Neuron23, a clinical-stage biotechnology company developing precision medicines for genetically defined neurological diseases, announced the completion of a $96.5 million Series D financing round and the dosing of the first patient in its Phase 2 clinical trial for a novel Parkinson's disease treatment. The funding round was led by a healthcare investor with significant participation from existing investors including Westlake Village BioPartners, SoftBank Vision Fund 2, Redmile Group, Blue Owl, Kleiner Perkins, HBM Healthcare Investments, and Acorn Bioventures.
Precision Medicine Approach for Parkinson's Disease
The company has initiated patient dosing in the global Phase 2 NEULARK clinical trial (NCT06680830) evaluating NEU-411, a brain-penetrant, potent, and selective LRRK2 inhibitor, in individuals with early Parkinson's disease. This represents the first precision medicine approach specifically targeting patients with LRRK2-driven disease.
"The dosing of the first patient in the NEULARK clinical trial marks a pivotal milestone in Parkinson's disease research. For the first time, we are using a precision medicine approach to identify and enroll patients with LRRK2-driven disease," said Dr. Aaron Ellenbogen, a movement disorders neurologist and Medical Director at Quest Research Institute who serves as a NEULARK study investigator.
Trial Design and Digital Innovation
The NEULARK trial is a randomized, double-blind, placebo-controlled study that will enroll approximately 150 participants with early-stage, LRRK2-driven Parkinson's disease. Participants will receive NEU-411 or placebo daily for a 52-week treatment period. The study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of the orally administered drug.
A key innovation in the trial design is the incorporation of digital biomarkers through smartphones equipped with proprietary software developed by Roche Information Solutions (RIS). This technology frequently measures Parkinson's disease symptoms including movement and tremor, as well as non-motor symptoms such as cognition. The primary endpoint is the change from baseline in the Roche digital biomarker score compared to placebo, with secondary measures including the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS).
Genetic Target and Patient Population
Mutations in the LRRK2 gene represent one of the most common genetic causes of Parkinson's disease, affecting approximately 2% of people with the condition. Individuals who inherit gain-of-function mutations in LRRK2 face higher risk of developing Parkinson's disease later in life. However, the therapeutic potential extends beyond familial cases.
Neuron23 has identified single-nucleotide polymorphisms (SNPs) that are predicted to drive LRRK2 overactivity in up to 30% of people with idiopathic Parkinson's disease. This broader population, combined with those carrying LRRK2 gene mutations, collectively represents what the company terms "LRRK2-driven PD" and constitutes the target population most likely to benefit from LRRK2 inhibition.
Patient Identification Partnership
To streamline patient identification and enrollment, Neuron23 has partnered with Sano Genetics. Under a separate protocol, Sano offers saliva test kits that can identify people with LRRK2-driven Parkinson's disease. Individuals identified through this testing will be referred to the nearest NEULARK clinical trial site for complete eligibility evaluation and potential enrollment.
Drug Mechanism and Therapeutic Rationale
NEU-411 is designed as a potent, highly selective, and brain-penetrant oral LRRK2 inhibitor with what the company describes as 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 Parkinson's disease, potentially offering a more precise and effective approach compared to existing treatments that only address symptoms.
"The Series D financing represents further endorsement of Neuron23's industry-leading approach, which leverages state-of-the-art precision medicine and patient identification to deliver NEU-411 to people with Parkinson's disease who are most likely to benefit from LRRK2 inhibition, increasing probability of success in the clinic and bringing the right therapy to the right patients," said Nancy Stagliano, Ph.D., Chief Executive Officer of Neuron23.
The company anticipates topline data from the NEULARK trial in 2027. Currently, no cure or therapy exists that impacts underlying Parkinson's disease progression, with available treatments limited to symptom management.
