Rona Therapeutics has submitted an Investigational New Drug (IND) application to the Australian Human Research Ethics Committee for RN3161. This is a GalNAc-conjugated small interfering RNA (siRNA) therapy designed to treat adults with overweight and obesity.
The Mechanism of Action
RN3161 works by targeting the INHBE gene, which is responsible for producing the protein Activin E in the liver. Activin E plays a role in regulating fat metabolism. Studies have shown that people with certain genetic variations in the INHBE gene have a more favorable fat distribution, healthier cholesterol levels, and a lower risk of conditions like type 2 diabetes and heart disease.
Key Features and Preclinical Results
The company reports that in preclinical studies on non-human primates, a single low dose of RN3161 achieved over 90% knockdown of the INHBE messenger RNA. The drug is designed with chemical modifications to minimize unwanted side effects. A significant advantage of RN3161 is its long-lasting effect, which could allow for dosing only once every six to twelve months. This infrequent dosing schedule could greatly improve patient adherence and the long-term maintenance of weight loss.
RN3161 is the fourth clinical candidate to come from Rona’s proprietary GAIA™ platform, which focuses on developing RNA interference therapeutics. This milestone highlights the company’s progress in advancing therapies for obesity and other cardiometabolic diseases.