On June 18, 2026 FivepHusion, an advanced clinical-stage biotechnology company, reported that an independent Human Research Ethics Committee (HREC) has approved progression to phase II of the Deflexifol at Relapse Trial (DART). The phase II trial is investigating Deflexifol monotherapy as a treatment for refractory or recurrent paediatric ependymoma. This HREC approval follows the successful completion of the phase I DART study in 2025 and incorporation of a recommended phase II dose and other updates into the phase II trial protocol.
Key design elements of the phase II DART study include:
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A multi-centre single-arm clinical trial involving all major paediatric oncology centres in Australia, with patients enrolled to determine the objective response rate to Deflexifol.
Recruitment of ten evaluable participants (aged 1-21 years) with refractory or recurrent paediatric ependymoma, including three participants currently evaluable from the phase I study.
Primary Objective: To evaluate the anti-tumour activity of Deflexifol as a single agent in patients with refractory or recurrent ependymoma.
Secondary Objectives: To assess overall survival and progression-free survival, and to further characterise the pharmacokinetics of Deflexifol monotherapy.
The phase II DART study is an Australian 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 phase II study follows the successful completion of the phase I DART study, the results of which were presented as a poster in November 2025 at the Society of Neuro-Oncology (SNO) Annual Meeting in Honolulu, Hawaii, USA. The phase II trial is designed to investigate Deflexifol monotherapy as a treatment for refractory or recurrent paediatric ependymoma. The trial is coordinated by the KOALA National Coordinating Centre at the Sydney Children’s Hospitals Network and all major Australian paediatric oncology centres are participating in the study. Significant trial funding has been provided by the Kids with Cancer Foundation and the Robert Connor Dawes Foundation.
Dr Christian Toouli, CEO and Managing Director of FivepHusion commented, "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."
Deflexifol is an innovative next-generation novel co-formulation of 5-fluorouracil (5-FU) and leucovorin (LV), a drug that significantly enhances 5-FU anti-tumour activity. Deflexifol has previously been evaluated in the phase I DART study in paediatric brain cancer patients and two successfully completed clinical trials in adults with a variety of solid tumours. 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 therapeutic 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 a novel optimised co-formulation of 5-FU and LV, may be efficacious against paediatric ependymoma and other brain cancers. Due to its superior safety, tolerability and anti-tumour efficacy, Deflexifol offers the exciting opportunity to address the limitations of current 5-FU formulations to enable development as potentially the first approved drug for ependymoma and possibly other brain tumours.
(Press release, FivepHusion, JUN 18, 2026, View Source [SID1234668795])