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 are preparing to initiate 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 expected to enroll 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. Final preparations are underway and enrollment will begin soon in 2018.

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 GLOBAL PHASE 3 CLINICAL SITES

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

At CureDuchenne Cares Family & Caregiver Workshops
September 15 in Salt Lake City, UT, and September 22 in Nashville, TN. This program provides information on the standard of care for loved ones with DMD. Register at www.cureduchenne.org/slc18 and www.cureduchenne.org/bna18.

Catabasis is proud to support World Duchenne Awareness Day!

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.