Roginolisib

Participation underscores growing excitement around company's momentum and potential in precision oncology GENEVA, AMSTERDAM and CAMBRIDGE, Mass., Jan. 9, 2026 /PRNewswire/ -- iOnctura, a clinical-stage biopharmaceutical company developing precision oral small molecules for neglected and hard-to-treat cancers, today announces that its Chief Executive Officer, Catherine Pickering, will present at the 44th Annual J.P. Morgan Healthcare Conference, taking place January 12–15, 2026, in San Francisco, California. Dr. Pickering will present on Thursday, January 15, 2026 at 11:00 PST. The presentation will highlight iOnctura's clinical progress with a focus on its lead program, roginolisib, the first allosteric modulator of PI3Kδ. Recognized as the world's largest and most influential healthcare investment conference, the J.P. Morgan Healthcare Conference is an invite-only event that serves as a vital forum for innovation and capital formation across the pharmaceutical and biotechnology sectors. Participation reflects iOnctura's growing visibility within the global oncology ecosystem and underscores the company's progress towards advancing transformative targeted therapies for patients with cancer. "We are honored to be invited to present at this year's J.P. Morgan Healthcare Conference," said Catherine Pickering, CEO and co-founder of iOnctura. "This recognition reflects the increasing interest in our science-driven approach and the meaningful clinical advances we have achieved with roginolisib. We look forward to sharing our progress and engaging with partners committed to transforming cancer care." Members of the management team will also be available for one-on-one meetings with investors and partners during the conference. For more information contact: Corporate Press Office: [email protected] About iOnctura iOnctura is a clinical-stage precision oncology company combating neglected and hard-to-treat cancers with a pipeline of first-in-class small molecules. The bold new treatments extend lives and improve healthspans, changing the outlook for patients and their families. Lead asset, roginolisib, is a non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. Roginolisib is being investigated in multiple randomized Phase II studies in solid and hematological malignancies. iOnctura is headquartered in Amsterdam, The Netherlands with subsidiaries located in Geneva, Switzerland and Cambridge, MA, USA. iOnctura is backed by specialist institutional investors including Syncona, M Ventures, Inkef Capital, EIC Fund, VI Partners, Schroders Capital and XGEN Venture. About roginolisib Roginolisib is a first-in-class, non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. The PI3K signaling pathway is one of the most commonly dysregulated pathways across multiple cancer types. The potential of roginolisib has been validated by positive clinical signals in Phase I in solid tumor and hematological malignancies, including a doubling of overall survival compared to historical controls in rare eye cancer, uveal melanoma. The company has carefully designed its clinical program to allow full development in uveal melanoma, while in parallel validating the program in larger market indications. The Phase II OCULE-01 study in uveal melanoma started in March 2025, the PULMO-01 study in non-small cell lung cancer (NSCLC) started in May 2025 and the HEMA-MED Phase I/II study in myelofibrosis began in November 2025. Logo : SOURCE iOnctura 21% more press release views with Request a Demo

First patient dosed in Phase I/II HEMA-MED clinical trial (NCT06887803) evaluating the tolerability and efficacy profile of roginolisib (PI3Kδ inhibitor) plus ruxolitinib in myelofibrosis (MF) patients unresponsive to Janus kinase (JAK) inhibitors Dual-target approach: combining PI3Kδ and JAK inhibition addresses complementary disease mechanisms and may overcome unresponsiveness to JAK inhibitors in MF New supporting non-clinical data to be presented at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida: Data from ex-vivo models of MF demonstrate roginolisib monotherapy activity and synergy with JAK inhibition GENEVA, AMSTERDAM and CAMBRIDGE, Mass., Dec. 4, 2025 /PRNewswire/ -- iOnctura, a clinical-stage biopharmaceutical company combating neglected and hard-to-treat cancers, today announces it has dosed the first patient in its Phase I/II clinical trial evaluating roginolisib in patients with MF who are no longer responding to JAK inhibition. Further details of the trial will be presented in a poster presentation at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida on Sunday, December 7 at 6pm EST. This milestone marks an important step toward addressing the significant unmet medical needs of MF patients who face diminishing therapeutic options, as their disease progresses or becomes resistant to standard-of-care JAK inhibitor treatment. The Phase I/II trial is part of a comprehensive development program evaluating next-generation PI3Kδ inhibitor roginolisib's tolerability and anti-tumor response across a number of solid and hematologic malignancies. "Based on non-clinical data being presented at ASH, we anticipate that targeting both the PI3Kδ and JAK pathways could have a synergistic effect in MF — an approach that has previously eluded older generation PI3Kδ inhibitors because of their unfavorable tolerability profiles." said Dr Michael Lahn, Chief Medical Officer of iOnctura. Overactivation of the PI3K-Akt signaling pathway contributes to the occurrence and progression of cancer[1]. In MF, pathway activation is a well described mechanism of resistance in response to JAK inhibition[2]. Inhibition of this pathway