- 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 TherapeuticsClinical UnitattheUniversityHospital ofVerona,Verona, Italy said,”These earlyfindings with cambritaxestat are encouraging. Clinical activity alongside a manageable safety profile is particularly meaningfulinadisease asaggressiveandfibroticasmetastaticpancreaticcancer.Thesefindings 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 GnPinpatients withpreviouslyuntreatedmPDAC.Sixteenpatients 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.
Theresultsshownodose-limitingtoxicities,andnotreatment-emergentadverseevents(TEAE) leading to drug discontinuation or dose modification.Pharmacodynamicanalysisshowed adosedependentreductionin 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 pathwaytoaddress the complexbiologyofpancreaticcancer, andpotentiallyother 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.”
Cambritaxestatisthefirstautotaxininhibitor tobeinvestigatedincancer patientsandisbeingdeveloped asa first-in-class therapy across multiple cancer indications.
Thisstudywasco-fundedbytheEuropean UnionandrecruitedpatientsinItalyandtheUnitedKingdom.
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AboutiOnctura
iOncturaisaclinical-stageprecisiononcologycompany combatingneglectedandhard-to-treatcancers witha pipeline of first-in-class small molecules. The bold new treatments extend lives and improve healthspans, changingtheoutlookforpatientsandtheirfamilies. Leadasset,roginolisib,isanallostericmodulatorof 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 generationsofinhibitors.RoginolisibisbeinginvestigatedinmultiplerandomizedPhaseIIstudies insolidand 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 investorsincluding Syncona,MVentures, InkefCapital,EICFund,VIPartners,Schroders CapitalandXGEN Venture.
Aboutcambritaxestat
Cambritaxestatisanorally administeredautotaxin 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.
Thisstudywasco-fundedbytheEuropean Union.
1ESMOAbstract#2227P:AQuinziietal.Safety andclinicalefficacy ofcambritaxestat (IOA-289), a novel autotaxin inhibitor, plus gemcitabineandnab-paclitaxel(GnP) in patients with previously untreated metastaticpancreaticductaladenocarcinoma(mPDAC). Abstract available, 13 October 2025.Poster presented at the European Society of Medical Oncology (ESMO) congress, 19 October 2025.
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SOURCE iOnctura