SOUTH SAN FRANCISCO, Calif. – March 24, 2025 – Neuron23® Inc., a clinical-stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, today announced that data from the Phase 1 healthy volunteer study of NEU-411 will be presented at the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™), taking place April 1-5 in Vienna. NEU-411 is a brain-penetrant, potent and selective inhibitor of LRRK2 for the treatment of Parkinson’s disease (PD).
Initial findings will be presented in a poster titled, “A Phase 1 Study of NEU-411, an Orally Available, Potent, Selective, and Brain-penetrant Small-molecule Inhibitor of LRRK2” (Poster ID: 1379). The Phase 1 clinical trial, designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NEU-411 in 150 healthy adults and elderly volunteers, showed robust LRRK2 target and pathway engagement after NEU-411 administration. NEU-411 was well-tolerated; no serious adverse events were observed, and all adverse events were mild or moderate. The administration of NEU‑411 resulted in dramatic reductions in pS935-LRRK2 in blood and in the cerebrospinal fluid (CSF). The Company plans to present full results from the study, including modulation of phosphorylated Rab10 and di-22:6-bis(monoacylglycerol)-phosphate (BMP), at a future conference.
About LRRK2’s Role in Parkinson’s Disease and NEU-411
Mutations in the LRRK2 gene are among the most common genetic causes of PD, affecting approximately 2% of people with the disease. Individuals who inherit gain-of-function mutations in LRRK2 are at higher risk of developing PD later in life. Additionally, there is emerging evidence that LRRK2 activity may play a role in a subset of the larger population of people with non-familial PD, known as idiopathic PD, suggesting that therapies targeting LRRK2 could be beneficial to a broader patient population than just individuals with rare, familial LRRK2 mutations.
Neuron23 has identified single-nucleotide polymorphisms (SNPs) – variations in an individual’s DNA sequence – that are predicted to drive LRRK2 overactivity in up to 30% of people with idiopathic PD. People with PD who have these SNPs, together with those who have LRRK2 gene mutations, make up the population collectively referred to as LRRK2-driven PD and represent who Neuron23 believes is most likely to benefit from LRRK2 inhibition.
NEU-411 is a potent, highly selective and brain penetrant oral LRRK2 inhibitor with best-in-class potential. By specifically inhibiting the overactive LRRK2 kinase pathway, NEU-411 aims to address an underlying cause of disease progression in people with LRRK2-driven PD, offering a more precise and potentially more effective approach compared to existing treatment options that only address some symptoms of PD.
About Parkinson’s Disease
Parkinson’s disease (PD) is a brain disorder that causes abnormal movements, such as shaking, stiffness and difficulty with balance and coordination. Symptoms usually begin gradually and worsen over time. As the disease progresses, people may have difficulty walking and talking. Additional symptoms can include mental and behavioral changes, such as sleep problems, depression, memory difficulties and fatigue.
Some cases of PD appear to be hereditary, and a few cases can be traced to specific genetic mutations. Currently, there is no cure or therapy that impacts underlying disease progression available for PD, and treatment options are only used to alleviate some symptoms.
About Neuron23
Neuron23 Inc. is a clinical-stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases. Neuron23 combines recent advances in human genetics with a state-of-the-art drug discovery and biomarker platform using advanced techniques in machine learning and artificial intelligence to advance therapeutics for devastating diseases. The Company’s focus areas are neurodegenerative diseases, neuroinflammatory diseases, and systemic autoimmune and inflammatory diseases. Founded in 2018, Neuron23 has assembled a world-class team of experts and entrepreneurs located in South San Francisco, CA. For more information, please visit www.neuron23.com, or follow us on LinkedIn and X (formerly Twitter).
Contact:
Josie Butler
1AB
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