US-based biopharmaceutical firm Akashi Therapeutics has reported preliminary positive clinical results from a Phase Ib/IIa trial of its drug candidate, HT-100 (delayed-release halofuginone), for treatment of Duchenne muscular dystrophy (DMD).
HT-100 is an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.
The preliminary data for HT-100 on the first DMD patient groups show promising signs of biological activity and a favourable safety profile to date in this ongoing Phase Ib/IIa multi-centre clinical programme to assess the safety and tolerability of increasing doses of the drug candidate and evaluate trends in a range of exploratory biomarkers and efficacy endpoints.
In addition, the company has also secured fast-track status for HT-100 from the US Food and Drug Administration (FDA).
Kennedy Krieger Institute's Center for Genetic Muscle Disorders director Kathryn Wagner said: "Fibrosis is a critical pathology of DMD directly linked to disease prognosis, muscle strength and motor function.
"In addition, fibrosis creates a physical barrier preventing DMD therapies from reaching target muscle tissue.
"By decreasing excess collagen formation associated with fibrosis, HT-100 could provide both direct clinical benefit in terms of physical performance and help pave the way for other DMD treatments to reach their target sites."
In the preliminary data, HT-100 showed a favourable safety profile in dosing groups ranging from 0.3mg/day to 1.2mg/day for up to 92 total dosing days with no serious adverse events and no clinically significant adverse events.
HT-100 also showed significant biologic activity and positive trends in serum biomarkers of muscle tissue remodelling in DMD including biomarkers of collagen formation (fibrosis) and collagen degradation, as well as positive effects on a biomarker of muscle damage.
Akashi Therapeutics chief medical officer Diana Escolar said: "This data suggests that HT-100 is having the predicted biological activity on collagen formation and degradation after a short time of exposure even at the lowest doses tested to date, as measured by these novel biomarkers of extracellular matrix remodelling.
"In addition, the favourable safety profile exhibited to date gives us confidence as we continue to treat patients in this clinical programme. We look forward to reporting on our continued progress with HT-100 as this study progresses."
Image: Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: courtesy of Splintercellguy.