Biotech

Montana lets firms sell unapproved drugs to patients

A private board with industry ties decides who can sell experimental medicine there.

Definium's LSD pill clears third straight Phase 3 trial

DT120, a dissolvable LSD tablet for anxiety, hit its main goal in a third Phase 3 trial, and the company is heading toward an FDA filing.

Diabetes Care editors boycott the ADA after police ouster

Police escorted five researchers out in June for handing out an editorial on research cuts. Now their journal's editors refuse to review new papers.

A gene therapy just reversed aging signs in senior dogs

Genflow's SIRT6 gene therapy hit its main goal in aged beagles, lowering their biological age and improving survival.

Kura spins off $50M diabetes biotech with Lilly's backing

The new company, Caspian Therapeutics, is chasing a treatment that could change how diabetes progresses, not just manage blood sugar.

Moonwalk raises $70M for a muscle-sparing fat drug

The Bay Area biotech's new injection aims to cut fat while keeping muscle, unlike today's popular GLP-1 weight-loss drugs.

A new cancer pill won FDA approval. Patients can't get it

Rasonque treats a genetic driver of pancreatic cancer, but insurance delays are leaving newly-approved patients stuck for weeks, STAT found.

Novo Nordisk halts two heart trials of ziltivekimab

A data-monitoring committee found the trials unlikely to succeed, another blow to the inflammation theory of heart disease.

Novartis' new heart drug fails its big trial

Pelacarsen lowered a key heart-disease marker, but that didn't mean fewer heart attacks or strokes in a big trial.

FDA approves first-ever drug for Alexander disease

Zanvastro is the first treatment that changes the course of Alexander disease, a rare brain disorder in kids and adults.

Akeso's drug beats Keytruda in a head-to-head trial

Ivonescimab outlived Merck's blockbuster in a Phase 3 lung cancer study, the first rival drug to do it.

Ultragenyx's Angelman syndrome drug fails Phase 3 trial

Ultragenyx's experimental drug for the rare disorder Angelman syndrome missed its main goals in a major trial, and shares dropped over 40%.

GSK moves its mRNA flu vaccine into Phase 3 trials

Early data show GSK's shot beats standard flu vaccines at triggering immune responses, in both younger and older adults.

Novartis halts CAR-T trials after three deaths

Three patients died from severe immune reactions in Novartis trials, prompting Bristol Myers to pause similar studies too.

Alnylam defends its drugs after a rival's trial flopped

AstraZeneca's heart-disease drug missed its goal in a major trial, and Alnylam is making the case its own medicine is the better bet.

CRISPR therapy keeps cholesterol down a year after one dose

CRISPR Therapeutics says a single infusion of its gene-editing drug CTX310 kept cholesterol and triglycerides lower a year later, per new trial data.

BioNTech's cancer vaccine trial fails after rival's win

BioNTech and Genentech shut down a mid-stage cancer vaccine trial for futility, a week after Merck and Moderna's rival vaccine succeeded in a similar disease.

Retro Biosciences raises the dose in its Alzheimer's trial

The Sam Altman-backed startup is testing higher doses of its brain-aging drug after early results underwhelmed, per Business Insider.

FDA approves Revolution Medicines' pancreatic cancer pill

The daily pill is the first drug to target the genetic mutation driving most pancreatic cancers, and it roughly doubled survival in a trial.

Precision BioSciences doses first patient in DMD gene trial

The company dosed its first patient in a gene-editing trial for Duchenne muscular dystrophy, a severe disease in boys.

FDA clears PrecivityAD2, an Alzheimer's blood test

C2N Diagnostics' test spots Alzheimer's biomarkers from a blood draw with over 90% accuracy, now cleared for symptomatic adults as young as 40.

Ramaswamy-backed biotech goes public via reverse merger

Ambros Therapeutics, co-founded by Vivek Ramaswamy, is merging with Werewolf Therapeutics and raising $150M to fund a rare pain drug's final trial.

FDA approves Ultragenyx's first gene therapy

Genglycos treats the genetic root of a rare blood sugar disorder, sparing patients a lifelong dependence on cornstarch.

A third patient dies in China's opaque trial system

RiboX confirmed a death in an unregulated China trial for its CAR-T therapy, the third such disclosed death in weeks.

Merck, Moderna's cancer vaccine succeeds in Phase 3 trial

Their personalized mRNA vaccine, paired with Keytruda, hit its goals in melanoma patients — the first Phase 3 win ever for this kind of cancer therapy.

Trump expected to pick Heidi Overton to lead FDA

The White House aide would take over an agency that's been without a permanent leader since its last commissioner quit.

New data dims Summit-Akeso's lung cancer drug in China

Ivonescimab won a third China approval last week, but longer follow-up shows its benefit fading versus earlier, more impressive data.

Quantum-Si delays its protein-reading device, cuts staff

Quantum-Si is cutting a fifth of its staff and pushing its next protein-sequencing machine, Proteus, back to 2027.

Cullinan's lung cancer drug clears Phase 3 trial

Zipalertinib, from Cullinan and Taiho, hit its goal in a trial for a hard-to-treat lung cancer, setting up a fight with J&J's Rybrevant.

Eli Lilly sues 6 sellers of black-market weight-loss drug

The pharma giant filed lawsuits accusing US vendors of selling fake versions of retatrutide, its unapproved weight-loss drug.