― On track to report topline data for Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in the fourth quarter of 2024 ―
― Restart of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) underway ―
― Conference call and webcast scheduled for 8:00 a.m. ET today ―
CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc.® (“Fulcrum”) (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today reported financial results for the fourth quarter and full year of 2023 as well as an update to the business.
“In 2023, we took important steps to advance our two key clinical programs which included completing enrollment for the Phase 3 REACH trial of losmapimod in FSHD and resolving the clinical hold for our Phase 1b PIONEER trial of pociredir in SCD,” said Alex C. Sapir, Fulcrum’s president and chief executive officer. “We are on track to report topline data for REACH in the fourth quarter of 2024, which could position losmapimod as the first approved treatment for patients with FSHD. The clinical data generated to date demonstrates losmapimod has the potential to slow progression and address the debilitating effects of this disease. Additionally, for PIONEER, we are encouraged by the level of physician engagement, and we are working diligently to activate additional trial sites and resume enrollment. We believe pociredir has the potential to shift the current standard of care and offer a differentiated, oral treatment option for patients with SCD.”
Recent Business Highlights
REACH, the Phase 3 clinical trial evaluating losmapimod in patients with FSHD continues to progress, and Fulcrum expects to report topline data in the fourth quarter of 2024. The trial enrolled 260 patients across sites in the United States, Canada, and Europe. There are currently no approved treatments for FSHD.
Clinical trial sites have been activated for the Phase 1b trial evaluating pociredir in patients with SCD. Cohort 3 of the Phase 1b trial will evaluate pociredir at the 12 mg once daily dose, followed by Cohort 4 at the 20 mg once daily dose. Each cohort is expected to enroll approximately 10 patients.
Fourth Quarter and Full Year 2023 Financial Results
Cash Position: As of December 31, 2023, cash, cash equivalents, and marketable securities were $236.2 million, as compared to $202.9 million as of December 31, 2022. The increase in our cash position is primarily due to net proceeds from our equity offering completed in January 2023 of $117.3 million, partially offset by our net cash used in operating activities in 2023.
Collaboration Revenue: Collaboration revenue was $0.9 million for the fourth quarter of 2023 as compared to $0.7 million for the fourth quarter of 2022. The increase of $0.2 million was attributable to an increase in revenues under our collaboration agreement with MyoKardia as we completed our research services during the fourth quarter of 2023.
Collaboration revenue was $2.8 million for the year ended December 31, 2023, as compared to $6.3 million for the year ended December 31, 2022. The decrease of $3.5 million was attributable to the completion of activities under our collaboration agreement with Acceleron, which terminated in October 2022, and due to a decrease in revenues under our collaboration agreement with MyoKardia as we completed our research services during the fourth quarter of 2023.
R&D Expenses: Research and development expenses were $19.0 million for the fourth quarter of 2023 as compared to $18.6 million for the fourth quarter of 2022. The increase of $0.4 million was primarily due to increased personnel costs.
Research and development expenses were $71.8 million for the year ended December 31, 2023 as compared to $76.8 million for the year ended December 31, 2022. The decrease of $5.0 million was primarily due to a $5.0 million obligation to GSK incurred in the second quarter of 2022 upon initiation of the REACH clinical trial.
G&A Expenses: General and administrative expenses were $9.9 million for the fourth quarter of 2023 as compared to $10.1 million for the fourth quarter of 2022. The decrease of $0.2 million was primarily due to decreased professional services costs.
General and administrative expenses were $41.7 million for each of the years ended December 31, 2023 and 2022.
Net Loss: Net loss was $24.8 million for the fourth quarter of 2023 as compared to $26.1 million for the fourth quarter of 2022.
Net loss was $97.3 million for the year ended December 31, 2023 as compared to $109.9 million for the year ended December 31, 2022.
Cash Runway Guidance
Fulcrum continues to expect that its existing cash, cash equivalents, and marketable securities will be sufficient to fund its operating requirements into 2026.
Conference Call and Webcast
Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to review the fourth quarter and full year 2023 recent business highlights and financial results. Individuals may register for the conference call by clicking the link here. Once registered participants will receive dial-in details and a unique pin which will allow them to access the call. The webcast will be accessible through the Investor Relations section of Fulcrum’s website at www.fulcrumtx.com or by clicking here. Following the live webcast, an archived replay will also be available for 90 days.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and pociredir (formerly known as FTX-6058), a small molecule designed to increase expression of fetal hemoglobin and in development for the treatment of sickle cell disease (SCD). Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. For more information, visit www.fulcrumtx.com and follow us on Twitter/X (@FulcrumTx) and LinkedIn.
About Losmapimod
Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Results reported from the Phase 2b ReDUX4 trial demonstrated slower disease progression and improved function, including positive impacts on upper extremity strength and functional measures supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications with no safety signals attributed to losmapimod. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD. Losmapimod is currently being evaluated in a Phase 3 multi-center randomized, double-blind, placebo-controlled, 48-week parallel-group study in people with FSHD (NCT05397470).
About FSHD
FSHD is a serious, rare, progressive, and debilitating disease for which there are no approved treatments. It is characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes relentless and accumulating muscle and functional loss impacting their ability to perform activities of daily living, loss of upper limb function, loss …
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