Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc.,announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s most advanced product candidate, 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.Fast track designation is granted by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
“We are pleased that the FDA supports our application for fast track designation for HT-100 for the treatment of DMD. This, along with the previously granted orphan drug designation for HT-100, is an important regulatory milestone for the program,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “We will continue to work closely with the FDA as we advance HT-100 through clinical development and the associated regulatory processes.”
Akashi is currently treating patients with DMD in a phase 1b/2a multi-center clinical program to evaluate HT-100 safety and tolerability and assess trends in a range of exploratory biomarkers and efficacy endpoints.
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-100has been granted orphan designation for DMD in both the U.S. and EU, 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 treatmentsfor 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.
Akashi Therapeutics Contact:
Marc B. Blaustein, CFA
Gina Nugent, The Yates Network