Fulcrum Therapeutics to Host Investor Event to Review New Clinical Data from the PIONEER trial of Pociredir in Sickle Cell Disease on December 7, 2025
CAMBRIDGE, Mass., Dec. 02, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, will host an investor event on Sunday, December 7, 2025 at 7:00 a.m. ET to review new clinical data from the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD). These data will also be presented at the 67th American Society of Hematology (ASH) Annual Meeting.
Members of Fulcrum management will be joined by Dr. Sheinei Alan, Director of the Inova Adult Sickle Cell Program and Assistant Professor at UVA School of Medicine, and Dr. Martin Steinberg, Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine at Boston University Chobanian and Avedisian School of Medicine.
To register, please click here or visit the "Events and Presentations" section of Fulcrum's website. A replay will be available on Fulcrum's website following the event.
The event will provide initial clinical data from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD, and full data from the 12 mg dose cohort.
A live question and answer session will follow the formal presentation.
About Sheinei Alan, MD, PhD
Dr. Sheinei Alan earned her MD and PhD from Virginia Commonwealth University, followed by an internal medicine residency at Georgetown University. She currently serves as Director of the Inova Adult Sickle Cell Program—one of the most comprehensive in the region, caring for more than 250 adults with sickle cell disease—and as an Assistant Professor of Medicine at the University of Virginia School of Medicine – Inova Campus. As principal investigator for multiple cutting-edge clinical trials in sickle cell disease, Dr. Alan leads both industry-sponsored studies of novel therapeutic approaches and an internally funded investigation into cardiac complications in this patient population. A key member of the National Alliance of Sickle Cell Centers, she plays an active role in developing consensus guidelines, standardizing care nationwide, and creating pathways to train the next generation of sickle cell disease specialists. She also contributed her expertise to the NIH-funded, St. Jude–sponsored development of the Sickle Cell Outcomes Grading System (SCOGS). Dr. Alan has authored numerous publications and is frequently invited to speak at national and international sickle cell conferences, positioning Inova as a leader in comprehensive, specialized care. Under her leadership, the program has strengthened community partnerships, expanded advocacy and education initiatives across Northern Virginia.
About Martin H Steinberg, MD
Dr. Martin H Steinberg, MD is a hematologist with a clinical and research focus on disorders of the red blood cell with special emphasis on sickle cell disease. He has published more than 450 articles and 3 textbooks on the science and clinical features of sickle cell disease and related disorders. A graduate of Cornell University and Tufts University School of Medicine he completed post-graduate training in New York and Boston. He has participated in basic, translational, and clinical studies devoted to understanding the pathophysiology and genetic basis of phenotypic heterogeneity in sickle cell disease. Using candidate gene, genome-wide association studies, next-generation sequencing, and induced pluripotent stem cells to understand the genetic determinants of sickle cell disease heterogeneity, Dr. Steinberg and his coworkers modeled disease severity and selected subphenotypes of disease to discover hitherto unsuspected genetic associations. He has also helped develop a widely accepted paradigm reimagining the pathophysiology of sickle cell disease as a combination of both sickle vasoocclusion and intravascular hemolysis. His most recent work focusses on the distribution of HbF concentrations among red cells of patients before and following HbF induction therapeutics.
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 lead clinical program is pociredir, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease. 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 http://www.fulcrumtx.com and follow us on X (@FulcrumTx) and LinkedIn.
About Pociredir
Pociredir 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 fetal hemoglobin (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 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.
Contact:
Kevin Gardner
LifeSci Advisors, LLC
[email protected]
617-283-2856
