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Sarepta's Black Box, 500 Pink Slips, and a Gene Therapy Franchise Fighting for Survival
The FDA slapped its most severe safety warning on Sarepta's Elevidys gene therapy for fatal liver failure in children, then pulled the non-ambulatory indication entirely. In the same breath, Sarepta announced it's cutting 36% of its workforce (roughly 500 people) as part of a broader restructuring expected to save $400 million a year. Revenue had already been cratering: from $375 million in Q1 2025 to just $110 million by Q4, as the company suspended shipments to non-ambulatory patients. The bizarre twist? Shares surged after hours because investors had been bracing for a full market withdrawal.
Why it matters: This isn't just a Sarepta story. It's a stress test for the entire gene therapy business model: one-time treatments that cost millions, treat tiny patient populations, and now carry the FDA's loudest alarm bell. If the commercial math doesn't work here, the ripple effects reach every rare disease gene therapy in development.
Read more →Clinical & Regulatory
The Eye Disease With Zero Treatments Just Got Its First Real Shot
Stargardt disease has robbed young people of their central vision for decades with exactly zero approved treatments. Belite Bio's tinlarebant just got FDA Priority Review after a Phase 3 trial showed a 35.7% reduction in retinal damage progression. A decision date of February 12, 2027, is now circled on the calendar of roughly 35,000 Americans living with the condition.
Read more →Bayer's PET Tracer Nailed 94% Sensitivity for the Heart Disease Hiding in Plain Sight
An estimated 35–45% of cardiac amyloidosis cases get initially misdiagnosed. Bayer's Phase 3 REVEAL trial showed its PET radiotracer hit 94% sensitivity and 86% specificity for detecting amyloid deposits in the heart. If approved, a single scan could replace a multi-step diagnostic obstacle course and unlock a massive underdiagnosed patient pool for every company selling ATTR drugs.
Read more →Five Years on Camzyos: Nearly Every Patient Still Responding
Bristol Myers Squibb dropped five-year follow-up data for Camzyos at ESC Congress, and the results held. A remarkable 97.4% of patients hit their target threshold, 59% became asymptomatic, and no new safety signals emerged. Revenue hit $1.068 billion in 2025 and is still accelerating, though competitor aficamten is warming up fast.
Read more →The First Pill for Dermatomyositis Crosses the Finish Line
The FDA approved Roivant's brepocitinib as the first-ever targeted oral therapy for dermatomyositis, a rare autoimmune disease that attacks muscles and skin. The Phase 3 trial hit all nine key secondary endpoints, and over half of patients slashed their steroid use. The stock dropped 7.6% anyway in a classic "sell the news" move.
Read more →Deals & Strategy
Teva Goes Dumpster Diving for a $145M Brain Drug
BioXcel filed for bankruptcy on a Wednesday; by Thursday, Teva had a $57.5 million stalking-horse bid on the table for its sublingual agitation drug IGALMI. The real prize is a pending FDA decision on at-home use due November 14. BioXcel's total 2025 revenue was $642,000, less than a single Chick-fil-A pulls in monthly.
Read more →Funding & Pipeline
$120M Bet That Hepatitis B Can Finally Be Cured
AusperBio raised $120 million in Series C funding to push its antisense drug AHB-137 into Phase 3 for hepatitis B. Phase 2 data showed a 30% functional cure rate at 72 weeks; the current standard of care delivers roughly 3% to 5% after a full decade. RA Capital joined as a new investor, bringing total funding to $360 million since 2024.
Read more →Gene Therapy Takes Its Biggest Swing at Heart Failure Since CUPID2
AskBio (a Bayer subsidiary) fully enrolled over 170 patients across 12 countries in its Phase 2 GenePHIT trial, one of the largest gene therapy studies ever in heart failure. Data are expected in early 2027. The field has been in retreat since Celladon's CUPID2 trial flopped a decade ago; this trial uses a different target and a newer, heart-seeking vector.
Read more →Tenax's Phase 3 Flopped Overall, but One Subgroup Tells a Different Story
Tenax's LEVEL trial for oral levosimendan in pulmonary hypertension missed its primary endpoint by a mile (3.5 meters, p=0.63), and the stock crashed roughly 88%. But a prespecified subgroup of sicker patients showed a 26-meter improvement over placebo. Intriguing biology, but the company has no revenue and a shrinking runway to prove it.
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