Fulcrum Completes IND-enabling Safety Studies for Sickle Cell Disease Treatment

Fulcrum Therapeutics announced it is on track for submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for FTX-6058 following the completion of preclinical safety studies and Good Laboratory Practices (GLP) toxicology work.

"Completed preclinical studies illustrate that FTX-6058, a small molecule upregulator of fetal hemoglobin, has the potential to provide distinct advantages over biologics and gene therapies currently being used or developed for the treatment of sickle cell disease," said Owen B. Wallace, Ph.D., Fulcrum’s chief scientific officer. "Based on its attractive profile in preclinical in vitro and in vivo models and its preclinical safety profile, we believe that FTX-6058 has the potential to be an effective oral therapy for people living with sickle cell disease."

In pre-clinical studies, treatment with FTX-6058 was shown to significantly increase HbF levels up to approximately 30% of total hemoglobin as measured by HPLC and mass spectrometry methods in erythroid progenitor cells from multiple human donors. FTX-6058 also elevated HbF in vivo in efficacy models at plasma concentrations reasonably expected to be achieved in humans. Fulcrum believes these results indicate that FTX-6058 could play a role in alleviating the burden of disease in people living with this devastating disorder.

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. 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.
 

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