Catabasis Pharmaceuticals announces top-line results for the phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy.
PolarisDMD Trial Did Not Achieve Primary or Secondary Endpoints – The development of edasalonexent will be stopped.
Original Press release BOSTON, MA, OCTOBER 26, 2020
Catabasis announced that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements. Edasalonexent was observed to be generally safe and well-tolerated in this trial. Catabasis is stopping activities related to the development of edasalonexent, including the ongoing GalaxyDMD open-label extension trial. The Company plans to work with external advisors to explore and evaluate strategic options in the future.
Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis – “We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial. I want to sincerely thank all of the boys, their families and caregivers, investigators and the trial sites that participated in and enabled this program. The entire Catabasis team has worked tirelessly to find a treatment for this progressive disease. We hope that our data and work to date can be used to benefit ongoing and future research in DMD.”
The Phase 3 trial was a one-year placebo-controlled trial designed to evaluate the safety and efficacy of edasalonexent in boys ages 4-7 (up to 8th birthday) with DMD. The global trial enrolled 131 boys across eight countries, with any mutation type, who were not on steroids. Edasalonexent was well-tolerated, consistent with the safety profile seen to date. The majority of adverse events were mild in nature, and the most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash. There were no treatment-related serious adverse events and no dose reductions. The global COVID-19 pandemic had no meaningful impact on the trial or its results. Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published.
Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD) – “These results are disheartening for the Duchenne community, and specifically for the boys who participated in this trial and their families. However, the results contribute to the natural history data of Duchenne and add to the knowledge base that will one day produce a foundational, long-term therapy for this disease. The continued advancement of research and the development of possible treatment options will remain of critical importance to our community. We appreciate Catabasis’ efforts and commitment to every family that is or has ever been affected by Duchenne.”
What is Edasalonexent?
Edasalonexent (CAT-1004 is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It is an investigational oral small molecule. Edasalonexent inhibits NF-kB, a protein activated by DMD and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. By inhibiting NF-kB, edasalonexent can decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression. Edasalonexent was designed as a stand-alone therapy and may also enhance the efficacy of dystrophin targeted therapies.
About Catabasis
The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families.
- Watch this video recorded in November 2016; Dr. Joanne Donovan answers our questions about edasalonexent (CAT-1004)
- CatabasisPharma on Facebook, Twitter and Instagram if you are interested in the latest updates
About La Force DMD
The Force’s mission is to unite the DMD community to raise awareness around a common objective: providing access to new treatments as fast as possible and participating in the funding of promising research projects. Where access to treatments for rare diseases is concerned, it is essential that our community be strong: each member must be an active spokesperson who helps raise awareness for DMD among the general public and the challenges associated with access to treatment.
Catabasis would like to sincerely thank everyone who participated in and enabled the edasalonexent program. Read more here.
