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.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
Invivyd Inc.’s VYD-222 produced positive initial top-line results in the ongoing pivotal phase III Canopy study for preventing symptomatic COVID-19. The results could reach a vulnerable population of patients who are immunocompromised and don’t get the same protection as other patients, the company’s CEO, Dave Hering, told BioWorld.
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.
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).
All treatment groups in Vertex Pharmaceuticals Inc.’s phase II study of the non-opioid VX-548 showed meaningful reductions in pain from baseline in treating diabetic peripheral neuropathy (DPN), pointing the company toward pivotal phase III studies and prompting analysts to applaud enthusiastically.
Multiple studies at the 65th American Society of Hematology Annual Meeting 2023 have the potential to change the treatment paradigm for first-line treatment of multiple myeloma.
It’s not every day you see a small drug company’s presentations get picked for both the plenary session and the late-breaker session at a conference, but Syndax Pharmaceuticals Inc. managed to do just that at the 65th American Society of Hematology Annual Meeting 2023 – with a little help from a friend.
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