– This randomised, double-blind, placebo-controlled trial was designed to assess the safety, tolerability, and efficacy of NLY01 in subjects with early, untreated PD.
– The primary endpoint, change from baseline in the sum of MDS-UPDRS Parts II and III after 36 weeks of NLY01 treatment, compared with placebo, did not reach statistical significance.
– Age was identified as a significant factor in response. Statistically significant and dose-related improvements in UPDRS Parts II + III were observed in patients under the age of 60.
– NLY01 was safe and well tolerated.
Neuraly, a clinical-stage biotechnology company developing disease-modifying agents for neurodegenerative disorders, announced on March 27th topline results from the Phase 2 trial with NLY01 in patients with early, untreated Parkinson’s disease (PD). The primary endpoint, change from baseline to Week 36 in the sum of MDS-UPDRS Parts II (Motor Experiences of Daily Living) and III (Motor Examination) after 36 weeks of NLY01 treatment, compared to placebo, did not reach statistical significance. NLY01 was safe and well tolerated.
Post hoc analysis of efficacy measures by age groups suggests a treatment effect in younger patients (<60 years). This group accounted for 37 percent of the study population (N=95). In patients <60 years of age, a clinically significant reduction in the sum of UPDRS Parts II and III was observed over the 36 weeks of treatment in subjects treated with NLY01 (an approximate 5-point reduction at 36 weeks, P<0.01 vs. placebo). The effect was statistically significant, dose-related, and appeared persistent over eight weeks after treatment was discontinued. These results suggest slower disease progression in NLY01-treated subjects compared to placebo-treated subjects in this age group. Detailed data will be presented at future medical meetings.
The Phase 2 study enrolled 255 patients across 55 clinical sites in the United States and Canada. The randomized, double-blind, placebo-controlled trial is designed to assess the safety, tolerability, and efficacy of NLY01 in subjects with early PD. Enrolled patients were randomized to receive either weekly injections of NLY01 (2.5 mg or 5.0 mg) or placebo for 36 weeks. Clinical assessments were made periodically during treatment and again eight weeks after treatment was discontinued. More information about the ongoing Phase 2 study of NLY01 in PD is available at www.clinicaltrials.gov under the identifier NCT04154072.
Although the study didn’t meet its primary endpoint, it is intriguing that NLY01 demonstrated a beneficial effect in patients under 60. This potentially may represent interest for further clinical evaluation of NLY01 in younger Parkinson’s patients.
Viktor Roschke, Head of R&D at Neuraly
Seulki Lee, CEO of Neuraly, also mentioned that the company will thoroughly analyse the data from the study to better understand the outcomes and determine the next steps for NLY01.
About NLY01
NLY01 is a proprietary long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor (GLP-1R) agonist that slows progression in animal models of Parkinson’s and Alzheimer’s disease. In clinically relevant animal models, NLY01 prevented neuronal cell death by inhibiting microglial activation and forming neurotoxic reactive astroglial cells. Treatment with NLY01 slowed disease progression, improved motor and cognitive functions, and extended the lifespan in mice with Parkinson’s disease. NLY01 penetrates the blood-brain barrier (BBB) in animal models, and its receptor (GLP-1R) is highly expressed on glial cells. NLY01 is being developed as a potentially disease-modifying agent for the treatment of neurodegenerative disorders, including Parkinson’s and Alzheimer’s.
About Neuraly
Neuraly is a clinical-stage company whose mission is to translate scientific discoveries in neurology into revolutionary new drugs that can radically improve and prolong the lives of people suffering from the devastating consequences of diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD) and other neurodegenerative disorders. The company is leveraging a deep understanding of the role of glia biology in neuroinflammation and neuroprotection in advancing a risk-diversified product portfolio for PD and AD. Neuraly is a subsidiary of D&D Pharmatech, a global biotech company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by top-tier medical research faculty and biotechnology veterans, please visit D&D Pharmatech (ddpharmatech.com).
