FivepHusion, an advanced clinical-stage biotechnology company, has received approval from the Human Research Ethics Committee to proceed to Phase 2 of the Deflexifol at Relapse Trial DART, advancing the novel co-formulation of 5-fluorouracil and leucovorin into a focused study for refractory or recurrent paediatric ependymoma.
The Phase 2 study follows completion of the Phase 1 DART study in 2025 and incorporates the recommended Phase 2 dose and protocol updates derived from that trial.
The Phase 2 DART study is a multi-centre single-arm Australian trial coordinated by the KOALA National Coordinating Centre at the Sydney Children’s Hospitals Network and led by Principal Investigators Professor David Ziegler and Dr Marion Mateos, with sponsorship from the Australian and New Zealand Children’s Haematology Oncology Group in collaboration with FivepHusion.
All major paediatric oncology centres in Australia will participate. Ten evaluable participants aged 1 to 21 with refractory or recurrent ependymoma will be recruited, including three participants currently evaluable from the phase I study.
The primary objective is to evaluate the anti-tumour activity of Deflexifol as a single agent, with secondary objectives to assess overall survival, progression-free survival, and to further characterise the pharmacokinetics of Deflexifol monotherapy.
Significant funding for the trial has been provided by the Kids with Cancer Foundation and the Robert Connor Dawes Foundation. Results from the Phase 1 study were presented as a poster at the Society of Neuro-Oncology Annual Meeting in November 2025 in Hawaii.
Dr Christian Toouli, CEO and Managing Director of FivepHusion, said, “Paediatric ependymoma is the third most common brain cancer in children, and with no approved drugs to treat this condition, presents a significant unmet medical need for a safe and efficacious therapy. The ethics approval of the Phase II DART protocol is an exciting milestone towards our goal of evaluating Deflexifol as a promising therapy for these patients."
