Alex’s Wish invests £30,000 into Edasalonexent

Alex’s Wish invests £30,000 into first study to examine Edasalonexent as a potential treatment for non-ambulant patients with DMD

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

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.

Alex Johnson, Joining Jack founder, along with co-founder of Duchenne UK, Emily Crossley, said:

“We first approached Catabasis earlier this 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 (https://dmdhub.org/trials/phase-3-polaris-dmd-trial/).

You can view the slides from Duchenne UK Patient Information to see the results:
https://www.duchenneuk.org/Handlers/Download.ashx?IDMF=5e0a155c-d6ee-4629-998d-b3a4df760fae

This project investment was lead by Duchenne UK and Joining Jack, alongside ourselves Alex’s Wish and family funds: Archie’s March, Help Harry, Jack’s Aim and Jacobi’s Wish.

Posted in Funding, Uncategorized