Fulcrum therapeutics, inc. announces multiple presentations of losmapimod data at world muscle society meeting

Fulcrum therapeutics, inc. announced that the company will present data from multiple studies of losmapimod in facioscapulohumeral dystrophy (fshd) patients during the international annual congress of the world muscle society being held from october 1-5 in copenhagen, denmark. michelle mellion, md, medical director at fulcrum therapeutics, will present data from the phase 1 trial to assess the pharmacokinetics, safety and tolerability, and target engagement of losmapimod in fshd patients during an oral presentation on october 4, 2019 from 10:30-12:30 cet. the company will highlight findings from the phase 1 trial as well as information related to plans for using biomarkers in the ongoing phase 2 clinical trials of losmapimod. update on data related to hemoglobinopathy program: fulcrum also announced recent progress in the company’s plan to advance the development program for ftx-6058, formerly referred to as ftx-hbf, for the potential treatment of sickle cell disease and beta-thalassemia. ftx-6058 is a novel upregulator of fetal hemoglobin. in pre-clinical research, treatment with ftx-6058 was shown to increase hbf levels to approx. 30% of total hemoglobin as measured by hplc and mass spectrometry methods in human erythroid progenitor cells from multiple donors. ftx-6058 also elevated hbf in vivo in animal models at plasma concentrations reasonably expected to be achieved in humans. fulcrum believes these results indicate that ftx-6058 could play a role in reducing the risk of crises in people living with sickle cell disease and may also help to address transfusion requirements in patients with beta-thalassemia as an oral therapeutic. fulcrum has initiated ind-enabling studies for ftx-6058 and anticipates filing an ind in mid-2020.
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