Rare Daily Staff
Catabasis Pharmaceuticals and the non-profit Duchenne UK have entered into a partnership for a phase 2 trial of edasalonexent, Catabasis’ experimental NF-kB inhibitor, in non-ambulatory patients with Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle deterioration and weakness that primarily affects boys with symptoms beginning as early as three years of age. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure. DMD occurs in about one out of every 3,600 male infants worldwide.
“We recognize the urgent need for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function by benefitting both skeletal muscle as well as cardiac function,” said Joanne Donovan, chief medical officer of Catabasis.
The exploratory phase 2 trial is designed to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients. The trial commencement is subject to the receipt of adequate funding with Duchenne UK granting more than $600,000 in funding to support patient and clinical trial site costs.
The phase 2 trial is designed to be a one-year, randomized, double-blind, placebo-controlled trial evaluating safety, pharmacokinetics and exploratory measures of function with edasalonexent in non-ambulatory boys and men affected by DMD. The trial expects to enroll approximately 16 non-ambulatory patients ages 10 and older regardless of mutation type who have not been on steroids for at least 6 months at clinical trial sites in the United Kingdom. The exploratory functional endpoints are anticipated to include assessments of cardiac function, upper limb skeletal muscle function and pulmonary function. In addition, the trial is also expected to explore patient reported outcomes. The intention is that upon completing this trial, patients will have the option to transition to the open-label extension trial and receive edasalonexent.
Edasalonexent is an experimental oral small molecule designed to inhibit NF-kB that Catabasis is developing as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Catabasis’ ongoing global phase 3 trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in an open-label extension trial. In Catabasis’ phase 2 trial and open-label extension, the company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation.
The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations and the European Commission has granted Orphan Medicinal Product designation to edasalonexent for the treatment of DMD.
Photo: Joanne Donovan, chief medical officer of Catabasis