FivepHusion, an advanced clinical-stage biotechnology company, is pleased to announce 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:

  • 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.

About FivepHusion

FivepHusion (www.fivepHusion.com) is an advanced clinical-stage, globally focused biotechnology company optimising cancer therapy to improve patient treatment outcomes and quality of life.

The FivepHusion lead asset is Deflexifol®, a proprietary, novel, and optimised co-formulation of the chemotherapeutic agent 5-FU and its biomodulator LV, a drug that significantly enhances 5-FU anti-cancer activity. These drugs are commonly used globally to treat various solid tumours including colorectal, pancreatic, gastric, and head & neck cancers, among others. However, due to their chemical incompatibility, current formulations of 5-FU and LV suffer from limitations in their safety, tolerability, effectiveness and pharmacological compatibility. These disadvantages contribute to limited efficacy, unpleasant side effects and toxicities, and a reduced quality of life experienced by cancer patients.

FivepHusion is developing Deflexifol® via the FDA 505(b)(2) and EMA Article 10b regulatory pathways as a next-generation replacement of standard of care formulations of 5-FU and LV for the first-line treatment of metastatic colorectal cancer (a ~US$13B market5) and other tumours with a projected global incidence of >8 million patients. Anticipated peak revenues for Deflexifol® are ~US$1.8B. Deflexifol® is also being developed as a new therapy for cancers with high unmet medical need, including paediatric ependymoma (part of the ~US$1.32B paediatric brain tumour market6).

In addition to Deflexifol®, FivepHusion has secured commercial rights to ResectAssist™, an innovative drug delivery platform technology enabling targeted delivery of anti-cancer drugs directly into solid tumours, maximising the drug efficacy while minimising side effects. The lead candidate, ResectAssist™-FOLFIRINOX, is in development to treat locally advanced pancreatic cancer, a significant segment of the >$7B pancreatic cancer market7.

Forward-Looking Statements
This announcement (and any attachments) may contain certain forward-looking statements that are based on any number of assumptions and estimates which may prove incorrect and relate to circumstances and events that may not take place. Forward-looking statements involve known and unknown risks, uncertainties, and other factors (such as significant business, economic and competitive uncertainties and contingencies, and regulatory and clinical development risks and uncertainties) which may cause the actual future plans, results or the performance of FivepHusion and its development pipeline opportunities Deflexifol® and ResectAssist™ to differ materially from the plans, results or performance expressed or implied by such forward-looking statements. Past performance is not a reliable indicator of future performance. There can be no assurance that any forward-looking statements will be realised. FivepHusion does not make any representation or give any warranty as to the likelihood of achievement or reasonableness of any forward-looking statements.

1 Atkinson et al. 2011, Cancer Cell 20(3):384.
2 Donson et al. 2018, Mol Cancer Ther. 17(9):1984
3 Wright et al. 2015, Neuro Oncol. 17(12):1620.
4 Griesinger et al. 2024, Clin Cancer Res. 30(8):1544.
5 DelveInsight 2025, Colorectal Cancer Market Insight, Epidemiology And Market Forecast – 2034.
6 Market Research Future 2026, Pediatric Brain Tumor Market.
7 Polaris Market Research 2022, Pancreatic Cancer Market Share, Size, Trends, Industry Analysis Report, 2022 – 2030.
Deflexifol® is a trademark of FivepHusion. ResectAssist™ is a trademark of University of Wollongong.