Original reporting and analysis on the stories shaping biotech.
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.
The NIH killed funding for 383 clinical trials and $1.81 billion in grants in 2025, leaving 74,000 patients in limbo. Now the entire playbook for how trials get designed, funded, and run is being rewritten, and execution quality is the new currency investors care about most.
Cyprium Therapeutics just sold a Priority Review Voucher for $205 million, without ever selling a single dose of the drug that earned it. It's the most fascinating business model in biotech, and the voucher market is only getting hotter.
MoonLake's stock crashed 90% after a catastrophic Phase 3 miss in 2025. Five months later, the company just posted an 81% response rate in spinal arthritis, with PET scans showing it might actually slow irreversible bone damage.
The FDA just fast-tracked a gene therapy that could replace the grueling, round-the-clock cornstarch regimen keeping GSDIa patients alive. Ultragenyx's DTX401 showed a 60% reduction in cornstarch intake at 96 weeks, and the company built its own factory to make sure supply doesn't become the bottleneck.
Vanda Pharmaceuticals got Bysanti approved for bipolar I and schizophrenia without running a single Phase 3 trial, and its stock surged 40%. After seven years of jet lag rejection heartbreak, this clever regulatory strategy tells a bigger story about how drugs really get approved.
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.
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.
Sanofi's board ousted CEO Paul Hudson after six years of one-drug dependence and a shrinking vaccine business. His replacement is Belén Garijo, a former Sanofi veteran turned Merck KGaA chief, and she's walking into one of pharma's biggest turnaround jobs.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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