Fulcrum Therapeutics, Inc. a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and β-thalassaemia, which has showed efficacy at raising the levels of fetal haemoglobin in cellular and animal models of the diseases, will now enter a a Phase 1 trial before the end of the year to assess the safety of this treatment in healthy volunteers.
« 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 », affirmed 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 haemoglobin. Should this elevation be seen in sickle cell patients, it has the potential to address multiple symptoms, including painful crises and anaemia. »
FTX-6058 is a small molecule that increases HbF levels by blocking the activity of a protein called embryonic ectoderm development (EED).
“An orally available small molecule therapeutic acting through a novel mechanism to induce increased pancellular [affecting all cell types] HbF should be an important disease-modifying agent for SCD”, said Martin H. Steinberg, MD, Professor of Medicine at Boston University School of Medicine.
New preclinical findings for this treatment were presented by the company in a presentation titled “FTX-6058, a novel HbF-inducing agent for the treatment of Sickle Cell Disease and β-Thalassemia,” at The 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting, held recently online.
“We are very encouraged by these in vitro and in vivo findings, as the preclinical data support our novel approach to treating haemoglobinopathies, such as sickle cell disease and β-thalassaemia”, added Owen Wallace, Chief Scientific Officer of Fulcrum.