iOnctura has dosed the first subject in the open-label Phase II OCULE-01 trial of oral roginolisib in individuals with rare eye cancer, metastatic uveal melanoma (UM).
The randomised, parallel-arm trial aims to evaluate the therapy as a single agent. It will enrol nearly 85 metastatic UM subjects who have progressed after at least one previous therapy.
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It will cover several sites across the US and Europe.
The primary objective is to assess overall survival (OS), with secondary objectives including the evaluation of response duration, progression-free survival (PFS), tolerability, time to response, clinical benefit rate, disease control rate, safety, quality of life, pharmacokinetics, and health care utilisation.
According to the company, this small molecule allosteric modulator of phosphoinositide-3-kinase δ (PI3Kδ) has shown promise in the Phase I DIONE-01 trial, where it demonstrated an encouraging safety profile and an OS doubling in metastatic UM individuals against historical controls.
iOnctura co-founder and CEO Catherine Pickering said: “We have achieved our first significant milestone for 2025 by starting one of several planned randomised Phase II studies for roginolisib.
“We are excited by the potential of roginolisib across a number of indications, including uveal melanoma, non-small cell lung cancer, and myelofibrosis, and we look forward to early data readouts by the end of the year.”
The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) granted orphan drug designation for the therapy in 2024 and 2023, respectively.
The Netherlands-headquartered company has a subsidiary in Switzerland and is supported by a consortium of institutional investors.
In June 2024, the company raised €80m ($85.8m) in Series B financing to fund the Phase II clinical trials for roginolisib and another lead cancer therapy, cambritaxestat.
