On September 5, 2023 FivepHusion, an advanced clinical-stage biotechnology company, reported that the first patient has commenced treatment as part of the Deflexifol at Relapse Trial (DART) (Press release, FivepHusion, SEP 5, 2023, View Source [SID1234634885]). The DART Study is a phase 1b/2a clinical trial investigating 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, which is being sponsored by the Australian and New Zealand Children’s Haematology / Oncology Group (ANZCHOG).
The DART Study is designed with two parts:
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Part A, to determine Deflexifol safety, tolerability, maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) in paediatric patients with refractory or recurrent central nervous system tumours, or newly diagnosed diffuse intrinsic pontine glioma (DIPG) / diffuse midline glioma (DMG) who have completed radiotherapy.
Part B, a phase 2 study at the RP2D to evaluate the Deflexifol anti-tumour activity in patients with refractory or recurrent ependymoma. Response rate, progression free survival and overall survival will be assessed.
Deflexifol is an advanced clinical-stage, next-generation co-formulation of the chemotherapy drug 5-fluorouracil (5-FU) and leucovorin (LV), a drug that significantly enhances 5-FU activity. FivepHusion aims to harness the proven cytotoxic activity of 5-FU, together with the unique optimised attributes of the Deflexifol co-formulation, to pursue development in strategic solid tumour indications presenting with significant unmet medical needs. The priority development indications for Deflexifol are paediatric ependymoma and the 1st line treatment of metastatic colorectal cancer (mCRC), with a global phase III registration trial planned to start in mCRC in late 2024.
Ependymomas are rare central nervous system tumours (~4 patients per million) originating from the ependymal cells that line the ventricles of the brain and central canal of the spinal cord. Tumours can manifest at any age, with peak incidence in young children (0-4 years) and older adults. The current standard of care treatment for ependymoma is surgery and radiotherapy, though relapse occurs in approximately half 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 safe and efficacious new treatments.
Previously, 5-FU has been reported to be a promising drug candidate for the treatment of paediatric ependymoma by independent labs1,2, and in a clinical trial run at the St Jude Children’s Research Hospital (Memphis, Tennessee, USA)3. Research by FivepHusion collaborators indicates that Deflexifol may also be efficacious against 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 the first approved drug for ependymoma and potentially other brain tumours.
The investigator-initiated DART Study is led by Principal Investigators Professor David Ziegler and Dr Marion Mateos at the Kids Cancer Centre. The trial is coordinated by the Sydney Children’s Hospital, Randwick, with support from the Cancer Institute NSW. Major funding to support the study 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, "Treatment of the first patient in the DART Study is a very important milestone for the development of Deflexifol as a promising new therapy for paediatric 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."