americanpharmaceuticalreviewOctober 12, 2020
Tag: Fulcrum , FTX-6058 , sickle cell disease
Fulcrum Therapeutics announced the Investigational New Drug application (IND) is now in effect for its Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease.
This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts. Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to six cohorts. Part B will be a randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study in up to four cohorts dosed once daily for 14 days. Part C will be an open label pilot food effect study in subjects randomized to take FTX-6058 with and without a high-fat meal, and Part D will be an open label study to evaluate the potential of FTX-6058 to induce CYP3A (using midazolam).
"We are pleased to leverage our expertise in the modulation of genetic drivers of disease to expand our clinical development efforts into a third area with sickle cell," said Robert J. Gould, Ph.D., president and chief executive officer of Fulcrum Therapeutics. “We believe FTX-6058 offers a differentiated approach to a potential treatment. A major unmet need remains for many sickle cell patients, and the availability of an effective and safe small molecule treatment option could represent a significant advancement. We are excited about the preclinical data that showed elevations of fetal hemoglobin up to 30% of total hemoglobin. Should this elevation be seen in sickle cell patients, it has the potential to address multiple symptoms, including painful crises and anemia."
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 leading to anemia. 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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