― Fulcrum plans to initiate a potential registration-enabling trial in the second half of 2026 ―
― Dosed first patient in an open-label, long-term dosing trial evaluating the long-term safety and durability of response to pociredir in participants previously enrolled in the PIONEER trial ―
― Appointed Josh Lehrer, M.D., M.Phil., FACC, an experienced leader in sickle cell disease drug development, to the Board of Directors ―
― Chief Financial Officer, Alan Musso plans to retire later this year and will continue in his role until a successor is named ―
― Ended the first quarter of 2026 with $333.3 million in cash, cash equivalents, and marketable securities; cash runway into 2029 ―
CAMBRIDGE, Mass., April 27, 2026 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules that improve the lives of patients with rare hematological disorders, today reported financial results for the first quarter of 2026 and provided a business update.
"The strength of the clinical data presented in the first quarter further reinforce our conviction in pociredir's potential to address the underlying biology of sickle cell disease," said Alex C. Sapir, Fulcrum's President and Chief Executive Officer. "The magnitude of HbF induction and improvements in markers of hemolysis and anemia observed to date support our upcoming discussions with the FDA as we prepare for a potential registration-enabling study in the second half of 2026. With a strong balance sheet extending our cash runway into 2029, we are well positioned to advance pociredir through the next phase of clinical development."
"I am also pleased to welcome Dr. Josh Lehrer to Fulcrum's Board of Directors. Josh's track record advancing transformative therapies for patients with sickle cell disease, most notably his experience with the development and approval of Oxbryta®, will be invaluable as we advance pociredir into the next phase of development. I would also like to thank Alan Musso, who will be retiring as CFO this year, for his years of dedication and unwavering commitment to Fulcrum's success. During his tenure, he not only strengthened Fulcrum's balance sheet through our recent financing, but also provided important strategic perspectives and instilled strong financial discipline across the organization."
Recent Business Highlights
Presented positive clinical data from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) during the first quarter of 2026, demonstrating robust and rapid HbF induction, progression toward pan-cellular distribution, improvements in markers of hemolysis and anemia, and encouraging trends in VOC reduction. Pociredir continues to be generally well-tolerated, with no treatment-related serious adverse events reported to date.
Fulcrum expects to provide an update on the design of its next trial in the second quarter of 2026 following receipt of meeting minutes from its End-of-Phase meeting with the U.S. Food and Drug Administration (FDA). Pending feedback from the FDA, Fulcrum plans to initiate a potential registration-enabling trial in the second half of 2026.
Dosed first patient in an open-label, long-term dosing trial designed to evaluate the long-term safety and durability of response to pociredir in participants previously enrolled in the Phase 1b PIONEER trial.
An abstract from the Phase 1b PIONEER trial of pociredir in sickle cell disease has been accepted for oral presentation at the Foundation for Sickle Cell Disease Research Symposium 2026, to be held in June 2026, featuring previously disclosed clinical data.
Announced a patient-focused collaboration with MedicAlert Foundation and the Sickle Cell Disease Association of America to help improve access to patient-specific care information in emergency department settings for individuals living with sickle cell disease.
Chief Financial Officer Alan Musso plans to retire later this year to spend more time with his family and other outside interests. Mr. Musso will remain in his role until a successor is named and has agreed to serve as a consultant thereafter to support a seamless transition. Fulcrum will initiate a search to identify a successor.
First Quarter 2026 Financial Results
Cash Position: As of March 31, 2026, cash, cash equivalents, and marketable securities were $333.3 million, compared to $352.3 million as of December 31, 2025. The decrease of $19.0 million was primarily due to cash used to fund operating activities in 2026.
R&D Expenses: Research and development expenses were $14.1 million for the three months ended March 31, 2026, compared to $13.4 million for the three months ended March 31, 2025. The increase of $0.7 million was primarily due to higher employee compensation costs, including $0.4 million of increased stock-based compensation expense.
G&A Expenses: General and administrative expenses were $8.1 million for the three months ended March 31, 2026, compared to $7.0 million for three months ended March 31, 2025. The increase of $1.1 million was primarily driven by higher employee compensation costs, including $0.3 million of increased stock-based compensation expense, as well as higher professional services costs.
Net Loss: Net loss was $18.9 million for the three months ended March 31, 2026, compared to a net loss of $17.7 million for the three months ended March 31, 2025.
Cash Runway Guidance
Based on its current operating plans, Fulcrum expects that its current cash, cash equivalents, and marketable securities will be sufficient to fund its operating requirements into 2029.
About Fulcrum TherapeuticsFulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules that improve the lives of people with rare hematological disorders. The company's lead clinical program is pociredir, a small molecule designed to ...
