Akashi Therapeutics is planning to initiate the HALO-DMD-04 clinical trial of HT-100 (delayed-release halofuginone) to treat patients with duchenne muscular dystrophy (DMD).
The move follows the clearance granted by the US Food and Drug Administration (FDA) to resume the clinical development of HT-100 in patients with any of the DMD causing genetic mutations.
HT-100 is a orally delivered, delayed-release therapeutic being developed to minimise fibrosis and inflammation, as well as improve regeneration of healthy muscle fibre in DMD patients.
The HALO-DMD-04 trial will assess the safety, tolerability, pharmacokinetic and pharmacodynamic activity of 150µg/day HT-100.
Akashi Therapeutics chief executive officer Marc Blaustein said: “Our goal continues to be improving the lives of patients with DMD and other muscle function diseases.
“We are pleased that the FDA has agreed with our conclusion that it is appropriate to resume development of HT-100 and look forward to moving ahead with the trial as quickly as possible.”
The new lower dose of the product being used in the trial is expected to eliminate the use of anti-emetic therapies by the subjects.
The results from previous clinical trials conducted in both ambulant and non-ambulant boys showed that the doses as low as 300µg/day were at least as effective as higher doses of up to 1,500µg/day.
Akashi is currently in discussions with potential investors and development partners to clinically develop and commercialise HT-100, along with its selective androgen receptor modulator DT-200 and cation channel modulator AT-300 for treating DMD patients.
HT-100 has already obtained orphan designation in the US and EU, as well as fast-track designation in the US for DMD.
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