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La Force is happy to share the latest edition of the Catabasis Connection newsletter – Communicating clinical trial results.

Catabasis understands the importance of clearly communicating clinical trial results with the Duchenne community. As they are getting prepared for the planned release of top-line results from their Phase 3 PolarisDMD trial in Q4 of this year, they are sharing in advance their plans for how those results will be communicated. As a public, pre-commercial stage company, Catabasis is making every effort to balance its reporting obligations and restrictions with the desire to inform the Duchenne community of the trial results quickly, efficiently, and in easily accessible ways. Here are their plans:

  • They will share top-line results in their newsletter and a press release simultaneously. You can sign up here.
  • They will be sharing both the newsletter and press releases on their social media. You can follow them on social media > FacebookTwitter and Instagram.
  • They will host a webinar for the Duchenne community in conjunction with PPMD within a couple of days of the release of the top-line results. The webinar will include a Q&A portion where you can ask questions.
  • Families participating in the Phase 3 PolarisDMD trial can hear about the next steps directly from their trial site personnel.

About Edalasolexent

Edasalonexent is an orally-administered small molecule designed to inhibit NF-kB. Activated NF-kB is a crucial link between the lack of dystrophin and resulting manifestation and progression of Duchenne. By inhibiting NF-kB in Duchenne, edasalonexent can limit muscle degeneration, promote muscle regeneration, and reduce inflammation and fibrosis. Edasalonexent is being developed as a monotherapy and for use with other therapies, such as exon-skipping. We believe that based on its mechanism of action, edasalonexent has the potential for use with different approaches in development, such as gene therapy. The Phase 3 PolarisDMD trial and GalaxyDMD open-label extension trial are both ongoing. Top-line results are expected in the fourth quarter of 2020.

About Catabasis

The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families. There lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and the Phase 3 trial, please visit www.catabasis.com.

About La Force DMD

The Force’s mission is to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate 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.

 

Edasalonexent is an investigational drug that is not yet approved in any territory.

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Pfizer DMD gene therapy