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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Truqap’s positive clinical data comes after it failed a late-stage study in metastatic triple-negative breast cancer. It helps AstraZeneca position itself as a top player in the prostate cancer space, alongside its Big Pharma colleagues.
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
Cassava Sciences has revealed the late-stage clinical failure of controversial Alzheimer’s drug simulfilam. The company had pledged to share the results whether “good, bad or ambiguous.”
The FDA has accepted Alnylam’s supplemental New Drug Application for Amvuttra on the heels of BridgeBio’s Attruby nod, potentially giving Pfizer’s tafamidis franchise another competitor.
Sage Therapeutics discontinued development of its lead candidate dalzanemdor after a third clinical failure, leading analysts to question the biotech’s future profitability.
ADC Therapeutics, Sutro Biopharma and Zai Lab are among those developing antibody-drug conjugates to address payload and toxicity challenges of current ADCs—and rapidly grow the multibillion-dollar market.
BridgeBio secured approval for Attruby Friday, along with the all-important mortality benefit that could give the drug a significant boost in the market against Pfizer’s tafamidis and potentially Alnylam’s Amvuttra.
A Reuters report on Friday revealed that FDA researchers have found additional molecular evidence tying blockbuster asthma therapy montelukast to adverse neuropsychiatric effects.
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401.
After previously failing studies in Parkinson’s and Alzheimer’s, dalzanemdor’s latest stumble in Huntington’s disease has pushed Sage Therapeutics to pull the plug on the NMDA receptor modulator.