Original reporting and analysis on the stories shaping biotech.
The FDA just handed BioMarin's achondroplasia monopoly three extra months of breathing room. Ascendis Pharma's once-weekly rival got delayed by a procedural amendment, and in rare disease, timing is everything.
Big pharma is spending record billions on rare disease, but only on drugs that are already proven. Early-stage rare disease biotechs are starving for capital, and the gap between winners and losers has never been wider.
After burning $10 billion on a Metsera buyout and years of obesity drug failures, Pfizer's monthly weight-loss shot just posted Phase 2 numbers that beat Wegovy's early benchmarks, with 75% fewer injections. The weight-loss curve still hadn't plateaued.
A UC Riverside team built a battery-powered gel that pumps oxygen deep into chronic wounds. In diabetic mice, untreated injuries were often fatal, but treated wounds healed in 23 days. The implications go way beyond band-aids.
Axsome Therapeutics trades at $9.3 billion, but claims its brain drug portfolio could generate $16 billion in peak sales. With an FDA decision on Alzheimer's agitation just weeks away and patents running into the 2040s, the gap between what the market sees and what the company believes is about to be tested.
India's pharma exports just blew past $30 billion, and the industry is gunning for double-digit growth next year. For Western biopharma, that means more pricing pressure on generics, and a whole new tier of manufacturing partners knocking on the door.
Abivax's oral Crohn's drug showed symptom relief in one week, while biologics typically take a month. Even wilder: preclinical data suggests it might also prevent the scar tissue that sends patients to surgery.
Daiichi Sankyo just hired John Tsai, the former Novartis CMO behind 15 global drug approvals, to run R&D. The move signals the ADC giant wants to become something much bigger, and it's betting a Novartis-trained pipeline architect can pull it off.
Ionis pulled the plug on an Alzheimer's trial for people with Down syndrome after enrolling just one patient in 14 months. The drug wasn't the problem; the brutal reality of rare disease recruitment was.
Roche's oral breast cancer drug giredestrant just got its FDA date, and its secret weapon isn't the drug itself. It's the combination strategy that cut disease progression by 62% in the patients who need it most.
RTW Investments just agreed to hand Candel Therapeutics $100 million to launch a cancer-killing virus, but only if the FDA approves it first. The deal's clever structure and the Phase 3 data behind it could be a blueprint for small biotechs everywhere.
Recursion's CFO sold shares near the 12-month low just days before Q4 earnings, while NVIDIA and Novo Holdings have already headed for the exits. The AI drug discovery firm's biggest moment of truth lands Tuesday.
A routine brain scan on a five-year-old, four years after gene therapy, revealed a tumor that may be linked to the treatment's viral vector. The FDA didn't just freeze that program. It froze a completely different one too.
Iberdomide just landed priority review with an August 17 FDA decision date, putting it on track to become the first cereblon E3 ligase modulator ever approved for myeloma. It binds its target 20 times tighter than Revlimid, and the way it got filed could change how myeloma drugs reach patients.
J&J's TREMFYA just posted three-year data showing 81% of ulcerative colitis patients stayed in remission, even the ones who'd failed every other treatment. In a disease where drugs tend to fade over time, these durability numbers are turning heads.
Grail's Galleri blood test detected deadly cancers at seven times the normal rate and cut Stage IV diagnoses by over 20%. The stock still crashed 47% because the trial's own design masked the very success it was measuring.
Novartis treated 30 cancer patients with its NKG2D-targeting drug QEQ278. Not a single tumor shrank. With similar programs from Nkarta and Astellas also crashing, an entire immunotherapy pathway might be headed for the graveyard.
The Supreme Court killed IEEPA tariffs in a landmark 6-3 ruling, but pharma's supply chain headaches are just getting started. Section 232 duties on APIs and finished drugs survived, and with the U.S. importing 88% of its active ingredients from abroad, the real cost reckoning hasn't even begun.
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