#None #Amsterdam #iOnctura #roginolisib #uveal melanoma

iOnctura Gets Underway with Phase II Study for Uveal Melanoma

Introduction
iOnctura, a pioneering clinical-stage biopharmaceutical company, has announced a significant milestone in its quest to combat hard-to-treat cancers. Recently, the company initiated a randomized Phase II clinical trial named OCULE-01, exploring its lead drug candidate, roginolisib, in patients diagnosed with metastatic uveal melanoma (UM), a rare and aggressive form of eye cancer.

Understanding Uveal Melanoma

Uveal melanoma, originating in the melanocytes of the uveal tract of the eye, is one of the rarest forms of cancer but known for its aggressive nature. The prognosis for patients with metastatic uveal melanoma remains challenging due to the limited options currently available for treatment. With the market for uveal melanoma therapies projected to reach $9.56 billion by 2032, the potential for innovative treatments is significant.

Roginolisib: A New Hope

Roginolisib stands out as an allosteric modulator of PI3Kδ, a pathway commonly involved in cancer progression. Unlike previous inhibitors that brought along adverse side effects, roginolisib is characterized by its favorable tolerability and precise target inhibition. Initial findings from the Phase I DIONE-01 study showed that it could double the overall survival rates for patients previously treated for metastatic uveal melanoma.

The Phase II OCULE-01 Study

The OCULE-01 study is set to be an open-label, randomized, parallel-arm clinical trial involving about 85 patients across various sites in Europe and the United States. Participants must have previously undergone at least one systemic treatment before entering this Phase II trial. The primary focus will be on evaluating overall survival rates, while secondary measures will include progression-free survival, objective response rates, safety, and the overall quality of life of the patients involved.

Orphan Drug Designation and Its Benefits

In late 2024, the European Medicines Agency (EMA) granted Roginolisib Orphan Drug Designation, which provides crucial benefits such as market exclusivity and financial incentives for drugs targeting rare diseases. In alignment with this, the U.S. Food and Drug Administration (FDA) awarded the same designation earlier in 2023. This recognition underscores the drug's potential in offering new treatment avenues for patients grappling with limited options.

Insights from Clinical Experts

Paul Nathan, the Principal Investigator for the Phase II OCULE-01 study, expressed confidence in roginolisib’s profile, highlighting that it has demonstrated impressive tolerability and an intriguing median overall survival rate of 16 months for patients who had exhausted previous therapies. Nathan's insights reveal the promising horizon for newer treatments against difficult cancers.

Future Prospects and Other Indications

Catherine Pickering, CEO and Co-Founder of iOnctura, confirmed that the initiation of this study marks a pivotal accomplishment for 2025. She noted their broader vision for roginolisib, which is also being investigated for treatment in non-small cell lung cancer and myelofibrosis. With plans for several randomized Phase II studies in motion, initial results are anticipated by the end of the year.

Conclusion

As iOnctura embarks on this Phase II study, the potential impact of roginolisib in treating metastatic uveal melanoma and other cancers could mark a turning point in oncology. Its innovative approach offers hope not just for patients but for the biopharmaceutical landscape as a whole. Clinical trials are essential for validating the efficacy of new therapies, and as these trials progress, the hope for improved patient outcomes remains bright.

For further information about iOnctura, its current studies, and future applications, visit their official website.