On June 12, 2025 FivepHusion, an advanced clinical-stage biotechnology company, reported the successful completion of the phase 1 (Part A) component of the phase 1/2 Deflexifol at Relapse Trial (DART) proudly supported by the Kids with Cancer Foundation, an Australian-led study of a new brain cancer treatment for children (Press release, FivepHusion, JUN 12, 2025, View Source [SID1234653831]).
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The phase 1/2 DART study is an Australian investigator-initiated trial, led by Principal Investigators Professor David Ziegler and Dr Marion Mateos and sponsored by the Australian and New Zealand Children’s Haematology / Oncology Group (ANZCHOG) in collaboration with FivepHusion. The trial is designed to investigate Deflexifol monotherapy as a treatment for paediatric ependymoma and other childhood brain cancers. All major paediatric oncology centres in Australia are participating in the trial, and major trial funding has been provided by the Kids with Cancer Foundation, through Sydney Children’s Hospitals Foundation, and the Robert Connor Dawes Foundation. An abstract summarising the results of the Phase 1 component has been submitted to the Society of Neuro-Oncology for consideration of presentation at their international meeting in November.
Dr Christian Toouli, CEO and Managing Director of FivepHusion commented, "Completion of Part A of the DART study, and confirmation of a safe and tolerable phase 2 dose, are major milestones in our plans to develop Deflexifol as a potential treatment for paediatric ependymoma and other brain cancers. We thank the patients and their families for their participation in this trial, and our collaborators and partners for conducting this important study."
Deflexifol is an advanced clinical-stage, next-generation co-formulation of 5-fluorouracil (5-FU) and leucovorin (LV), a drug that significantly enhances 5-FU activity. Deflexifol has previously been evaluated in two successfully completed clinical trials in adults with a variety of solid tumours; the DART study is the first clinical evaluation of Deflexifol in paediatric patients. FivepHusion is harnessing the proven cytotoxic activity of 5-FU together with the unique, optimised attributes of the Deflexifol co-formulation to pursue Deflexifol development in a range of strategic solid tumour indications presenting with significant unmet medical needs, including paediatric ependymoma.
Ependymomas are rare central nervous system tumours (annual incidence of ~4 patients per million) that are more common in young children 0-4 years of age. The current standard treatment for ependymoma is surgery and radiotherapy, though relapse occurs in one third of all paediatric patients and is associated with a poor prognosis. Currently, there are no drugs approved for the treatment of ependymoma, presenting a significant unmet medical need for the development of safe and efficacious new treatments for this disease.
Previously, 5-FU has been reported as a promising drug candidate for the treatment of paediatric ependymoma by independent research groups1,2, and in a clinical trial conducted at the St Jude Children’s Research Hospital (Memphis, Tennessee, USA)3. Recently, independent studies have gained further insights into understanding the susceptibility of paediatric ependymoma to 5-FU4. Research by FivepHusion collaborators indicates that Deflexifol, as an optimised co-formulation of 5-FU and LV, may be efficacious against paediatric ependymoma and other brain cancers. Due to its improved safety, tolerability, and potentially superior anti-tumour efficacy, Deflexifol offers the exciting opportunity of addressing the limitations of current 5-FU formulations to enable development as potentially the first approved drug for ependymoma and possibly other brain tumours.