Parkinson's Cure Journey

Landmark 141 · Trial in people

Lixisenatide Phase 2 (LixiPark)

In 156 people, movement scores stayed flat while placebo scores worsened by 3 points. The gap was small, and nausea was common. It needs a larger test.

In short

In 156 people with early Parkinson's, the diabetes drug lixisenatide left motor scores flat over 12 months while placebo scores worsened by 3 points, but the gap was small and side effects may have unblinded the trial.

What it is
Trial in people
Outcome
Mixed or early results
When
2024
Size
156 people · Phase 2
Approach
Look beyond the brain
Research lane
Metabolic drug repurposing
Trial registry
NCT03439943
Last checked
19 Oct 2026

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

Wassilios Meissner (Bordeaux) and Olivier Rascol (Toulouse) with colleagues in France's NS-Park network. Sponsor: Toulouse University Hospital. The paper lists the French Ministry of Health among its funders; the registry names Cure Parkinson's, NS-Park, EUCLID and Sanofi as collaborators. Published in the New England Journal of Medicine 2024;390(13):1176–1185.

What happened

GLP-1 is a gut hormone that helps control blood sugar. Drugs that mimic it, such as exenatide and lixisenatide, are used for diabetes and appeared to protect neurons in animal models (the story is in landmark 20; the first exenatide pilot is landmark 101). Lixisenatide is a daily injection that is thought to enter the brain better than exenatide.

  • Design. Double-blind, placebo-controlled Phase 2. 156 people (78 per arm), diagnosed less than 3 years earlier, on stable symptom medication, no motor complications. Daily injection for 12 months (10 micrograms for 14 days, then 20 micrograms, per the registry), then a 2-month washout. Only one dose was tested. Started 13 June 2018.
  • Main result. The main measure was the motor score on medication at 12 months (MDS-UPDRS Part III), which changed by −0.04 points on lixisenatide and +3.04 on placebo, a difference of 3.08 points (95% CI 0.86–5.30, p=0.007). After the 2-month washout the off-medication scores were 17.7 (95% CI 15.7–19.7) versus 20.6 (18.5–22.8), so some gap remained.
  • Weak spots. Other measures (daily living, non-motor symptoms) did not clearly favour the drug. Commentators noted that 3 points is below what is usually counted as clinically meaningful. A post hoc split suggested the effect was larger in people under 60 (5.2 points, 95% CI 1.95–8.48) than in those 60 or over (1.0, CI −2.01 to 4.03), but such splits are unreliable.
  • Side effects. Nausea 46% versus 12% on placebo; vomiting 13% versus 3%; reflux 8% versus 1%. Any adverse event 86% versus 71%. Five people in each arm had serious events; one case of pancreatitis in the lixisenatide arm and one of fainting on placebo were judged treatment-related. Obvious nausea may have revealed who was on the drug, which could bias symptom ratings (my inference; not raised in the sources read).

Why it moved us toward a cure

It was the second independent positive Phase 2 of a GLP-1 drug after exenatide (landmark 20), and used a different drug and a different team. That gave the GLP-1 idea much of its momentum. The exenatide Phase 3 that followed found no benefit (landmark 142).

Outcome

Open. Positive on its main measure, but small, and not backed by secondary measures. The paper itself calls for longer and larger trials. A ClinicalTrials.gov search (19 Oct 2026) finds no lixisenatide Parkinson's trial other than this one (status completed; no results posted), so no Phase 3 is registered. The exenatide Phase 3 result means this one needs a larger, longer test before it can be taken as slowing the disease, as opposed to a mild symptom effect.

Sources

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