Date: 2015-06-12
Type of information: Interim results
phase: 1b - 2a
Announcement: results
Company: Akashi Therapeutics (USA - MA)
Product: HT-100 (delayed-release halofuginone)
Action
mechanism: 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.
Disease: Duchenne muscular dystrophy (DMD)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: USA
Trial
details: The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken. (NCT01847573)
Latest
news: * On June 12, 2015, Akashi Therapeutics, a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), announced positive interim clinical data from an ongoing Phase 1b/2a clinical program with HT-100 (delayed-release halofuginone) an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD. 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. 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.
