• Plans to initiate trial in second half of 2018
• Edasalonexent significantly slowed progression in Ph II trial
• Edasalonexent is being developed as monotherapy or for use in combination

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB) announced on 7/09/2018 plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy (DMD). Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in the second quarter of 2020.

The POLARIS DMD trial will evaluate the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent. The trial design was informed by discussions with the U.S. Food and Drug Administration (FDA) as well as input from treating physicians and families of boys affected by Duchenne.

“We have designed a robust study with POLARIS DMD to evaluate edasalonexent as a potential new treatment for Duchenne. We have benefited from input from many people that are part of the Duchenne community and we are well underway with our preparations to begin the trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We are very excited to advance edasalonexent through this potentially last phase of clinical development with the hope of providing a new treatment option to all boys affected by this disease. We believe that edasalonexent has great potential as a therapy to be taken on its own as well as in combination with other treatments.”

Edasalonexent is a potential oral foundational therapy that is being developed for all patients affected by DMD. Edasalonexent is being developed for use as monotherapy and in possible combination with dystrophin upregulation therapies. Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD Phase 2 trial and open-label extension. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been safe and well tolerated through more than 45 patient-years of treatment.

More specifically, edasalonexent, administered orally as 100 mg/kg dose for 48 weeks — resulted in a statistically significant delay in disease progression and signs of lower inflammation and muscle fat accumulation compared to measures taken before the start of treatment.

Edasalonexent inhibits NF-kB, a protein that is activated in DMD and drives inflammation, fibrosis and muscle degeneration and suppresses muscle regeneration.