DAWN - Day One Reports Positive Initial Data from Pivotal FIREFLY-1 Trial of Tovorafenib in Relapsed Pediatric Glioma

Day One Biopharmaceuticals (NASDAQ: DAWN) has announced positive initial data for the first 22 Response Assessment for Neuro-Oncology evaluable patients enrolled in the on going phase 2 FIREFLY-1 clinical trial.

Initial data from the first 25 patients enrolled in the trial demonstrate:

64% ORR and 91% CBR (partial response/unconfirmed partial response + stable disease) in the 22 RANO-evaluable patients:

14 partial responses (13 confirmed responses and 1 unconfirmed response)

6 patients with stable disease

All patients with stable disease (n=6) were noted to have tumor shrinkage, ranging between 19% and 43%

Responses were observed in patients with both BRAF fusions and BRAF V600E mutations who received prior MAPK-targeted therapy

The median-time-to-response was 2.8 months

A heavily-pretreated population, with a median of 3 prior lines of therapy (range: 1-9)

All patients who responded remain on therapy (n=14) and no patients have discontinued treatment due to treatment-related adverse events.

“These initial findings underscore the potential of tovorafenib monotherapy to become a significant and transformative new option for relapsed/progressive pLGG, a pediatric brain tumor with no approved treatments today,” said Samuel Blackman, M.D., Ph.D., co-founder and chief medical officer of Day One. “With the registrational cohort fully enrolled, patient follow-up is ongoing, and we look forward to the topline data from the complete study population in the first quarter of 2023. Based on these positive initial data, we plan to begin the pivotal Phase 3 FIREFLY-2 clinical trial evaluating tovorafenib as a front-line therapy in pLGG to evaluate whether tovorafenib can provide benefit early in the disease development.”

“These initial data demonstrate significant anti-tumor activity in children with relapsed/progressive pLGG, including children who are refractory to available therapies. Pediatric low-grade glioma is a truly challenging disease in which children face years of aggressive regimens that can carry a long-term impact on learning, cognition, and quality of life,” said Roger Packer, M.D., senior vice president, Center for Neuroscience and Behavioral Medicine, and director, Brain Tumor Institute, Children’s National Hospital.

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