Tovorafenib exhibits ‘encouraging’ activity in pediatric low-grade glioma
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Tovorafenib monotherapy induced response among approximately two-thirds of patients with recurrent or progressive pediatric low-grade glioma, according to topline data released by the agent’s manufacturer.
Tovorafenib (DAY101, Day One Biopharmaceuticals) is an investigational, brain-penetrant, highly selective type II pan-RAF kinase inhibitor that targets a key enzyme in the MAPK signaling pathway.
The open-label phase 2 FIREFLY-1 trial evaluated the efficacy of once-weekly tovorafenib monotherapy for patients aged 6 months to 25 years with recurrent or progressive pediatric low-grade glioma.
Most (86%) trial participants had BRAF fusion alternations, for which no systemic therapies are approved. The other 14% of trial participants had BRAF mutations.
Overall response rate served as the primary endpoint.
Analyses of 69 evaluable patients showed a 64% ORR and a 91% clinical benefit rate.
Researchers reported a median therapy duration of 8.4 months, with 77% of patients remaining on treatment at data cutoff.
The most common adverse events reported in the trial included change in hair color (75%), increased creatine phosphokinase (64%), anemia (46%), fatigue (42%) and maculopapular rash (42%).
Complete data from the trial will be presented at a medical meeting.
“The responses we’ve observed in the FIREFLY-1 study with weekly monotherapy tovorafenib in children with recurrent or progressive low-grade gliomas are very encouraging,” Samuel Blackman, MD, PhD, co-founder and chief medical officer of Day One Biopharmaceuticals, said in a company-issued press release. “As tovorafenib progresses in the clinic, we want to thank the patients, their families, the clinical investigators and the advocates who have chosen to participate in the FIREFLY-1 clinical trial and support the development of a potential new treatment for children in need of new therapeutic options.”
Day One Biopharmaceuticals intends to expand development of tovorafenib as a front-line therapy for patients with newly diagnosed pediatric low-grade glioma through the phase 3 FIREFLY-2/LOGGIC clinical trial. The trial will evaluate once-weekly monotherapy tovorafenib for patients with known activating RAF alterations.
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