Akashi Therapeutics, Inc., a clinical stage biopharmaceutical company developing treatments for patients with Duchenne muscular dystrophy (DMD), reported that the U.S. Food and Drug Administration (FDA) has completed its review and concluded that Akashi may resume clinical development of HT-100 (delayed-release halofuginone) in patients with any of the genetic mutations that cause DMD, a rare disease that results in muscle degeneration and premature death in boys. Akashi plans to initiate a new study, HALO-DMD-04.
“We are pleased to learn that the FDA is allowing Akashi to resume the clinical development of HT-100,” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD). “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.”
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 potential to treat all DMD patients independent of their specific genetic mutation.
“Our goal continues to be improving the lives of patients with DMD and other muscle function diseases,” said Marc Blaustein, Chief Executive Officer, Akashi Therapeutics. “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.”
HALO-DMD-04 will evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic activity of HT-100 at a new lower dose of 150 µg/day and eliminates the use of antiemetic therapies by participants. Data from previous clinical studies of HT-100 in DMD have indicated the lowest doses tested (as low as 300 µg/day) were at least as effective as higher doses (up to 1500 µg/day) investigated in a subject population that included both ambulant and non-ambulant boys.
HT-100, a delayed-release, orally-delivered therapeutic, is being researched for its ability to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. In a previously-released analysis of results from trials in patients with DMD, data showed promising improvements in muscle strength for boys treated in the lowest three dose cohorts. These strength improvements, rather than just a slowed rate of decline, are a highly unusual finding in ambulant DMD boys age 7 and older and in non-ambulant DMD boys.
Duchenne muscular dystrophy (DMD) is an X-linked recessive, inheritable disease that affects approximately 1 in 3,600 boys. DMD results in muscle degeneration and premature death. Symptoms usually become visible in early childhood: progressive proximal muscle weakness of the legs and pelvis associated with loss of muscle mass is observed first, and this weakness spreads to other parts of the body.
As the disease progresses, muscle tissue is replaced by fat and fibrotic tissue (fibrosis). Untreated, most patients are wheelchair dependent by their teenage years. Due to progressive deterioration of muscle, patients lose ambulation, then arm function, and ultimately experience respiratory and/or cardiac failure. While life expectancy varies, patients typically survive until the third decade.
(Source: Business Wire)