Agios won a decisive hemoglobin response in sickle cell but missed the pain-crisis endpoint. A liver-safety question has now pushed the mitapivat decision to February.
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Its $1.275 billion upfront commitment to a Phase 1 program puts the spotlight on oral peptide developers, including Entera as it approaches Phase 3 in osteoporosis.
Intismeran plus Keytruda cut melanoma recurrence or death risk by 42% versus Keytruda alone, a first Phase 3 win for a personalized mRNA vaccine. Moderna still fell as much as 25%.
A sign-and-close reverse merger leaves Bolt's original stockholders with 6.9% of the company as Prana's IL-4Rα/TSLP bispecific inherits the Nasdaq listing, backed by a $120 million placement closing today.
In depth
FDA expanded Anktiva's use to BCG-unresponsive, high-grade papillary-only bladder cancer without a dedicated trial, leaning on earlier carcinoma in situ data and scientific similarities. The move sets a precedent developers of rare indications will study closely.
Read the storyThe FDA's 321-page review of Rasonque documents the survival win that earned a broad pancreatic cancer label, and the mutation subgroups whose response data ran against the drug.
CSL is paying $355 million upfront for an antibody whose best evidence is a 26-patient interim readout. The deal prices claudin-1 biology, not proven data, across kidney and liver fibrosis.
PhRMA sued CMS on October 7 over the GLOBE most-favored-nation rule for Medicare Part B. The final rule covers as few as four manufacturers and $440 million in projected savings. The suit's real target is the CMMI test authority beneath it.
A Nature paper finds that one arginine transfer RNA, tRNA1 Arg(UCU), keeps prostate tumors on androgen receptor signaling. When it falls, cells slip into a lineage-plastic, AR-inhibitor-resistant state.
