- Cambritaxestat, the first autotaxin (ATX) inhibitor to be investigated in cancer patients, meets its primary endpoint in a study to assess safety and anti- tumour responses in patients with metastatic pancreatic cancer
- Combining cambritaxestat with gemcitabine/nab-paclitaxel (GnP) was tolerable and associated with anti-tumor responses
- Cambritaxestat administration combined with GnP demonstrated reduction in pharmacodynamic markers associated with fibrosis, immune regulation and tumor progression
GENEVA and AMSTERDAM and CAMBRIDGE, Mass., Oct. 14, 2025 /PRNewswire/ -- iOnctura, a clinical-stage precision oncology company focused on neglected and hard-to-treat cancers, today announces the Phase Ib study investigating oral autotaxin (ATX) inhibitor, cambritaxestat (IOA-289), has met the primary endpoint: demonstrating safety, tolerability and anti-tumor responses, in combination with standard-of-care chemotherapy, in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC). These data are being presented as a poster at the European Society for Medical Oncology (ESMO) Congress in Berlin, Germany.1
Lead investigator, Davide Melisi, M.D., Ph.D, Associate Professor of Medical Oncology, and Director of the Digestive Molecular and Clinical Oncology Research Unit, University of Verona, and Investigational Cancer Therapeutics Clinical Unit at the University Hospital of Verona, Verona, Italy said, "These early findings with cambritaxestat are encouraging. Clinical activity alongside a manageable safety profile is particularly meaningful in a disease as aggressive and fibrotic as metastatic pancreatic cancer. These findings warrant continued scientific exploration in autotaxin inhibition."
The Phase Ib dose escalation study, AION-02 (NCT05586516) evaluated cambritaxestat in combination with standard-of-care chemotherapy GnP in patients with previously untreated mPDAC. Sixteen patients received cambritaxestat orally, twice daily at doses of 100 mg (n=4), 200 mg (n=4), 400 mg (n=5) and 800 mg (n=3). GnP was administered by IV infusion, weekly for three weeks of a four-week cycle.
The results show no dose-limiting toxicities, and no treatment-emergent adverse events (TEAE) leading to drug discontinuation or dose modification. Pharmacodynamic analysis showed a dose dependent reduction in the ATX-dependent plasma lipid LPA C18:2 over 24 hours supporting cambritaxestat's on-target effects. Patients in the higher-dose cohorts had consistent and durable reductions of the tumor marker CA19-9. These changes were associated with radiographic responses and survival.
Dr. Michael Lahn, Chief Medical Officer at iOnctura said, "These data reinforce the therapeutic promise of targeting the autotaxin pathway to address the complex biology of pancreatic cancer, and potentially other tumors with high expression of autotaxin and its associated signaling. As we advance development, we remain focused on unlocking the potential of cambritaxestat to improve outcomes for patients facing some of the most challenging and hard-to-treat cancers."
Cambritaxestat is the first autotaxin inhibitor to be investigated in cancer patients and is being developed as a first-in-class therapy across multiple cancer indications.
This study was co-funded by the European Union and recruited patients in Italy and the United Kingdom.
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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 an allosteric modulator of PI3Kd with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kd, 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 cambritaxestat
Cambritaxestat is an orally administered autotaxin inhibitor being developed for the treatment of highly fibrotic cancers, including metastatic pancreatic cancer. By targeting the autotaxin pathway, cambritaxestat offers a novel three-pronged approach-directly inhibiting tumor growth, stimulating immune effector cells, and reducing fibrosis to enhance drug and immune cell access to the tumor. Cambritaxestat received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), in March 2024.
This study was co-funded by the European Union.
1 ESMO Abstract #2227P: A Quinzii et al. Safety and clinical efficacy of cambritaxestat (IOA-289), a novel autotaxin inhibitor, plus gemcitabine and nab-paclitaxel (GnP) in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC). Abstract available, 13 October 2025. Poster presented at the European Society of Medical Oncology (ESMO) congress, 19 October 2025.
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