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Monday, January 9, 2023

Day One: Pediatric Brain Cancer Candidate Shows Encouraging Responses

 

  • Day One Biopharmaceuticals  announced topline results from the ongoing Phase 2 FIREFLY-1 trial evaluating tovorafenib (DAY101) as a monotherapy in recurrent or progressive pediatric low-grade glioma (pLGG). 
  • Pediatric low-grade glioma is the most common brain tumor diagnosed in children.
  • Additional data will be submitted for presentation at an upcoming medical meeting in the second quarter of 2023.
  • Overall response rate (ORR) of 64% and clinical benefit rate (CBR) of 91% were observed in 69 heavily-pretreated, RANO-evaluable patients.
  • Based on 77 treated patients, safety data indicated monotherapy tovorafenib to be generally well-tolerated. 
  • The most common side effects reported related to tovorafenib were change in hair color (75%), increased creatine phosphokinase (64%), anemia (46%), fatigue (42%), and maculopapular rash (42%).
  • In addition to FIREFLY-1, Day One is expanding the development of tovorafenib as a front-line therapy for patients newly diagnosed with pLGG. 
  • The global, Phase 3, registrational FIREFLY-2/LOGGIC clinical trial is evaluating once-weekly monotherapy tovorafenib in newly-diagnosed patients with pLGG harboring a known activating RAF alteration.

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