Company: DAWN
Filing Date: 2025-11-04
Form Type: 10-Q
Source: 0001193125-25-264649
Chunk: 133

Company: Day One Biopharmaceuticals, Inc.
Filing Date: 2025-11-04
Form: 10-Q
Item: Item 2
Chunk 133
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, harboring an activating RAF alteration.

On April 23, 2024, we announced that the FDA approved OJEMDA™ (tovorafenib) for the treatment of patients 6 months of age and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. The indication was approved under accelerated approval based on a regulatory endpoint of overall response rate and the totality of the Company’s pivotal open-label Phase 2 trial, or FIREFLY-1, data, including duration of response. With the approval, we received, and subsequently sold, a rare pediatric disease priority review voucher, or PRV, from the FDA. We have commenced the commercial launch of OJEMDA in the United States. OJEMDA is the only systemic therapy for pLGG that offers once-weekly dosing, with or without food, as a tablet or oral suspension.

The accelerated approval of OJEMDA is based on data from FIREFLY-1, which enrolled a total of 137 relapsed or refractory BRAF-altered pLGG patients across two study arms. Arm 1, which accrued 77 patients, was used for the efficacy analyses. Arm 2 provided additional safety data from an incremental 60 patients and was initiated to enable access to OJEMDA once Arm 1 had fully accrued. Details of this trial were presented in November 2023 at the Society for Neuro-Oncology meeting through two oral plenary presentations and in parallel through a publication in Nature Medicine. 

The approval of OJEMDA was based, in part, on the major efficacy outcome measure of overall response rate, or ORR, defined as the proportion of patients with complete response, partial response, or PR, or minor response, or MR, by independent review based on Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma, or RAPNO LGG.

 In Arm 1, data from the 76 RAPNO LGG evaluable patients include (May 2024 data cutoff to support the ongoing evaluation of OJEMDA): 

•A best ORR of 52.6% (95% CI: 40.8 - 64.2), which included 29% PRs and 11% MRs. 

•The ORR for OJEMDA was 53% among the 64 patients with BRAF fusions or rearrangements and 50