Cardiology
BridgeBio's victory holds up
A would-be blockbuster heart medicine from BridgeBio reduced the risk of death and hospitalization for patients with a progressive cardiovascular disease, researchers said at a medical conference yesterday, setting the stage for competition against a multibillion-dollar medicine from Pfizer.
BridgeBio's detailed data, presented at the European Society of Cardiology meeting in Amsterdam, supported the company's previously disclosed success in a Phase 3 study. The drug, acoramidis, met its goals in a trial enrolling patients with ATTR-CM, an increasingly prevalent disease in which misfolded proteins lead to irreversible and often fatal heart damage.
The question now is whether BridgeBio's drug, once it's approved, should be used instead of tafamidis, the Pfizer treatment that brings in more than $2.5 billion a year. There is no head-to-head study of the two medicines, and history suggests there never will be, but BridgeBio's successful trial enrolled patients who weren't as sick as those in the Pfizer study, suggesting its drug is more potent. Furthermore, the study allowed patients to take tafamidis for the final 18 months of the 30-month trial. More patients in the placebo group received Pfizer's drug than those in the treatment arm, another datapoint suggesting acoramidis' superiority.
R&D
Bayer makes inroads on a big idea in Parkinson's
For decades, researchers have hoped they might cure Parkinson's by replacing patients' dying neurons with neurons derived from stem cells. But trials in the 1980s with embryo-derived stem cells showed little promise, and tests with the newer, fancier, lab-built stem cells have been largely limited to (dramatic) single-patient cases.
This morning, however, Bayer's stem cell subsidiary Bluerock released detailed data from a trial of 12 patients, one of the first rigorous looks into how the long-gestating approach might one day change Parkinson's. Although still quite early, the study showed evidence that replacement cells survived in the patients' brains and suggested Parkinson's symptoms improved. One patient suffered a seizure attributed to the surgery required to implant the cells.
"I think it's quite exciting for the field of cell therapy in the brain," said Bob Carter, chair of neurosurgery at Mass General, who was not involved in the work. Bluerock is now planning to begin a larger study next year.
Washington
AstraZeneca gets creative with its IRA lawsuit
British pharmaceutical giant AstraZeneca became the sixth drug company to sue over the U.S.'s plan to let Medicare negotiate certain drug prices, but its legal challenge came with a twist.
As STAT Rachel Cohrs reports, AstraZeneca echoes previous claims that the Inflation Reduction Act is unconstitutional but also argues that the Centers for Medicare and Medicaid Services overstepped its authority in how it interpreted parts of the law. The company, which paid about $40 billion for the rare disease drugmaker Alexion Pharmaceuticals, also took issue with how the law applies to certain medicines for rare conditions.
The lawsuit comes just days before Medicare is expected to publish the first 10 medicines that will be subject to negotiation when the program takes effect in 2026. Symbicort, AstraZeneca's treatment for asthma and COPD, could end up on the list.
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