{"id":5080,"date":"2017-07-10T14:09:58","date_gmt":"2017-07-10T14:09:58","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=5080\/"},"modified":"2022-05-03T18:21:23","modified_gmt":"2022-05-03T18:21:23","slug":"portrait-of-duchenne-edasalonexent","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/portrait-of-duchenne-edasalonexent\/","title":{"rendered":"Portait of Duchenne \u2013 Edasalonexent: The potential to modify DMD"},"content":{"rendered":"

Joanne M. Donovan, MD, PhD<\/strong><\/h4>\n

Chief Medical Officer at Catabasis Pharmaceuticals, Cambridge, Massachusetts, USA<\/h4>\n

In this fifth interview of our series \u201cPortrait of Duchenne,\u201d<\/em> La Force DMD talks with Joanne Donovan<\/strong>, <\/strong>Chief Medical Officer at Catabasis Pharmaceuticals. <\/strong>She has been working on a treatment for DMD called <\/strong>edasalonexent (formerly known as CAT-1004)<\/strong>. She earned her MD at Harvard Medical School. <\/strong><\/p>\n

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Understanding how edasalonexent works<\/b><\/h4>\n

Edasalonexent (CAT-1004) is an investigational oral drug that targets NF-\u0138appa B (NF-\u0138B). What is NF-\u03baB, and how does its inhibition benefit patients with Duchenne muscular dystrophy (DMD)? NF-kB is a protein complex that controls the transcription of DNA. In people with DMD, the absence of dystrophin, combined with mechanical stress in muscle, leads to an activation of NF-\u0138B. When activated, NF-\u0138B transcribes proteins that drive muscle damage and prevent muscle regeneration. Ultimately, the mission of edasalonexent is to prevent NF-kB from being activated. Inhibiting NF-kB can potentially protect muscles and have an important disease-modifying effect in DMD. This treatment works in patients with any mutation for DMD.<\/strong><\/p>\n

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Updated information about clinical trials<\/b><\/h4>\n

Parts A and B of the MoveDMD trial with edasalonexent (CAT-1004) in DMD are complete. Part A reported that edasalonexent was well-tolerated with no significant safety issues. Catabasis has reported that, in Part B, with 12 weeks of edasalonexent treatment, numerical improvements were observed in well-established and pre-specified functional assessments. These statistical improvements in the functional assessments demonstrated reductions in the rate of functional decline in both placebo-controlled and crossover analyses. The crossover analysis compared changes during an off-treatment period to edasalonexent treatment for boys who were also in Phase 1 of the trial. The primary endpoint in the 12-week Phase 2, which was an exploratory MRI biomarker endpoint, was not met. Edasalonexent was well-tolerated with no safety signals observed. Edasalonexent is currently in the open-label extension of the MoveDMD trial with results expected in Q3 2017. Catabasis anticipates announcing plans for a Phase 3 trial in the second half of 2017.<\/p>\n

\u00a0<\/strong><\/p>\n

\u00a0<\/strong><\/h4>\n

This\u00a0video was\u00a0recorded in November 2016, Dr. Joanne Donovan answers our questions about edasalonexent (CAT-1004)<\/u><\/strong><\/h4>\n