Mirum Pharmaceuticals Phase 2 Trial in Fragile X Syndrome

Ages: 13–45, Male Only

Study Drug: Oral Medication Twice Daily

Enrollment Start: November 2025

MRM-3379 is designed to selectively inhibit phosphodiesterase 4D (PDE4D), an enzyme highly active in brain regions crucial for learning, memory, and emotional regulation. PDE4D breaks down cAMP, a key signaling molecule between nerve cells, which is found at lower levels in individuals with fragile X syndrome . By blocking PDE4D, MRM-3379 aims to elevate cAMP levels and rebuild neural networks affected by the disease, potentially alleviating fragile X symptoms. 

Genentech Phase 2 Trial in Coronary Artery Disease (CAD)

Ages: 18 and up

Study Drug: Oral Medication Twice Daily

Enrollment Start: December 2025

This is a Phase II, multicenter, randomized, participant- and investigator-blinded, placebo-controlled study designed to evaluate the efficacy, safety, and pharmacodynamics of selnoflast in adults with CAD, with and without elevated hsCRP, and at high-risk for MACE, who are currently receiving standard-of-care therapies. The study consists of a screening period of up to 28 days, a treatment period of up to 90 days, and a minimum follow-up period of 28 days. Approximately 162 participants are expected to be enrolled in this study.