DART completes successful trials on Duchenne biomarker

DART Therapeutics CEO Gene Williams says the company is competing against DMD, a fatal disease for which there is no treatment.

Wednesday, October 10, 2012

DART Therapeutics LLC, a biotech focused on finding therapies for Duchenne Muscular Dystrophy (DMD), a progressive and fatal neuromuscular disease for which there is no current treatment, has completed tests on a new biomarker intended to speed drug development by cutting the number of patients needed for new drug trials by half.

Cambridge-based DART conducted the trials from February through August of this year on 61 boys with DMD and 31 without. Newly appointed CEO Gene Williams told Mass High Tech that the biomarker - a device which measures a muscles response to a tiny electrical impulse - provides a more exact way to measure a potential drugs affects than the current standard for muscular diseases, which is to measure how far a patient can walk in six minutes. The so-called six-minute walk measurement can vary widely among boys, who might walk faster or slower depending on mood, said Williams. That means trials on potential DMD drugs must be large enough to factor out such variables, and as a result, many companies never test promising compounds, he said.

I think in the industry, we have a lot of drugs that work, but trials that fail to show it, Williams said.

The device was developed by Seward Rutkove, chief of the division of neuromuscular disease at Beth Israel Deaconess Medical Center and professor of neurology at Harvard Medical School. Convergence Medical Devices, the Woburn-based company Rutkove co-founded, plans to submit a 510(k) application to the U.S. Food and Drug Administration in 2013, according to the statement from DART.

DART, which has 20 employees and is run as a virtual biotech, was founded in 2010 by patient foundations, through which it continues to be funded. While its a for-profit business, Williams - a former executive at Genzyme - said, We consider ourselves to be competing against the disease.

DMD affects one in 3,500 boys - about 15,000 in the U.S. -, according the company. Williams said that he believes any successful treatment for the disease will likely involve several drugs used in combination, so developing a biomarker will enable many drugs to be tested more quickly and with less expense.

DMD is like most diseases in that there not going to be one single silver bullet, he said. Just one trial isnt enough.

Excerpt from:
DART completes successful trials on Duchenne biomarker