Weekly Rundown: Huntington’s disease slowed in gene therapy trial

The new gene therapy became the first to demonstrate slowed progression of Huntington’s disease.

Credit: iStock.com/drafter123

Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.

Huntington’s disease slowed for first time in pivotal gene therapy trial

UniQure’s gene therapy AMT-130 became the first treatment to demonstrate a statistically significant slowing of disease progression in a pivotal study of Huntington’s disease, marking a major step forward in tackling the fatal genetic disorder. At three years, patients given the highest dose of AMT-130 experienced a 75 percent slowing of decline on a standard clinical scale compared with matched external controls, with results also pointing to improved motor and cognitive outcomes and a 60 percent slowing of functional decline. The one-time treatment, delivered via a 12- to 20-hour brain surgery, uses an AAV5 vector to silence the huntingtin (HTT) gene at the root of the disease. While other experimental drugs have attempted to lower levels of the toxic huntingtin protein, AMT-130 is the first therapy to show durable clinical benefit, raising hopes it could transform care for patients and families affected by the condition. Shares of uniQure jumped nearly 250 percent with this news, and the company said it plans to meet with the FDA to discuss a biologics license application in early 2026. – Bree Foster

Capricor clears regulatory path for Duchenne therapy deramiocel

Capricor Therapeutics said an agreement has been reached with the FDA on the use of its completed HOPE-3 trial data to support resubmission of its biologics license application for deramiocel, its investigational therapy for Duchenne muscular dystrophy. Following a July complete response letter (CRL), the agency confirmed HOPE-3 meets requirements as the additional study, with the Performance of the Upper Limb Scale (PUL v2.0) as the primary endpoint and left ventricular ejection fraction as a key secondary measure. Capricor expects to submit the data, due in mid-Q4 2025, as part of its CRL response with the aim of securing approval covering both cardiac and skeletal muscle function. This update is significant as it positions deramiocel to advance toward potential FDA approval for Duchenne muscular dystrophy. – Andrea Corona

Pfizer strengthens obesity portfolio with $4.9B deal to buy Metsera

Pfizer announced it will acquire Metsera and their weight loss drugs — including a GLP-1 agonist and an amylin analog in clinical trials — at an enterprise value of $4.9 billion. After emerging from stealth 16 months ago, Metsera’s stock surged over 60 percent following the announcement. Pfizer’s own attempts to develop obesity drugs has seen setbacks, with the decision to stop work on their oral GLP-1 danuglipron this April, which followed their discontinued oral pill, lotiglipron, in 2023. “The proposed acquisition of Metsera aligns with our focus on directing our investments to the most impactful opportunities and propels Pfizer into this key therapeutic area,” said Albert Bourla, Chairman and CEO of Pfizer, in a statement. – Allison Whitten

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First-in-class CELMoD therapy shows promise in major myeloma trial

Bristol Myers Squibb (BMS) announced that its Phase 3 EXCALIBER-RRMM trial of iberdomide in combination with daratumumab and dexamethasone demonstrated a statistically significant improvement in minimal residual disease negativity rates in patients with relapsed or refractory multiple myeloma. Iberdomide is the first in a new class of cereblon E3 ligase modulators (CELMoDs), designed to degrade disease-driving proteins rather than just blocking their activity. BMS is the first company to successfully develop and commercialize protein degrader drugs, known as immunomodulatory drugs (IMiDs). IMiDs are a class of therapies that target cereblon, a protein in cells that acts as part of an E3 ubiquitin ligase complex, tagging specific proteins for destruction. By binding to cereblon, IMiDs both trigger degradation of harmful proteins and stimulate the immune system to attack cancer cells. CELMoDs build on this approach, binding more selectively and potently to cereblon to increase protein degradation and potentially overcome resistance to earlier therapies. – Bree Foster

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First Barth syndrome drug FDA-approved after rejection

After rejecting Stealth BioTherapeutics’ Forzinity (elamipretide) back in May, the FDA has now granted an accelerated approval in Barth syndrome patients that weigh over 66 pounds — with the requirement that Stealth complete a post-approval randomized controlled trial to prove clinical benefit to patients. The drug becomes the first to treat Barth syndrome, an ultra-rare genetic condition occurring mainly in males that affects the heart, immune system, and muscles. Forzinity works by binding to a phospholipid in the mitochondrial membrane to improve mitochondrial function. The earlier decision was part of a long and growing list of FDA rejections this year that suggest greater regulation, but also delays and longer timeframes. – Allison Whitten

Lilly scraps obesity study of Versanis-acquired antibody bimagrumab

Eli Lilly has quietly terminated a planned Phase 2b trial of bimagrumab in obesity and type 2 diabetes before patient enrollment. The antibody, which blocks activin and myostatin signaling to promote muscle growth, was gained through Lilly’s $1.9 billion Versanis acquisition and was set to be tested alone and in combination with Lilly’s blockbuster tirzepatide across nine study arms. Bimagrumab has previously shown potential to drive fat-specific weight loss when paired with GLP-1 therapies, prompting interest in its use alongside tirzepatide. The cancellation does not end development of the drug, which remains in an ongoing Phase 2 trial with results expected in 2026. – Andrea Corona