Duchenne UK is partnering with Catabasis Pharmaceuticals to provide financial support for MoveDMD, a Phase 2 clinical trial testing the experimental oral therapy edasalonexent in non-ambulatory boys and men with Duchenne muscular dystrophy (DMD).
The charity is granting more than $600,000 to support participants and trial expenses. The study will measure edasalonexent’s safety, pharmacological profile, and effectiveness.
“We first approached Catabasis last year to ask if we could encourage them to advance a trial to look at the non-ambulant patient population and we are delighted to be able to announce this collaboration today,” Emily Crossley and Alex Johnson, Duchenne UK’s co-founders, said in a press release.
“Duchenne UK is committed to developing medicines for all boys and men with DMD, regardless of their physical stage, mutation or age. This trial will represent an important step in that direction,” Crossley said.
Joanne Donovan, MD, PhD, chief medical officer of Catabasis, said “We are thrilled to announce plans to expand our knowledge of edasalonexent to non-ambulatory boys and men affected by Duchenne. We recognize the urgent need for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function by benefiting both skeletal muscle as well as cardiac function.”
Edasalonexent, developed by Catabasis, is an orally available small molecule administered in oral capsules. It works by blocking a protein called NF-kappa B, which suppresses a pathway that links the loss of the dystrophin protein — a hallmark of DMD — and disease progression. By targeting NF-kappa B, edasalonexent is intended to prevent muscle fiber breakdown.
The trial will compare edasalonexent to a placebo over one year. It will be conducted at sites around the U.K. and is expected to enroll about 16 non-ambulatory males age 10 and older affected by DMD — regardless of mutation type — who have stopped taking steroids for at least six months.
Clinical assessments will include tests of cardiac function, upper limb skeletal muscle function and pulmonary function, as well as patient-reported outcomes. Upon completing the trial, patients will have the option to continue treatment as part of an open-label extension study called GalaxyDMD (NCT03917719).
“We are incredibly fortunate to have the opportunity to partner with Duchenne UK for this important work and appreciate their deep commitment as we work together to bring treatment options to all patients,” Donovan said.
Results of the MoveDMD Phase 2 trial (NCT02439216) in boys age 4–8 showed that edasalonexent helped preserve muscle function and slowed disease progression at 72 weeks of treatment.
Currently, the effectiveness and safety of edasalonexent is being evaluated in the Phase 3 PolarisDMD trial (NCT03703882) in boys age 4–7. As in MoveDMD, participants of PolarisDMD also will have access to continued treatment in GalaxyDMD.
The U.S. Food and Drug Administration has granted orphan drug, fast track and rare pediatric disease designations to edasalonexent for the treatment of DMD. The European Commission awarded the therapy orphan medicinal product designation for the same purpose.