Parent Project Muscular Dystrophy (PPMD) has awarded Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and efficacy of an investigational new drug known as HT-100 (delayed-release halofuginone).
HT-100 is an orally available small molecule drug candidate being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne patients. The application of HT-100 to Duchenne and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. Akashi, led by CEO Marc Blaustein, has been granted orphan designation for Duchenne in both the U.S. and EU, as well as Fast Track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals across the U.S.
The grant will be used to evaluate the safety, tolerability and pharmacokinetics of HT-100 in patients with Duchenne in both a phase 1b open-label, single and multiple ascending dose study and a phase 2a extension study.
Funding for the project from PPMD has been awarded based on a positive review by PPMD’s Scientific Advisory Committee, composed of key clinicians and scientists in the field and upon the basis of a review by the TREAT-NMD Advisory Committee on Therapeutics (TACT), whose review activities are supported, in part, by PPMD.
Preclinical studies in young mice that lack dystrophin demonstrated that HT-100 reduced the amount of fibrosis that accumulates in the heart and diaphragm muscles and improved the ability of the mice to cling to a rotating rod. In older mice with established fibrosis in the diaphragm muscle the compound was able to reverse fibrosis and improve respiratory and cardiac function.
“Marc Blaustein and the team at Akashi are innovators and leaders in the Duchenne space. We are thrilled to be able to support this next critical step in the journey of HT-100, a therapy that we believe in. PPMD is excited to partner with Akashi and appreciate that, like us, they believe it is as important for a drug to be safe, as it is to be effective.”
-- PPMD’s Founding President and CEO, Pat Furlong
“PPMD was an early supporter of our efforts to develop HT-100 and this funding—following a thorough scientific review—comes at a crucial time as we continue to generate both safety and efficacy data from the HT-100 clinical program in boys with DMD. We are grateful for PPMD’s continued financial support and pleased to build on our already-strong relationship with PPMD and the broader DMD patient community.”
-- Akashi CEO Marc Blaustein
To learn more about other projects PPMD is currently funding, visit our website.