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What’s in Today’s Brief? (March 3rd Preview)
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FDA Seeks Sham-Controlled Study – UniQure's Huntington Program Stalls
The FDA has told UniQure it expects a randomized, double-blind, sham surgery–controlled trial before it will consider a marketing application for AMT‑130, the company’s gene therapy for Huntington’s disease. UniQure disclosed the agency’s recommendation after a meeting with regulators, which raises the prospect of a two‑ to three‑year setback and renewed ethical and operational hurdles for the program. Investors reacted quickly: the company’s shares plunged after the announcement, reflecting heightened investor concern about regulatory stringency for rare-disease gene therapies. UniQure plans to request a Type B meeting with the FDA to clarify trial design and next steps. The agency’s stance echoes recent FDA demands for gold‑standard controls in other gene‑therapy programs and signals tighter scrutiny of externally controlled data.
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FDA Lifts Hold on Intellia’s CRISPR Trial... Safety Conditions Apply
Regulators have lifted an FDA hold on Intellia’s Magnitude Phase 3 study of nexiguran ziclumeran for transthyretin amyloidosis with cardiomyopathy, allowing the trial to resume under new safety conditions. The agency cleared the study after reviewing a prior serious liver‑toxicity incident, but imposed exclusions for patients with certain liver issues, low ejection fraction or recent cardiovascular instability. Intellia will increase liver‑enzyme monitoring and provide guidance on short‑term steroid treatment for enzyme elevations. The company said the changes are intended to enhance patient safety while letting enrollment continue toward completion of both Magnitude trials.
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FDA Clears Ascendis’ Weekly Dwarfism Shot – Market Battle Begins
The FDA granted accelerated approval to Ascendis Pharma’s TransCon CNP (now Yuviwel) for children two years and older with achondroplasia, using annualized growth velocity as the surrogate basis for clearance. The decision ends BioMarin’s effective monopoly in the indication and positions Ascendis to compete directly with Voxzogo, which remains the incumbent therapy. Ascendis must confirm clinical benefit in post‑marketing studies to maintain approval. The company expects to begin physician distribution in the second quarter; analysts project modest 2026 uptake with potential acceleration in 2027 and beyond, depending on confirmatory data and label expansions.
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Cardiac Signal Halts Aardvark’s Phase 3 Hunger Trial – Data Review Underway
Aardvark Therapeutics voluntarily paused dosing and enrollment in its Phase 3 HERO trial of ARD‑101 for hyperphagia in Prader‑Willi syndrome after routine safety monitoring in a healthy volunteer study revealed reversible cardiac observations at supra‑therapeutic doses. The company said the pause is precautionary while it conducts a comprehensive review of the safety data to determine next steps and optimal therapeutic dosing. The halt has squeezed the company’s market value and delayed an expected topline readout. Aardvark emphasized patient safety as its highest priority and will provide an update following the internal review and any additional external safety analyses.
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Candid to Go Public via Rallybio Reverse Merger – $505M War Chest
Candid Therapeutics agreed to a reverse merger with Rallybio that will leave the combined company publicly traded and well‑capitalized after a $505 million private financing. The deal transfers Candid’s T‑cell engager (TCE) platform and four‑asset pipeline onto a Nasdaq listing, enabling rapid advancement of clinical programs including cizutamig, slated for Phase 2 testing in autoimmune indications. Company executives framed the transaction as an ‘efficient path’ to scale: the financing and public listing are intended to support global trials and commercialization of TCE candidates in autoimmune and oncology settings. Rallybio stockholders will own a stake in the combined entity, which expects to trade under the Candid name.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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