Unlocking gene control mechanisms to develop small molecule therapies

Fulcrum Therapeutics is dedicated to addressing genetically defined diseases through the modulation of gene expression. The company focuses on developing small molecules aimed at treating the underlying causes of these diseases, particularly in areas where there is a significant unmet medical need. Their lead program, losmapimod, is currently undergoing clinical trials for the treatment of facioscapulohumeral muscular dystrophy (FSHD), showcasing their commitment to advancing therapeutic options for patients with rare genetic disorders.

In addition to losmapimod, Fulcrum Therapeutics is actively exploring a diverse pipeline of programs and discovery efforts aimed at improving patient outcomes. The company's approach emphasizes the importance of understanding the genetic basis of diseases to create targeted therapies that can effectively address the root causes of these conditions. By focusing on small molecule development, Fulcrum Therapeutics aims to provide innovative solutions that enhance the quality of life for individuals affected by genetically defined diseases.

United States

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.

Cardiovascular Diseases

Genetic Diseases

Hematologic Diseases

Musculoskeletal Diseases

Rare diseases

Fulcrum Therapeutics

Sickle cell disease

FTX-6058 is a highly potent small molecule inhibitor of EED capable of &nbsp;inducing robust HbF protein expression in cell and murine models. &nbsp;Fulcrum believes the pharmacokinetics and human dose simulations support &nbsp;that FTX-6058 may be given as a once daily oral compound. The &nbsp;validation of EED as a target for sickle cell disease and the discovery &nbsp;of FTX-6058 as a novel HbF-inducing small molecule were conducted using &nbsp;Fulcrum’s proprietary Product Engine. The company’s composition of &nbsp;matter patent covering FTX-6058 and related structures has been granted.
<a href="https://ir.fulcrumtx.com/news-releases/news-release-details/fulcrum-therapeutics-presents-updated-data-sickle-cell-disease">Source</a>

Pociredir (FTX-6058)

COVID-19

Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds.

Losmapimod 

Facioscapulohumeral muscular dystrophy

Muscle degeneration

Muscle weakness

Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds.

Cambridge, Massachusetts, United States of America

Fulcrum Therapeutics

Company Details