Drug Development
Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.
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Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
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While new late-stage data point to some liver toxicity signals, analysts at BMO Capital Markets said Tukysa’s efficacy outcomes “appear to more than make up for any safety concerns.”
While overall survival remains immature, results so far show a clear trend in favor of Roche’s giredestrant.
In this episode of Denatured presented by AnaptysBio, Jennifer Smith-Parker speaks to Dr. Joe Murray, Mayo Clinic; Marilyn Geller, the Celiac Disease Foundation; and Dr. Paul Lizzul, AnaptysBio, about the challenges and opportunities facing celiac disease treatment.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying.
Structure’s aleniglipron elicited over 11% weight loss in a Phase II trial, sending the biotech’s stock up nearly 103% as markets closed on Monday.
A mid-stage study for ABI-5366 will begin mid next year, while Assembly continues to assess the Phase II potential of ABI-1179.
Although still in Phase I, Wave Life Sciences’ injectable RNA weight loss treatment achieved results that impressed analysts, with 4% fat reduction after three months, beating Novo Nordisk’s semaglutide at a similar time point.
The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases.
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.