PolarisDMD: Phase 3 study of Edasalonexent (formerly CAT-1004) in people with Duchenne

Catabasis’ Phase 3 PolarisDMD Trial of edasalonexent in Duchenne muscular dystrophy

Questions and Answers

What stage is this research?

PolarisDMD is enrolling boys in a global Phase 3 trial in Duchenne muscular dystrophy to evaluate the efficacy and safety of edasalonexent.

What is the goal or purpose of this research?

Edasalonexent inhibits NF-kB, a protein which plays a fundamental role in skeletal and muscle disease in Duchenne. By inhibiting NF-kB, edasalonexent has the potential to decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression. Our goal is for edasalonexent to become an oral new foundational therapy to slow disease progression for all people affected by Duchenne at all ages as a single agent and in combination with other therapies.

Who is eligible to participate in the trial?

 The trial is enrolling approximately 125 patients with the following criteria:
 Boys ages 4 to 7 (up to 8th birthday) with a diagnosis of Duchenne (any confirmed mutation)
 Ability to complete the timed function tests, including stand from supine without assistance in < 10 seconds, 10MWT and 4-stair climb  Not on corticosteroids for at least 6 months  The key inclusion and exclusion criteria for this clinical trial is available on www.clinicaltrials.gov (NCT# 03703882).

 What do I have to do if I decide to participate in the trial?

 The trial is a randomized, double-blind and placebo-controlled study with 2 boys receiving edasalonexent for each boy receiving placebo. All boys may have the opportunity to receive edasalonexent after 12 months through our open-label extension, GalaxyDMD.
 Edasalonexent capsules are taken orally, three times per day with food. The soft-gel capsules are available in two sizes, one similar to the size of a tic-tac and another similar to the size of a jelly bean. A special straw is provided to help facilitate capsule swallowing.
 Clinical trial site visits are every 3 months to measure key assessments and safety, including labs. Neither muscle biopsies nor MRIs are required for this study.

How long will this study last, and will I have access to the drug/treatment once the study has ended?

After the 12-month PolarisDMD trial, all boys may have the opportunity to receive edasalonexent in our open-label extension, GalaxyDMD.

Is there any funding to help pay for site travel?

Yes, Catabasis will fund travel-related expenses as described by the clinical study coordinator at the clinic site and through our patient travel coordinating agency.

What are the results from the Phase 2 MoveDMD® trial and open-label extension?

 Preserved muscle function and slowed disease progression were observed in all assessments of muscle function (North Star Ambulatory Assessment, time to stand, 4-stair climb and 10-meter walk/run) through 72 weeks of edasalonexent treatment compared to the off-treatment control period.
 Supportive changes in measures of muscle health were seen, with significant reductions in muscle enzymes, C-reactive protein (CRP), and MRI T2 rate of change compared to the off-treatment control period, supporting the durability of edasalonexent treatment effects.
 Significantly decreased heart rate towards age-normative values, supporting potentially beneficial cardiac
effects of edasalonexent.
 Boys grew similarly to unaffected boys. Height increased by an average of 2.1 inches/year, while weight increased by an average of 2.9 lbs/year, both in line with typical height and weight increases of unaffected boys.

Who is funding this research?

This research is being done by Catabasis, a clinical-stage biopharmaceutical company with a mission to bring hope and life-changing therapies to patients and their families.

Where does the clinical trial take place?

PolarisDMD has clinical trial sites in North America, Europe, Israel, and Australia to improve patient access. For more information on expected and active clinical sites, please visit www.catabasis.com/patients-families/polaris-dmd-clinical-trial.php.

Where can I learn more about this research?

 You can learn more at www.catabasis.com/patients-families/polaris-dmd-clinical-trial.php
 You can contact Catabasis directly with any questions at DMDTrials@catabasis.com.
 Please check www.ParentProjectMD.org for updates to this FAQ sheet.

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