Day One Biopharmaceuticals has concluded patient enrolment in the Phase III FIREFLY-2 trial assessing tovorafenib for paediatric low-grade glioma (pLGG).
The randomised, global, open-label, multi-centre trial is evaluating the safety, efficacy, and tolerability of tovorafenib, compared to standard of care chemotherapy, as a first-line therapy for patients aged six months to 25 years with low-grade glioma.
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It is a collaborative effort involving the European Society for Paediatric Oncology (SIOPe) Brain Tumor Group LOGGIC Consortium.
Also called Ojemda, tovorafenib is currently approved for treating patients six months and older with relapsed or refractory pLGG with b-raf proto-oncogene, serine/threonine kinase (BRAF) fusion, rearrangement, or V600 mutation.
The trial will explore oral tovorafenib monotherapy for patients with RAF-altered pLGG who require initial systemic therapy.
It is being carried out at 140 sites across Australia, Asia, Canada, Europe, the Middle East, South America, and the US.
In more than three years, the study has enrolled around 400 participants, randomising them to receive either tovorafenib weekly or one of four standard chemotherapy regimens.
The primary endpoint is overall response rate, including duration of response per Response Assessment in Paediatric Neuro-Oncology Low-Grade Glioma (RAPNO-LGG) criteria.
Key secondary endpoints comprise progression-free survival, event-free survival, time to next treatment, overall survival, and patient-reported outcomes.
Preliminary insights from the Phase III trial are anticipated to be available in 2027.
Day One chief medical officer Elly Barry said: “Reaching full enrolment 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.
“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 tumour afflicting children.”
