MHT/BBJ – Jul 3, 2014, 10:54am EDT
Cambridge biotech changes its name, reports favorable treatment from FDA
By Patricia Resende, Special to the Journal
It’s been a busy few months for Akashi Therapeutics, a patient/parent-founded startup focused on Duchenne muscular dystrophy, a fatal neuromuscular disorder affecting one in 3,500 boys worldwide with no current cure.
The Cambridge company, formerly known as DART Therapeutics, has changed its name, secured funding and its DMD candidate HT-100 received U.S. Food & Drug Administration Fast Track status, the company announced today.
The company’s new name is derived from the Akashi-Kaikyo bridge in Japan, which has the longest central span of any suspension bridge in the world, according to the company. The new name symbolizes how the company was formed to save children’s lives by bridging the gap between research and treatments for DMD.
Four years ago, a motivated group of parents who formed Charley’s Fund and the Nash Avery Foundation created the company to make DMD research and drug development faster and less expensive. They connected with experts in the biotech industry who reviewed a molecule that they believe have great promise in DMD. They acquired the molecule in March 2011 and formed the company to develop it.
“Our focus was to get into clinic and to patients as quickly as possible,” said CEO Marc Blaustein. “That was our driving motivation.”
Before getting it to patients, the company had to address the fact that patients were getting sick to their stomach. “It was always a feature of this drug, which prevented it from being a viable therapeutic,” Blaustein said.
That resulted in the development of HT-100, an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. Akashi got it into the clinic and early data shows that HT-100 has addressed the gastrointestinal tolerability issue.
“There is no cure for the disease and there is not one on the near-term horizon,” Blaustein said. “What we are hoping to do is improve the quality of life and extend life.”
The hope is that, like AIDS — which was once a death sentence for many but has since become a manageable disease — patients with DMD will be able to manage the disease with a cocktail of drugs.
“Our focus is on developing therapies in important parts of the cocktail that are underinvested in by the industry,” Blaustein said.
Now that 16 disease foundations have come together to provide $1.5 million in funding to support the clinical development of HT-100, the plan for Akashi is to complete the current clinical program. The hope is to have enough data to move into a pivotal trial for safety and efficacy to submit to the FDA.
But before Akashi can design the registration trial, it will need to see what the data from the existing trial shows.
“Until we know that information, it’s difficult to project,” Blaustein said.