Catabasis Pharmaceuticals and Duchenne Uk Announce Partnership to Evaluate Edasalonexent in A Phase 2 Non-Ambulatory Duchenne Muscular Dystrophy Trial
January 08, 2020 at 08:00 am EST
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Catabasis Pharmaceuticals, Inc. and Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), have entered into a partnership for a Phase 2 trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory DMD patients. This exploratory Phase 2 trial, which is subject to the receipt of adequate funding, is designed to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients. 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 GalaxyDMD open-label extension trial and receive edasalonexent. Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed 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. ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In MoveDMD 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 FDA 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.
Astria Therapeutics, Inc. is a biopharmaceutical company focused on developing therapies for allergic and immunological diseases. The Company's lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema (HAE), a rare, debilitating and potentially life-threatening disease. Its second program, STAR-0310, is a monoclonal antibody OX40 antagonist in preclinical development for the treatment of atopic dermatitis (AD). The Company owns two patent families directed to STAR-0215. The first patent family is directed to the composition of matter of its product candidate STAR-0215 and its use in treating various plasma kallikrein associated disorders including HAE. In the second patent family, the Company owns one International (PCT) patent application directed to methods of treating various plasma-kallikrein associated disorders, including HAE, with specific dosing regimens of the STAR-0215 antibody.