FivepHusion, an advanced clinical-stage biotechnology company, is pleased to announce 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.
  • Nine patients with a variety of brain cancers were treated with Deflexifol® monotherapy at paediatric oncology centres around Australia
  • An independent Data Safety Monitoring Committee consisting of adult and paediatric oncologists reviewed the trial participants’ safety and tolerability data and declared a Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) for Deflexifol® in paediatric brain cancer patients
  • Confirmation of a safe and tolerable dose of Deflexifol® represents the successful completion of the primary objective of DART Part A
  • The MTD, RP2D and other clinical and scientific data from DART Part A is being collated and will be presented at a paediatric oncology conference later this year
  • Deflexifol® is now poised to be investigated further in DART Part B, an ethics-approved phase 2 study designed to evaluate the anti-tumour activity of Deflexifol® in patients with refractory or recurrent ependymomas

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.

About FivepHusion

FivepHusion (www.fivepHusion.com) is an advanced clinical-stage, globally focused biotechnology company whose purpose is to optimise 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-fluorouracil (5-FU) and its biomodulator leucovorin (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, head & neck, and breast cancers. 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 treatment response rates, 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$12.6B market5) and other tumours with a projected global incidence of >6 million patients. Anticipated revenues for Deflexifol® reach peaks of >US$1B. Deflexifol® is also being developed as a new therapy for cancers with high unmet medical need, including paediatric ependymoma; a rare and deadly brain cancer which afflicts very young children. The underserved paediatric brain cancers market is reported to be US$1.8B6.

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 efficacy of proven and investigational drugs while minimising side effects. The lead candidate, ResectAssist™-FOLFIRINOX, is in development to treat unresectable, locally advanced pancreatic cancer.

Deflexifol® is a trademark of FivepHusion. ResectAssist™ is a trademark of the University of Wollongong.

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 2025 Colorectal Cancer Market Insight, Epidemiology And Market Forecast – 2034.
6 Market Research Future 2023.