Sanofi’s Genzyme division will make an upfront payment of $22.5m to Alnylam Pharmaceuticals under a newly-signed deal that will see the firms develop and commercialise RNAi therapeutics in Asia.
The investigational drugs will target transthyretin for the treatment of transthyretin-mediated amyloidosis (ATTR), a rare and hereditary condition affecting the nervous system and heart, which severely reduces someone’s life expectancy.
Genzyme and Alnylam will develop the ALN-TTR programme to target variations of ATTR, including familial amyloidotic polyneuropathy (FAM), which can occur in patients carrying the V30M TTR mutation.
Other ATTR-related conditions also touted as potential indications include familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA).
According to Alnylam, ATTR is an orphan condition, with FAP thought to affect about 10,000 people worldwide and FAC though to affect at least 40,000 people.
However, despite the significant morbidity and mortality of the conditions, there is a considerable unmet medical need, with liver transplantation and Pfizer’s Vyndaqel (tafamidis) often the only two treatment options.
Specific drugs in development as part of the ALN-TTR programme include ALN-TTR02, which is currently in phase II development following promising results in phase I where the drug demonstrated it was able to suppress transthyretin. A phase III trial is expected to begin by the end of 2013.
In addition, ALN-TTRsc, a subcutaneously administered RNAi therapeutic is in late stage pre-clinical development.
“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,” said David Meeker, president and CEO of Genzyme.
“The results to date demonstrate impressive clinical activity and support advancement of this promising therapeutic into pivotal studies and toward the market.”
As well as the upfront payment, Genzyme will also pay Alnylam development milestones and tiered royalties.
The Genzyme-Alnylam deal applies only to Japan and other Asian countries, with Alnylam harbouring alternative intentions in the US, Europe and rest of the world.