Fulcrum Therapeutics Reports Q1 2024 Business Highlights and Financial Results

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, recently announced its financial results for the first quarter of 2024 and provided significant business updates. Specializing in the development of small molecules aimed at treating genetically defined rare diseases, Fulcrum has made substantial strides in its research and development efforts.

In a major development, Fulcrum has entered into a strategic collaboration and licensing agreement with Sanofi for the development and commercialization of losmapimod. Under this agreement, Sanofi will hold exclusive rights to commercialize losmapimod outside the United States. Fulcrum will receive an initial payment of $80 million and stands to gain up to an additional $975 million based on regulatory and sales milestones. Both companies will share global development costs equally.

Fulcrum is progressing well with its Phase 3 REACH trial, which is evaluating losmapimod in patients with facioscapulohumeral muscular dystrophy (FSHD). The company anticipates reporting topline data from this trial in the fourth quarter of 2024. This trial, which enrolled 260 patients across the United States, Canada, and Europe, aims to address a significant unmet need as there are currently no approved treatments for FSHD. Previous studies, such as the Phase 2 ReDUX4 trial, have shown promising results, including improvements in functional and structural outcomes for patients treated with losmapimod.

In addition to losmapimod, Fulcrum is advancing its Phase 1b PIONEER trial of pociredir, a novel oral treatment for sickle cell disease (SCD). The trial has activated additional clinical sites, and Fulcrum is working closely with leading physicians in the SCD community. Pociredir, which aims to increase fetal hemoglobin expression, has shown potential benefits in initial data and has been granted Fast Track and Orphan Drug Designation by the U.S. FDA for the treatment of SCD.

The company also announced the appointment of Dr. Patrick Horn as its new chief medical officer. Dr. Horn brings over two decades of experience in late-stage rare disease drug development and will help steer Fulcrum's clinical programs and regulatory strategies.

Financially, Fulcrum reported a cash position of $213.3 million as of March 31, 2024, down from $236.2 million at the end of 2023. This decrease is attributed to net cash used in operating activities. However, with the upfront payment from Sanofi, the company’s pro forma cash position is approximately $293.3 million. Research and development expenses increased to $19.8 million for the first quarter of 2024, up from $16.7 million in the same period last year, primarily due to the ongoing advancement of the REACH trial. General and administrative expenses decreased slightly to $10.1 million, compared to $11.5 million in the first quarter of 2023, mainly due to reduced employee compensation costs. The net loss for the quarter was $26.9 million.

Based on current operating plans, Fulcrum expects its cash, cash equivalents, and marketable securities, combined with the $80 million payment from Sanofi, to fund its operations into 2027.

Fulcrum Therapeutics is committed to advancing its pipeline of promising therapies for patients with rare genetic diseases. With strategic collaborations, robust clinical programs, and a strengthened leadership team, the company is well-positioned to make significant strides in its mission to improve patient outcomes.