Any skepticism that might have been lingering in the wake of Alnylam Pharmaceuticals Inc.’s February decision to tweak the analysis plan for the Helios-B trial testing vutrisiran in transthyretin amyloidosis with cardiomyopathy appeared to dissipate as the company reported top-line data showing the study met the primary and all secondary endpoints.
Shares of Avidity Biosciences Inc. hit a 52-week high on reports of promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD), including impressive biomarker results that could indicate a path for potential accelerated approval.
In a deal that could exceed $1 billion for Ochre Bio Ltd., the U.K.-based liver disease therapy developer will collaborate with Boehringer Ingelheim GmbH. In the meantime, Hepion Pharmaceuticals Inc. shut down its lead candidate’s phase IIb study while looking for a lifeline.
Glaxosmithkline plc (GSK) tapped Arrowhead Pharmaceuticals Inc. for its foray into nonalcoholic steatohepatitis (NASH), as the pair signed an exclusive global licensing deal (excluding China) for GSK to develop and commercialize phase I/II-stage ARO-HSD, Arrowhead’s RNAi therapy targeting HSD17B13.
RNAi therapeutics specialist Sirnaomics Inc. has wrapped up a $105 million series D financing that will support ongoing clinical development of its cancer and fibrotic disease programs plus work on early stage programs targeting metabolic disease and viral infections. But with both new and existing investors jumping in, the company is also preparing for something bigger: its next phase of growth, including a near-term IPO.
RNAi therapeutics specialist Sirnaomics Inc. has wrapped up a $105 million series D financing that will support ongoing clinical development of its cancer and fibrotic disease programs plus work on early stage programs targeting metabolic disease and viral infections. But with both new and existing investors jumping in, the company is also preparing for something bigger: its next phase of growth, including a near-term IPO.
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.
HONG KONG – Suwon-based Olix Pharmaceuticals Inc. is completing the final preclinical work before starting clinical trials for its as-yet-unnamed COVID-19 drug candidate, developed inside three months using RNA interference (RNAi) technology.
LONDON – Evox Therapeutics Ltd. sealed a $1.2 billion collaboration with Eli Lilly and Co. to apply its exosome technology to the doubly difficult task of systemically delivering RNA interference and antisense oligonucleotide drugs, to reach central nervous system targets.
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