Investigational New Drug Application for Sickle Cell Disease Treatment

Fulcrum Therapeutics has recently announced that it is on track to submit an Investigational New Drug application for their medication, FTX-6058. The application will likely be filed in the second half of 2020, following positive results from preclinical studies.

About Sickle Cell Disease

Sickle cell disease includes a group of disorders that are characterized by malformed red blood cells that look like sickles. The most common and severe form of this disease is sickle cell anemia. In sickle cell disease, deformed cells cause blockages and restricting blood flow as they get caught along the walls of blood vessels.

This disease is caused by a mutation in the gene responsible for producing hemoglobin, which carries oxygen throughout the body. This gene is inherited in an autosomal recessive pattern, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African and Hispanic descent.

Symptoms of sickle cell disease include pain crisis, swelling of the hands and feet, and symptoms associated with anemia like fatigue, jaundice, and delayed growth. Adults tend to constantly feel the effects of this disease, but children usually only experience them during pain crisis. Regardless of age, damage usually occurs to the organs that are affected by the blocked blood flow. The most commonly damaged organs are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones.

There is no cure for sickle cell disease. While some people qualify for bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.

FTX-6058 and Preclinical Data

FTX-6058 is a small molecule upregulator of fetal hemoglobin and is administered orally. While research is still being conducted, it has the potential to be preferable over biologics and gene therapy.

Preclinical data showed that this treatment increased HbF levels by about 30%. In vitro and in vivo models were used, and they both gave positive results that have led researchers to believe that FTX-6058 is a viable treatment for sickle cell disease.

Find the source article here.

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