## 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 notable 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 request 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.
Lilly Halts Phase 2b trial of obesity Drug Bimagrumab, Growth Continues
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Eli Lilly has quietly terminated a Phase 2b clinical trial evaluating bimagrumab for the treatment of obesity and type 2 diabetes before any patients were enrolled. The decision does not signal the end of the drug’s development,as another Phase 2 trial is ongoing with results anticipated in 2026. Bimagrumab, acquired through Lilly’s $1.9 billion acquisition of Versanis in 2022, works by blocking activin and myostatin signaling pathways, aiming to promote muscle growth.
Background on Bimagrumab and Versanis Acquisition
Lilly acquired Versanis Bio in November 2022 for $1.9 billion upfront, with potential for up to $1.7 billion in future payments tied to clinical and regulatory milestones [1]. The primary asset gained in this acquisition was bimagrumab, an antibody designed to selectively inhibit activin and myostatin signaling. Thes proteins normally suppress muscle growth, and blocking them is intended to increase muscle mass and improve metabolic health.
planned Trial Design and rationale for Combination Therapy
The terminated Phase 2b trial was designed to assess bimagrumab both as a standalone treatment and in combination with Lilly’s highly successful drug, tirzepatide. The study was to include nine different study arms, exploring various dosages and combinations [2].
The interest in combining bimagrumab with tirzepatide stemmed from earlier research suggesting bimagrumab could drive fat-specific weight loss when used alongside GLP-1 receptor agonists – the class of drugs to which tirzepatide belongs.Tirzepatide, marketed as Mounjaro and Zepbound, is already a blockbuster medication for diabetes and weight loss, and researchers hoped the combination with bimagrumab could offer even greater benefits.
Why the Trial Was Stopped
Lilly has not publicly disclosed the specific reasons for terminating the Phase 2b trial. It is common in drug development for companies to reassess their clinical trial portfolios based on emerging data, strategic priorities, and resource allocation.
Current Development Status and Future Outlook
Despite the cancellation of the Phase 2b trial, bimagrumab’s development is not over. An ongoing Phase 2 trial is still underway, and results are expected in 2026 [3]. This trial is likely focused on a different patient population or treatment regimen.
The ongoing trial will be crucial in determining the future of bimagrumab and its potential role in addressing obesity and related metabolic disorders. Lilly continues to invest heavily in its pipeline of obesity and diabetes treatments, and bimagrumab remains a possibly valuable asset.
Key Takeaways:
* Eli Lilly has terminated a Phase 2b trial of bimagrumab in obesity and type 2 diabetes.
* Bimagrumab was acquired through the $1.9 billion Versanis acquisition.
* The drug aims to increase muscle mass by blocking activin and myostatin signaling.
* Development continues with an ongoing phase 2 trial, with results expected in 2026.
* The cancellation does not necessarily indicate a lack of confidence in the drug, but rather a strategic reassessment of clinical trial priorities.
Sources:
1][LillyAcquiresVersanistoImprovePatientOutcomesinCardiometabolicDisease[LillyAcquiresVersanistoImprovePatientOutcomesinCardiometabolicDisease
2][Lillyquietlyterminatedaplannedphase2btrialofbimagrumabinobesityandtype2diabetesbeforepatientenrollment[Lillyquietlyterminatedaplannedphase2btrialofbimagrumabinobesityandtype2diabetesbeforepatientenrollment
3][Bimagrumab[Bimagrumab