Full Press Release Details
CAMBRIDGE, Mass., May 29, 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 upcoming presentations at the 5th Global Congress on Sickle Cell Disease and the 2025 European Hematology Association Congress.
The Company’s presentations are listed below. Following congress publication, copies of the presentations will be available in the "Publications & Presentations" section of the Company's website at https://www.fulcrumtx.com/publications-presentations.
5th Global Congress on Sickle Cell Disease, Abuja, Nigeria, June 3-6, 2025:
Poster Presentation: Preclinical and Early Clinical Results of Pociredir, a Novel, Oral, Potent Fetal Hemoglobin Inducer for the Treatment of Sickle Cell Disease (SCD) (Presentation ID: 103, Tuesday, June 4)
Poster Presentation: A Phase 1b, Open-Label, Multiple-Dose Study Evaluating Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Pociredir in Patients with Sickle Cell Disease (SCD): Trial Design (Presentation ID: 101, Tuesday, June 4)
2025 European Hematology Association Congress, Milan, Italy, June 12-15, 2025
Poster Presentation: Pharmacokinetics (PK), Pharmacodynamics (PD), and Safety of the Novel Oral Fetal Hemoglobin (HBF) Inducer Pociredir in Healthy Adults in a Phase 1 study (PF1170 – Friday, June 13)
Poster Presentation: Pociredir, a Potent and Selective EED Inhibitor for the Treatment of Sickle Cell Disease, Induces Target Engagement and Gene Expression Changes that are Specific and Reversible in Wild-Type Mice (PS2175 – Saturday, June 14)
About Fulcrum Therapeutics
Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (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 fetal hemoglobin (HbF). Pociredir is being developed for the treatment of SCD. Initial data in SCD demonstrated proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. In clinical trials conducted prior to the clinical hold, which was lifted by the FDA in August 2023, pociredir was generally well-tolerated in people with SCD with up to three months of exposure, with no serious treatment-related adverse events reported. Pociredir has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about these trials please visit ClinicalTrials.gov.
About Sickle Cell Disease
Sickle cell disease (SCD) 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.
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