Phrixus to study P-188 NF in Phase II trial to treat DMD

17th September 2017 (Last Updated September 17th, 2017 18:30)

US-based Phrixus Pharmaceuticals is set to conduct a Phase II clinical trial of Poloxamer 188 NF (P-188 NF) for the treatment of non-ambulatory patients with Duchenne muscular dystrophy (DMD).

US-based Phrixus Pharmaceuticals is set to conduct a Phase II clinical trial of Poloxamer 188 NF (P-188 NF) for the treatment of non-ambulatory patients with Duchenne muscular dystrophy (DMD).

The firm has entered agreements to perform the trial at various DMD patient organisations and Cincinnati Children’s Hospital in the country.

Formulated as a subcutaneous injection, P-188 NF is an active pharmaceutical ingredient of Carmeseal-MD.

The single-centre, open-label Phase II trial will investigate 5mg/kg once-daily P-188 NF over 52 weeks in eight non-ambulatory boys and young men who had early heart failure and respiratory dysfunction on stable background therapies such as corticosteroids.

During the trial, subjects will be evaluated through cardiac magnetic resonance imaging (MRI), pulmonary function and different upper body function tests, including performance of upper limb (PUL).

"This pilot study will generate initial data in the Duchenne population for a promising treatment that targets a major unmet need: Duchenne-related cardiac and pulmonary failure."

The trial will include multiple assessments such as P-188 NF effects on forced vital activity and other respiratory endpoints, as well as secondary endpoints associated with performance of cardiac and skeletal limb muscles.

Funding for the trial had been led by Charley’s Fund and Team Joseph, while the National Heart Lung and Blood Institute will supply the drug product.

Charley’s Fund president Dr Benjamin Seckler said: “We are excited to join forces with our colleagues in the non-profit community to support the development of Carmeseal-MD as a treatment for Duchenne.

“This pilot study will generate initial data in the Duchenne population for a promising treatment that targets a major unmet need: Duchenne-related cardiac and pulmonary failure.”