Fractyl Health Announces Promising Preclinical Findings for GLP-1 Gene Therapy in Diabetes and Obesity

Fractyl Health, Inc., a company focused on metabolic therapeutics, has announced promising preclinical findings for its first clinical candidate, RJVA-001, from the Rejuva® pancreatic gene therapy platform. This candidate demonstrated up to 50% blood sugar lowering and 11% weight loss in db/db mice, a standard model for type 2 diabetes (T2D) and obesity, four weeks after a single administration. In comparison, chronic semaglutide treatment resulted in 32% glucose lowering and 2% weight loss.

Dr. Timothy Kieffer, Chief Scientific Officer of Fractyl Health, highlighted the significance of these findings, stating that a single-dose administration of a human GLP-1 transgene can achieve durable effects on blood sugar and body weight. The company has aligned with European regulators on using this efficacy model to support a Clinical Trial Application (CTA) submission in Europe.

Dr. Harith Rajagopalan, CEO of Fractyl Health, emphasized the potential of RJVA-001 to change the trajectory of obesity and T2D treatment by offering a single administration therapy that could lead to durable remission of metabolic diseases. Fractyl Health plans to progress RJVA-001 through IND-enabling toxicity studies in 2024 and initiate First-in-Human clinical studies in 2025.

The Rejuva® platform aims to develop next-generation adeno-associated virus (AAV)-based, locally delivered gene therapies for T2D and obesity, focusing on addressing the underlying root causes of these metabolic diseases. Despite the promising preclinical results, the Rejuva platform is still in development and has not yet been evaluated by regulatory agencies for investigational or commercial use.