The RNAi therapeutic zilebesiran, which was the focus of a 2023 $2.8 billion deal between Alnylam Pharmaceuticals Inc. and Roche Holding AG, demonstrated significant systolic blood pressure reductions in patients with mild to moderate hypertension who were part of the Kardia-2 phase II study.
Boehringer Ingelheim GmbH’s start to the new year includes two fresh deals across two continents. BI struck one deal with Kunshan, China-based Suzhou Ribo Life Science Co. Ltd. and its Mölndal, Sweden-based subsidiary, Ribocure Pharmaceuticals AB, to develop small interfering RNA (siRNA) treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis. It struck a second deal with San Francisco-based 3T Biosciences Inc. to develop cancer immunotherapies, which builds on an earlier collaboration formed last year. Combined, the two deals are worth more than $2.5 billion.
Announcing two licensing deals with Swiss pharma giant Novartis AG, Shanghai-based Argo Biopharmaceutical Co. Ltd. said on Jan. 7 that it stands to gain up to $4.165 billion for two of its cardiovascular assets combined. Marking the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company,” the deal includes an up-front payment of $185 million from Novartis to Argo.
Announcing two licensing deals with Swiss pharma giant Novartis AG, Shanghai-based Argo Biopharmaceutical Co. Ltd. said on Jan. 7 that it stands to gain up to $4.165 billion for two of its cardiovascular assets combined. Marking the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company,” the deal includes an up-front payment of $185 million from Novartis to Argo.
Boehringer Ingelheim GmbH’s start to the new year includes two fresh deals across two continents. BI struck one deal with Kunshan, China-based Suzhou Ribo Life Science Co. Ltd. and its Mölndal, Sweden-based subsidiary, Ribocure Pharmaceuticals AB, to develop small interfering RNA (siRNA) treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis. It struck a second deal with San Francisco-based 3T Biosciences Inc. to develop cancer immunotherapies, which builds on an earlier collaboration formed last year. Combined, the two deals are worth more than $2.5 billion.
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).