using roginolisib may overcome the lack of response and lead to a beneficial therapeutic effect. Non-clinical evidence in support of this mechanism is being presented at ASH on Monday, December 8, 2025 at 6pm EST. These data demonstrate monotherapy activity of roginolisib against MF cell lines and in vitro models of primary MF cells. Further, roginolisib and JAK inhibition (ruxolitinib or momelotinib) exerts a synergistic anti-cancer effect. These data support the rationale behind the ongoing HEMA-MED clinical trial. Professor Alessandro Vannucchi, Professor of Hematology and Department Head of the Center for Research and Innovation in Myeloproliferative Neoplasms (CRIMM) at the University of Florence, Florence, Italy and Principal Investigator of the HEMA-MED study said: "Myelofibrosis patients often have suboptimal responses to ruxolitinib and/or acquire resistance over time. Roginolisib's intriguing mechanism of action and favorable toxicity profile position it as a compelling partner for ruxolitinib. By combining these agents, we could unlock more durable responses and redefine therapeutic expectations for this challenging rare disease." In addition to the two posters related to MF, trials in progress posters for the ongoing investigations of roginolisib in peripheral T-cell lymphoma and chronic lymphocytic leukemia (CLL) will also be presented at ASH. iOnctura poster presentations at the 67th ASH Annual Meeting in Orlando, Florida, USA: 1. A Vannucchi et al. Trial in progress: Sunday, December 7, 06:00 PM - 08:00 PM EST Abstract 25-4153: A study of roginolisib in combination with ruxolitinib in patients with myelofibrosis who are unresponsive to JAK inhibitors (HEMA-MED) 2. M Balliu et al.: Monday, December 8, 06:00 PM - 08:00 PM EST Abstract 25-7102: The selective PI3Kd inhibitor roginolisib synergizes with ruxolitinib against progenitor cells from naïve and JAK-inhibitor-refractory/resistant patients with myelofibrosis 3. D Sibon et al. Trial in progress: Sunday, December 7, 06:00 PM - 08:00 PM EST Abstract 25-11050: PlatΤform: A multicenter, multi-arm, academic platform trial evaluating novel agents and combinations in relapsed or refractory peripheral T-cell lymphomas 4. J Brown et al. Trial in progress: Sunday, December 7, 06:00 PM - 08:00 PM EST Abstract 25-7765: Roginolisib (IOA-244), an orally bioavailable, selective PI3Kδ inhibitor, in combination with venetoclax and rituximab in patients with relapsed chronic lymphocytic leukemia (CLL) About myelofibrosis (MF) MF is a rare myeloproliferative neoplasm marked by activation and growth of mutated cells in the bone marrow, with approximately 0.5 cases per 100,000 individuals diagnosed globally a year[3]. JAK inhibitors are a key treatment option for MF patients and improve symptoms and quality of life, but approximately half of patients discontinue therapy within three years[4]. Reasons for discontinuation include disease progression, tolerability and adverse events and death resulting from resistance to the JAK therapy[5],[6]. About the HEMA-MED clinical trial The HEMA-MED trial (NCT06887803) is a prospective, multi-center, open-label Phase I/II single arm trial consisting of two parts. Part 1 (Phase 1) will enroll 13 patients to assess the safety of the combination of the PI3Kδ inhibitor roginolisib in combination with the JAK inhibitor ruxolitinib, and Part 2 (Phase 2) will expand to enroll 13 additional patients to further allow the assessment of benefit/risk for all 26 patients. In addition to safety, the secondary endpoints of the HEMA-MED trial include blood biomarker and spleen reduction responses, and improvements of MF related symptoms. Exploratory measures will assess endpoints associated with the mechanism of action (MOA) of roginolisib. About iOnctura iOnctura is a clinical-stage precision oncology company combating neglected and hard-to-treat cancers with a pipeline of first-in-class small molecules. The bold new treatments extend lives and improve healthspans, changing the outlook for patients and their families. Lead asset, roginolisib, is a non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. Roginolisib is being investigated in multiple randomized Phase II studies in solid and hematological malignancies. iOnctura is headquartered in Amsterdam, The Netherlands with subsidiaries located in Geneva, Switzerland and Cambridge, MA, USA. iOnctura is backed by specialist institutional investors including Syncona, M Ventures, Inkef Capital, EIC Fund, VI Partners, Schroders Capital and XGEN Venture. About roginolisib Roginolisib is a first-in-class, non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. The PI3K signaling pathway is one of the most commonly dysregulated pathways across multiple cancer types. The potential of roginolisib has been validated by positive clinical signals in Phase I in solid tumor and hematological malignancies, including a doubling of overall survival compared to historical controls in rare eye cancer, uveal melanoma. The company has carefully designed its clinical program to allow full development in uveal melanoma, while in parallel validating the program in larger market indications. The Phase II OCULE-01 study in uveal melanoma started in March 2025, the PULMO-01 study in non-small cell lung cancer (NSCLC) started in May 2025 and the HEMA-MED Phase I/II study in myelofibrosis began in November 2025. For more information contact: Corporate Press Office: [email protected] [1] He et al., 2021 [2] Moyo et al., 2023 [3] Titmarsh et al., 2014 [4] Verstovsek et al., 2015 [5] Kuykendall et al., 2018 [6] Newberry et al., 2017 Logo - SOURCE iOnctura 21% more press release views with Request a Demo