Uniqure hails 'groundbreaking' results in gene therapy trial in Huntington's disease

An experimental gene therapy from Uniqure slowed the progression of Huntington's disease by 75% after three years — study results reported this morning that are likely to support the first approval of a genetic treatment for the rare neurodegenerative condition.

"These data suggest the potential for a disease-modifying drug," Rachel Harding, a Huntington's researcher and assistant professor at the University of Toronto, told my colleague Adam Feuerstein. She has no financial ties to Uniqure, and, like STAT, was provided embargoed access to the study results by the company.

Uniqure plans to submit AMT-130 to the Food and Drug Administration in the first quarter of next year. If approved, the therapy could become available to patients later in 2026. "We view these data as groundbreaking and close to the best-case scenario for us and HD patients," said Uniqure CEO Matt Kapusta.

infectious disease

Deal reached for generic versions of Gilead's HIV prevention drug

Several prominent health organizations including the Gates Foundation and the Clinton Health Access Initiative will provide backing for two generic manufacturers to produce and distribute a low-cost version of Gilead's HIV prevention drug in dozens of low-income countries.

The arrangements call for generic drugmakers Dr. Reddy's Laboratories and Hetero to make versions of Gilead's Yeztugo available at $40 per person a year in 120 countries starting in early 2027.

The drug has been hailed as groundbreaking after study data showed that a single set of injections every six months could provide virtually complete protection against infection. But patient advocacy groups have raised concerns about pricing and access, particularly in countries with fast-growing HIV populations.

obesity

Veru update shows FDA still values weight loss over body composition

A new update from Veru, a biotech developing a muscle-preserving drug for obesity, suggests that the FDA will continue to focus on overall weight loss when evaluating obesity candidates, instead of changes in specific body components like fat and muscle, despite calls from some doctors and companies for the agency to change its approach.

Veru said the FDA has provided guidance that "an acceptable primary endpoint to support approval" is to show its drug, enobosarm, can enhance the weight loss of a GLP-1 treatment. While the company included changes in lean and fat mass as secondary endpoints in its proposed development plan, the FDA said "none of these is a clinically meaningful clinical endpoint, nor a validated surrogate endpoint," Veru told STAT in an email.

As GLP-1s have taken off, some doctors have raised concerns that patients may lose too much lean mass as they lose weight, which could affect not only physical function, but also their metabolism. As a result, several companies including Eli Lilly, Regeneron, and Scholar Rock have started repurposing drugs initially developed for muscle diseases to treat patients using GLP-1s.

Critics of muscle-preserving drugs say it's not yet clear if they're needed because there hasn't yet been robust evidence that the lean mass loss while taking GLP-1 drugs is actually harmful. Also, the current way to measure body composition, the DEXA scan, is not precise and does not differentiate between different types of lean mass (for example, organ mass or muscle).

According to Veru, the FDA told the biotech, "We are still not aware of data informing what magnitude of imaging-based changes in lean mass (or fat mass) could be considered clinically meaningful, and that such imaging changes would need to be directly linked to improvement in how a patient feels, functions, or survives."

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