PhaseOut is a Phase 2 proof of concept clinical trial of its utrophin modulator ezutromid in patients with DMD.
Dosing is expected to follow for US patients within 28 days.
The PhaseOut trial is already underway in the UK.
John Jefferies, at Cincinnati Children’s Hospital and the US trial’s coordinating investigator said: “Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow or stop disease progression in all patients with DMD, regardless of their underlying dystrophin gene mutation.”
DMD (Duchenne Muscular Dystrophy) is caused by different mutations in the dystrophin gene that result in patients being unable to produce the protein, which is essential for maintaining healthy muscle function.
Ezutromid is designed to increase the levels of another, similar, protein call utrophin to compensate from the lack dystrophin. The system is called utrophin modulation.
Story by ProactiveInvestors