MDA Funds Development of Drug to Fight Inflammation, Scarring in DMD

An MDA Venture Philanthropy grant of $500,000 to Halo Therapeutics will support the development of HT-100, an experimental drug for Duchenne muscular dystrophy

Article Highlights:
  • HT-100 combats inflammation and scar tissue formation in muscle, both of which can impair muscle regeneration and be a barrier to therapy, and promotes regeneration of healthy muscle fibers.
  • HT-100 is currently being evaluated in a five-site clinical study of 30 boys and young men with Duchenne MD.
  • The MDA Venture Philanthropy (MVP) program, which is helping to fund development of HT-100, is designed to move promising compounds out of the laboratory and into clinical testing.
by Margaret Wahl on November 14, 2013 - 7:00am

Update (June 12, 2014):  In a June 12, 2014, announcement, Halo Therapeutics said that it has resumed testing of HT-100, an experimental oral drug intended to reduce scar formation and inflammation and promote healthy muscle regeneration in DMD. The company will now continue to enroll and dose DMD ptients in its ongoing clinical trial and extension study.

Update (Jan. 6, 2014): In a Dec. 23, 2013, announcement, Halo Therapeutics said that the U.S. Food and Drug Administration (FDA) had placed the HT-100 trial on "clinical hold," based on adverse events that occurred in a study being conducted with this compound in dogs. A clinical hold means no further dosing of patients will take place until new data are generated and evaluated and a new decision is made. See the Dec. 23 Q&A from Halo for details.

MDA has awarded a grant of $500,000 to Newton, Mass., biopharmaceutical company Halo Therapeutics for development of HT-100, an experimental drug for Duchenne muscular dystrophy (DMD).

HT-100, now in a 30-person clinical trial in boys and young men with DMD, is an oral compound designed to reduce damaging inflammation and fibrosis (scar tissue formation) in DMD-affected muscles, and to promote regeneration of healthy muscle fibers. The study is being conducted at five leading academic research sites across the U.S. Recruitment for this trial is complete.

The award, which will run through Nov. 15, 2014, was made through MDA Venture Philanthropy (MVP), the drug development arm of the organization’s program in translational research. The goal of translational research is the movement of promising therapeutic compounds from the laboratory to the clinic.

"Excessive inflammation and fibrosis in muscle can interfere with muscle regeneration and can be a significant barrier to certain therapies now in development, such as gene replacement," said Jane Larkindale, MDA's vice president of research. "Drugs like HT-100 have the potential to be used on their own or in conjunction with other treatments. A rigorous review process conducted by MVP has determined that Halo has the capability of taking HT-100 forward in an efficient and safe manner."

For more information

To learn more, read Creating a Hospitable Environment for Muscle Regeneration, part of a special Quest series called In Focus: Preserving and Building Muscle Fibers (April 2011).

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