Early phase clinical trial results offer new hope for dyskinesia treatment

We're excited to announce positive results of a trial for a potential new treatment for people with Parkinson’s with levodopa-induced dyskinesia. 

Dyskinesia is a debilitating side effect of current Parkinson’s medication, with around half (40 to 50%) of all people with Parkinson’s experiencing it after 5 years of taking levodopa, the main drug used to treat the condition. Up to 80% experience it after 10 years.

With dyskinesia everyday tasks, such as eating, writing and walking, can become extremely difficult. In fact, uncontrolled movement was voted the third most important issue to be addressed by research in a recent Parkinson’s UK survey on quality of life. The main medication available to manage dyskinesia is amantadine, which can have side effects and does not work for everyone. 

The study tested whether a drug called NLX-112 is safe to use in people with Parkinson’s. It also looked at how effective it may be at reducing dyskinesia in people who take levodopa to manage their Parkinson's. Its safety has previously been confirmed in people with other conditions.

Serotonin cells in the brain have the ability to convert levodopa into dopamine. These cells are thought to contribute to the development of dyskinesia when they start to release dopamine erratically. NLX-112 works by targeting serotonin cells inside the brain, and decreasing the amount of dopamine the cells release.

Supported by the Parkinson’s Virtual Biotech

The clinical trial was co-funded by the Parkinson’s Virtual Biotech, the drug discovery arm of Parkinson’s UK, and The Michael J Fox Foundation for Parkinson’s Research. The projects the Parkinson’s Virtual Biotech funds are entirely driven by the Parkinson’s community and their priorities. 

This is the first completed clinical trial funded by the Parkinson’s Virtual Biotech, a global partnership with the Parkinson’s Foundation. The promising results show that this innovative way of working to fast-track the most promising breakthroughs through the drug development pipeline is bringing us closer to new treatments. 

What did the research set out to do?

The phase 2a clinical trial investigated how safe and well tolerated the drug was on a small number of people with Parkinson’s. The trial also took a first look at the drug’s efficacy, which is how well it does its job. 

What did the clinical trial involve?

22 participants with Parkinson’s with levodopa-induced dyskinesia completed the 8-week trial in Sweden. 15 participants received NLX-112 and 7 participants received a dummy drug. 

Participants either received NLX-112 or the dummy drug in increasing doses during the initial 4 weeks, to minimise the potential side effects. They stayed on the maximum dose for 2 weeks, and then were weaned off the drug over 2 weeks. 

What were the results?

The results achieved the first objective to suggest that NLX-112 was safe and well tolerated in people with Parkinson’s. 

The second aim of the study was to show that NLX-112 was effective in treating dyskinesia. The results suggest participants who received NLX-112 showed significant reduction in their scores for dyskinesia, whereas those who received the dummy drug did not show significant reduction in their scores. 

The side effects of participants who received NLX-112 were mild, which confirmed previous results. 

What's next?

Now that the phase 2a clinical trial has been completed, the researchers will complete a full analysis of the results. They will then progress to a phase 2b clinical trial where they will investigate the safety and efficacy of the drug in a larger group.

Dr Arthur Roach, Director of Research at Parkinson’s UK, said:

"We’re incredibly proud and excited by these early results from Neurolixis. They were one of the first companies that the Parkinson’s Virtual Biotech invested in, in partnership with The Michael J Fox Foundation, so to see it making positive progress just reiterates why this brave and innovative approach is right for the Parkinson’s community. It really is bringing us closer to new treatments that address the symptoms that the Parkinson’s community have told us are the most urgent and levodopa-induced dyskinesias is one of those. 

"Further studies will be necessary for regulatory approval and routine clinical use of NLX-112. But now people with Parkinson’s can have hope that a much needed new treatment for levodopa-induced dyskinesias may be coming to them soon, and know that their support of the Parkinson’s Virtual Biotech has made this possible."