Company: DAWN
Filing Date: 2025-02-25
Form Type: 10-K
Source: 0000950170-25-026654
Chunk: 80

Company: Day One Biopharmaceuticals, Inc.
Filing Date: 2025-02-25
Form: 10-K
Item: Item 1
Chunk 80
---
 2.1 per 100,000 in the United States. Given the incidence of this disease, our team recognized the market opportunity for developing tovorafenib in this patient population based on the following rationale:

•Potential for tovorafenib, a type-II RAF inhibitor, to be a high-impact targeted therapeutic in pLGG where approximately 70% of tumors are BRAF-altered pLGG in the United States.

•Premium reimbursement precedents for high impact therapeutics in rare diseases, oncology and pediatrics.

•Chronic duration of treatment required over many years to address these slow-growing and relentless tumors.

•High unmet medical need with limited current treatment alternatives for patients.

•Strong value proposition for physicians, patients and families.

We believe tovorafenib, if approved, could become the standard of care for the treatment of pLGG. Due to the need for chronic administration, potentially over many years, the standard of care should be an effective, long-term therapeutic while providing a tolerability profile that minimizes long-term morbidity and treatment-associated toxicity. We believe that tovorafenib has the potential to provide long-term benefit—similar to effective therapies for more traditional chronic rare diseases—to patients with pLGG. We also believe that tovorafenib’s oral, once-weekly dosing regimen would appeal to physicians, patients and their parents.

Investigator-initiated trials of tovorafenib

We intend to leverage our relationships with academic investigators and pediatric oncology cooperative groups and consortia to explore the potential for tovorafenib in other rare pediatric tumor types. The following investigator-sponsored trials are currently open to enrollment as of January 2025:

A Phase 2 trial in relapsed Langerhans cell histiocytosis was initiated in March 2022 by the Children’s Oncology Group, a National Cancer Institute-supported clinical trials group and the world’s largest organization 

14

devoted exclusively to childhood and adolescent cancer research. This study is part of a Written Request issued by the FDA, and is planned as a single-arm non-randomized trial which has an estimated enrollment of 56 participants, who will be administered tovorafenib to determine overall response rate for children and young adults with recurrent or progressive Langerhans cell histiocytosis.

A second Phase 2 trial in craniopharyngioma was initiated in July 2022 by the Pacific Pediatric Neuro-Oncology Consortium. This study was originally designed as a randomized multi-arm trial, where patients