Alnylam Pharmaceuticals Inc. said the 164-patient Helios-A phase III study with next-generation RNAi drug vutrisiran hit its primary endpoint as well as both secondary goals in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months as compared to historical placebo data from the Apollo phase III study of Alnylam’s Onpattro, cleared by the FDA for ATTR polyneuropathy in August 2018.
Four days after receiving European approval, the FDA has followed suit for Alnylam Pharmaceuticals Inc.’s Oxlumo (lumasiran), the first treatment for primary hyperoxaluria type 1, an ultra-rare genetic disorder that contributes to kidney stones and deposits.
In a deal that could bring the company as much as $1.04 billion, Arrowhead Pharmaceuticals Inc. will collaborate with Takeda Pharmaceutical Co. Ltd. to co-develop and co-commercialize an investigational RNAi-based liver disease treatment. Arrowhead’s candidate, ARO-AAT, is designed to reduce mutant alpha-1 antitrypsin protein production, which causes the disease to progress. Arrowhead will receive a $300 million up-front payment on closing in addition to development, regulatory and commercial milestones that could total $740 million.
In a deal that could bring the company as much as $1.04 billion, Arrowhead Pharmaceuticals Inc. will collaborate with Takeda Pharmaceutical Co. Ltd. to co-develop and co-commercialize an investigational RNAi-based liver disease treatment. Arrowhead’s candidate, ARO-AAT, is designed to reduce mutant alpha-1 antitrypsin protein production, which causes the disease to progress. Arrowhead will receive a $300 million up-front payment on closing in addition to development, regulatory and commercial milestones that could total $740 million.
Positive preclinical data from Phio Pharmaceuticals Inc., of Marlborough, Mass., propelled the company stock (NASDAQ:PHIO) dramatically upward, about 86%, at midday Friday only to see it finally settle at a more modest 36.16% increase to close May 29 at $3.05.
A $2 billion deal with Blackstone Group Inc. is setting Alnylam Pharmaceuticals Inc. up so that it doesn’t have worry about future equity financing and instead can concentrate on RNAi R&D.
Vir Biotechnology Inc. and Alnylam Pharmaceuticals Inc. are expanding an infectious disease collaboration begun three years ago to take on the coronavirus. The effort now includes developing and commercializing RNAi therapies targeting SARS-CoV-2, the virus that triggers COVID-19, by developing siRNAs identified by Alnylam.
Every year at the Cleveland Clinic Medical Innovation Summit, the Top 10 list of technologies that are predicted to come to the market and change patient care is revealed. Last year's October event saw RNA-based therapies take the No. 10 spot.
