Mirum Pharmaceuticals has officially enrolled the first participant in its BLOOM Phase 2 clinical study evaluating MRM-3379, a potential new treatment for Fragile X syndrome (FXS).
What’s Happening
The BLOOM study is now underway, enrolling males living with FXS to evaluate the safety, tolerability, and early signs of benefit of MRM-3379 over a 12-week period. This is the first time this investigational therapy is being tested in individuals with Fragile X.
Why This Matters
MRM-3379 targets a biological pathway known to be disrupted in Fragile X. Early preclinical research suggests it may improve key challenges related to behavior, cognition, and daily functioning. With no FDA-approved treatments that address the underlying biology of FXS, any drug showing potential in this area represents a major step forward.
What’s Next
As the study progresses, updates on safety, enrollment, and early outcomes will help determine whether MRM-3379 advances to later-stage trials. Mirum expects top-line Phase 2 data in 2027.
This milestone underscores continued forward movement in Fragile X research — and the community’s ongoing drive toward better treatments and brighter futures.
Read the full press release here.
