Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no longer walk.
The trial, to take place at Cincinnati Children’s Hospital, will evaluate the drug’s performance on respiratory measures such as forced vital activity, and on preventing cardiac and skeletal limb muscle damage.
The goal is to demonstrate Carmeseal-MD’s beneficial effects in patients with DMD and Becker muscular dystrophy. It aims to do this by documenting an improvement in cardiac and respiratory function via protection of heart muscle and diaphragm, as well as an improvement in upper body strength.
Carmeseal-MD has been shown in animal models to improve the efficiency of damaged hearts and diaphragm performance with once-daily subcutaneous administration at low doses. When infused into the bloodstream, the drug binds to microscopic tears in the muscle and prevents the pathological leakage of calcium into cells, which keeps the muscle from performing as it should. Carmeseal-MD is expected to benefit DMD patients egardless of their genetic mutation.
“Older, non-ambulatory patients with DMD have few treatment options, especially with regard to heart failure and respiratory dysfunction, the two leading causes of death,” John L. Jefferies, the study’s principal investigator, said in a press release. “Therefore, they are in urgent need of additional therapies that can slow or arrest disease progression.”
According to DuchenneConnect, one of the study’s partners, Carmeseal-MD has an open investigational new drug application in the United States but it is already available to patients in Europe, Argentina and New Zealand as an unlicensed medicinal product, when prescribed by a specialist.
The single-center, open-label study will enroll eight patients, including non-ambulatory boys and young men with early heart failure and respiratory dysfunction on a stable regimen of background therapies, including corticosteroids.
For 52 weeks, patients will be subcutaneously injected 5 mg/Kg of P-188 NF daily. Monitoring will be conducted by cardiac magnetic resonance imaging, pulmonary function testing and a number of upper body function tests, including performance of upper limbs.
“We are excited to join forces with our colleagues in the nonprofit community to support the development of Carmeseal-MD as a treatment for Duchenne,” said Benjamin Seckler, president of Charley’s Fund, one of the DMD groups taking part. “This pilot study will generate initial data in the Duchenne population for a promising treatment that targets a major unmet need.”
The study will be funded by the SMARTT (Science Moving towards Research Translation and Therapy) program at the National Heart, Lung and Blood Institute. For more information on this therapy, please contact firstname.lastname@example.org or visit www.phrixuspharmaceuticals.com.