Health and Healthcare

Catabasis Gears Up for Late-Stage Duchenne Muscular Dystrophy Trial

Chris Lange

Published: January 3, 2019 1:15 pm

Last Updated: January 11, 2020 1:01 pm

Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) shares jumped on Thursday after the company provided an update from its late-stage trial for edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD). Look for top-line results from the Phase 3 PolarisDMD trial in the second quarter of 2020.

Ultimately, the PolarisDMD trial is evaluating the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent.

The Phase 3 PolarisDMD clinical trial sites are actively enrolling patients. Specifically, a total of nine PolarisDMD clinical trial sites are now open for enrollment across the United States and at least 10 additional sites are expected to open shortly.

Additionally, Catabasis has received approval of clinical trial applications to conduct the PolarisDMD trial in Canada and multiple countries in Europe. Clinical trial sites are expected to open in Canada this month and sites in Europe as well as Australia and Israel, pending regulatory approval, are expected to open in first quarter of this year. In total, the PolarisDMD trial is expected to include roughly 40 clinical trial sites globally with enrollment expected to be completed in 2019.

Joanne Donovan, M.D., Ph.D., chief medical officer of Catabasis, commented:

We are very excited to be screening and dosing patients in our Phase 3 PolarisDMD trial as we believe edasalonexent has tremendous potential for all those affected by Duchenne, regardless of mutation type and from the time of diagnosis throughout their lifespan. We have received extensive inbound interest from families interested in the study and we are actively referring these families to the enrolling clinical trial sites. Edasalonexent inhibits NF-kB, which plays a fundamental role in skeletal and cardiac muscle disease in Duchenne. We are monitoring bone and heart health in the PolarisDMD trial, in addition to the assessments of skeletal muscle function, as we see these as important potential differentiating benefits of edasalonexent.

Shares of Catabasis were last seen up about 6% at $4.70, with a consensus analyst price target of $38.33. The stock has a 52-week trading range of $0.39 to $23.80.

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