Carmeseal-MD (Poloxamer-188 NF, or P-188 NF) is the first disease-modifying agent in diseases characterized by membrane instability such as Duchenne MD or limb girdle muscular dystrophy, among others.
Phrixus has a leading global patent portfolio for the use of poxamers in DMD, heart failure, and respiratory dysfunction. The pharmaceutical company is developing Carmeseal-MD for subcutaneous injection in DMD and Carmeseal-HF for intravenous administration, for acute decompensated heart failure.
DMD is estimated to affect 20,000 boys and young men in the U.S. and a similar number in Europe. Hallmarks of DMD include skeletal muscle weakness, respiratory distress, and heart failure.
Carmeseal MD has the potential to protect all three dystrophic target muscles: skeletal limb muscle, diaphragm, and heart.
In animal models of DMD and heart failure, Carmeseal-MD has been proven to improve the efficiency of damaged hearts. In the same models, the drug candidate has also been shown to improve the performance of damaged diaphragms and to protect skeletal limb muscle.
Carmeseal-MD is expected to be effective in all DMD patients, regardless of the genetic defect.
The studies that enabled the IND application were supported by the National Institutes of Health’s (NIH) National Heart, Lung and Blood Institute (NHLBI) and by Coalition Duchenne.
The basis for the IND include a two-arm, randomized, double-blind clinical trial with 120 participants at several sites. One dose of Carmeseal-MD will be evaluated against the standard of care over 48 weeks.
The study’s primary endpoint will be forced vital capacity (FVC), and a broad set of secondary endpoints will include cardiac endpoints and additional respiratory endpoints and measures of upper body strength, besides safety measures.
The principal investigator leading the study will be Dr. John L. Jeffries, the director of Advanced Heart Failure and Cardiomyopathy, and a cardiology professor at the Heart Institute and a professor of human genetics at the Cincinnati Children’s Hospital Medical Center.
“This novel therapeutic strategy offers a unique opportunity to potentially favorably impact outcomes in boys and young men with Duchenne muscular dystrophy,” Jeffries said in a press release.
Carmeseal-MD is already being sold in Europe through Ethicor Pharma as part of Phrixius’ European Access Program.