Akashi Therapeutics, Inc. announced today that the Muscular Dystrophy Association (MDA) has made an investment through the MDA Venture Philanthropy program to help fund clinical development of HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The investment from MDA is part of collective funding totaling $1.5 million that Akashi has received in its most recent fundraising round from MDA and 15 other non-profit organizations to support clinical development of HT-100. The current funding is in addition to more than $1 million in non-profit support from an earlier fundraising round, for a cumulative total of more than $2.5 million raised by Akashi from 25 patient advocacy organizations.
This approach to attracting cooperative funding reflects Akashi’s distinct business model, based on the company’s founding by two leading patient organizations, Charley’s Fund and the Nash Avery Foundation. The organizations that support Akashi’s clinical development efforts are listed on the company’s website at www.akashirx.com/financial-supporters.
‘MDA is pleased to provide this funding to evaluate the clinical potential of HT-100 for the treatment of DMD,’ said MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D. ‘Significant need remains for safe and effective treatment options for DMD and we look forward to working together with Akashi Therapeutics to explore the potential of HT-100 to help individuals living with this progressive muscle disease.’
HT-100 is currently being tested in patients with DMD in a phase 1b/2a multi-center clinical program to evaluate the safety and tolerability and assess trends in a range of exploratory biomarkers and efficacy endpoints.
‘We are delighted that these disease foundations, including MDA, are joining forces with our mission at Akashi to realize the potential of HT-100 and seek new treatment options for muscular dystrophy,’ said Marc B. Blaustein, CEO of Akashi Therapeutics. ‘We are continuing to treat patients in the phase 1b/2a multi-center clinical program and anticipate reporting additional progress in the months to come.’
About HT-100
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.
About Duchenne muscular dystrophy (DMD)
Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.
About Akashi Therapeutics
Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.
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