The full results of the international REACH study, a phase 3 clinical trial investigating the drug Losmapimod for FSHD, have recently been published. The study was conducted across nine countries and included a total of 260 participants with FSHD1 or FSHD2.
Purpose of the study
The REACH study examined the efficacy and safety of Losmapimod in FSHD. It was the first large registration trial to use Reachable Workspace (RWS) as the primary endpoint, a measure of arm and shoulder function that reflects the challenges of people with FSHD experience in daily life.
Key findings
Although Losmapimod was generally safe and well tolerated, the study did not meet its primary endpoint: no difference was observed between the Losmapimod and placebo groups in improvement of RWS after 48 weeks. Notably, both groups showed improvement in RWS, likely due to a combination of placebo effects, learning effects, and high participant expectations. MRI data revealed small, nondefinitive advantages for Losmapimod in some subgroups, but these were insufficient to confirm a clinical benefit.
Valuable insights for future research
Despite the lack of demonstrated efficacy, the REACH study provides important insights for future FSHD research. The study highlights:
- the need for better and more sensitive outcome measures, particularly for long‑term studies
- the importance of managing patient expectations to minimize placebo effects
- that careful patient selection (for example based on disease activity) may be essential
- that longer trial durations may be necessary to detect treatment effects.
Conclusion
Losmapimod appears to be safe, however, the REACH study found no evidence of clinical efficacy within the 48-week study period. Even so, the trial represents an important milestone, providing new insights that can help accelerate and improve the development of future FSHD therapies.
Click here for the official publication, including the full background, methodology, results, and conclusions.
FSHD Europe would like to express our sincere appreciation to all the patients who participated in the REACH study. We remain committed and dedicated to working together toward our shared goal of finding an effective treatment for FSHD.