Alnylam Pharmaceuticals Inc. and Genzyme said today they have formed an exclusive alliance to develop and commercialize RNA interference therapeutics targeting transthyretin for treating transthyretin-mediated amyloidosis in Asia.
Under terms of the agreement, Genzyme will pay $22.5 million in cash upfront 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.
Each company will also be responsible for development and commercialization activities in their respective territories.
Known as ATTR, transthyretin-mediated amyloidosis is a rare hereditary disease that damages the nervous system and heart, resulting in a life expectancy of five to 15 years. The companies said ATTR is an endemic disease in Japan, with a significant number of patients carrying the V30M TTR mutation that leads to an onset of a severe form of the disease known as familial amyloidotic polyneuropathy.
Alnylam said its 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.
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