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Day One Biopharmaceuticals Begins Pivotal Pan-RAF Inhibitor Trial in Pediatric Brain Cancer Patients

NEW YORK – Day One Biopharmaceuticals said on Tuesday said that it has dosed the first pediatric, low-grade glioma patient in its registration-directed trial of the pan-RAF kinase inhibitor, DAY101.

Specifically, the Phase II trial, dubbed FIREFLY-1, is designed to evaluate DAY101 as a treatment for roughly 60 pediatric or young adult patients with recurrent or progressive low-grade gliomas harboring known BRAF alterations. The primary aim of the trial, for which Day One is collaborating with the Pacific Pediatric Neuro-Oncology Consortium, is to assess patients' overall response rates.

Secondary endpoints include progression-free survival, duration of response, and the drug's safety and tolerability, among other measures. Investigators will also evaluate the concordance between centralized BRAF molecular profiling and patients' prior BRAF testing results.

According to Day One, prior studies have demonstrated DAY101's anti-tumor activity and tolerability. FIREFLY-1 follows in the wake of a Phase I trial, dubbed PNOC014, in which nine patients with pediatric, low-grade glioma received DAY101 monotherapy.

"Studies have demonstrated that DAY101 has high brain distribution and exposure, thus potentially benefiting patients with brain tumors," Samuel Blackman, Day One's cofounder and chief medical officer said in a statement. "We believe DAY101 has the potential to become an important treatment advance for pediatric patients with RAF-altered low-grade gliomas."

Later this year, Day One also plans to evaluate DAY101 combined with a MEK inhibitor that it recently in-licensed from MERK KGaA as a treatment for solid tumors harboring MAPK pathway alterations.

Commencement of the pediatric glioma trial follows an announcement in February that Day One had raised $130 million in a Series B funding to put toward DAY101's commercial launch.