Healthcare Business

Major Duchenne Muscular Dystrophy Trial Is Ready to Launch

Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) has announced that it completed the enrollment for its late-stage Duchenne muscular dystrophy (DMD) study. Specifically, the enrollment is for the Phase 3 PolarisDMD trial of edasalonexent in DMD. Its shares jumped early on Tuesday as a result.

The target enrollment of 125 boys was exceeded due to strong interest from 40 clinical sites in eight countries and the support of patient advocacy organizations. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020m and the trial is anticipated to support a new drug application filing in 2021.

The PolarisDMD trial enrolled 130 boys ages four to seven (up to the eighth birthday) with any mutation type and who had not been on steroids for the past six months.

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

We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive. At a time when there are multiple trials for Duchenne, we are very pleased that physicians and families chose the Phase 3 PolarisDMD trial for edasalonexent. Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health. We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients.

Shares of Catabasis Pharma traded up more than 5% early Monday to $5.69, in a 52-week range of $3.60 to $9.76. The consensus price target is $35.00.