Day One Announces Three Year Follow-Up Data From OJEMDA™ (tovorafenib) Phase 2 FIREFLY-1 Trial at the 2025 Society for Neuro-Oncology (SNO) Annual Meeting

Core Insights - The updated three-year results from the FIREFLY-1 trial demonstrate that 77% of patients who entered a treatment-free observation period after OJEMDA treatment remained off therapy for at least 12 months [1][5] - The median time to next treatment (TTNT) following the initiation of OJEMDA exceeded 3.5 years [1][5] - Tovorafenib shows potential as a second-line standard of care for pediatric low-grade glioma (pLGG) [3][4] Trial Results - In the FIREFLY-1 trial, 76 evaluable patients showed an overall response rate of 53% (40/76), with a median duration of response of 19.4 months [3][14] - The median time to response was 5.4 months, and the median progression-free survival was 16.6 months [3][5] - Among 39 patients who entered a treatment-free observation period, the median treatment-free interval was not reached, with minimal tumor rebound observed [5] Safety Profile - No new safety signals were identified in the updated analysis, with common grade 3 or higher adverse events including decreased growth velocity and anemia [6][14] - The most frequently reported adverse reactions (≥30%) included rash, fatigue, and viral infections [14][15] Regulatory Status - Tovorafenib (OJEMDA) is approved by the FDA for pediatric patients aged 6 months and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement [7][8] - The drug has received Breakthrough Therapy and Rare Pediatric Disease designations from the FDA [10] Industry Context - Pediatric low-grade glioma is the most common brain tumor in children, with significant unmet medical needs due to the lack of approved treatments targeting BRAF alterations [11][12] - The FIREFLY-1 trial is part of a broader effort to evaluate tovorafenib in pediatric patients with pLGG, highlighting the importance of targeted therapies in this demographic [13][18]