Catabasis Pharmaceuticals provides update on edasalonexent, qualitative research
Catabasis Pharmaceuticals shared an update on the clinical development of edasalonexent, a novel NF-kB inhibitor for the treatment of Duchenne muscular dystrophy, or DMD, this week at the Parent Project Muscular Dystrophy, or PPMD conference. Catabasis shared progress in the edasalonexent Phase 3 PolarisDMD trial. Screening is ongoing for the randomized, double-blind, placebo-controlled trial in the United States, Canada, Australia, the United Kingdom, Ireland, Sweden, Germany and Israel and making progress. Clinical trial sites globally are enrolling and sites in the United Kingdom, Ireland, Sweden, Germany and Israel are at capacity and no longer accepting additional patients. The trial is active in all planned countries with 37 sites open for enrollment. Top-line results from the study are expected in the second half of 2020 and are anticipated to support an NDA filing in early 2021. Catabasis also shared insights from a recent blinded qualitative research project that collected information from in-depth interviews with approximately 30 physicians treating boys affected by DMD, caregivers and patient advocacy representatives in the United States that was sponsored by Catabasis. A key observation from the research was that members of the DMD community prioritized treatments that will go beyond skeletal muscle and positively impact additional important aspects of DMD, including cardiac and pulmonary effects. Participants also shared their hope that treatments will provide durable benefits and improve quality of life. Additionally, physicians predicted that the majority of boys will receive combination therapy for the treatment of DMD within the next few years. Data from the edasalonexent MoveDMD Phase 2 open-label extension study were also presented at the PPMD conference. Through 72 weeks of treatment compared to an off-treatment control period, patients treated with edasalonexent demonstrated preserved muscle function and substantially slowed DMD disease progression across all four assessments of muscle function. Preclinical data and clinical biomarker data from the MoveDMD trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been well tolerated through more than 55 patient-years of treatment. In the Phase 3 PolarisDMD trial, Catabasis plans to enroll approximately 125 patients ages 4 to 7 regardless of mutation type who have not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen may be eligible to enroll. Boys from the MoveDMD open-label extension and their eligible siblings can now enroll in the GalaxyDMD open-label extension trial. Boys who complete the Phase 3 PolarisDMD trial as well as their eligible siblings will also have the opportunity to participate in the GalaxyDMD open-label extension trial. Boys can begin or continue treatment with an approved exon skipping therapy in the GalaxyDMD trial. The GalaxyDMD trial has a streamlined schedule with visits to trial sites every six months and is designed to collect long-term safety data to support registration filings. The trial is also monitoring assessment of muscle function and bone health.