Our drug development approach focuses on identifying and advancing small molecules designed to achieve clinically translatable endpoints in neurodegenerative diseases. We believe that the well-established paradigm of small molecule development has the potential to be a straightforward treatment modality for neuroregeneration that is capable of providing a meaningful restorative clinical benefit to patients.
Our approach involves three components:
- Focusing on neural pathways that allow for the reactivation of innate repair mechanisms in order to achieve neuroregeneration;
- Prioritizing objective clinical outcomes, rather than patient-reported measures, and trial designs to facilitate proof-of-concept early in clinical development; and
- Selecting neurodegenerative disease preclinical models with functional outcomes that we believe are reflective of appropriate measures used to assess clinical disease responses in patients.