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PBTF-Funded Therapy for Pediatric Brain Cancer Shows Promise in Phase 1 Trial; Day One Biopharmaceuticals Announces New Phase 2 Study and FDA Designation

11/19/20

The Pediatric Brain Tumor Foundation (PBTF), the nonprofit leader dedicated to children battling brain tumors, joins the research community in congratulating Day One Biopharmaceuticals, Dana-Farber Cancer Institute, and the Pacific Pediatric Neuro-Oncology Consortium (PNOC) on the preliminary results of PNOC014, an ongoing Phase 1 study of DAY101 in children with radiographically recurrent/progressive low-grade gliomas and other MAP kinase pathway activated tumors.

DAY101, an oral, once-weekly CNS penetrant inhibitor of BRAF and CRAF fusions and mutations, received critical seed funding from the PLGA Fund at PBTF through its early development and Phase 1 trial stage. 

"There is no cure without collaboration. In partnership with families and supporters across the country, the Pediatric Brain Tumor Foundation has had a hand in funding the game-changing science that resulted in the discovery of DAY101 as a targeted therapy for children with brain cancer. We are thrilled that our grassroots efforts have led to such a promising clinical trial, and we are encouraged by Day One’s commitment to apply the industrial horsepower needed to take this treatment to the next level for the children we serve,” says Courtney Davies, PBTF’s President and CEO.

Preliminary results from PNOC014 were presented at the Society for Neuro-Oncology’s 25th Annual Scientific Meeting and Education Day, held virtually November 19-22, 2020.

“We found in an independent analysis of the nine patients treated in the ongoing Phase 1 study that the majority had responses or stable disease based upon Response Assessment for Neuro-Oncology (RANO) criteria,” says Dr. Karen D. Wright, Senior Physician at Dana-Farber, Assistant Professor of Pediatrics at Harvard Medical School, and principal investigator and author of the Phase 1 study.

Because of these results and additional clinical data from the DAY101 development program, the US Food and Drug Administration (FDA) has granted DAY101 a Breakthrough Therapy Designation (BTD) for the treatment of pediatric patients with advanced low-grade glioma harboring RAF alteration and Orphan Drug Designation for the treatment of malignant glioma.

Additionally, noted Dr. Jeremy Bender, CEO of Day One Biopharmaceuticals, in today's announcement, “Day One is launching a global Phase 2 monotherapy trial concurrent with the ongoing Phase 1 trial, evaluating the efficacy and safety of DAY101 administered orally, once-a-week in pediatric patients with relapsed or progressive low-grade glioma harboring a BRAF-alteration. The study will be conducted in collaboration with PNOC, is called FIREFLY-1 and PNOC026, and is intended to support DAY101 registration.”

More children are diagnosed with a low-grade glioma than any other type of brain cancer. Although the survival rate is higher than other brain tumor types, children who do survive face a lifetime of physical, cognitive and psychological side effects. Clinical trials of DAY101 and other targeted therapies offer families a promising alternative to the aggressive, toxic chemotherapies that have been the “gold standard” since the 1970s. 

“Children battling brain tumors have waited too long for the scientific breakthroughs other childhood cancer patients have experienced. While the Pediatric Brain Tumor Foundation celebrates this incredible research milestone, we know that our work is far from done,” says Davies. “The partnership between PBTF, Dana-Farber’s scientists, and Day One that led to Day101 offers a model of success that can be duplicated as we fund research to accelerate the number of targeted therapies for children battling brain tumors today.”

In addition to PBTF’s support, the development of DAY101 would not have been possible without the support of the Team Jack FoundationThea’s Star of Hope, and Why Not Me? organizations, as well as Geared Up For KidsStarry Night KnoxvillePar For Kids and Think Fit For Kids fundraising events. The PNOC014 trial is supported by the PLGA Fund at the Pediatric Brain Tumor Foundation, Team Jack Foundation, Team Nathan, Day One, the Takeda Pharmaceutical Company, Dana-Farber Cancer Institute (DFCI), and the National Cancer Institute of the National Institutes of Health under award number P50 CA 165962 as part of the prestigious SPORE grant for Targeted Therapies in Gliomas awarded to Dana-Farber and Brigham and Women’s Hospital under Drs. Tracey Batchelor and Mario Suva and for which Dr. Wright is co-Principal Investigator on project 1 with co-collaborators Dr. Michael Eck and Dr. Daphne Haas-Kogan.

About the Pediatric Brain Tumor Foundation
Every day, 13 children and teens are diagnosed with a brain tumor, the deadliest form of childhood cancer. Every day after, they are in a fight for their life. It is a fight the Pediatric Brain Tumor Foundation is here to help families win. A leader in the brain tumor and childhood cancer communities, PBTF’s mission of Care. Cure. Thrive. reflects its commitment to curing all pediatric brain tumors and transforming how children and their families are cared for. Since 1991, PBTF has provided strategic leadership and funding to accelerate the number of targeted therapies for children battling brain tumors today, while equipping families with the educational resources, financial relief and emotional support they need to navigate their child’s journey. A world without childhood brain tumors is possible when we stand together to effect real, meaningful change. Learn more at www.curethekids.org.

About Day One Biopharmaceuticals
Day One Biopharmaceuticals is committed to leading the development of promising new therapies in children and to improving the standard of care for patients of all ages living with cancer. Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop emerging cancer treatments that can help both children and adults living with cancer. The company raised a $62M series A financing from Canaan, Access Biotechnology, and Atlas Venture. Through Day One and its collaborators, cancer drug development comes of age. For more information, please visit www.dayonebio.com.

About the Pacific Pediatric Neuro-Oncology Consortium
The Pacific Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with study sites within the United States, Canada, Europe and Australia dedicated to bringing new therapies to children and young adults with brain tumors.