Portait of Duchenne – Edasalonexent: The potential to modify DMD

PORTRAIT OF DUCHENNE – Edasalonexent (CAT-1004): The potential to modify DMD

Joanne M. Donovan, MD, PhD

Chief Medical Officer at Catabasis Pharmaceuticals, Cambridge, Massachusetts, USA

In this fifth interview of our series “Portrait of Duchenne,” La Force DMD talks with Joanne Donovan, Chief Medical Officer at Catabasis Pharmaceuticals. She has been working on a treatment for DMD called edasalonexent (formerly known as CAT-1004). She earned her MD at Harvard Medical School.


Understanding how edasalonexent works

Edasalonexent (CAT-1004) is an investigational oral drug that targets NF-ĸappa B (NF-ĸB). What is NF-κB, 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-ĸB. When activated, NF-ĸB 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.


Updated information about clinical trials

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.



This video was recorded in November 2016, Dr. Joanne Donovan answers our questions about edasalonexent (CAT-1004)


What is edasalonexent (CAT-1004)?

Yes, so we have been working on edasalonexent, which is an NF-kB inhibitor. And the reason that we are targeting NF-kB for Duchenne muscular dystrophy is that that protein is central to the progression of the disease. In infants, NF-kB is active in the muscle, so we know it happens very early before there is progression, and while every boy lacks dystrophin in all of their muscles, we know that the muscles that are subjected to more mechanical stress have faster disease progression. So, we also know that NF-kB is activated by mechanical stress in muscles. So, if we can protect, if we can inhibit NF-kB, we can potentially protect the muscles and have a very important disease-modifying effect.


How do we take it?

It is an oral medicine, and the boys take it as small gel capsules. And even the boys who are aged 4 to 7 in the study have been able to take the capsules.


About clinical trials

This is an initial phase 2 study, and it’s to understand whether the drug affects muscles. And it’s a small study: it’s 31 boys, and we have done this at five sites in the United States. What, with the results of that study, we are then looking to plan studies – a more significant phase 3 study, which is a global study, again in 4 to 7-year-old boys that are not yet on steroids, and we are also potentially looking to start a study next year in non-ambulatory patients that are no longer on steroids. We know that there are a significant number of young men who are no longer on steroids after they become non-ambulatory. So, we’re looking at those two patient groups, and we anticipate that we will start those studies next year in 2018.


About the connection with the DMD community

I have now met many boys, many many parents, and it makes an enormous difference. At Catabasis, we have been fortunate to have several parents come in – we’re a small company – and go in and talk to the whole company. And it’s incredibly meaningful for the company. It gives us very much an understanding of the urgency of why we need to move things forward as fast as we can. So, we appreciate the opportunity always to talk to you as parents and to meet patients, because it does drive us, which is essential.


Interesting links

About Catabasis Pharmaceuticals

Up-to-date information about clinical trials

About NF-kb


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We thank Action Duchenne, who received us with open arms to conduct a series of interviews.

Special Thanks to Daniel K Cooper and Allain Lagadic


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