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.
FEATURED STORIES
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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The FDA granted rare pediatric disease designation to AVR-RD-04, an experimental gene therapy treatment for the lysosomal storage disease cystinosis, AVROBIO announced Tuesday.
Pres. Joe Biden declared the COVID-19 pandemic over. Biopharma stocks react as vaccines and antivirals are still in development.
Alzheon Inc. unveiled promising data from a Phase II biomarker trial of its investigational drug ALZ-801 to treat early Alzheimer’s disease.
Wave Life Sciences presented promising early data from its Phase Ib/IIa SELECT-HD trial in Huntington’s disease with evidence of biomarker improvements.
First Wave BioPharma announced that it is restarting its adrulipase program for the treatment of exocrine pancreatic insufficiency in cystic fibrosis and chronic pancreatitis.
The past week saw promising developments in candidate therapies for cancer, congenital hyperinsulinism, chronic cough and COVID-19 vaccines.
Virios Therapeutics announced Monday its lead asset IMC-1 fell short of its primary endpoint in the Phase IIb FORTRESS study in patients with fibromyalgia.
Research on Alzheimer’s, specifically in neuroscience, continues with the Alzheimer’s Drug Discovery Foundation, Critical Path Institute, Abbvie, AlzeCure Pharma and more.
The FDA has two PDUFA dates on its calendar this week, with two extra days slotted for an advisory committee meeting to evaluate drug applications from three companies.
Intellia Therapeutics reported early, positive signs from its in vivo gene therapies for both transthyretin (ATTR) amyloidosis and hereditary angioedema Friday.