Company: DAWN
Filing Date: 2025-02-25
Form Type: 10-K
Source: 0000950170-25-026654
Chunk: 68

Company: Day One Biopharmaceuticals, Inc.
Filing Date: 2025-02-25
Form: 10-K
Item: Item 1
Chunk 68
---
 FHCT, began between 2006 to 2011 found that of the 185 drugs relevant to the pediatric population, only about a one third were evaluated in the pediatric population, of which 30 ultimately received FDA approval (29 were adult only indications; only one received a pediatric indication within nine years of following evaluation in adults). This analysis found a median lag between FHCT and first-in-child trial, or FCCT, for agents that received FDA approval of 5.6 years. In October 2023, the World Health Organization published an analysis spanning 15 years from 2007 to 2022 concluding that of 440 agents used in 2,519 cancer treatment trials worldwide, approximately 41% have also been approved for use in children. Furthermore, only five have been approved for use exclusively in children. The median time between adult and pediatric approvals in this analysis was approximately three years.

We believe that now is the right time to revisit and correct historic assumptions about pediatric oncology drug development. In doing so, we believe there are unique advantages to developing new oncology products and product candidates in pediatric patients, in parallel with, or even in advance of, adult indications:

•Enriched responder populations.    The generation of large-scale molecular profiling datasets necessary to define addressable subpopulations in pediatric oncology has accelerated over the last decade. This has 

5

allowed scientists and drug developers to identify oncogenic drivers underlying numerous pediatric tumor types, and has revealed druggable oncogenic drivers in nearly 50% of pediatric cancers. Moreover, pediatric tumors are less heterogeneous and genomically more stable compared to highly heterogeneous adult tumors. Directly targeting these mutations may lead to deep and sustained anti-tumor activity, as demonstrated by other targeted oncology products.

•Ability to efficiently advance clinical development.    Recently, global regulatory authorities have established paths for accelerated feedback on the design and execution of clinical trials in pediatrics. As part of the FDA Reauthorization Act, 205 relevant molecular targets were identified for pediatric cancers. In addition, new tumor-specific pediatric oncology consortia and cooperative groups have been established, allowing industry to sponsor pediatric clinical trials in the same manner as adult clinical trials. Further, the potential to achieve proof-of-concept and regulatory approval can be obtained with relatively smaller-sized clinical trials with clear endpoints.

•Regulatory and commercial tailwinds.    The scarcity of approved products or an established standard of care, particularly in relapsed disease in pediatric oncology, provides multiple opportunities to bring new therapeutics to market