We are disheartened to learn today the results from the Catabasis Phase 3 trial for edasalonexent (PolarisDMD) did not meet its primary or secondary endpoints.
The unfortunate results from the Catabasis study is yet another discouraging blow to our community and devastating to those who participated in the study. We are all well-aware inflammation is significant in Duchenne, present at birth and increasing over time as muscle cells deteriorate. This persistent inflammation is one of the reasons that steroids (which help reduce inflammation) are part of the standard of care for Duchenne. Edasalonexent is an NFkB inhibitor, suggested to “tamp down” inflammatory pathways while hopefully not inducing the side-effect profile seen with chronic steroid use. The study included 131 participants and started in October, 2018. It was 52 weeks in duration with a primary outcome of change in the NSAA compared to placebo.
The Polaris 3 study is complete and data suggests there are positive trends, but not statistically meaningful to reach the required P-value. While there are many questions left to be answered, we are grateful that the Catabasis team will continue to analyze the data. Families participating in the GalaxyDMD open-label extension trial are encouraged to reach out to their site directly.
Please join PPMD and Catabasis as we host a webinar tomorrow, October 27 at 1 PM ET to discuss the trial and results in more detail, as well as the company’s plans for the edasalonexent program going forward.Join the Webinar >
Our Work Continues
We have many questions for everyone involved in the drug development space working diligently to create treatments for individuals with Duchenne. Is 52 weeks sufficient time to demonstrate improvement or even stability? Can we design better trials to understand if these investigational products provide long-term benefit? And perhaps a bigger question remains around willingness for regulatory flexibility for a rare disease with so much heterogeneity.
We are grateful to all the families who have participated in clinical trials with edasalonexent, and to the Catabasis team for their work to advance therapy development in Duchenne. The failure of a trial is always difficult for our community, but we remain hopeful that the forthcoming data from this trial will inform other potential treatments and that these experiences will lead us to the day that we end Duchenne.
Read the Catabasis Community Letter:
We are very sorry to share today that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy did not meet its primary endpoint, which was to measure the change in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent treatment compared to placebo. The secondary endpoints were the timed function tests (time to stand, 10-meter walk/run, and 4-stair climb) and also did not show statistically significant improvements. Muscle enzymes and heart rate did not show significant differences between edasalonexent and placebo.
Edasalonexent was well-tolerated, which is consistent with the safety profile we have 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. There were no adverse trends in blood tests and growth was maintained.
We would like to sincerely thank everyone who participated in and enabled the edasalonexent program. We are greatly appreciative of all the boys that participated in the trial and their families and caregivers. Thank you to everyone who helped make PolarisDMD possible: boys and their families, site personnel, investigators, trial sites and advocacy groups. We are extremely grateful for your support and understand the sacrifices made when participating in a clinical trial and even more so during the COVID-19 pandemic.
We are profoundly saddened and disappointed to share the difficult decision that development of edasalonexent, including the ongoing GalaxyDMD open-label extension trial, will be stopped. In addition to the muscle function and safety information, we will be analyzing the bone and cardiac data, and we are committed to sharing the data from the PolarisDMD trial with the Duchenne community to contribute to natural history data. We plan to submit the data from the Phase 3 PolarisDMD trial for presentation at upcoming scientific conferences as well as to publish these results in an effort to advance Duchenne research for the entire community.
We wish that we could be in touch with each trial participant individually, however due to confidentiality we are unable to reach out directly. We will be in close communication with the investigators and study site personnel. Families involved in the PolarisDMD and GalaxyDMD trials who have questions are encouraged to contact their study site directly.
All in the Duchenne community are welcome to join our webinar with PPMD on Tuesday, October 27 at 1:00pm ET where we will share more details and you can ask questions about the trial and the results.
Joanne M. Donovan, M.D., Ph.D.
Chief Medical Officer, Catabasis Pharmaceuticals
Read the Catabasis Press Release:
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 —
BOSTON–(BUSINESS WIRE)–Oct. 26, 2020– Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, which was 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 going forward.
“We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “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.
“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,” said Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD). “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.”
The Company expects to report Q3 2020 financials in November of 2020. As of September 30, 2020, Catabasis had cash and cash equivalents of approximately $52.9 million.