Date: 2012-10-22
Type of information: Development agreement
Compound: RNAi therapeutics targeting transthyretin (TTR)
Company: Genzyme (USA - MA), a Sanofi company (France) - Alnylam Pharmaceuticals (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: development - commercialisation
Action mechanism:
Disease: transthyretin-mediated amyloidosis (ATTR)
Details:
- Alnylam Pharmaceuticals and Genzyme, a Sanofi company, have formed an exclusive alliance to develop and commercialize RNAi therapeutics targeting transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries. ATTR is a rare, debilitating, hereditary disease that damages the nervous system and heart, resulting in a life expectancy of 5 to 15 years.
Genzyme will advance the ALN-TTR program in the Japanese and broader Asian market, while we maintain our plans to develop and commercialize this potential breakthrough medicine in the U.S., Europe, and rest of world. Together, Alnylam and Genzyme intend to maximize the value of ALN-TTR worldwide by developing the program in FAP and other ATTR indications, such as familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA). Alnylam’s ALN-TTR program currently includes ALN-TTR02, which is in a Phase II clinical trial, and ALN-TTRsc, a subcutaneously administered RNAi therapeutic in late stage pre-clinical development.
Financial terms:
- Under the terms of the agreement, Genzyme will make an upfront cash payment of $22.5 million to Alnylam. The agreement also includes development milestone payments and tiered royalties expected to yield an effective rate in the mid-teens to mid-twenties on Genzyme’s sales of ALN-TTR products in their territory. In addition, each party will be responsible for the development and commercialization activities in their respective territories.
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