Day One Biopharmaceuticals presented the first publicly available three‑year follow‑up data from its pivotal FIREFLY‑1 trial for OJEMDA (tovorafenib) at the 30th Annual Meeting of the Society for Neuro‑Oncology on November 24, 2025. The presentation showed that 77% of patients who entered the treatment‑free observation period remained off therapy for at least 12 months, and the median time to next treatment was 42.6 months—exceeding 3.5 years. Forty‑four of 76 evaluable patients completed 26 or more cycles of OJEMDA, underscoring the drug’s tolerability and the feasibility of extended therapy.
The safety profile remained consistent with earlier reports. No new safety signals emerged, and grade 3 or higher adverse events were limited to previously observed categories such as decreased growth velocity, anemia, and rash. The durability data confirm that OJEMDA can maintain disease control over long periods, a critical attribute for a second‑line therapy in pediatric low‑grade glioma.
These results reinforce OJEMDA’s position as a durable second‑line treatment and provide a strong evidence base for payer negotiations and prescriber confidence. The data support the company’s raised 2025 net product revenue guidance of $145‑$150 million, up from the prior $140‑$150 million range, and align with the $38.5 million net product revenue reported in Q3 2025—a 15% increase from the previous quarter. By demonstrating long‑term efficacy and manageable safety, Day One strengthens its case for broader market share in the pediatric oncology space.
OJEMDA is the first FDA‑approved targeted therapy for children six months or older with relapsed or refractory low‑grade glioma harboring BRAF alterations, having received accelerated approval in April 2024. The FIREFLY‑1 data are the first to show sustained benefit beyond two years, a milestone that can accelerate adoption and justify the company’s optimistic revenue outlook.
"These three‑year data showed that patients were able to maintain disease control during extended periods off therapy, with the option to reinitiate tovorafenib treatment if clinically indicated," said Dr. Cassie Kline, Director of Clinical Research in the Division of Neuro‑Oncology at the Children’s Hospital of Philadelphia. "This approach has the potential to offer patients and their families meaningful time away from treatment," she added. Dr. Elly Barry, Chief Medical Officer of Day One, noted that the findings "highlight the potential for a treatment approach to help support patients through the long‑term course of their disease and further support our view that tovorafenib has the potential to become the second‑line standard of care in pLGG."
The long‑term durability and safety profile demonstrated by the FIREFLY‑1 trial strengthen Day One’s commercial strategy and validate its guidance for 2025. With a proven ability to keep patients off therapy for extended periods, OJEMDA is well positioned to capture a larger share of the pediatric oncology market and to secure payer coverage that reflects its clinical value.
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