Carmeseal-MD™, the first disease-modifying agent with the potential to protect all three dystrophic target muscles: skeletal limb muscle as well as diaphragm and heart
ANN ARBOR, Mich., Sept. 07, 2016 (GLOBE NEWSWIRE) — Phrixus Pharmaceuticals is announcing that FDA allowed Phrixus’s IND for Carmeseal-MD (P-188 NF) in Duchenne muscular dystrophy (DMD). The protocol that forms the basis of this IND includes a two-arm, randomized, double-blinded design with 120 patients in several centers in which one dose of P-188 NF will be evaluated against standard of care over 48 weeks. The primary endpoint will be forced vital capacity (FVC); a broad set of secondary endpoints will include cardiac endpoints and additional respiratory endpoints and measures of upper body strength in addition to safety measures. “This novel therapeutic strategy offers a unique opportunity to potentially favorably impact outcomes in boys and young men with Duchenne muscular dystrophy,” stated Dr. John L. Jefferies, Director, Advanced Heart Failure and Cardiomyopathy, Professor, Pediatric Cardiology and Adult Cardiovascular Diseases, The Heart Institute, Professor, Division of Human Genetics, Cincinnati Children’s Hospital Medical Center, who will be the Principal Investigator.
The IND-enabling studies were supported by the NIH’s National Heart, Lung and Blood Institute and by Coalition Duchenne.
Carmeseal-MD is already available in Europe as part of Phrixus’s European Access Program. Please contact Ethicor Pharma Ltd. at firstname.lastname@example.org or visit www.ethicorpharma.com.