Full Press Release Details
― Announced encouraging results in July 2025 from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) ―
― Enrollment complete in the 20 mg dose cohort (n=12) of the PIONEER trial; on track to provide data from the 20 mg dose cohort by year-end ―
― Ended Q3 2025 with $200.6 million in cash, cash equivalents, and marketable securities; cash runway into 2028 ―
CAMBRIDGE, Mass., Oct. 29, 2025 (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 third quarter of 2025 and provided a business update.
“We are extremely pleased with the compelling data from the 12 mg dose cohort of the PIONEER trial, which demonstrated that pociredir has the potential to meaningfully improve outcomes for people living with sickle cell disease,” said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer. “The strength of those results has generated significant interest and engagement from investigators and patients, reflected in the over-enrollment of the 20 mg dose cohort. We look forward to sharing results from the 20 mg dose cohort by the end of 2025.”
Recent Business Highlights
Third Quarter 2025 Financial Results
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 2028.
About Fulcrum TherapeuticsFulcrum 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 lead clinical program is pociredir, a small molecule designed to increase expression of HbF for the treatment of 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, visitwww.fulcrumtx.comand follow us on X (@FulcrumTx) and LinkedIn.
About PociredirPociredir is an investigational oral small-molecule inhibitor of EED that was discovered using Fulcrum’s proprietary discovery technology. Inhibition of EED leads to potent downregulation of key fetal globin repressors, including BCL11A, thereby causing an increase in HbF. Pociredir is being developed for the treatment of SCD. Initial data in SCD in the PIONEER Phase 1b clinical trial showed proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. Through the completion of the 12 mg dose cohort, pociredir was demonstrated to be generally well-tolerated in people with SCD with up to three months of exposure, with no treatment-related SAEs reported. Pociredir has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about clinical trials of pociredir please visitClinicalTrials.gov.
About Sickle Cell DiseaseSCD is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. People with SCD typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease, and reduced life expectancy.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, including express or implied statements regarding Fulcrum’s Phase 1b PIONEER clinical trial of pociredir, including planned data announcement for such trial; the potential of pociredir to increase HbF to levels that could ameliorate symptoms of SCD and transform the standard of care; Fulcrum’s ability to progress its early stage development programs and planned IND filings related thereto; and its projected cash runway, among others. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum’s ability to continue to advance its product candidates in clinical trials, including progressing early stage candidates into the clinic; initiating and enrolling clinical trials on the timeline expected or at all; obtaining and maintaining necessary approvals from the FDA and other regulatory authorities; replicating in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials; obtaining, maintaining or protecting intellectual property rights related to its product candidates; managing expenses; realizing the anticipated benefits of the workforce reduction and strategic realignment and managing risks associated therewith; and raising the substantial additional capital needed to achieve its business objectives, among others. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.
| Fulcrum Therapeutics, Inc.Selected Consolidated Balance Sheet Data(In thousands)(Unaudited) | ||||||||
| September 30,2025 | December 31,2024 | |||||||
| Cash, cash equivalents, and marketable securities | $ | 200,645 | $ | 241,021 | ||||
| Working capital(1) | 194,231 | 238,879 | ||||||
| Total assets | 214,858 | 260,718 | ||||||
| Total stockholders’ equity | 198,366 | 243,034 |
(1) Fulcrum defines working capital as current assets minus current liabilities.
| Fulcrum Therapeutics, Inc.Consolidated Statements of Operations(In thousands, except per share data)(Unaudited) | ||||||||||||||||
| Three Months EndedSeptember 30, | Nine Months EndedSeptember 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Collaboration revenue | — | — | — | 80,000 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 14,296 | 14,639 | 40,687 | 51,673 | ||||||||||||
| General and administrative | 7,562 | 8,424 | 21,389 | 28,732 | ||||||||||||
| Restructuring expenses | — | 2,063 | — | 2,063 | ||||||||||||
| Total operating expenses | 21,858 | 25,126 | 62,076 | 82,468 | ||||||||||||
| Loss from operations | (21,858 | ) | (25,126 | ) | (62,076 | ) | (2,468 | ) | ||||||||
| Other income, net | 2,263 | 3,430 | 7,530 | 9,311 | ||||||||||||
| Net (loss) income | $ | (19,595 | ) | $ | (21,696 | ) | $ | (54,546 | ) | $ | 6,843 | |||||
| Net (loss) income per share, basic | $ | (0.31 | ) | $ | (0.35 | ) | $ | (0.87 | ) | $ | 0.11 | |||||
| Net (loss) income per share, diluted | $ | (0.31 | ) | $ | (0.35 | ) | $ | (0.87 | ) | $ | 0.11 | |||||
| Weighted-average common shares outstanding, basic | 62,597 | 62,409 | 62,537 | 62,200 | ||||||||||||
| Weighted-average common shares outstanding, diluted | 62,597 | 62,409 | 62,537 | 63,688 |