Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
In two phase III studies, Merck & Co. Inc.’s oral, once monthly proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor produced statistically significant and clinically meaningful cuts in low-density lipoprotein cholesterol levels. The PCSK9 inhibitor is looking to fit into a crowded market that already has well-established therapies from other big pharmas and a potential competitor in development to treat another indication.
More phase III data coursed through the annual American Society of Clinical Oncology (ASCO) conference on Sunday, as Protagonist Therapeutics Inc. and Takeda Pharmaceutical Co. Ltd. led the charge with positive results for its potential blockbuster rusfertide in treating a rare leukemia.
More phase III data coursed through the annual American Society of Clinical Oncology (ASCO) conference on Sunday, as Protagonist Therapeutics Inc. and Takeda Pharmaceutical Co. Ltd. led the charge with positive results for its potential blockbuster rusfertide in treating a rare leukemia.
Atherosclerotic cardiovascular disease (ASCVD) remains a leading cause of mortality worldwide despite advances in managing classic risk factors and therapies lowering LDL-cholesterol. Inhibiting angiopoietin-like protein 4 (ANGPTL4), a key regulator of triglyceride metabolism, is emerging as a potential strategy to treat atherogenic dyslipidemia and lower ASCVD risk.
A former board member of Chinook Therapeutics Inc. and four others were charged May 22 in a 19-count indictment stemming from an alleged insider trading scheme that produced more than $600,000 in profits after the June 2023 announcement that Novartis AG was acquiring the Seattle-based Chinook in a $3.5 billion deal.
Orionis Biosciences Inc. is sticking with Genentech Inc. in a second deal to discover small-molecule monovalent glue therapies for treating cancer. Privately held Orionis is getting $105 million up front and could earn more than $2 billion in R&D, development, commercial and net sales milestones, plus royalties. The multiyear collaboration calls for Orionis to handle discovery and optimization of molecular glues, with Genentech in charge of later-stage preclinical and clinical development, regulatory filing and commercialization of any small molecules the partnership produces.
Regulus Therapeutics Inc. CEO Jay Hagan told investors in a January call the company had no interest in “simply out-licensing” rights to lead candidate farabursen, an oligonucleotide targeting autosomal dominant polycystic kidney disease set to start phase III testing on a path to a potential accelerated approval. And now, there’s no need to, as Regulus found a buyer for the whole company in a deal with Novartis AG valued at about $1.7 billion.
Roche AG has become the latest pharmaceutical company to respond to the Trump administration’s threat to impose tariffs, saying it will invest $50 billion in drug and diagnostics manufacturing in the U.S. over the next five years. That figure matches a similar commitment by its Basel, Switzerland-based neighbor, Novartis AG, which on April 11 said it would be investing almost $50 billion in the U.S., also over the next five years.
The U.S. FDA issued a complete response letter in October 2023 for Dupixent (dupilumab) in treating chronic hives but has now approved the monoclonal antibody for the indication. The approval makes Dupixent the first targeted biologic the agency has approved in the past 11 years for chronic spontaneous urticaria.
