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Duchenne treatment receives FDA clearance for clinical development

March 24, 2017

Akashi Therapeutics now have the greenlight from the FDA to continue clinical development of HT-100. The biopharmceutical company is developing treatments for patients suffering from Duchenne muscular dystrophy (DMD). HT-100 (delayed-release halofuginone) development will continue under the new study name HALO-DMD-04.

Following the clearance from the FDA, Marc Blaustein, Chief Executive Officer at Akashi Therapeutics said, “Our goal continues to be improving the lives of patients with DMD and other muscle function diseases…We are pleased that the FDA has agreed with our conclusion that it is appropriate to resume development of HT-100 and look forward to moving ahead with the trial as quickly as possible.”

HT-100 is not the only treatment to head into clinical development. DT-200 (selective androgen receptor modulator) as well as AT-300 (cation channel modulator) are also in the pipeline as treatment options for all DMD patients.

The company plans to initiate the new clinical trial as quickly as possible, and is in discussions with potential investors and development partners regarding clinical development and commercialization of HT-100, as well as DT-200 (selective androgen receptor modulator) and AT-300 (cation channel modulator), all of which are “novel, complementary compounds with the potential to treat all DMD patients independent of their specific genetic mutation.”

Pat Furlong, the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD) commented on the announcement saying, “Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be required to end Duchenne. We are pleased to be partnering with Akashi to develop important therapies for Duchenne.”

About HALO-DMD-04

HT-100 will undergo evaluation for safety, tolerability, pharmacokinetic, and pharmacodynamic activity. The study will test lower doses of HT-100 (150 µg/day) after earlier studies at higher doses were proven to be “as effective as higher doses.” Additionally, the study will eliminate the use of antiemetic therapies.

HT-100 has received orphan designation for DMD in the US and EU. The drug is designed to reduce the thickening and scarring of connective tissue and reduce inflammation and promote healthy muscle fiber regeneration. While there is no cure for DMD, the HALO-DMD-04 hopes to transform DMD from a fatal condition to a treatable one.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy an X-linked disease in which there is an abnormal DMD gene on the X-chromosome. The DMD gene regulates the production of dystrophin protein. On the spectrum of dystrophinopathies Duchenne is the most severe.

The onset of DMD is commonly seen in males aged 3-5 years. Symptoms include weakness, swollen calves, delays in reaching developmental milestones and reduced bone density. It is a progressive disease that works its way up the body, leaving patients wheelchair bound by their teens. Not much is know about the role of dystrophin, but it is thought to maintain the membrane (sarcolemma) of muscle cells.