Our lead compound OT-003
Our lead asset OT-003, a proprietary small molecule, shows compelling results across a range of cellular and in vivo models for CNS diseases. We plan to initiate Phase 1 clinical studies with OT-003 by 2025 and we are building a pipeline of NCEs to treat a wide range of neurological and neuropsychiatric 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.
People suffer from dementia globally
Approved disease modifying therapies for Parkinson's Disease
Of schizophrenia patients are treatment resistant
Years life expectancy after diagnosis with ALS
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.
