University of Michigan spinoff Phrixus Pharmaceuticals has won an award from the National Institutes of Health (NIH) that will help the company advance to clinical trials for Carmeseal-MD, its developmental drug to treat cardiac and respiratory problems in patients with Duchenne muscular dystrophy.
Thomas Collet, Phrixus’ president and CEO, says the company will receive approximately $2.5 million in preclinical regulatory, pharmacology, and toxicology services as part of the NIH’s National Heart, Lung, and Blood Institute program called Science Moving Towards Research Translation and Therapy. RTI International in North Carolina will provide regulatory affairs support. Pre-clinical studies will be conducted over the next year by SRI International in Menlo Park, CA.
“We view this as a significant validation of our approach,” Collet notes. “The NIH, in general, has supported us quite well.”
Collet and Bruce Markham founded Phrixus in 2006 to determine the potential of Carmeseal-MD to treat heart failure. Describing it as “a molecular band-aid,” Carmeseal was initially developed in the 1990s to treat heart attacks. Repurposed as Carmeseal-MD, the drug is infused into the bloodstream, where it finds microscopic tears in the heart muscle and binds with them. Collet says this could prevent the deadly leakage of calcium into heart cells; calcium overload can keep the heart from delivering sufficient oxygen to vital organs.
“Heart attack and heart failure are two very separate diseases,” Collet explains. “Heart failure is acute, while heart attacks are chronic. We’re quite confident that, at lower doses, Carmeseal-MD will prevent a diseased heart from expanding.”
Collet says Duchenne muscular dystrophy affects about 20,000 boys in the United States, and is usually fatal by the time a patient reaches his 20s. If Phrixus is able to get funding in place, it hopes to begin clinical trials on Carmeseal-MD by early next year. Collet adds that the drug has already been tested successfully in mice and dogs thanks to an earlier NIH grant of $2 million.
