Fulcrum Therapeutics Suspends Rare Muscular Disease Candidate After Disappointing Data From Pivotal Trial

Fulcrum Therapeutics Inc. FULC stock is trading lower on Thursday after the company released data from the Phase 3 REACH trial evaluating losmapimod in patients with Facioscapulohumeral Muscular Dystrophy (FSHD).

The study did not achieve its primary endpoint of change from baseline in relative surface area (RSA) with losmapimod compared to placebo.

Also Read: Fulcrum Therapeutics Upgraded: BofA Debates Pivotal Study Success For Losmapimod In Rare Genetic Muscle Disease.

In addition, secondary endpoints did not achieve nominal statistical significance. Losmapimod’s safety and tolerability profile was consistent with previously reported studies.

The company will complete a full evaluation of the data it received this week and plans to share the results at an upcoming medical meeting.

• Participants receiving losmapimod demonstrated a 0.013 (±0.007) improvement in RSA at week 48 compared to placebo patients, who showed a 0.010 improvement in RWS (p-value = 0.75).
• RSA measures reachable workspace (RWS), an outcome measure used to track disease progression in neuromuscular disorders that affect the upper extremities.
• Participants receiving losmapimod demonstrated an increase of 0.42% in Muscle Fat Infiltration (a measurement indicating the quality of the muscle) at week 48 compared to participants receiving placebo, who showed an increase of 0.576% (p-value = 0.16).
• Participants receiving losmapimod demonstrated a 9.63% improvement in abductor strength at week 48 compared to a 2.24% improvement in the placebo arm of the study (p-value = 0.51).

The rate of treatment-related adverse events was similar in the two treatment arms, and there were no treatment-related serious adverse events in participants receiving losmapimod.

“We are deeply disappointed that the REACH trial did not replicate the clinical results observed in the Phase 2 ReDUX4 trial,” said Alex Sapir, Fulcrum’s president and CEO. “In light of these results, we plan to suspend the losmapimod program in FSHD.”

As previously disclosed, Fulcrum had approximately $273.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024.

Fulcrum intends to use these resources to advance pociredir for sickle cell disease, novel therapeutic agents for Diamond-Blackfan Anemia, and early discovery programs.

Price Action: FULC stock is down 59.30% at $3.60 at the last check on Thursday.

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