The Jett Foundation Fighting Duchenne Muscular Dystrophy

Industry News: Akashi Therapeutics Announces Plans to Have Three Compounds for Duchenne Muscular Dystrophy in the Clinic in 2017

January 9, 2017

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Two new compounds are poised for the clinic, and the company is in discussions with FDA regarding HT-100

CAMBRIDGE, Mass., January 5, 2017 – Akashi Therapeutics, Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today reported that the company is working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all DMD patients independent of their specific genetic mutation.
“DMD is a challenging condition that will require a multi-therapy cocktail to transform it into a chronic, manageable disease,” said Diana Escolar, MD, Chief Medical Officer, Akashi Therapeutics. “Our early-stage research demonstrates the potential of each of our three investigational compounds, either as monotherapies or in combination with other molecules, to make unique contributions to DMD treatment.”
Tackling the most critical issues in DMD, Akashi’s current pipeline contains three compounds that comprise a diversified portfolio: HT-100 (delayed-release halofuginone), DT-200 (selective androgen receptor modulator) and AT-300 (cation channel modulator). Collectively, these compounds aim to reduce fibrosis and inflammation, increase muscle strength, and reduce muscle necrosis by restoring calcium level balance. Next steps for each:

  • HT-100: The company is in discussion with the FDA regarding reintroducing HT-100 into the clinic in the coming months. HT-100 is unique in its ability to reduce fibrosis and inflammation, promote healthy muscle fiber regeneration, and upregulate utrophin. Early evidence from a clinical study of DMD patients suggests that treatment with HT-100 results in an improvement from baseline in muscle strength. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and received fast track designation in the U.S.
  • DT-200: In early 2017, the company plans to initiate a randomized double-blind, placebo-controlled proof-of-concept trial of DT-200, an oral SARM (selective androgen receptor modulator) being explored for its potential to improve muscle strength and function in DMD patients. Phase 1 data have demonstrated preliminary safety and tolerability. Efficacy data are expected before the end of 2017 if dosing begins as planned in the first half of the year.
  • AT-300: Following demonstration of unparalleled effects on muscle protection in the preclinical mdx model of DMD, Akashi is poised to initiate an IND-enabling program for AT-300 in early 2017. AT-300 addresses calcium level imbalance, an early trigger of critical pathologies that lead to loss of muscle function and muscle cell death in DMD patients. AT-300 is the only known specific inhibitor of the stretch-activated class of calcium ion channels. This patented new molecular entity has been granted orphan drug designation in the U.S. The initiation of the IND-enabling program early in 2017 is expected to allow AT-300 clinical trials to begin by the end of year.

“Our comprehensive, multi-pathway approach to tackling Duchenne is unique,” said Marc Blaustein, Chief Executive Officer, Akashi Therapeutics. “We look forward to making substantial progress across our entire development portfolio in 2017.”

While research of all three treatments has been focused on DMD, a rare disease that results in muscle degeneration and premature death in boys, the compounds may also have applications for conditions beyond DMD.

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 age 10. 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 late in the second or the third decade.

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 www.akashirx.com.

For more information please contact:
Tara DiMilia
On behalf of Akashi Therapeutics
908-369-7168
tara.dimilia@tmstrat.com

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