Hoth Therapeutics Reports Positive GDNF Data for Fatty Liver Disease

GDNF Reprograms Liver Metabolism, Outperforms Semaglutide in Key Gene Markers

Apr. 14, 2026 at 7:07am

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A highly detailed, translucent X-ray photograph of a human liver, with glowing lines and shapes representing the complex genetic pathways involved in fat production and fat burning. The image conveys a sense of scientific discovery and the potential for a new therapeutic approach to metabolic disorders.Cutting-edge genetic research reveals how a novel therapy can fundamentally reprogram the liver's fat metabolism, offering new hope for treating fatty liver disease and obesity.NYC Today

Hoth Therapeutics, a clinical-stage biopharmaceutical company, announced positive data from its HT-VA study, conducted under a CRADA with the U.S. Department of Veterans Affairs and Emory University. The data shows that parenteral GDNF (Glial Cell-Derived Neurotrophic Factor) directly reprograms liver fat metabolism at the genetic level, positioning GDNF as a potentially differentiated therapeutic approach targeting the root cause of fatty liver disease and metabolic dysfunction.

Why it matters

Unlike existing therapies that primarily focus on weight loss, GDNF directly targets the biological mechanisms responsible for fat accumulation in the liver, demonstrating a dual mechanism of reducing fat production and increasing fat metabolism. This suggests GDNF may offer a disease-modifying approach for metabolic-associated fatty liver disease (MAFLD), obesity, and related metabolic disorders.

The details

The HT-VA study evaluated the effects of parenteral GDNF in a diet-induced obesity and MAFLD model. Key observations include a statistically significant reduction in Srebf1, a key gene driving fat production in the liver, and increased expression of Pparα, a central regulator of fat metabolism and fat burning. GDNF also outperformed semaglutide in key gene expression markers tied to liver fat regulation.

  • The HT-VA study was conducted in 2026.

The players

Hoth Therapeutics, Inc.

A clinical-stage biopharmaceutical company dedicated to developing innovative treatments to improve patient quality of life.

U.S. Department of Veterans Affairs

A government agency that collaborated with Hoth Therapeutics on the HT-VA study through a Cooperative Research and Development Agreement (CRADA).

Emory University

A research university that collaborated with Hoth Therapeutics on the HT-VA study through the CRADA.

Robb Knie

Chief Executive Officer of Hoth Therapeutics.

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What they’re saying

“HT-VA represents a major milestone for Hoth as we expand into high-value metabolic indications. These results demonstrate that GDNF is not simply reducing fat, but fundamentally reprogramming how the body produces and metabolizes fat at the genetic level. The ability to shut down fat creation while activating fat metabolism differentiates GDNF from existing therapies, including GLP-1 agonists.”

— Robb Knie, Chief Executive Officer of Hoth Therapeutics

What’s next

Hoth plans to advance the HT-VA findings into additional preclinical validation studies, evaluate clinical development pathways for metabolic and liver diseases, and explore strategic partnerships and collaborations to accelerate development.

The takeaway

Hoth's GDNF therapy has the potential to be a disease-modifying approach for metabolic-associated fatty liver disease, obesity, and related metabolic disorders by directly targeting the biological mechanisms responsible for fat accumulation in the liver, rather than just focusing on weight loss.