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Good morning! It’s an ASCO-full newsletter today (and be sure to peruse the More Reads for more reads) but there’s so much more this week. We’re previewing tomorrow’s advisory committee on the closely watched MDMA therapy from Lykos Therapeutics, and both Jonathan Wosen and I will be at BIO! Hope to see you there.
The need-to-know this morning
- Agios Pharmaceuticals said its drug called mitapivat reduced the need for blood transfusions in patients with a severe form of beta-thalassemia, an inherited blood disorder. The results achieved the primary goal of a placebo-controlled Phase 3 clinical trial, and if the drug is eventually cleared by regulators, could accelerate sales.
- Structure Therapeutics reported that its oral GLP-1 drug led to weight loss in two studies, leading shares to surge in pre-market trading.
- Arrowhead Pharmaceuticals reported positive results from a Phase 3 study of its RNAi-based therapy for familial chylomicronemia syndrome, an ultra-rare disorder that causes high levels of triglycerides.
What to expect from the MDMA advisory committee
Later this summer, the FDA will decide whether or not to approve MDMA as a therapy for PTSD. It’s a tremendous step for validating psychedelic drugs as therapies, but it’s one being weighed with extreme caution. On Tuesday, a panel of independent scientific advisers will review data on MDMA’s safety and efficacy, and share their feedback with regulators. STAT’s Olivia Goldhill outlines the key issues you should look out for in this high-profile ad comm.
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