Neuron23 Launches Innovative Phase 2 Trial for Early Parkinson’s Disease

SOUTH SAN FRANCISCO, Calif.--(
BUSINESS WIRE
)--Neuron23® Inc., a biotechnology company in the clinical stages, is making strides in its development of precision medicines aimed at neurological and immunological diseases. Recently, the company announced the initiation of its global Phase 2 NEULARK clinical trial. This trial will focus on NEU-411, a potent and selective inhibitor of LRRK2, which is being tested on individuals with early-stage Parkinson's disease (PD).

The NEULARK trial represents a notable breakthrough in precision medicine, leveraging advanced digital biomarkers and a patient stratification strategy. This strategy aims to identify individuals with PD who have LRRK2-driven pathology and are thus more likely to benefit from NEU-411. While mutations in the LRRK2 gene account for 2% of PD cases, it is estimated that up to 30% of PD patients might experience disease driven by LRRK2.

Nancy Stagliano, Ph.D., CEO of Neuron23, emphasized the trial's innovative approach. She highlighted that while current PD therapies focus mainly on alleviating symptoms without addressing disease progression, NEULARK aims to measure therapeutic impact using digital biomarkers. This approach could revolutionize how treatment efficacy is identified and measured in PD patients with LRRK2-driven pathology. The trial is expected to start in early 2025.

NEU-411 has shown promise as a potent LRRK2 inhibitor, having demonstrated safety and tolerance in a Phase 1 clinical trial involving over 100 healthy volunteers. The trial also showed that NEU-411 effectively engaged its target pathway, evidenced by fluid-based biomarkers in both healthy volunteers and animal studies. These results provide a robust foundation for further clinical development of NEU-411.

LRRK2 mutations are a significant genetic contributor to PD, increasing the risk for those with gain-of-function mutations. Emerging evidence suggests LRRK2 activity might also be relevant in a subgroup of patients with idiopathic, or non-familial, PD. Neuron23 has identified specific single-nucleotide polymorphisms (SNPs) that drive LRRK2 overactivity, potentially affecting up to 30% of idiopathic PD patients. The NEULARK trial aims to target this broader patient population, not just those with familial LRRK2 mutations.

The Phase 2 trial is designed to be a global, double-blind, placebo-controlled study. It will evaluate the safety and efficacy of NEU-411 in early-stage PD patients. To identify trial candidates, Neuron23 has collaborated with QIAGEN to develop a clinical trial assay using next-generation sequencing (NGS) and an algorithmic model. This assay will detect specific SNPs to predict elevated LRRK2 activity.

Quest Diagnostics will implement this assay in their advanced diagnostics lab, ensuring rigorous screening to enroll patients most likely to benefit from NEU-411. Neuron23 has also partnered with Sano Genetics to streamline patient referrals. Sano will provide saliva test kits and genetic counseling, referring eligible individuals to the nearest clinical trial site.

Roche Information Solutions has developed an innovative digital biomarker to monitor disease progression in enrolled participants. This biomarker uses smartphone technology to measure PD symptoms such as tremors and cognitive difficulties. According to Sam Jackson, M.D., Chief Medical Officer of Neuron23, this technology allows for continuous, real-time monitoring of PD symptoms, offering insights that traditional in-clinic assessments cannot match.

Parkinson’s disease is a progressive brain disorder characterized by involuntary movements, stiffness, and balance issues. It can also lead to cognitive and behavioral changes. There is currently no cure for PD, and treatments mainly focus on symptom management.

Neuron23® Inc. was founded in 2018 and focuses on neurodegenerative, neuroinflammatory, and systemic autoimmune diseases. The company combines human genetics, drug discovery, and biomarker technologies to develop new therapies. They are based in South San Francisco, CA.