On May 8, 2026 Day One Biopharmaceuticals, Inc., now part of Servier Group, reported that it has completed enrollment in the FIREFLY-2 clinical trial, which is evaluating the efficacy, safety and tolerability of tovorafenib vs standard of care chemotherapy in the front-line setting of therapy for patients aged 6 months to 25 years with low-grade glioma. The trial is being conducted in collaboration with the European Society for Paediatric Oncology (SIOPe) Brain Tumor Group LOGGIC Consortium (LOGGIC). Tovorafenib, known as OJEMDATM, is currently indicated for treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
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"Reaching full enrollment in this trial is a critical step toward our goal of establishing OJEMDA as standard of care across all lines of therapy for individuals with BRAF-altered pLGG. By moving earlier in the treatment journey, we aim to intervene when we can have the greatest impact on the burden of this challenging cancer," said Elly Barry, M.D., Chief Medical Officer at Day One, now part of Servier Group. "Success in this study would not only further validate the efficacy and safety profile of OJEMDA, but also fundamentally evolve the pLGG treatment paradigm, and potentially establish a new standard of care for patients newly diagnosed with pLGG, the most common brain tumor afflicting children."
FIREFLY-2 is a Phase 3, global, randomized, multicenter, open-label study to evaluate the efficacy, safety, and tolerability of monotherapy tovorafenib, an oral, Type II RAF inhibitor, in patients ages 6 months to 25 years with RAF-altered pLGG requiring first-line systemic therapy. It is being conducted at approximately 140 sites across the U.S., Canada, Europe, Australia, South America, Middle East and Asia, and in just over three years has enrolled approximately 400 participants who are receiving either tovorafenib once weekly or one of four standard of care (SoC) chemotherapy regimens.
"Completing enrollment in FIREFLY-2 is a powerful early signal of momentum. It reflects what’s possible when we bring together deep scientific expertise, a patient-first culture, and the scale to execute globally," said David K. Lee, CEO of Servier Pharmaceuticals. "So soon after Servier’s acquisition of Day One, this milestone reinforces our conviction that joining forces was the right decision for patients and for our oncology strategy. As we aim to accelerate the development of targeted medicines and advance them with the rigor, speed and reach needed to make a meaningful difference, we hope this study helps unlock the potential to improve outcomes for children living with pediatric low-grade glioma."
The primary endpoint of the trial is overall response rate (ORR), including duration of response (DOR), based upon Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO-LGG) criteria, with key secondary endpoints including progression-free survival (PFS) and event-free survival (EFS) per RAPNO-LGG, time to next treatment (TTNT), overall survival (OS) and other measures, including patient-reported outcomes. The primary analysis is expected to occur approximately 12 months after the last patient is enrolled; preliminary insights are expected to be available in 2027. More information on the trial can be found at The FIREFLY-2 Trial – Day One Clinical Trials or View Source
About tovorafenib
Tovorafenib (known as OJEMDATM in the U.S.) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. Tovorafenib is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based, in part, on response rate and duration of response according to multiple response assessment criteria: Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO LGG) criteria, and Response Assessment for Neuro-Oncology Low-Grade Glioma (RANO LGG) criteria. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.
Please see full prescribing information including important safety information about OJEMDA at www.OJEMDA.com.
About Pediatric Low-Grade Glioma
Pediatric low-grade gliomas (pLGG) are the most common brain tumor with an estimated US incidence of 1,100 and Europe incidence of 700 children per year who are eligible for front-line systemic therapy.i, ii BRAF is the gene most commonly altered in pLGG, which includes two primary types of BRAF alterations – a BRAF gene fusion and BRAF point mutation. These BRAF alterations account for >50% of pLGG cases worldwide and prior to the introduction of OJEMDA, there were no approved treatments for people with pLGG driven by BRAF fusions.i, iii
Pediatric low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact their life over the long term, and may include motor deficiencies, vision loss, hormone deficiency and alterations in growth and development. Most children with pLGG will survive their cancer, but for the majority of those in whom a complete surgical resection is not possible, these tumors tend to recur frequently throughout childhood, necessitating multiple treatments. The cumulative toxicity of numerous therapies, along with the damage caused by multiple episodes of tumor progression, take a significant toll on the children and their families.
(Press release, Day One, MAY 8, 2026, View Source [SID1234665394])