Alnylam Pharmaceuticals has said it is modeling itself after Genzyme, and now it has formed a new partnership with the pioneering developer of drugs for rare diseases.
Cambridge, MA-based Alnylam (NASDAQ: ALNY), the developer of RNA interference drugs, said today it has agreed to give Genzyme an exclusive license to develop and market ALN-TTR02 as a treatment for transthyretin-mediated amyloidosis in Japan and other Asia-Pacific countries. In exchange, Alnylam will get $22.5 million in upfront cash, milestone payments, and a ‘mid-teens to mid-20s’ percentage royalty from Genzyme’s product sales in the territory. Alnylam retains its rights to the drug in the rest of the world.
The disease is known for the accumulation of excessive amyloid proteins that build up in tissues throughout the body. Alnylam likes going after this disease partly because it’s a single gene disorder of the TTR protein, and the protein in question is concentrated in the liver, where RNAi therapeutics can be delivered. People typically start developing symptoms of this disease in their 30s and 40s, suffer progressive damage to their nerves and heart, and usually live nine to 11 years after symptoms appear, Alnylam has said. About 8,000 people around the world are thought to have a form of TTR amyloidosis that damages the nerves, while about 40,000 have a form that harms the heart.
Alnylam has run a couple of clinical trials for drugs in this program, one with a first-generation delivery system and another with a second-generation delivery technology. Using the newest delivery technology, Alnylam showed in July it could reduce the amount of TTR proteins circulating in the blood by as much as 94 percent. The TTR knockdown lasted for at least a month with a single shot.
“We are encouraged by Alnylam’s progress with their ALN-TTR program 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, CEO of Genzyme, in a statement. “The results to date demonstrate impressive clinical activity and support advancement of this promising therapeutic into pivotal studies and toward the market.”