Mirum Pharmaceuticals Advances in Fragile X Syndrome Treatment Study

Mirum Pharmaceuticals ((MIRM)) announced an update on their ongoing clinical study.

Study Overview: Mirum Pharmaceuticals is conducting a Phase 2 clinical trial titled ‘Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome.’ The study aims to evaluate the safety and tolerability of MRM-3379 in males aged 16 to 45 with Fragile X Syndrome (FXS), with a parallel cohort of younger participants aged 13 to under 16 receiving open-label treatment. This research is significant as it explores a potential new treatment to alleviate symptoms of FXS, a genetic condition affecting cognitive and behavioral development.

Intervention/Treatment: The study tests various doses of MRM-3379, an oral capsule, across five groups: low, middle, and high doses for the main cohort, a placebo group, and a low-dose open-label group for younger participants. MRM-3379 is intended to improve FXS symptoms by targeting underlying biological pathways.

Study Design: This interventional study employs a randomized, parallel assignment model with double masking for participants and investigators. The primary purpose is treatment-focused, aiming to establish proof-of-concept for MRM-3379’s efficacy in treating FXS symptoms.

Study Timeline: The study began on October 2, 2025, with the latest update submitted on October 23, 2025. These dates mark the study’s initiation and the most recent progress update, indicating ongoing recruitment and data collection efforts.

Market Implications: The ongoing study could impact Mirum Pharmaceuticals’ stock performance positively if MRM-3379 shows promising results, potentially increasing investor confidence. As FXS lacks effective treatments, successful outcomes could position Mirum as a leader in this niche market, influencing the competitive landscape.

The study is currently recruiting, with further details available on the ClinicalTrials portal.