In the clinical program, statistically significant differences in muscle strength as compared to a matched external control cohort and a favorable safety profile were observed.
"We are excited by these interim Phase 1b/2a clinical data for HT-100, a powerful anti-inflammatory and anti-fibrotic, which further demonstrate its potential in the treatment of DMD," said Marc B. Blaustein, CEO of Akashi Therapeutics.
"As a group, the boys in this study showed an increase in muscle strength over their baseline and a statistically significant increase relative to a comparable external control cohort, and we look forward to further evaluating and reporting on the progress of HT-100 as a promising treatment option for all boys with DMD as the study continues."
Highlights of the interim data as of June 12 include:
- The 10 DMD patients participating in the trial for 18 to 22 months and with at least six months of continuous dosing achieved mean total muscle strength 22.3% greater than levels predicted by comparable steroid-treated external control (p=0.027) as measured by quantitative muscle testing (QMT) of upper and lower extremity muscle groups.
- The mean increase in total muscle strength compared to baseline (study entry) over 18-22 months was 11.7%.
- These efficacy findings are in the trial’s 2 lowest dose cohorts (mean age[SD]=10.4[2.55]). All study participants are on a stable dose of corticosteroids.
- HT-100 continues to be well-tolerated with no serious adverse events. The safety database in this study reflects a cumulative 10.5 years of dosing, with 6 patients dosed for a total of 12-13 months, and 10 patients dosed continuously for 9-10 months.
HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and fast track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals across the U.S.