Clinical trial starts for potential Becker muscular dystrophy drug

A clinical trial is under way for EPM-01 – a potential treatment for boys and men with Becker muscular dystrophy that has shown early promise.

There are currently few clinical trials being carried out for the condition, which affects about 2,400 people in the UK.

While it is still early days and EPM-01 would not be a cure, it is hoped that the drug, also known as (+)-epicatechin, could slow the progression of Becker muscular dystrophy.

What is Becker muscular dystrophy?

Becker muscular dystrophy usually affects boys and men. It causes muscles to weaken and waste over time, leading to increasing and often severe disability.

In some cases, Becker muscular dystrophy can lead to life-threatening health problems, as heart and breathing muscles weaken. There is currently no treatment for the condition.

How does EPM-01 work?

EPM-01 is a man-made drug manufactured by Epirium Bio Inc that appears to improve some functions common in Becker muscular dystrophy, such as muscle regeneration and cell energy balance.

While it would not be a cure, it is hoped it could slow the progression of the condition. You can read more in this news release from the company.

What do we know about the clinical trial?

The early-phase clinical trial involves boys and men aged between 16 and 60 and is taking place in the USA.

This is an open-label study, which means researchers and participants will know which dose of the drug they are taking. The participants will be given up to three different doses that will increase every two months over a six-month period.

The main aim of the trial is to look at EPM-01’s safety and tolerability – the degree to which the side effects of a drug can be “tolerated” by the participant.

The study will also look at early measures of how effective the drug is by examining changes in blood and various muscles through muscle biopsy, heart imaging and tests of muscle function.

What do we think about this?

Although both Becker muscular dystrophy and Duchenne muscular dystrophy are caused by mutations in the dystrophin gene, very few clinical trials are carried out for Becker muscular dystrophy.

This is a significant study for people with the condition – however, it is important to remember that not all clinical trials result in the creation of a drug. This could be because the expected outcomes might not be reached, or there are safety concerns.

Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, says:

While it is still very early days, this will be welcome news for the Becker muscular dystrophy community. We will be following the clinical trial with interest and will continue to keep our supporters updated on the progress of EPM-01 and other potential treatments.


You can read more about the clinical trial here