Our lead compound OT-003
Our lead asset OT-003, a proprietary small molecule activating the TrkB receptor, shows compelling results across a range of cellular and in vivo models for CNS diseases. We are planning our regulatory submission for Phase1 studies in 2025. In addition, we are developing a pipeline of next-generation TrkB activators to treat a wide range of neurological and mental health disorders.
By 2050 the global patient population with Central Nervous System (CNS) disorders will experience significant growth, adding to the huge unmet medical need of today.
Of schizophrenia patients are treatment resistant
People suffer from dementia globally
Approved disease modifying therapies for Parkinson's Disease
Do not fully recover from Major Depressive Disorder on antidepressant medication
Problem
Multiple disease hallmarks underlie CNS disorders including neuronal loss, synaptic and neuronal dysfunction, pathological protein aggregation, inflammation and mitochondrial dysfunction. These processes drive progressive loss of neurons, leading to impairment in memory, cognition, behavior and motor function.
Target
Our most advanced R&D program focuses on the TrkB receptor pathway. Impaired TrkB signaling is implicated in a range of neurological and psychiatric conditions. Activation of the TrkB receptor activates downstream signaling pathways enhancing neuronal survival, promoting neuronal and synaptic plasticity, reducing neuroinflammation and pathological protein aggregates including α-synuclein, amyloid-β and tau. In addition, TrkB activation leads to an improvement in mitochondrial function and an increase in BDNF production, the endogenous ligand for the TrkB receptor.
Compound
Our lead compound, OT-003, binds to the TrkB receptor, leading to activation of the downstream signaling pathways and promoting the above effects in both cellular and in vivo experiments. This pharmacology ultimately leads to significant improvements in memory, cognition and motor function as shown in a range of in vivo studies.
To further complement our lead molecule, we are developing a portfolio of TrkB-activating compounds, to treat a wide range of neurological disorders with high unmet medical need, and which can have a transformative impact on patients and their families.
