Issue #76·

Biotech stocks are rallying because the FDA chief might get fired

The industry that's supposed to hate regulatory uncertainty is cheering the potential ouster of its top regulator. That tells you everything about how the last 14 months went. Plus: a gene therapy that killed four kids is back in the clinic, and Amgen bets big on a computer that doesn't fully exist yet.

Top Story Today

The FDA Is About to Lose Its Boss. Wall Street Is Throwing a Party.

Biotech stocks rallied on reports that Trump plans to fire FDA Commissioner Marty Makary after just 14 months. The twist: an industry that normally dreads regulatory shakeups is relieved, after Makary's inconsistent drug rejections, staff exodus, and political clashes left companies unable to predict whether their therapies would get a fair review. Several major approval decisions now face a leadership vacuum.

Why it matters: When the regulated industry actively roots for the regulator's removal, it signals a broken relationship that will take months to repair, with billions in pipeline value hanging on pending decisions during the transition.

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Clinical and Regulatory

The Gene Therapy That Killed Four Boys Gets a Second Chance

Five years after four boys died of liver failure in Astellas' XLMTM gene therapy trial, the first patient has been dosed in a new study called VALOR. The redesigned therapy uses a muscle-targeted vector at 100-fold lower doses, essentially rerouting the delivery truck away from the liver. Nine patients will decide if that's enough to tip the risk calculus for a disease with zero approved treatments and near-certain early death.

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Vyvgart Becomes the First Drug Approved for Every Type of Myasthenia Gravis

About 10% of generalized myasthenia gravis patients had no targeted therapy because their antibody subtype didn't match existing labels. The FDA just fixed that, approving argenx's Vyvgart for all serotypes. The $4.2 billion franchise (up 90% year-over-year) keeps widening its moat as the first-in-class FcRn inhibitor pulls further ahead of UCB and Janssen.

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Funding and Deals

CellCentric Raises Europe's Largest Private Biotech Round of 2026

A $220 million Series D (with Pfizer on the cap table) funds a first-in-class oral pill for myeloma patients who've failed everything else. Phase 2 data showed 69% response rates in heavily pretreated patients, roughly triple what existing options deliver. The target market: patients who relapse after CAR-T and bispecific antibodies, a population that grows every time those therapies succeed earlier in treatment.

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Amgen-Backed Quantum Computing Firm Files for Landmark IPO

Quantinuum, valued at $10 billion and backed by Amgen, JPMorgan, and NVIDIA, filed for what could be the largest quantum computing IPO ever. The biotech angle: Amgen is an investor in the company, betting quantum simulation can eventually shave years off drug development timelines.

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