DMD research

Duchenne UK supports first study to examine Edasalonexent as a potential treatment for non-ambulant patients with DMD

8th January 2019

  • Duchenne UK, with support from Joining Jack and Alex’s Wish, grant US$615,056 to Catabasis Pharmaceuticals to study Edasalonexent, a new drug for DMD to be tested in non-ambulant boys
  • Duchenne UK approached Catabasis last year to offer funding to extend their clinical trials to the non-ambulant population

This trial is an extension to Cababasis’ current ongoing study looking at their drug, Edasalonexent – currently being tested of 4-7 year olds. It’s important to us to do this. Not only because our boys and young men are living longer and want to be part of research. But also because we owe a debt of thanks to the parents who came before us and who started this fight for treatments. They and their sons MUST be included in research.

Edasalonexent is a novel NF-kB inhibitor, that is being studied in patients not taking steroids.

The drug is currently in a global Phase 3 pivotal study in 4 to 7 year old ambulant boys not on corticosteroids. Last year, we approached Catabasis to offer funding to extend their clinical trial into the non-ambulant population.

This will be a pilot study in non-ambulatory patients, which means that it will be tested on older boys and young men who can no longer walk. We know that 40% of these patients are not on steroids, so we can look to see if this drug can have an impact there.

The results from Edasalonexent being tested in young patients who are not on steroids, so far, have been positive.

We have seen that it has the potential to slow the progression of the disease by improving muscle function, preserving cardiac function and reducing risk of fractures, so we hope that it can help patients with any mutation and the older population as well. Plus, it doesn’t work through the routes that steroids do. So, we hope it will have functional benefits, as well as anti-inflammatory and muscle protective effects, but without the side effects of steroids. 

Emily Crossley & Alex Johnson, Duchenne UK cofounders, said:

“We first approached Catabasis last year to ask if we could encourage them to advance their trial to look at the non-ambulant patient population and we are delighted to be able to announce this collaboration today. Duchenne UK is committed to developing medicines for ALL boys and men with DMD, regardless of their physical state, mutation or age.  This trial will represent an important step in that direction.”

Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, said:

“We are thrilled to announce plans to expand our knowledge of edasalonexent to non-ambulatory boys and men affected by Duchenne. We recognize the urgent need for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function by benefitting both skeletal muscle as well as cardiac function. We are incredibly fortunate to have the opportunity to partner with Duchenne UK for this important work and appreciate their deep commitment as we work together to bring treatment options to all patients.”

Emma Hallam, DMD parents and founder of Alex’s Wish, said:

“We are so encouraged to be able to invest in the extension of this trial into the non-ambulant population of the Duchenne community, it is a very important step for us to ensure that we move forward the development of medicine for all those living with Duchenne Muscular Dystrophy, despite age or stage of progression.”

Catabasis is currently conducting the PolarisDMD trial, a Phase 3 pivotal study for edasalonexent in 4 to 7-year-old boys not on corticosteroids.

View the PolarisDMD trial on the DMD Hub website: https://dmdhub.org/trials/phase-3-polaris-dmd-trial/

Click here to view the slides from our Patient Information to see the results.

We would like to thank our partner charities Joining Jack and Alex’s Wish for supporting this project. We would also like to thank our family funds who have supported this project: Archie’s March, Help Harry, Jack’s Aim and Jacobi’s Wish.


What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org

Published on 8 January 2019

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