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Morning! With STAT reporting from ASH this year, we have some stories in the blood space. Also, a potential Dupixent competitor is in the works, and we examine the fallout from Friday’s ACIP meeting. Plus, a SPAC!
The need-to-know this morning
- Agios Pharma said the FDA has delayed a decision on the expanded approval of its drug Pyrukynd to treat thalassemia, an inherited blood disorder. The agency missed its Dec. 7 review deadline.
- Dyne Therapeutics said its experimental drug for Duchenne muscular dystrophy — a potential next-generation version of Sarepta Therapeutics’ much-debated Exondys 51 — hit the mark in a late-stage study, setting up a potential approval.
- Cogent Biosciences said a Phase 3 study of bezuclastinib in advanced systemic mastocytosis achieved its primary goal.
- Structure Therapeutics reported results from mid-stage study of its oral GLP-1 drug candidate.
Early update shows Wave’s RNA drug cut visceral fat
From my colleague Elaine Chen: Wave Life Sciences said today that interim data show its investigational siRNA therapy cut visceral fat in a small Phase 1 study, though it’s not yet clear how meaningful the reduction is.
Continue to STAT+ to read the full story…
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