Fulcrum Therapeutics Inc (NASDAQ:FULC) announced earlier this month the launch of its Phase 1 trial to evaluate its pipeline sickle cell disease treatment called FTX-6058.
FTX-6058 is a small molecule drug whose mechanism is designed to facilitate the release of fetal hemoglobin, potentially treating sickle cell disease and other hemoglobinopathies. The Phase 1 study aims to identify FTX-6058’s safety and efficacy, and it will be divided into four parts. The first part will be labeled Part A, and it will feature a placebo-controlled, double-blind, randomized single ascending dose (SAD).
Part B of the study will feature a placebo-controlled, randomized, double-blind multiple ascending dose (MAD) that will have 4 cohorts with patients dosed daily for 14 days. Part C will feature an open-label pilot food effect study in which randomized patients will receive FTX-6058 accompanied by a high-fat meal and some without high-fat meals. Fulcrum will conduct part D as an open-label study that will determine FTX-6058’s ability to induce CYP3A.
“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,” stated Fulcrum Therapeutics’ CEO, Robert J. Gould.
Why Fulcrum excited for FTX-6058 and its phase 1 trial
Dr. Gould also noted that sickle cell patients still have major unmet needs as far as therapeutic options are concerned. He believes that the availability of a safe and effective small molecule treatment option for those patients might offer them significant improvements. The CEO also revealed that the company is excited for 30 percent fetal hemoglobin elevation observed in the preclinical data.
The reason for the excitement about FTX-6058 is because of what it will mean if the clinical trial data is consistent with previous findings; it could deliver significant benefits. If the sickle cell patients in the phase 1 trial experience as much as 30 percent fetal hemoglobin elevation, it will be an indicator that FTX-6058 can be used to treat other symptoms such as anemia and painful crises. It will also be great news for patients suffering from sickle cell anemia, especially those whose condition is currently not catered to by existing treatments.