Tovorafenib (DAY101) – our lead investigational therapy
Tovorafenib (DAY101) is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor.
Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which may offer an important alternative for people with primary brain tumors or brain metastases of solid tumors.
Tovorafenib has been studied in over 300 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) for the treatment of pediatric low-grade glioma (pLGG). pLGG is the most common form of childhood brain cancer. We have also initiated a Phase 1/2 study (FIRELIGHT-1) with tovorafenib in patients with recurrent or progressive solid tumors with activating RAF alterations.
Tovorafenib has the potential to become an important alternative for people with primary brain tumors or brain metastases of solid tumors.
Additional studies are planned with tovorafenib alone or in combination with other agents that target key signaling nodes in the MAPK pathway, such as our investigational MEK inhibitor pimasertib, in patient populations where RAS and RAF alterations are believed to play an important role in driving disease.
Tovorafenib has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. The FDA has also granted Rare Pediatric Disease Designation to tovorafenib for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. Tovorafenib has also received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA) for the treatment of malignant glioma.