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TMCNet:  Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

[July 03, 2014]

Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

CAMBRIDGE, Mass. --(Business Wire)--

Akashi Therapeutics, Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy, today announced the presentation of preliminary positive clinical data for its most advanced drug candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne muscular dystrophy (DMD). Preliminary clinical data for HT-100 on the first DMD patient cohorts suggests promising signs of biological activity and a favorable safety profile to date in this ongoing phase 1b/2a multi-center clinical program to evaluate the safety and tolerability of increasing doses of HT-100 and assess trends in a range of exploratory biomarkers and efficacy endpoints. The preliminary Phase 1b/2a data was presented at the New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference held in Chicago June 29 through July 2, 2014.

"The preliminary biomarker data from this study is encouraging and could indicate that HT-100 is having the desired activity on fibrosis formation and degradation as well as muscle damage," said Kathryn Wagner, M.D., Ph.D., director, Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and an associate professor of neurology and neuroscience at the Johns Hopkins School of Medicine. "Fibrosis is a critical pathology of DMD directly linked to disease prognosis, muscle strength and motor function. In addition, fibrosis creates a physical barrier preventing DMD therapies from reaching target muscle tissue. By decreasing excess collagen formation associated with fibrosis, HT-100 could provide both direct clinical benefit in terms of physical performance and help pave the way for oter DMD treatments to reach their target sites."


"These data suggest that HT-100 is having the predicted biological activity on collagen formation and degradation after a short time of exposure even at the lowest doses tested to date, as measured by these novel biomarkers of extracellular matrix remodeling," said Diana M. Escolar, Chief Medical Officer of Akashi Therapeutics. "In addition, the favorable safety profile exhibited to date gives us confidence as we continue to treat patients in this clinical program. We look forward to reporting on our continued progress with HT-100 as this study progresses."

In an oral presentation entitled "Preliminary Safety, PK and Novel Biomarker Data from a Phase 1b/2a Clinical Program of HT-100 in Duchenne Muscular Dystrophy," Dr. Escolar presented clinical data which demonstrated preliminary safety and signs of biologic activity of HT-100 in the first two DMD patient cohorts with six patients per cohort (n=12).

  • In the preliminary data, HT-100 demonstrated a favorable safety profile in dosing cohorts ranging from 0.3 to 1.2 mg/day for up to 92 total dosing days with no serious adverse events and no clinically significant adverse events.
  • In the preliminary data, HT-100 demonstrated significant biologic activity and positive trends in serum biomarkers of muscle tissue remodeling in DMD including biomarkers of collagen formation (fibrosis) and collagen degradation, as well as positive effects on a biomarker of muscle damage.

The HT-100 phase 1b/2a multi-center clinical program is enrolling up to 30 boys and young men aged six through 20 years old with DMD, both ambulatory and non-ambulatory, enabling investigators to evaluate HT-100 in a broad patient population. The study is designed to evaluate the safety and tolerability of a series of increasing doses of HT-100. In addition, the study has also been designed to assess trends in a range of exploratory biomarkers and efficacy endpoints. The study's biomarkers include plasma-derived measures of fibrosis formation and degradation in muscle that have been validated in other disease states. The clinical outcome endpoints include motor function and muscle strength tests as well as measures of pulmonary and cardiac function.

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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