iOnctura awarded UK’s MHRA Innovation Passport for entry into innovative licensing and access pathway (ILAP)

Geneva, Switzerland and Amsterdam, The Netherlands, 28 March - iOnctura, a clinical stage biotechnology company developing breakthrough therapies for patients suffering with cancer, today announces that the innovative medicine designation, the Innovation Passport, has been awarded for roginolisib, for the treatment of metastatic uveal melanoma by the Medicines & Healthcare products Regulatory Agency (MHRA).

The Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP) which aims to accelerate time to market and thereby patient access to novel treatments in the UK. Reserved for innovative therapies for life-threatening or seriously debilitating conditions, ILAP provides applicants with a toolkit to support all stages of the design, development, and approval process.

Roginolisib is a first-in-class, non-ATP-competitive, allosteric modulator of PI3Kd which prevents tumor proliferation and breaks immune tolerance in patients with solid and hematological tumors.

Catherine Pickering, Chief Executive Officer of iOnctura, said: “The Innovation Passport is an exciting step in the clinical development programme for roginolisib, a drug with a game-changing clinical safety and activity profile. Being awarded this passport will allow us to work closely with the MHRA and its partner agencies to chart out a roadmap for regulatory and key development milestones with the primary goal of achieving early patient access to roginolisib.”

PI3Kδ inhibition in solid tumors has recently emerged as a novel approach to treating cancer because of its potential in targeting multiple tumor survival pathways. First-generation PI3Kδ inhibitors are used to treat hematological tumors, but safety concerns and limited target selectivity have curbed their clinical usefulness. These concerns are even more aggravated in patients with solid malignancies where rapid onset of toxicities have been observed. In contrast, roginolisib has a favorable toxicity profile with less than 5% Grade 3/4 toxicities at the biologically effective dose in clinical studies. Importantly, these toxicities have to-date been transient in nature without the need for dose reductions.

Clinical activity, including partial and complete responses, are being seen in patients with both solid and hematologic malignancies. Further details on clinical responses will be released at a future international clinical conference in 2023. Fourteen of 43 patients (including 12 of 28 uveal melanoma patients) are still on treatment, with two patients having been on treatment for more than two years. The one-year OS rate is currently 70%; median OS has not been reached.

A research paper recently published in Cancer Research Communications highlights that roginolisib has immune-modulatory properties that can be exploited in solid tumors and further reinforced the conclusion that roginolisib inhibits regulatory T cell proliferation while having limited anti-proliferative effects on conventional CD4+ T cells and no effect on CD8+ T cells; both immune cell types key to a robust immune response to tumors. You can access the link to the paper here.