Top Story Today
Cancer's Most Famous "Undruggable" Target Just Got Drugged
Revolution Medicines reported that its drug zoldonrasib shrank tumors in 52% of lung cancer patients carrying the KRAS G12D mutation, with median progression-free survival of 11.1 months. That nearly triples what chemotherapy delivers. The trick: instead of blocking the protein directly (like plugging a keyhole on a smooth, round ball), zoldonrasib recruits a second protein and glues it onto KRAS while it's active, essentially booting a speeding car's wheel. At 12 months, 73% of patients were still alive, and the FDA already granted Breakthrough Therapy designation.
Why it matters: If these results hold in Phase 3 trials, they validate an entirely new strategy for drugging proteins the field spent four decades calling impossible, opening the door for a wave of therapies targeting other "undruggable" cancer drivers.
Read more →Clinical and Regulatory
The Two-Drug HIV Pill That Wasn't Supposed to Make It
The FDA approved Idvynso, a two-drug HIV pill from Merck that matched Gilead's three-drug blockbuster Biktarvy in two Phase 3 trials (1% detectable virus in both arms). The kicker: islatravir, the pill's backbone, was nearly killed after clinical holds over dropping immune cell counts. Merck slashed the dose to a tiny 0.25 mg, and the safety concerns vanished. It's the first simplified HIV regimen that completely avoids the integrase inhibitor class most treatments depend on.
Read more →PBMs Tell Trump Administration That Drug Price Transparency Is Illegal
Pharmacy benefit managers are pushing back hard on a federal rule that would force them to disclose drug pricing, rebates, and fees to employers, calling the mandate illegal under ERISA. Noncompliance carries fines up to $10,000 per violation per day. With three PBMs controlling 80% of the market and roughly 87 million Americans on self-insured plans, the outcome could reshape the entire drug pricing supply chain.
Read more →Funding and Launches
Sage Therapeutics Veterans Launch Brain Drug Startup With $106M
Two former Sage leaders (with two FDA approvals under their belts) launched Tortugas Neuroscience with $106 million and four clinical-stage drugs licensed from Hansoh and Eisai. Targets include schizophrenia, tinnitus (which has no approved medications), focal epilepsy, and rare encephalopathies. In a therapeutic area where 85% of drugs fail, they're betting experience and a diversified portfolio beat the odds.
Read more →$125M Flows Into a Gene Therapy That Could Treat All Forms of Inherited Blindness
Ray Therapeutics closed an oversubscribed $125 million Series B for RTx-015, an optogenetic therapy that reprograms surviving retinal cells to detect light, sidestepping the 100-plus genetic mutations that cause retinitis pigmentosa. The FDA granted RMAT designation in April, and the approach could treat patients the only approved retinal gene therapy (Luxturna) can't reach.
Read more →Flagship Launches Serif Biomedicines to Build DNA That Acts Like mRNA's Longer-Lasting Cousin
Flagship Pioneering publicly launched Serif Biomedicines with $50 million to develop "modified DNA" therapeutics: chemically engineered DNA that aims to combine mRNA's redosability with gene therapy's durability. Co-founded by Noubar Afeyan (yes, the Moderna guy), Serif uses lipid nanoparticles for delivery and plans to target rare diseases first. Still preclinical, but the concept addresses genetic medicine's biggest trade-off.
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