Our Pipeline
New Breakthroughs Require New Methods
We leverage our strengths in chemistry, genetic target selection and validation, and AI-enabled drug discovery to uncover promising new ways to fight disease.
| Program | Target | Therapeutic Area | Discovery | Lead Optimization | Phase 1 | Phase 2 |
|---|---|---|---|---|---|---|
| NEU-411 | LRRK2 | Parkinson’s Disease | ||||
| NEU-111 | Systemic TYK2 | Systemic Inflammation | ||||
| NEU-627 | Central TYK2 | Neuroinflammation, MS | ||||
| Undisclosed | Target 4 | Not disclosed | ||||
| Undisclosed | Target 5 | Not disclosed |
About LRRK2
Leucine-rich repeat kinase 2 (LRRK2) is a large, multidomain protein that plays a role in many cellular functions including vesicular trafficking, autophagy, lysosomal and mitochondrial function, neurotransmission and immune and microglial responses.
Mutations in the LRRK2 gene are among the most common genetic causes of Parkinson’s disease (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 overactivity 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 believed 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.
Mutations in the LRRK2 gene are among the most common genetic causes of Parkinson’s disease (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 overactivity 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 believed 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.
Our Programs
NEU-411
NEU-411is a brain-penetrant, potent and selective inhibitor of LRRK2 currently being evaluated in the global Phase 2 NEULARK study in people with early Parkinson’s disease. Learn more
By specifically inhibiting the overactive LRRK2 kinase, 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.
The NEULARK study will also utilize an investigational clinical trial assay developed in collaboration with QIAGEN to select people with LRRK2-driven PD for the trial, representing the first precision medicine approach for people with PD.
NEU-411
NEU-411is a brain-penetrant, potent and selective inhibitor of LRRK2 currently being evaluated in the global Phase 2 NEULARK study in people with early Parkinson’s disease. Learn more
By specifically inhibiting the overactive LRRK2 kinase, 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.
The NEULARK study will also utilize an investigational clinical trial assay developed in collaboration with QIAGEN to select people with LRRK2-driven PD for the trial, representing the first precision medicine approach for people with PD.
About TYK2
Tyrosine kinase 2 (TYK2), a non-receptor intracellular signaling kinase, mediates signaling pathways that are critical in the cycle of chronic inflammation in immune-mediated diseases.
TYK2 is implicated in numerous peripheral autoimmune and CNS disorders with significant unmet medical need. Loss of function mutations in TYK2 are protective for CNS disorders such as multiple sclerosis and peripheral autoimmune disorders such as psoriasis, AS and UC. Neuron23 is developing both CNS-penetrant and peripherally restricted TYK2 inhibitors to address both neurological autoimmune and peripheral autoimmune disorders.
TYK2 is implicated in numerous peripheral autoimmune and CNS disorders with significant unmet medical need. Loss of function mutations in TYK2 are protective for CNS disorders such as multiple sclerosis and peripheral autoimmune disorders such as psoriasis, AS and UC. Neuron23 is developing both CNS-penetrant and peripherally restricted TYK2 inhibitors to address both neurological autoimmune and peripheral autoimmune disorders.
Our Programs
NEU-627
NEU-627 is a highly potent and selective central TYK2 inhibitor currently in preclinical development for multiple sclerosis and potentially other neuroinflammatory diseases.
NEU-111
NEU-111 is a peripherally restricted TYK2 inhibitor currently being evaluated in a Phase 1 study in healthy volunteers. Learn more
NEU-627
NEU-627 is a highly potent and selective central TYK2 inhibitor currently in preclinical development for multiple sclerosis and potentially other neuroinflammatory diseases.
NEU-111
NEU-111 is a peripherally restricted TYK2 inhibitor currently being evaluated in a Phase 1 study in healthy volunteers. Learn more
