program is also a key part of building Alnylam for the future and driving towards commercialization,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We have made tremendous progress with this program in recent months, including reporting positive Phase II results, and initiating both an open-label Phase II extension study as well as our Phase III APOLLO study. We look forward to continued progress with Genzyme as our collaborator for the development and commercialization of our TTR program in the Japanese and Asian markets, and we are pleased to achieve this milestone based on successful completion of our Phase II study.”

“We continue to be very encouraged by Alnylam’s progress with their ATTR program and are excited by the potential for this innovative drug candidate to make a difference in the lives of patients,” said David Meeker, M.D., President and Chief Executive Officer of Genzyme. “The results to date demonstrate impressive clinical activity and support advancement of this promising therapeutic into pivotal studies and toward the market. We very much look forward to working with Alnylam on this important program.”

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