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Clinical Development of HT-100 for Duchenne Has Resumed

Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 

Preliminary Clinical Data to be presented at Upcoming Medical Conference 

Read the news release from DART Therapeutics:

DART Therapeutics Continues Clinical Development of HT-100 for Duchenne Muscular Dystrophy 

Cambridge, Mass.—June 12, 2014—DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-release halofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose DMD patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints. Preliminary clinical data on the first patient cohorts in this study will be presented at the 2014 New Directions in Biology and Disease of Skeletal Muscle Conference being held June 29 to July 2, 2014 in Chicago. 

“We are pleased to resume clinical development of HT-100, following positive interactions with the FDA,” said Marc B. Blaustein, CEO of DART Therapeutics. “HT-100 has the potential to not only address major pathologies associated with DMD, including fibrosis and inflammation, but also to promote healthy muscle regeneration. We look forward to presenting preliminary data on the on-going Phase 1b/2a clinical study in the coming weeks.” 

The phase 1b/2a multi-center clinical program is enrolling 30 boys and young men aged six through 20 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, which has been granted orphan designation for DMD in both the U.S. and E.U., is being developed by DART subsidiary Halo Therapeutics, LLC. 

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Comment by David on June 17, 2014 at 11:02am
The history of this organization is very unique. This is not your typical drug company.

http://dartrx.com/about-us/
Comment by David on June 17, 2014 at 10:59am
http://en.m.wikipedia.org/wiki/Orphan_drug

It means in theory that the treatment has an easier path through regulators once trials are successful.
Comment by Dawn Steele on June 17, 2014 at 8:48am
What does orphan designation mean?
Comment by Mindy on June 16, 2014 at 8:34pm
Yay, that's so awesome, I can't wait until my son can get it.
Comment by David on June 12, 2014 at 6:38pm
Woooooooooo!!! Go DART go

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