Phrixus Pharmaceuticals receives services from National Institutes of Health, NHLBI SMARTT Program to produce Carmeseal-MD™ for clinical trial to treat Duchenne muscular dystrophy
Carmeseal-MD™ (P-188 NF) to be tested in US multicenter study starting in early 2016, already available under European Access Program
ANN ARBOR, MI – 17 June 2015
Phrixus Pharmaceuticals, Inc., today announced that the NIH National Heart, Lung, and Blood Institute (NHLBI) SMARTT Program will provide services to produce clinical-‐grade Carmeseal-‐MD™ (P-‐188 NF) for the company’s first study involving non-‐ambulatory boys with Duchenne muscular dystrophy (DMD). SMARTT stands for “Science Moving towards Research Translation and Therapy.” The program is designed to facilitate the development of promising new therapies such as Carmeseal-‐MD™, which has the potential to protect the heart from the damaging effects of DMD.
“Duchenne muscular dystrophy is a debilitating and fatal genetic disease,” said SMARTT Project Officer Dr. Narasimhan Danthi. “Significant heart disease is present in a majority of patients by age 18, and once the heart damage occurs, it is irreversible. Interventions that slow or stop this process are needed.”
Thomas A. Collet, President and Chief Executive Officer of Phrixus, emphasized that time is of the essence for the patients. “The sooner they receive treatment for this disease the better will be their prospects for survival,” he said.
Collet explained that the manufacture of clinical-‐grade Carmeseal-‐MD is the last step before the Food and Drug Administration will accept Phrixus’s investigational new drug application (or IND) in anticipation of beginning the first trial of the drug in the US in early 2016. Preclinical studies of Carmeseal-‐MD in the U.S. were funded by NHLBI’s Small Business Innovation Research grants program. Currently, Carmeseal-‐ MD™ is available in Europe as part of Phrixus’s European Access Program.