Hereditary disease is endemic in Japan, resulting in a life expectancy of 5 to 15 years
Genzyme will pay Alynlam US$22.5m upfront as part of an exclusive alliance to develop and market transthyretin (TTR)-targeting RNAi therapeutics for the treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries.
ATTR is a hereditary disease that is endemic in Japan. It damages the nervous system and heart, resulting in a life expectancy of 5 to 15 years. Japanese patients often carry a V30M TTR mutation, which leads to a severe form of ATTR known as familial amyloidotic polyneuropathy (FAP).
Genzyme and Alynlam aim to maximise the value of ALN-TTR worldwide by developing the programme in FAP and other ATTR indications, including familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA).
The agreement also includes development milestone payments and tiered royalties. In addition, each firm will be responsible for the development and commercialisation activities in their respective territories.
John Maraganore, CEO of Alnylam, said: ‘In this important collaboration, Genzyme will advance our ALN-TTR programme with their proven capabilities in the Japanese and broader Asian market, while we maintain our plans to develop and commercialise this potential breakthrough medicine in the US, Europe, and rest of world. In addition, a key part of the value proposition in this alliance for Alnylam is the potential for significant royalty payments on sales of products.’
David Meeker, president and CEO of Genzyme, said: ‘We are encouraged by Alnylam’s progress with their ALN-TTR programme and are excited by the potential for this innovative drug candidate to make a difference in the lives of patients with ATTR.
‘The results to date demonstrate impressive clinical activity and support advancement of this promising therapeutic into pivotal studies and toward the market.’