Phase 2 trial results of lixisenatide published

The full results of the one-year, phase 2 clinical trial of Type 2 diabetes drug lixisenatide have now been published. This Cure Parkinson’s co-funded trial has reported positive results, indicating that the treatment may slow the progression of motor symptoms. Researchers have reported that the LixiPark study has met its primary endpoint – the efficacy measures that the trial set out to achieve – as results showed that the progression of motor (or movement) symptoms in those receiving the lixisenatide treatment slowed, while motor symptoms in participants receiving the placebo, or dummy drug, continued to progress. Preliminary results of the LixiPark study were initially presented at the 2023 International Parkinson and Movement Disorder Society annual congress in Copenhagen, with the full results now published in The New England Journal of Medicine. Led by Professors Olivier Rascol and Wassilios Meissner at the University Hospitals of Toulouse and Bordeaux, the trial involved 156 people within 3 years of Parkinson’s diagnosis (early-stage) across 21 different research centres of the NS-Park network in France. The study, sponsored by the Toulouse University Hospital, was co-funded by Cure Parkinson’s with Van Andel Institute (VAI; in Michigan, US), and the French Ministry of Health, with drug and placebo support from pharmaceutical company Sanofi. Lixisenatide was prioritised for clinical testing through Cure Parkinson’s and Van Andel Institute’s International Linked Clinical Trials (iLCT) programme. Lixisenatide belongs to a group of Type 2 diabetes medicines called glucagon-like peptide 1 receptor agonists (or GLP-1R agonists) that function by mimicking the action of a naturally occurring gut hormone that is produced after eating food. GLP-1R agonists are clinically approved for the treatment of diabetes, and, importantly, have demonstrated beneficial actions in the brain as well, making them an area of interest for Parkinson’s research. These results are important because they represent the second clinical trial of GLP-1R agonists in people with Parkinson’s to demonstrate a positive result at phase 2. The first GLP-1R agonist to show potential for slowing motor symptoms in Parkinson’s is exenatide, which was investigated in both a small pilot study in 2008 and a phase 2 clinical trial in 2017. The recent LixiPark study was much larger than both studies but replicates the earlier exenatide results, strengthening support for some of this class of drugs. There is now a large, multicentre phase 3 clinical trial of exenatide for Parkinson’s being run across the UK, and the top-line results are due to be reported in the second half of 2024. While these results show promising potential, it is important to note that there are currently no GLP-1R agonists, including lixisenatide, that are approved for use in Parkinson’s and further testing is required before this drug can be reviewed by regulators for use in Parkinson’s. Cure Parkinson’s is now working closely with the principal investigators of the LixiPark study to plan the next phase in the development of this interesting drug for Parkinson’s.