Fulcrum Therapeutics Reports Q2 2024 Business Highlights and Financial Results

Fulcrum Therapeutics, Inc., based in Cambridge, Massachusetts, has declared its financial outcomes for the second quarter of 2024 and provided a business update. The clinical-stage biopharmaceutical firm, listed on Nasdaq under the ticker FULC, specializes in developing small molecules to enhance the lives of patients with rare, genetically defined diseases. 

President and CEO Alex C. Sapir asserted that the company is on track to disclose topline results for the Phase 3 REACH trial by the end of October 2024. This trial assesses the efficacy of losmapimod in treating facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle-wasting condition for which there are currently no approved treatments. Fulcrum is also gearing up for the potential New Drug Application (NDA) filing and subsequent U.S. launch of losmapimod. In collaboration with Sanofi, Fulcrum is preparing for regulatory filings and the launch of losmapimod outside the U.S.

The Phase 3 REACH trial has enrolled 260 patients across sites in the United States, Canada, and Europe. By June 30, 2024, 234 patients had completed the 48-week treatment phase, with 232 opting for the open-label extension. Fulcrum is on schedule to complete activities agreed with the U.S. Food and Drug Administration (FDA) to define the clinical importance of RWS, the trial's primary endpoint, at the time of reporting topline data.

In addition to progressing with losmapimod, Fulcrum is advancing its Phase 1b PIONEER trial evaluating pociredir for sickle cell disease (SCD). This trial will explore different dosing levels, with Cohort 3 and Cohort 4 administering 12 mg and 20 mg once daily, respectively. Interim results from this trial were presented at the European Hematology Association's 2024 Hybrid Congress.

Financially, Fulcrum has seen significant changes. As of June 30, 2024, the company’s cash, cash equivalents, and marketable securities totaled $273.8 million, up from $236.2 million on December 31, 2023. This rise is mainly attributed to an $80 million upfront payment received from Sanofi. Collaboration revenue for the three months ending June 30, 2024, was $80.0 million, a substantial increase from $0.9 million during the same period in the previous year, mainly due to the Sanofi payment. Research and development expenses were slightly down to $17.3 million, influenced by cost-sharing with Sanofi, whereas general and administrative expenses remained nearly stable at $10.2 million. Net income for the second quarter of 2024 was $55.4 million, compared to a net loss of $23.8 million in the same period in 2023, largely driven by the Sanofi collaboration payment.

Fulcrum anticipates that its current cash reserves will sustain its operations into 2027, based on existing plans. 

A conference call and webcast were scheduled at 8:00 a.m. ET on the announcement day to review the second quarter financial results and business highlights.

Fulcrum Therapeutics focuses on developing small molecules for the treatment of genetically defined rare diseases. Its leading programs include losmapimod for FSHD and pociredir for SCD. Losmapimod has demonstrated potential benefits in previous trials and has received FDA Fast Track and Orphan Drug Designations. Pociredir is designed to increase fetal hemoglobin levels, potentially alleviating symptoms of SCD, and has also received FDA Fast Track and Orphan Drug Designations.

FSHD is a severe, progressive disease characterized by muscle atrophy and involves a significant impact on daily living activities due to relentless muscle and functional loss. Meanwhile, SCD is a genetic disorder affecting hemoglobin, leading to severe clinical consequences such as anemia, pain, and organ damage.

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