DMD: FDA Gives Fast Track Designation to HT-100

An experimental drug designed to reduce scarring in Duchenne MD-affected muscles shows signs of safety and desired activity; FDA will speed its review

Preliminary results from 17 DMD-affected trial participants receiving HT-100 are encouraging, and the FDA has given fast track designation to this investigational drug.
Article Highlights:
  • HT-100 is an experimental drug for Duchenne muscular dystrophy whose development by Akashi Therapeutics is being supported by MDA through a $500,000 grant.
  • Akashi recently announced encouraging preliminary results for HT-100 from 17 trial participants with Duchenne muscular dystrophy.
  • A trial of HT-100 is active but not currently recruiting additional patients.
by Margaret Wahl on July 3, 2014 - 12:52pm

HT-100, an experimental drug being developed for Duchenne muscular dystrophy (DMD) with support from MDA, has shown preliminary safety and signs of the desired effects on scar tissue formation in the first 17 trial participants, and it has received "fast track" designation from the U.S. Food and Drug Administration (FDA).

Cambridge, Mass.-based Akashi Therapeutics (formerly Halo Therapeutics) announced these and related developments in three July 3, 2014, press releases.

"MDA is pleased to provide funding for the development and testing of HT-100," said neurologist Valerie Cwik, MDA's chief medical and scientific officer, "and we're especially happy to see that the FDA considers this compound worthy of its fast track designation. We look forward to working with Akashi Therapeutics to move this drug forward."

Fast track is a mechanism the FDA can use to speed up the agency's review of experimental drugs for serious or life-threatening disorders when there is early evidence that a new medication may provide benefit over available therapies.

MDA is supporting the development of HT-100 through a $500,000 grant to Akashi.

About HT-100

HT-100 is an experimental compound given by mouth that is intended to reduce inflammation and scar tissue formation and promote regeneration in muscles affected by DMD, a disease resulting from a deficiency of the dystrophin protein. Unlike some other experimental drugs in development to treat DMD, HT-100 does not target a specific mutation (flaw) in the gene for the dystrophin protein, making it potentially useful for all patients with DMD.

Positive preliminary results announced

Akashi announced encouraging interim results from an ongoing phase 1b/2a clinical trial of HT-100 in boys and young men with DMD at the 2014 New Directions in Biology and Disease of Skeletal Muscle Conference, held in Chicago June 29-July 2.

At the conference, the company said the following:

  • There were no serious or clinically significant adverse events in the first 17 DMD patients to receive HT-100 for up to 92 days at doses ranging from 0.3 to 1.2 milligrams per day.
  • The preliminary data on the 12 boys who received repeated doses of HT-100 indicate that the drug is having the desired effects on muscle tissue with respect to scar formation ("fibrosis").

Data from the presentation are summarized in a July 3, 2014, press release from Akashi.

HT-100 trial information

A trial of HT-100 in boys and young men with DMD is being conducted at five U.S. sites. An extension study is open for those who have already participated in the main trial of HT-100. However, Akashi says these studies are not currently enrolling new patients as of July 3.

For more details about the main study, see Safety, Tolerability and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy; or enter NCT01847573 in the search box at

For details about the extension study, see Open-Label Extension Study of HT-100 in Patients With DMD; or enter NCT01978366 in the search box at

Akashi can be contacted at or (617) 431-7250. Email is preferred.

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