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uniQure's Huntington's Gene Therapy Loses Its Signal at Year Four
A $1 billion market wipeout in a single morning. uniQure's Huntington's gene therapy AMT-130 showed 80% disease slowing at three years, but four-year data revealed that benefit had dropped to 44%, and the result was no longer statistically significant. The stock cratered 37%. The awkward part: the FDA is currently reviewing a marketing application built on the stronger three-year numbers from high-dose patients. If the agency approves a therapy whose signal appears to be fading in real time, it sets an uncomfortable precedent. If it doesn't, the only promising treatment for a devastating disease goes back to square one.
Why it matters: This isn't just a uniQure problem. If a one-time gene therapy's effect fades in the brain (where neurons don't divide and the payload should theoretically last forever), it challenges the foundational promise of AAV-based gene therapy in neurodegenerative disease.
Read more →The Obesity Arms Race
Lilly's Triple-Agonist Retatrutide Posts 20.8% Weight Loss in Phase 3
The first triple-agonist obesity drug just delivered pivotal data, and the numbers are big. Patients on Lilly's retatrutide lost up to 20.8% of their body weight over 80 weeks; among those with BMI above 35, the average was 60.8 pounds. By targeting GLP-1, GIP, and glucagon receptors simultaneously, the drug doesn't just suppress appetite, it may also boost fat burning. Filing is expected in Q1 2027.
Read more →Roche Crashes the Obesity Duopoly With 15.5% Weight Loss
Roche's enicepatide posted 15.5% weight loss in a mid-stage diabetes trial, establishing the company as a credible third player in a market Novo Nordisk and Lilly have dominated. Blood sugar results were equally striking: 90% of patients on the top dose hit well-controlled diabetes levels. Roche has spent billions assembling its obesity pipeline through acquisitions, and Phase 3 trials are already underway.
Read more →Zealand's Obesity Drug Worked Better at a Lower Dose (Wait, What?)
Zealand Pharma's amylin-based drug petrelintide posted 9.8% weight loss in Phase 2, but the middle dose (5 mg) outperformed the highest one (9 mg). That inverted dose-response curve complicates Phase 3 planning considerably. The upside: GI side effects were comparable to placebo, a rarity in obesity drugs. Zealand is developing petrelintide both alone and in combination with Roche's enicepatide.
Read more →Clinical Wins and Regulatory Milestones
Amgen Survives Biotech's Most Notorious Graveyard: Lupus
Lupus has destroyed more drug programs than almost any disease in biotech. Amgen's daxdilimab just posted a clean Phase 2 win in discoid lupus, with a 6-point reduction in disease severity versus placebo and no serious adverse events. The catch: an earlier study in systemic lupus (the bigger commercial prize) didn't deliver the efficacy Amgen wanted.
Read more →The Peanut Patch Finally Reaches the FDA's Door, Eight Years Later
DBV Technologies submitted its VIASKIN Peanut Patch for FDA approval after one of biotech's wildest regulatory sagas: seven years, a rejection, a patch redesign controversy, and a complete trial restart. With Palforzia (the only competing treatment) now off the U.S. market, DBV could become the sole option for pediatric peanut allergy if approved.
Read more →A Pill That Wants to Replace Your Psoriasis Injection
Ascletis reported that its oral IL-17A inhibitor ASC50 delivered a 48.9% placebo-adjusted PASI reduction in 28 days, with a half-life long enough to support once-weekly dosing. No liver safety signals, which matters because Lilly's competing oral IL-17 program was killed by liver toxicity. It's early, but in a $30 billion injectable-dominated market, the proof of concept turns heads.
Read more →Industry and Policy Moves
Beam Therapeutics Says a Scientist Walked Out With Its Secret Recipe
Beam is suing a former scientist and two biotech companies, alleging they stole proprietary base-editing technology to build a competing gene therapy for alpha-1 antitrypsin deficiency. The complaint claims the scientist secretly co-founded a rival startup while still employed at Beam. The case could set a precedent for how aggressively gene-editing companies protect trade secrets as talent moves between firms.
Read more →Big Pharma's New Lobbying Chief Is a Former House Majority Leader
PhRMA tapped former House Majority Leader Eric Cantor as its next CEO, choosing a Washington power player over a healthcare insider. With Medicare drug-price negotiations expanding to 20 drugs annually by 2029 and PhRMA's lobbying spend topping $38 million, the hire signals that the industry sees its biggest fight happening in Congress, not in the lab.
Read more →Roche Is Building Autonomous AI Labs to Discover Drugs Without Humans
Roche announced plans for fully autonomous AI-driven laboratories, with its internal tool Target Nexus on track to influence 80% of research portfolio decisions by year-end. The company has amassed 2,176 NVIDIA GPUs (the largest footprint in pharma) and is redirecting roughly CHF 2 billion in R&D savings toward new programs. The goal: 20 new molecular entities by 2030.
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