GENEVA - iOnctura, a clinical-stage biopharmaceutical company, has announced positive results from the Phase I DIONE-01 study of roginolisib, its lead asset, in treating uveal melanoma (UM), a rare cancer of the eye. The data was presented at the European Society for Medical (TASE:PMCN) Oncology Immuno-Oncology (ESMO-IO) congress.
The study met its primary objective, establishing the safety of the anticipated optimal biologically effective dose (BED) of roginolisib. The recommended Phase II dose (RP2D) of 80mg was well tolerated over long periods, up to 4.5 years. Notably, the median overall survival (OS) for the 29 UM patients treated with roginolisib was 16 months, surpassing the seven-month median OS observed in historical controls undergoing second-line immunotherapies. Additionally, median progression-free survival (PFS) was five months, compared to less than three months for historical controls.
These encouraging results have led iOnctura to initiate the OCULE-01 Phase II study of roginolisib in UM, with a data readout anticipated in CY2026. Syncona Ltd, a leading life science investor and a backer of iOnctura, highlighted the potential of roginolisib to become a clinically meaningful medicine targeting the PI3K cancer pathway.
Roginolisib is a first-in-class allosteric modulator of PI3K delta (PI3Kδ), a protein implicated in a variety of cancers. Its unique chemical structure and binding mechanism confer high specificity for PI3Kδ, which may offer an improved pharmacology and safety profile over previous generations of PI3Kδ inhibitors.
The UM market is rapidly growing and is projected to be valued at USD 9.56B by 2032. The Phase II trial will further investigate roginolisib's efficacy and safety, offering hope for patients with few available treatments.
The press release statement from iOnctura, which also operates a subsidiary in Amsterdam, Netherlands, underscores the company's commitment to developing treatments that extend lives and improve healthspans for patients with neglected and hard-to-treat cancers. The upcoming randomized Phase II study is a significant step in understanding the potential of roginolisib, which has shown promise in early trials. Site activation for the Phase II study is underway, with additional trials for other cancers, including non-small cell lung cancer and myelofibrosis, in planning.
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