Pharma Focus Europe

IOnctura Granted FDA Clearance for IND Application of Roginolisib, a Novel Allosteric Modulator Targeting PI3Kδ

Wednesday, November 01, 2023

iOnctura, a biotechnology company in the clinical stage, is dedicated to developing targeted therapies for cancer that disrupt crucial tumor survival pathways. It has obtained approval from the US Food and Drug Administration (FDA) to proceed with clinical investigations of roginolisib in the United States.

Roginolisib (IOA-244) is currently under development for treating solid and hematologic malignancies, including uveal melanoma, a rare cancer that originates in the uveal tract of the eye. In about 50% of patients with uveal melanoma, the cancer metastasizes, and existing treatment options are limited, with an estimated overall survival of only one year.

Roginolisib represents a significant advancement in drug development within the PI3Kδ class, as it is the first novel allosteric modulator of PI3Kδ. Its unique binding mechanism, combined with a high degree of selectivity for PI3Kδ, is expected to result in a better safety and tolerability profile compared to earlier-generation inhibitors.

iOnctura, expressed the company's commitment to advancing roginolisib through clinical trials, with final clinical data from the initial patients expected in 2024. She highlighted the potential of roginolisib to slow or halt the progression of uveal melanoma, offering a vital treatment option for patients who have exhausted their first-line therapy options.

Roginolisib is currently under investigation in the DIONE-01 trial, a two-part, first-in-human Phase I study with identifier NCT04328844. The study is fully enrolled, and final data is anticipated in the first quarter of 2024. Among all patients treated with roginolisib monotherapy so far, only 7% experienced Grade 3/4 toxicities, with no dose-limiting toxicities, drug-related serious adverse events, or drug-related adverse events leading to dose interruptions or discontinuations. Although the median overall survival has not yet been reached, 62% of patients were alive at the 12-month mark, which is a favorable outcome compared to historical controls where only 34% of patients in a similar setting survived.

Roginolisib has demonstrated good tolerability during long-term administration, with some patients being treated for up to 40 months in the study. Promising clinical responses have been observed in patients with both solid and hematological cancers.



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