Topline results announced from phase 2 trial of treatment for early Parkinson’s disease

Key takeaways:

• The primary endpoint of statistically significant change from baseline to week 36 in sum of MDS-UPDRS Parts II and III was not met.
• Efficacy analysis suggests a positive impact in patients younger than 60 years.

Neuraly has announced that its investigational NLY01 did not meet the primary study endpoint of improvement in motor symptoms in patients with early, untreated Parkinson’s disease.

According to a company release, NLY01 is a long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor agonist. In an ongoing phase 2 trial, NLY01 did not achieve statistically significant change from baseline to week 36 in the sum of MDS-UPDRS Parts II (Motor Experiences of Daily Living) and III (Motor Examination) compared with placebo, although it did have a favorable safety profile and was well-tolerated.

The randomized, double-blind, placebo-controlled trial included 255 participants from 55 clinical sites in the United States and Canada and sought to assess safety, tolerability and efficacy of NLY01 in patients with early PD. Participants were given weekly injections of 2.5 mg or 5 mg of NLY01 or placebo for 36 weeks, with clinical assessments made throughout the trial and 8 weeks after treatment.

Notably, a post-hoc analysis stratified by age revealed a beneficial treatment effect in participants younger than 60 years, with significant reduction in the sum of UPDRS Parts II and III over 36 weeks. Per the release, the effect was statistically significant, dose-related and persisted over 8 weeks after treatment, suggesting NLY01 resulted in slower disease progression compared with placebo-treated individuals of similar age.

“Although the study didn’t meet its primary endpoint, it is intriguing that NLY01 demonstrated a beneficial effect in patients under 60,” Viktor Roschke, head of research and development at Neuraly, said in the release. “This potentially may represent interest for further clinical evaluation of NLY01 in younger PD patients.”

The company will engage in rigorous data analysis to determine next steps for NLY01, the release stated.