Positive Interim Findings in Early Sickle Cell Disease Trial

According to a press release from Street Insider, the biopharmaceutical company Fulcrum Therapeutics, Inc., has recently announced positive interim findings from its ongoing phase I clinical trial, which consists of single and multi-ascending dose cohorts. This trial is evaluating FTX-6058, an experimental EED inhibitor designed to treat sickle cell disease and beta thalassemia. This trial consisted of healthy volunteers. Fulcrum focuses on developing treatments for rare, genetic diseases.

About Sickle Cell Disease

Sickle cell disease is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. The disease most frequently affects people of African ancestry. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.

Interim Findings

This phase I trial is evaluating the tolerability, pharmacokinetics, and safety of FTX-6058, with patients in the single-dose portion receiving a dose of either 2, 4, 10, 20, 30, or 40 mg. In the multi-dose portion, patients were given a dose of 2, 6, or 10 mg every day for 14 days in a row. Findings from the multi-dose cohort identified therapeutic target engagement, which is defined as change in H3K27me3/Total Histone H3 ratio. Levels dropped by 70 to 80 percent. There have been no discontinuations or serious adverse effects reported.

The company anticipates beginning enrollment for another clinical trial that will include sickle cell disease patients later in the year. This is anticipated to be a multi-dose, open-label trial which will start a 6 mg daily dose and include a three-month period of treatment.

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