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FIREFLY-1 is a pivotal Phase 2, multicenter, open-label study designed to evaluate the safety and efficacy of tovorafenib in patients aged 6 months to 25 years with relapsed or progressive low-grade glioma harboring a known activating BRAF alteration. The study aims to enroll approximately 60 patients. The primary endpoint will be overall response rate (ORR), defined as the proportion of patients with best overall confirmed response rate based upon Response Assessment for Neuro-Oncology (RANO) criteria. Secondary and exploratory endpoints include the overall response rate based on Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria and volumetric analyses, event free survival, safety, functional outcomes, and quality of life measures.

Additional information about FIREFLY-1 may be found at ClinicalTrials.gov, using Identifier NCT04775485.

FIREFLY-2 is a phase 3, randomized, multicenter, open-label study to evaluate the efficacy, safety, and tolerability of monotherapy tovorafenib, an investigational, oral, pan-RAF inhibitor versus standard of care (SoC) chemotherapy in pediatric patients with RAF-altered low-grade glioma requiring front-line systemic therapy. The primary endpoint will be overall response rate (ORR) based upon Response Assessment for Neuro-Oncology (RANO) criteria. Secondary endpoints include progression-free survival (PFS) by independent review committee (IRC) per RANO-LGG, duration of response (DOR) assessed by IRC per RANO-LGG, and overall survival.

Additional information about FIREFLY-2 may be found at ClinicalTrials.gov, using Identifier NCT05566795.

FIRELIGHT-1 is a multi-center, open-label umbrella master study designed to evaluate the safety and efficacy of tovorafenib in patients with recurrent or progressive solid tumors with activating RAF alterations. This study will accrue adults as well as adolescent patients as young as 12 years of age. In the Phase 2 monotherapy portion (sub-study 1), patients will be enrolled either into a melanoma cohort or a “tissue agnostic” cohort. The study also includes a Phase 1b combination (sub-study 2) portion of tovorafenib with the Day One’s investigational oral MEK inhibitor, pimasertib. The primary endpoint will be overall response rate (ORR) as assessed by RECIST version 1.1 criteria. Secondary and exploratory endpoints include safety, duration of response, progression-free survival, and time to response.

Additional information about FIRELIGHT-1 may be found at ClinicalTrials.gov, using Identifier NCT04985604.