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Fulcrum Therapeutics Presents Updated Data On Sickle Cell Disease Program At The 62nd American Society Of Hematology (ASH) Annual Meeting And Exposition

CAMBRIDGE, Mass., Dec.

CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.  (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that preclinical data with FTX-6058 for the treatment of sickle cell disease will be presented in three posters at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 5-8, 2020. FTX-6058 is a highly potent small molecule EED inhibitor that induces expression of fetal hemoglobin (HbF). Elevating HbF can compensate for the mutated adult hemoglobin that has been identified as the root cause of several hemoglobinopathies and can ameliorate or eliminate the symptoms of sickle cell disease.

"We are encouraged by the robust preclinical data package and unique mechanism of action of FTX-6058, which has the potential to be a transformative therapy for sickle cell patients," said Owen B. Wallace, Ph.D., Fulcrum's chief scientific officer. "Through internal research and discussions with key opinion leaders, we have identified areas within the sickle cell disease landscape where FTX-6058 has the potential to address significant unmet need. Enrollment has begun in our Phase 1 trial in healthy volunteers and we look forward to progressing FTX-6058 in clinical development."

FTX-6058 Results at ASHPreclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. FTX-6058 inhibits PRC2 via binding to EED, which induces a robust HbF protein expression in cell and murine models. Increasing HbF has the potential to prevent or reduce disease-related pathophysiology, resulting in reduction of recurring events such as vaso-occlusive crises and hemolysis. Human genetic data indicates that individuals with the sickle cell mutation but who have high HbF levels may have asymptomatic disease, underscoring the protective effect of increased HbF.

Key highlights include:

  • Demonstrated potent target engagement and HbF induction in vivo in animal models at plasma concentrations reasonably expected to be achieved in the clinic.
  • Pharmacological activity in target cells can be readily monitored in the clinic since target engagement in bone marrow correlates with target engagement in peripheral monocytes in animals.
  • Demonstrated an impressive preclinical pharmacological profile with the potential to be a disease-modifying therapeutic for sickle cell patients.

The posters will be available in the "Publications" section of following the sessions.

About FTX-6058FTX-6058 is a highly potent small molecule inhibitor of EED capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary Product Engine. The company's composition of matter patent covering FTX-6058 and related structures has been granted. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

About Sickle Cell DiseaseSickle 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 leading to anemia. SCD patients 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.

About Fulcrum Therapeutics Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia, into Phase 1 clinical development.

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