Mirum Pharmaceuticals Begins Phase 2 Trial of MRM-3379 for Fragile X Syndrome
Mirum Pharmaceuticals has initiated the Phase 2 BLOOM clinical trial for MRM-3379, an investigational oral therapy for Fragile X syndrome. The study will evaluate the safety, tolerability, and potential benefits of MRM-3379 in male participants with Fragile X syndrome. Preclinical models showed cognitive and behavioral improvements. Top-line data is expected in 2027. This announcement was made by Mirum Pharmaceuticals via Business Wire.
Mirum Pharmaceuticals Inc. has announced the enrollment of the first participant in the BLOOM Phase 2 clinical study evaluating MRM-3379, an investigational oral therapy for Fragile X syndrome (FXS). This proof-of-concept study will assess the safety, tolerability, and potential clinical benefit of MRM-3379 in male participants with a confirmed diagnosis of FXS. According to Mirum, preclinical models have shown improvements in cognitive and behavioral domains associated with FXS, along with favorable tolerability in healthy volunteers. Top-line data from this Phase 2 study are expected in 2027; results have not yet been presented. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Mirum Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251201663577) on December 01, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT)
