After Patient Death a Year Ago, Cambridge's Akashi Finally Gets the OK to Resume DMD Study

Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Akashi Therapeutics, Inc. a clinical stage biopharmaceutical company developing treatments for patients with Duchenne muscular dystrophy (DMD), today reported that the U.S. Food and Drug Administration (FDA) has completed its review and concluded that Akashi may resume clinical development of HT-100 (delayed-release halofuginone) in patients with any of the genetic mutations that cause DMD, a rare disease that results in muscle degeneration and premature death in boys. Akashi plans to initiate a new study, HALO-DMD-04.

“We are pleased to learn that the FDA is allowing Akashi to resume the clinical development of HT-100”

“We are pleased to learn that the FDA is allowing Akashi to resume the clinical development of HT-100,” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD). “Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be required to end Duchenne. We are pleased to be partnering with Akashi to develop important therapies for Duchenne.”

The company plans to initiate the new clinical trial as quickly as possible, and is in discussions with potential investors and development partners regarding clinical development and commercialization of HT-100, as well as DT-200 (selective androgen receptor modulator) and AT-300 (cation channel modulator), all of which are novel, complementary compounds with potential to treat all DMD patients independent of their specific genetic mutation.

“Our goal continues to be improving the lives of patients with DMD and other muscle function diseases,” said Marc Blaustein, Chief Executive Officer, Akashi Therapeutics. “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.”

HALO-DMD-04 will evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic activity of HT-100 at a new lower dose of 150 µg/day and eliminates the use of antiemetic therapies by participants. Data from previous clinical studies of HT-100 in DMD have indicated the lowest doses tested (as low as 300 µg/day) were at least as effective as higher doses (up to 1500 µg/day) investigated in a subject population that included both ambulant and non-ambulant boys.

HT-100, a delayed-release, orally-delivered therapeutic, is being researched for its ability to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. In a previously-released analysis of results from trials in patients with DMD, data showed promising improvements in muscle strength for boys treated in the lowest three dose cohorts. These strength improvements, rather than just a slowed rate of decline, are a highly unusual finding in ambulant DMD boys age 7 and older and in non-ambulant DMD boys.

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is an X-linked recessive, inheritable disease that affects approximately 1 in 3,600 boys. DMD results in muscle degeneration and premature death. Symptoms usually become visible in early childhood: progressive proximal muscle weakness of the legs and pelvis associated with loss of muscle mass is observed first, and this weakness spreads to other parts of the body. As the disease progresses, muscle tissue is replaced by fat and fibrotic tissue (fibrosis). Untreated, most patients are wheelchair dependent by their teenage years. Due to progressive deterioration of muscle, patients lose ambulation, then arm function, and ultimately experience respiratory and/or cardiac failure. While life expectancy varies, patients typically survive until the third decade.

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. For more information, please contact Akashi Therapeutics (

HT-100 development is generously supported by patient advocacy organizations. A list of these organizations can be found at

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy. Akashi was founded by leading patient organizations Charley’s Fund and Nash Avery Foundation in collaboration with biotechnology industry veterans 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


On behalf of Akashi Therapeutics
Tara DiMilia, 908-369-7168

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