Fulcrum Therapeutics

Dyne Therapeutics' DYNE-302 demonstrated functional improvement in a severe FSHD mouse model, with a single intravenous dose restoring treadmill running ability at peak muscle weakness.

Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD).

Fulcrum Therapeutics has successfully enrolled 10 patients in the 12mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease, with data expected by mid-2025.

• Pfizer has voluntarily withdrawn Oxbryta (voxelotor) from all global markets due to safety concerns, citing an imbalance in vaso-occlusive crises and fatal events. • The decision follows a review of clinical data that indicated the overall benefit of Oxbryta no longer outweighs the risks for sickle cell disease patients. • Regulatory agencies, including the EMA and FDA, have alerted healthcare professionals and patients about the withdrawal, advising discussions on alternative treatments. • The withdrawal impacts ongoing clinical trials and expanded access programs, leaving the sickle cell community seeking alternative treatment options and raising concerns about trust.

• Fulcrum Therapeutics' Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) did not meet its primary endpoint, showing no significant improvement in reachable workspace compared to placebo. • Secondary endpoints, including muscle fat infiltration and shoulder strength, also failed to achieve statistical significance in the losmapimod arm. • Fulcrum plans to suspend the losmapimod program and shift focus to pociredir for sickle cell disease and other early-stage programs. • The company's stock price plummeted following the announcement, raising concerns about its future prospects despite a solid cash position.