Veru (VERU) investor relations material

Clinical development strategy

• Focus on muscle preservation in older patients with obesity, targeting those over 65 and BMI >35, a group at high risk for muscle loss and functional decline.

• Phase II-B QUALITY trial demonstrated that combining enobosarm with semaglutide preserved muscle mass and function, with 3 mg enobosarm maintaining 100% lean mass and reducing stair climb decline by up to 60%.

• Ongoing PLATEAU trial extends treatment to 68 weeks, focusing on the same high-risk population to amplify differences in muscle and function outcomes.

• Interim analysis at 32 weeks will assess lean and fat mass changes, guiding phase III design.

• Phase III will require 4,500 patients, with phase II-B serving as a mini phase III to refine endpoints and patient selection.

Regulatory and clinical endpoints

• FDA prefers functional endpoints like stair climb over cosmetic or strength measures; stair climb is sensitive to anabolic interventions and predictive of adverse outcomes.

• Quality of life is measured using SF-36 PF-10 and IWQOL-Lite questionnaires, linking functional changes to patient-reported outcomes.

• Statistical significance alone is insufficient; endpoints must demonstrate meaningful functional benefit to patients.

• The PLATEAU trial incorporates expanded functional and quality of life endpoints based on learnings from QUALITY.

Mechanism of action and combination rationale

• Enobosarm is a selective androgen receptor modulator (SARM) that is anabolic on muscle, catabolic on fat, and anti-resorptive on bone, with minimal masculinizing effects.

• Oral dosing allows for combination with oral semaglutide, maximizing market and patent opportunities.

• Combination with semaglutide targets incremental fat loss while preserving muscle, addressing the plateau in weight loss seen with GLP-1s alone.

• No significant drug-drug interaction is expected due to different molecular classes.