Somewhat of a comparison here - with this market cap A$1.4 billion company - called ONE DAY - who is approaching the FDA for marketing approval, following a Phase 2 study in a type of childrens brain cancer, with BRAF mutations.
Of significance,
this 69 patient, phase 2 study was also undertaken by the Pacific Pediatric Neuro-Oncology Consortium (PNOC) - same group who are conducting the paxalisib DIPG combo study, in over 300 kids.Self-explanatory - except really there is no comparison in actual fact, I would say
- as this study is ONE DAY s primary value driver - whereas paxalisib may well prove, to be only a small portion of Kazia revenues. (and also of course
$1.4 billion verse $36million)
Day One Announces Topline Data from Pivotal Phase 2 FIREFLY-1 Trial Demonstrating Meaningful Responses with Tovorafenib (DAY101) in Recurrent or Progressive Pediatric Low-Grade GliomaJanuary 08, 2023 17:00 ET| Source:
Day One Biopharmaceuticals, - Overall response rate (ORR) of 64% and clinical benefit rate (CBR) of 91% in 69 heavily-pretreated, RANO-evaluable patients
- Median duration of 8.4 months on therapy as of September 28, 2022, with 77% of patients remaining on treatment
- Additional data to be presented at a medical meeting in the second quarter of 2023
- New Drug Application submission planned for first half of 2023
SOUTH SAN FRANCISCO, Calif., Jan. 08, 2023 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced positive topline results from the ongoing, open-label, pivotal Phase 2 FIREFLY-1 trial evaluating the investigational agent, 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 and for which there is no standard of care, and for which there are no approved therapies for the majority of patients. Additional data will be submitted for presentation at an upcoming medical meeting in the second quarter of 2023.
The primary endpoint of the FIREFLY-1 trial is overall response rate (ORR) by Response Assessment for Neuro-Oncology (RANO) criteria as assessed by blinded independent central review. Topline results as of September 28, 2022 include:
Among 69 RANO-evaluable patients:
- 64% ORR and 91% clinical benefit rate (complete response + partial response/unconfirmed partial response + stable disease)
- 4% (n=3) confirmed complete responses
- 59% (n=41) partial responses (31 confirmed and 10 unconfirmed)
- 28% (n=19) patients with stable disease
- 86% (n=59) of patients had a BRAF fusion alteration, for which there are no approved systemic therapies, while the remaining 14% (n=10) had a BRAF mutation
Safety data, based on 77 treated patients, 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%).
About the Pacific Pediatric Neuro-Oncology ConsortiumThe 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.
