Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
A U.S. FDA advisory committee’s backing keeps Alnylam Pharmaceuticals Inc.’s Onpattro (patisiran) on the road to a supplemental approval in treating a rare heart disease, but it couldn’t stop the company stock from sliding. Shares (NASDAQ:ALNY) closed Sept. 14 down 8.8% at $193.06, the day after the Cardiovascular and Renal Drugs Advisory Committee voted 9-3 that patisiran’s benefits outweigh the risks in treating cardiomyopathy of transthyretin-mediated amyloidosis.
Sirnaomics Ltd. reported progress with its Galahead RNAi delivery platform for developing novel therapeutic products focused on complement-related diseases.
With Alnylam Pharmaceuticals Inc.’s FDA clearance for Amvuttra (vutrisiran) in the rearview mirror, investors are looking ahead to potentially label-widening phase III data related to another, already approved drug: Onpattro (patisiran).
Arrowhead Pharmaceuticals Inc. and Vivo Capital LLC have launched a joint venture named Visirna Therapeutics for RNA interference therapeutics in the greater China market. Arrowhead is the majority shareholder of the new entity, while Vivo invested $60 million in the new entity.
Arrowhead Pharmaceuticals Inc. and Vivo Capital LLC have launched a joint venture named Visirna Therapeutics for RNA interference therapeutics in the greater China market. Arrowhead is the majority shareholder of the new entity, while Vivo invested $60 million in the new entity.
Sirnaomics Ltd. became the first RNA therapeutics company to list on the Hong Kong Stock Exchange (HKEX) with a $64 million initial public offering. Its shares (HKEX:2277) have since surged, rising to HK$93.90 ($12.04) on Jan. 4 following their debut at HK$65.90 per share.
LONDON – The global pandemic has put the term mRNA on lips worldwide, but vaccines apart, the field of RNA therapeutics has failed to overcome the problem of delivery and is stuck at first base, targeting the liver. Newco Sixfold Bioscience Ltd. is the latest company working to end this constraint, applying a mixture of computation and advanced chemistry to design tissue-specific RNA delivery systems.
Qilu Pharmaceutical Co. Ltd. licensed rights to AB-729, an RNA interference (RNAi) therapeutic from Arbutus Biopharma Corp., in a deal worth up to $300 million. Qilu obtained rights to develop and commercialize the phase II asset for the treatment of hepatitis B (HBV) in mainland China, Hong Kong, Macau, and Taiwan. Under terms of the agreement, Qilu will pay Arbutus $40 million up front, as well as up to $245 million in development, regulatory and sales milestones. In addition, Qilu will make a $15 million equity investment in Arbutus at a price of $4.19 per share.
