Adam Lenkowsky, chief commercial officer for Bristol Myers Squibb Co. (BMS), said his firm plans to launch Karxt (xanomeline-trospium) in the U.S. as soon as it’s approved by the U.S. FDA, and “expect[s] to accumulate sales in early 2025.” BMS tied a bow on the year by disclosing its plan to pay $330 per share to take over Karuna Therapeutics Inc. in a deal valued at $14 billion to bring aboard Karxt, which acts as a dual M1/M4 muscarinic acetylcholine receptor agonist. The FDA has assigned Sept. 26, 2024, as the PDUFA date for Karxt as a new treatment for schizophrenia in adults.
Calliditas Therapeutics AB’s full approval from the U.S. FDA for Tarpeyo (budesonide) delayed release capsules in immunoglobulin A nephropathy (IgAN) revived speculation about competitor Travere Therapeutics Inc. which, like Calliditas, has gained accelerated approval for its prospect.
Argenx SE CEO Tim Van Hauwermeiren said that, with subcutaneous efgartigimod (efgartigimod alfa and hyaluronidase-qvfc) in pemphigus, the company is “facing a situation where, even with a strong scientific hypothesis and well-executed trial, we encountered the unknown-unknown,” and the phase III experiment failed. “We are committed to doubling down” on the execution of the firm’s business plan, he added.
On-again, off-again investor enthusiasm for Uniqure NV’s Huntington’s disease (HD) gene therapy AMT-130 got another boost as the company followed this summer’s news from phase I/II trials with additional interim data. Shares of Uniqure (NASDAQ:QURE) closed Dec. 19 at $6.64, down $1.34, or 17%, as the company offered results on up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European experiment.
Given what CEO Raymond Stevens called a space that’s “evolving extremely rapidly,” Structure Therapeutics Inc. chose – rather than wait for next year’s 12-week data – to unblind the eight-week obesity findings with GSBR-1290, an oral glucagon-like peptide-1 (GLP-1) agonist for which the firm also provided a phase IIa update in patients with type 2 diabetes.
Pfizer Inc. proved its ongoing interest in antibody-drug conjugates (ADCs), as well as at least tentative faith in mesothelin as a target, by snatching up Nona Biosciences Inc. for up to $53 million in up-front and near-term cash along with possibly $1.05 billion in milestone payments as the drug advances.
The advent of glucagon-like peptide-1 (GLP-1) receptor agonists has meant new hope for people with type 2 diabetes and obesity, but also has brought the challenge of muscle loss, which various drug developers are trying to ameliorate by way of mixed approaches.
Wall Street will be watching closely for such adverse effects as anemia that foiled Gilead Sciences Inc.’s CD47-binding magrolimab earlier this year, but so far Shattuck Labs Inc.’s SL-172154 looks strong in combination with azacitidine to treat front-line higher-risk myelodysplastic syndrome (HR-MDS) and TP53-mutant (TP53m) acute myeloid leukemia (AML).
Astrazeneca plc, which seemed to have backed away from vaccine development after the COVID-19 pandemic, clearly took a shine to Icosavax Inc.’s virus-like particle technology and signed a deal to take over the firm for as much as $1.1 billion.
Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%. Data from the ongoing phase II Summit trial testing bezu in non-advanced systemic mastocytosis rolled out during the American Society of Hematology (ASH) meeting in San Diego. Waltham, Mass.-based Cogent’s prospect turned up a rapid and continuing improvement in patient symptoms, with a 57% median best improvement on Mast Cell Quality-of-Life.
