Alnylam Earns $7 Million Milestone Payment from Genzyme

Alnylam Pharmaceuticals, Inc. recently announced 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 9th 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 5×15 product strategy, this program is also a key part of building Alnylam for the future and driving toward commercialization,” said John Maraganore, PhD, 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, MD, 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.”

In 2012, Alnylam entered into an exclusive alliance with Genzyme to develop and commercialize RNAi therapeutics, including patisiran and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam plans to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.

Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for retinol binding protein. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. ATTR represents a major unmet medical need with significant morbidity and mortality; familial amyloidotic polyneuropathy (FAP) affects approximately 10,000 people worldwide and familial amyloidotic cardiomyopathy (FAC) affects at least 40,000 people worldwide.

FAP patients have a life expectancy of 5 to 15 years from symptom onset, and the only approved treatment options for early stage disease are liver transplantation, and tafamidis (approved in Europe). The mean survival for FAC patients is approximately 2.5 years, and there are no approved therapies. There is a significant need for novel therapeutics to treat patients who have inherited mutations in the TTR gene.

Alnylam has licenses to Tekmira LNP intellectual property for use in RNAi therapeutic products using LNP technology.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers. For more information, visit www.alnylam.com.