PolarisDMD Clinical Trial

We are honored and proud to be members of the Duchenne muscular dystrophy (DMD) community – one that is built on strength and determination. We strive to discover, develop and bring to patients therapies that will make a meaningful difference in the lives of those who are affected by this disease.

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We have recently initiated a single global Phase 3 trial called PolarisDMD to evaluate the efficacy and safety of edasalonexent as a potential novel treatment for Duchenne. The trial is enrolling approximately 125 boys ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months. Clinical trial sites across the United States will open for enrollment through this year and into next year. Additional sites in Australia, Canada, Europe and Israel are also expected to open early next year. In total, the PolarisDMD trial is expected to have approximately 40 clinical trial sites globally.

The trial will be randomized, double-blind and placebo-controlled with 2 boys receiving edasalonexent for every 1 boy receiving placebo. The primary endpoint for the trial will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. After 12 months in the study, all boys are expected to have the opportunity to receive edasalonexent in an open-label extension.

EXPECTED & ACTIVE GLOBAL PHASE 3 CLINICAL SITES

  Now Launching: SAN ANTONIO, TX!

For additional information, please visit clinicaltrials.gov

ADDITIONAL INFORMATION

Edasalonexent is a potential foundational therapy for all affected by Duchenne regardless of mutation type. Edasalonexent inhibits NF-kB and is taken by mouth.

Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD Phase 2 trial and open-label extension. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart.

Edasalonexent is being developed as a therapy that could be taken on its own and may also enhance the efficacy of dystrophin upregulation therapies such as exon skipping and gene therapies.

For more information about edasalonexent, visit our pipeline.

Clinical trial site visits and key assessments will be every 3 months during the trial and costs for travel to the trial sites will be provided.

MAKING COMMUNITY CONNECTIONS

Catabasis and PPMD Webinar
We recently co-hosted a webinar with Parent Project Muscular Dystrophy to share information about our PolarisDMD Phase 3 clinical trial of edasalonexent in Duchenne Muscular Dystrophy. Slides from our presentation are available here, and if you have any questions, feel free to reach out to our clinical team DMDtrials@catabasis.com.

Jett Foundation Family Workshops
This national education program brings together clinicians, researchers, and families affected by Duchenne to learn about care, crucial information, and resources. Coming up, Catabasis will attend workshops held in Little Rock, Arkansas on March 9, and Phoenix, Arizona on March 23.

PPMD End Duchenne Tours
In an effort to reach every single family facing a Duchenne diagnosis in the US, PPMD hosts a community experience called the End Duchenne Tour to bring updates on research, advocacy, and care. Catabasis will be on the tour in Omaha, Nebraska on March 30.

CureDuchenne Cares
These events provide an immersive education experience where clinicians and experts share their wealth of Duchenne knowledge and advancements in the field. Caregivers learn about physical therapy, get important updates about clinical trials, and learn valuable best practices in Duchenne Care. Catabasis will attend in Portland, Oregon on February 23, and Columbia, Missouri on March 16.

EXPANDED ACCESS

Catabasis is committed to developing safe and effective therapies for patients with rare diseases. To do this, we conduct clinical trials to evaluate the safety and efficacy of investigational medicines to obtain the necessary marketing approvals that allow patients broad access to these medicines.

In some extreme circumstances, patients with serious diseases may seek access to investigational therapy outside of a clinical trial setting. These situations are typically referred to as expanded access or compassionate use.

When considering whether to support expanded access of an investigational therapy, a number of factors consistent with the US Food and Drug Administration guidelines should be taken into account. These factors include:

  • The disease or condition being studied must be serious and life-threatening.
  • There should be no adequate alternative therapies available.
  • Sufficient efficacy and safety data should exist for the drug in order for Catabasis to make a benefit-risk analysis consistent with the establishment of an expanded access program.
  • Adequate drug supplies must exist to support both the ongoing clinical trials and an expanded access program.
  • The patient must not be eligible for one of the Catabasis clinical trials.
  • The expanded access will not adversely impact the clinical development program, in particular, the conduct of clinical trials required for regulatory approval.

In consideration of our goal to provide access to our investigational therapies at the appropriate time and in the correct manner for all patients, at this time Catabasis believes that participation in one of our clinical trials is the most appropriate way for patients to access our investigational therapies. Our aim is to make our investigational medicines available to as many patients as possible as quickly as possible.

If you have additional questions, please speak with your treating physician or contact DMDtrials@catabasis.com. We anticipate acknowledging receipt of any expanded access inquiries sent to this email address within five (5) business days.

For further information on our current clinical trials, please visit clinicaltrials.gov.

Consistent with the 21st Century Cures Act, Catabasis may revise this policy at any time.