Company: DAWN
Filing Date: 2025-02-25
Form Type: 10-K
Source: 0000950170-25-026654
Chunk: 66

Company: Day One Biopharmaceuticals, Inc.
Filing Date: 2025-02-25
Form: 10-K
Item: Item 1
Chunk 66
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•Establish a leadership position in targeted oncology therapeutics for patients of all ages through our unique expertise in pediatrics.    We have built a targeted oncology company with differentiated business and clinical development capabilities. We leverage these capabilities to navigate the unique challenges and nuances of pediatric and adult drug development. We initially focused on pediatric patients as we believe this provides a favorable pathway to approval for tovorafenib. We have established trusted relationships within the pediatric and adult oncology community, and we seek their advice on aligning our clinical development plans with the needs of the patients and their families. We believe we are a leader in this development space and to further this position, we plan to continue to consult and strategically partner with biopharmaceutical companies, academic oncologists and scientists, and patient advocacy groups to identify areas of unmet need and then acquire high-impact assets to address underserved patients.

•Advance our first commercial product candidate, tovorafenib, through clinical development towards full regulatory approval in pLGG.    We demonstrated clinical proof-of-concept of tovorafenib in pediatric patients for cancers that harbor genetic alterations in RAF. Oral, once-weekly dosed tovorafenib was also well-tolerated in the Phase 1 trial in pLGG, which is important for achieving 

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and maintaining long-term, durable responses in these patients. Further, tovorafenib received FDA breakthrough therapy designation for the treatment of pediatric patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. Tovorafenib also received orphan drug designation from the FDA for the treatment of malignant glioma. We conducted a pivotal Phase 2 FIREFLY-1 trial of tovorafenib as a monotherapy for pediatric patients with relapsed or refractory pLGG harboring an activating BRAF alteration. In April 2024, OJEMDA received accelerated approval from the FDA for the treatment of patients 6 months of age and older with relapsed or refractory pediatric LGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation based on the FDA’s evaluation of the data from FIREFLY-1 as pivotal study, and other supportive data. Results 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. FIREFLY-2 is an ongoing Phase 3