Our Approach
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.
We are pioneering the development and commercialization of first-in-class therapies for precision neuroregeneration including myelin restoration, synaptogenesis and axonal repair. The Company has a broad pipeline of programs to address multiple CNS disorders. Our flagship program, PIPE-307, has completed two Phase 1 clinical trials – a SAD/MAD study and a PET study – in healthy volunteers, and has received Investigational New Drug clearance from the Food and Drug Administration to initiate clinical development in relapsing-remitting MS patients. We have also entered into a global license and development agreement with Janssen to develop PIPE-307 for nervous system disorders.
