First And Only FDA-Approved Type Ii Raf Inhibitor For Patients With Relapsed Or Refractory Plgg Harboring A Braf Fusion Or Rearrangement, Or Braf V600 Mutation Made Possible Through PNOC Clinical Trial 

FOLLOWING SUCCESSFUL FIREFLY-1 TRIAL IN PARTNERSHIP WITH THE PNOC SCIENTIFIC CONSORTIUM AND DAY ONE BIOPHARMACEUTICALS

SAN RAFAEL, Calif., April 24, 2024 /PRNewswire/ -- The Pediatric Neuro-Oncology Consortium Foundation (PNOC Foundation), a platinum rated non-profit dedicated to supporting advancements in childhood brain cancer research and clinical trials, announces the FDA approval of Tovorafenib (DAY101), a significant advancement in the fight against pediatric low-grade glioma brain tumors. This approval is a direct result of the pivotal Phase 2 FIREFLY-1 trial/PNOC026, which demonstrated meaningful responses and a favorable safety profile, marking a promising development in pediatric neuro-oncology. This trial built on the initial PNOC phase 1 trial, PNOC014.

This approval marks a significant milestone as the first and only FDA-approved medicine for children with BRAF fusions or rearrangements in pediatric low-grade glioma. Before Tovorafenib, there was no standard of care for these children.

FIREFLY-1/PNOC026 underscored the effectiveness of Tovorafenib (DAY101). This breakthrough therapy offers a great alternative therapy option for children with low grade gliomas.

Dr Sabine Mueller, PNOC's scientific co-founder said "PNOC is proud of our role in this collaborative achievement and remains committed to advancing research, enhancing treatment options, and improving the lives of children affected by brain tumors. We extend our heartfelt gratitude to all the patients, families, researchers, and healthcare professionals who contributed to the success of PNOC014 and FIREFLY-1/PNOC026 and the development of Tovorafenib (DAY101)."

"This is a landmark development for pediatric brain cancer research and treatment," said PNOC Foundation co-founder and patient parent Bruce Campbell. "It shows what's possible when doctors, scientists, researchers, biopharma, and philanthropists truly collaborate and work together. This is a victory for kids and their families fighting pediatric low-grade gliomas, however, there is still a huge unmet need. More courage to invest in and fund new therapies and trials is needed to bring further breakthroughs to other types of pediatric brain cancer, the deadliest of childhood cancers."

"Seeing the promise of PNOC realized with advancements like Tovorafenib confirms the crucial role of our foundation," stated Allyn Campbell, co-founder of the PNOC Foundation. "This milestone, made possible by our community's support and the generosity of our donors, empowers us to continue our quest to revolutionize pediatric neuro-oncology and improve the lives of children affected by brain tumors."

For more information contact: media@pnocfoundation.org 

To View the DayOne Press Release, Click HERE.

To View the FDA Press Release, Click HERE.

To learn more about the PNOC Foundation please visit www.pnocfoundation.org.

About the PNOC Foundation:

The PNOC Foundation is a non-profit dedicated to supporting the advancements in childhood brain cancer research. The PNOC Foundation's vision is to lose no child to brain cancer, and to improve the quality of life for those fighting and surviving pediatric brain cancer. The PNOC Foundation was founded to support the work of Pacific Pediatric Neuro-Oncology Consortium (PNOC), formed in 2012 at UCSF, to provide access to personalized treatment strategies and ultimately improve overall outcomes for children with brain cancer and tumors.

Since its inception, PNOC has pushed beyond older paradigms and historic trial designs, expanding nationally and globally to accelerate clinical trial completion and find cures faster. Through the cooperative open-source network, PNOC is fostering worldwide collaboration with the world's leading pediatric brain tumor specialists. In the pursuit of enhanced clinical trial development and treatments for CNS cancer. PNOC has grown from a 7-member group along the West Coast to a global consortium with 43 participating sites, funded largely by philanthropy.

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SOURCE PNOC. Foundation