Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The initiation of this trial is possible due to FDA approval of Fulcrum’s Investigational New Drug application.

About Sickle Cell Disease (SCD)

Sickle cell disease (SCD) 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 restricted 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.

About the Trial

The Phase 1 trial aims to test the pharmacokinetics, safety, and tolerability of FTX-6058, a small molecule that is intended to increase the expression of fetal hemoglobin. It will be conducted in four parts.

• The first section of this trial, Part A, is randomized, placebo-controlled, single ascending dose, and double-blind.
• Part B will be placebo-controlled, double-blind, randomized, and multiple ascending doses. Participants will be dosed once a day for two weeks.
• Part C will be open label and evaluate FTX-6058 when taken with and without high-fat meals.
• Part D, the final section, will study the medications ability to induce CYP3A.

Researchers working on the trial believe that this treatment offers a different approach to sickle cell disease treatments, hopefully one that will be able to better the lives of patients.

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