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Acumen's Alzheimer's Drug Cleared Plaque but Didn't Slow Decline, and the Stock Fell 89%
Acumen's antibody sabirnetug failed its Phase 2 trial in 542 people with early Alzheimer's disease. Neither dose beat placebo on the iADRS, a combined score of thinking and daily function, after 18 months, and key secondary measures such as the CDR-SB missed too. The odd part: brain scans showed significant drops in amyloid plaque at both doses. About 10% of treated patients had ARIA-E, a form of brain swelling seen on scans. Acumen is stopping development, conserving cash and exploring strategic alternatives, and its shares fell almost 89% on Friday.
Why it matters: A drug that clears plaque yet doesn't slow decline adds to the doubts about whether lowering amyloid is enough on its own, and will make investors warier of the next amyloid antibody. It also shows how fast one readout can erase a small biotech's value.
RTTNews →Clinical & Regulatory
Gilead Pauses a Lymphoma CAR-T After Severe Nerve Side Effects
Two trials of KITE-753, a Gilead CAR-T for diffuse large B-cell lymphoma, are on partial clinical hold. On Thursday's earnings call, executives said a small number of patients had grade 3 or 4 ICANS, a serious neurotoxicity, and that no patients have died. KITE-753 uses a three-day manufacturing process; Gilead's autoimmune CAR-T, which doesn't, carries on.
The Motley Fool (Gilead earnings call transcript) →FDA Approves the First Plasmid DNA Immunotherapy, for a Rare Airway Disease
Inovio's Voceryq won accelerated approval for adults with recurrent respiratory papillomatosis, in which HPV causes wart-like growths in the airway that keep returning after surgery. Plasmids, small pieces of DNA, are injected into muscle with a device that uses brief electrical pulses, prompting the immune system to attack infected cells. In the trial, 7 of 26 patients with at least three baseline surgeries needed none for a year.
FDA →FDA Pushes Agios's Sickle Cell Decision to February Over Liver Safety Questions
The FDA was due to rule on mitapivat for sickle cell disease by November 1; now the date is February 1, 2027. The agency asked whether the drug needs a REMS, a mandatory safety program, after liver injuries in thalassemia trials, and deemed Agios's answer a major amendment. Agios says the drug's benefit-risk is unchanged and it is launch-ready.
HCPLive →Deals & Money
AstraZeneca Will Spend Up to $2 Billion to Save $1.5 Billion a Year
AstraZeneca launched a three-year restructuring, Reshaping for Sustainable Growth, with its third-quarter results. It expects up to $2 billion in one-time costs and $1.5 billion a year in savings, mostly reinvested in the pipeline, with the work essentially done by the end of 2029. Double-digit growth in oncology and rare disease is offsetting generic competition for Farxiga and Brilinta; the cuts are about funding what comes next.
AstraZeneca (SEC filing) →Jazz Bought Actio's Epilepsy Drug. The Rest of the Pipeline Just Raised $90 Million.
Setara Biosciences launched Friday with $90 million from Deerfield, Canaan and Jazz itself. Jazz bought Actio for its epilepsy program in August; Setara takes the remaining pipeline, under the same CEO, David Goldstein. Its pill STAR-0871 blocks TRPV4, an ion channel, and is headed for a Phase 1b in a severe form of Charcot-Marie-Tooth, an inherited nerve disease, and a Phase 2 in overactive bladder.
BioPharma Dive →Science
Clinics Sell 'Three-Parent IVF' to Older Women With No Evidence It Works for Them
Mitochondrial replacement moves a patient's DNA into a donor egg, and it was designed to prevent inherited disease. An eight-month STAT investigation found it sold abroad as a fix for age-related infertility, with an Illinois facilitator sending most clients to an Albanian clinic for packages now costing $30,000. An ethicist says no published study shows it works over 40; the facilitator has paused new patients.
STAT →