iOnctura commences randomized Phase II study in metastatic uveal melanoma
- First patient dosed in Phase II OCULE-01 study with roginolisib, an allosteric modulator of PI3Kδ
- The European Medicines Agency (EMA) has granted Orphan Drug Designation for roginolisib for treatment of uveal melanoma - benefits include 10 years market exclusivity in the European Union
Geneva, Switzerland and Amsterdam, The Netherlands, 20 March 2025 - iOnctura, a clinical-stage biopharmaceutical company combating neglected and hard-to-treat cancers, today announces it has dosed the first patient in the randomized Phase II OCULE-01 study investigating lead asset roginolisib in patients with metastatic uveal melanoma (UM), a rare cancer of the eye.
Roginolisib is an orally dosed small molecule allosteric modulator of PI3Kδ. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. In the Phase I DIONE-01 study roginolisib demonstrated an excellent safety profile and a doubling of overall survival in metastatic uveal melanoma patients compared to historical controls. With few available treatments, eye melanoma is a rapidly growing market which is projected to be worth USD 9.56B by 2032[1].
The Phase II open label, randomized, parallel-arm OCULE-01 study (NCT06717126), has been designed to assess roginolisib as a monotherapy with the primary objective to evaluate overall survival in patients. The study will have multiple sites across Europe and the US, enrolling approximately 85 patients with metastatic UM, who have progressed following at least one prior therapy.
The secondary objectives of the Phase II study will assess progression free survival, objective response rate, duration of response, time to response, disease control rate, clinical benefit rate, safety and tolerability, pharmacokinetics, safety, health care utilization and quality of life.
Late in 2024, iOnctura also received Organ Drug Designation (ODD) from the European Medicines Agency (EMA) for roginolisib. ODD provides privileged status to drugs that show promise for the treatment of rare diseases in the European Union and qualifies iOnctura for benefits including protocol assistance, market exclusivity and fee reductions. Additionally, in early 2023 the US Food and Drug Administration (FDA) granted ODD for roginolisib in UM.
Paul Nathan, Principal Investigator of the Phase II OCULE-01 study said: ‘Roginolisib has so far shown impressive tolerability and an interesting median overall survival of 16 months in patients with uveal melanoma who had progressed on prior systemic therapy. With roginolisib’s attractive safety profile and the encouraging survival data observed in the first-in-human dose study, we look forward to continuing to investigate the potential of roginolisib in patients with limited therapeutic options.’
Catherine Pickering, CEO and Co-Founder of iOnctura added: ‘We have achieved our first significant milestone for 2025 by starting one of several planned randomized 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.’
[1] Emergen Research, Jan 2024
