Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) saw its shares leap over 20% in pre-market trading after the drugs firm and its French partner Sanofi SA revealed positive results from a late-stage trial of a treatment for Hereditary ATTR (hATTR) Amyliodosis patients with polyneuropathy.
In a joint statement, the companies said an investigational RNAi therapeutic met its primary and secondary endpoints.
The disease is an inherited one that is progressively debilitating and often fatal and is caused by mutations in the TTR gene - TTR protein is produced in the liver and usually acts as a carrier for vitamin A.
Significant milestone
About 50,000 people worldwide suffer from the disease, which currently has no approved treatment apart from liver transplant.
Elias Zerhouni, president, global R&D at Sanofi said: “This is a significant milestone that supports our belief that RNAi therapeutics have the potential to become an innovative new class of medicines for patients with rare genetic diseases."
In pre-market trading in New York, Alnylam shares were up nearly 22% at US$91.53.
Later they gained over 35% to stand at US$101.87 each.