Halofuginone: Phase Ib/IIa halted

Akashi said that a patient treated with HT-100 in the open-label, U.S. Phase Ib/IIa HALO trial in boys and young men with ambulatory or non-ambulatory DMD has died. In late January, Akashi suspended dosing and enrollment in all cohorts of the trial after the patient began experiencing undisclosed “serious, life-threatening health issues” after receiving once-daily 60 ug/kg HT-100 -- the highest dose tested -- for about 2 weeks. Akashi said it has initiated an investigation, including detailed data reviews, in vitro studies and reviews of patient records to evaluate the extent to which the patient’s health issues are related to HT-100 and/or to other factors (see BioCentury, Feb. 1). ...