NEW YORK – Neuron23 this week detailed the design of its global Phase II trial, dubbed NEULARK, in which it is investigating a precision treatment strategy in early Parkinson's disease.
Within the clinical trial, which the company plans to launch early next year, investigators will evaluate NEU-411, a selective inhibitor of LRRK2, and its potential to slow disease progression.
LRRK2 mutations are the most common cause of familial Parkinson's, representing about 2 percent of cases. Additionally, there's emerging evidence that LRRK2 activity plays a role in disease for a larger subset of patients with idiopathic Parkinson's, according to Neuron23. Taking these populations together, South San Francisco, California-based Neuron23 estimates that up to 30 percent of patients with Parkinson's could benefit from its LRRK2-inhibiting drug.
Within the Neuron23 trial, investigators will utilize an investigational assay that Neuron23 developed with Qiagen to identify people with LRRK2-driven Parkinson's based on SNPs that are associated with LRRK2 overactivity. The whole-exome next-generation sequencing-based assay detects these SNPs and uses a predictive model to identify patients who are likely to have elevated LRRK2 pathway activity.
Quest Diagnostics will deploy the assay at its laboratory in San Juan Capistrano, California, to identify candidates for the clinical trial under an exclusive agreement.
The primary endpoint of the clinical trial is disease progression as measured by a digital biomarker developed by Roche, called Navify. Patients will use a smartphone equipped with software that measures Parkinson's symptoms such as slowed movement, tremors, and cognition. Roche will provide device hardware, software, and site support for the clinical trial.
"By utilizing a digital biomarker as the primary endpoint and the first precision medicine patient stratification approach in Parkinson's clinical development, we are shedding new light on how we can effectively identify and measure therapeutic impact in people with LRRK2-driven Parkinson's disease," Neuron23 CEO Nancy Stagliano said in a statement.
Neuron23 has also partnered with Sano Genetics, a biotech company that offers genetic testing and recruits patients for clinical trials, to offer saliva test kits to identify patients with LRRK2-driven Parkinson's. Sano will provide genetic counseling and engagement tools to these patients and refer patients to NEULARK trial sites for potential enrollment as part of the partnership.