CAMBRIDGE, Mass., Aug. 26, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (the “Company”) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that company management will attend and present at the following investor conferences: Cantor Fitzgerald 2025 Annual Global Healthcare Conference Location: New York, NY, U.S.Date: September 4, 2025Time: 1:35-2:05 PM ETType: Fires ...

Core Insights - Fulcrum Therapeutics reported promising results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD), showing a robust mean increase of 8.6% in fetal hemoglobin (HbF) and improvements in key markers of hemolysis and anemia [1][5] - The company ended Q2 2025 with $214.1 million in cash, providing a cash runway into 2028 [1][6] - Collaboration revenue was zero for Q2 2025, a decrease from $80 million in Q2 2024, primarily due to the recognition of an upfront license payment from Sanofi in the previous year [5][11] Recent Business Highlights - The 12 mg dose cohort of the PIONEER trial demonstrated no treatment-related serious adverse events, indicating that pociredir was generally well-tolerated [1][5] - The ongoing 20 mg dose cohort is expected to provide clinical data by the end of 2025 [1][5] - Fulcrum is advancing its program for inherited aplastic anemias and plans to submit an investigational new drug application for Diamond-Blackfan anemia in Q4 2025 [5] Financial Results - Research and development expenses for Q2 2025 were $13 million, down from $17.3 million in Q2 2024, attributed to reduced employee compensation costs and the discontinuation of the losmapimod program [5][11] - General and administrative expenses decreased to $6.8 million in Q2 2025 from $10.2 million in Q2 2024, reflecting lower professional services and employee compensation costs [11] - The net loss for Q2 2025 was $17.3 million, compared to a net income of $55.4 million in Q2 2024 [11][16]

Core Insights - Fulcrum Therapeutics reported positive results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) [2][3] Efficacy Data - The mean absolute fetal hemoglobin (HbF) increased by 8.6% from a baseline of 7.6% to 16.2% after 12 weeks of treatment, with 7 out of 16 patients achieving HbF levels greater than 20% [4][10] - The proportion of F-cells increased from 34% at baseline to 67% at 12 weeks, indicating pan-cellular induction of HbF [4][10] - Key markers of hemolysis showed meaningful improvements, including a mean increase in total hemoglobin of 0.9 g/dL, from 7.8 g/dL to 8.7 g/dL [10] - A trend of reduced vaso-occlusive crisis (VOC) rates was observed, with 50% of patients reporting no VOCs during the treatment period [10] Safety Profile - Pociredir was generally well-tolerated, with no treatment-related serious adverse events (SAEs) reported, and all treatment-related adverse events (AEs) were Grade 1 [3][10] - The safety profile observed in the 12 mg dose cohort was consistent with previously reported data [10] Company Overview - Fulcrum Therapeutics is focused on developing small molecules for genetically defined rare diseases, with pociredir as its lead clinical program aimed at increasing HbF expression for SCD treatment [8][9]

Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases, particularly in areas with high unmet medical need [3] - The company's lead clinical program is pociredir, aimed at increasing fetal hemoglobin (HbF) levels for the treatment of sickle cell disease (SCD) [3][4] Upcoming Event - Fulcrum will host a conference call and webcast on July 29, 2025, at 8:00 a.m. ET to present topline results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD [1] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), discovered using Fulcrum's proprietary technology [4] - The drug works by downregulating fetal globin repressors, leading to increased HbF levels, which may benefit patients with SCD [4] - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for SCD treatment [4] Sickle Cell Disease (SCD) Background - SCD is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [5] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [5]

Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases with high unmet medical needs [2][3] - The company's lead clinical program is pociredir, designed to increase fetal hemoglobin expression for treating sickle cell disease (SCD) [2][3] Upcoming Presentations - Fulcrum Therapeutics will present at the 5th Global Congress on Sickle Cell Disease in Abuja, Nigeria, from June 3-6, 2025, and at the 2025 European Hematology Association Congress in Milan, Italy, from June 12-15, 2025 [2] - Specific presentations include preclinical and early clinical results of pociredir, a Phase 1b study evaluating safety and pharmacokinetics in SCD patients, and pharmacokinetics and safety in healthy adults [5] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), leading to downregulation of fetal globin repressors and increased fetal hemoglobin levels [3] - Initial data showed proof-of-concept for pociredir in SCD, with well-tolerated results in clinical trials prior to the FDA lifting the clinical hold in August 2023 [3] Sickle Cell Disease Overview - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [4] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [4]

Core Insights - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [3] - The company will participate in a fireside chat at the 46th Annual Goldman Sachs Global Healthcare Conference on June 11, 2025 [1] - Fulcrum's lead clinical program is pociredir, aimed at increasing fetal hemoglobin expression for treating sickle cell disease [3] Company Overview - Fulcrum Therapeutics specializes in small molecule development to address high unmet medical needs in rare diseases [3] - The company employs proprietary technology to identify drug targets that modulate gene expression [3] - More information about the company can be found on their website and social media platforms [3]

Core Insights - Fulcrum Therapeutics has completed enrollment in the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) and has initiated the 20 mg dose cohort [1][6] - The company plans to share data from the 12 mg dose cohort in early Q3 2025 and from the 20 mg dose cohort by the end of 2025 [2][5] Recent Business Highlights - The PIONEER trial shows strong engagement with 16 patients enrolled in the 12 mg cohort and adherence rates exceeding 90% [6] - Two abstracts related to pociredir have been accepted for presentation at the 2025 European Hematology Association Congress [6] - The company is advancing its program for inherited aplastic anemias and plans to submit an IND for Diamond-Blackfan anemia in Q4 2025 [6] Financial Results - As of March 31, 2025, Fulcrum had $226.6 million in cash, cash equivalents, and marketable securities, down from $241.0 million at the end of 2024 [5][14] - Research and development expenses for Q1 2025 were $13.4 million, a decrease from $19.8 million in Q1 2024, primarily due to the discontinuation of the losmapimod program [5][15] - General and administrative expenses decreased to $7.0 million in Q1 2025 from $10.1 million in Q1 2024, attributed to reduced employee compensation costs [5][15] - The net loss for Q1 2025 was $17.7 million, compared to a net loss of $26.9 million in Q1 2024 [5][15] Cash Position and Guidance - Fulcrum expects its current cash position to fund operations into at least 2027 [7]