US FDA grants Orphan Drug Designation for iOnctura's first-in-class autotaxin cancer therapy
07.08.2025 - 18:05:43
Autotaxin inhibitor, IOA-289, recently received the proposed INN cambritaxestatCambritaxestat is in the Phase I AION-02 trial in combination with chemotherapy in metastatic pancreatic cancerIt is the first autotaxin inhibitor to be investigated in cancer patientsis a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways. This pioneering approach to drug development is expected to offer significant clinical benefits over the traditional approach of targeting a single pathway alone. iOnctura has progressed two therapeutic candidates into mid-stage clinical development: Roginolisib, an allosteric modulator of PI3K?; and cambritaxestat, a highly selective, non-competitive autotaxin inhibitor. IOA-359, a TGF–? pathway inhibitor is also undergoing an extensive pre-clinical program in preparation for first-in-human studies. iOnctura is backed by specialist institutional investors including M Ventures, Inkef Capital, VI Partners, Schroders Capital, European Innovation Council and 3B Future Health Fund. iOnctura BV is headquartered in Amsterdam, The Netherlands with its wholly owned Swiss subsidiary, iOnctura SA, located in Geneva, Switzerland.
About cambritaxestat (IOA-289)
Cambritaxestat is an orally dosed molecule that has shown preclinically to inhibit the growth and proliferation of cancer cells, stimulate immune cell infiltration into tumors and inhibit the development of fibrosis. Cambritaxestat is being developed as a first-in-class therapy for highly fibrotic cancer indications that overexpress autotaxin including pancreatic, liver, colorectal, ovarian and breast cancers. A Phase I study of cambritaxestat in combination with chemotherapy in metastatic pancreatic cancer started in Q4 2022 (NCT05586516).
View original content:https://www.prnewswire.co.uk/news-releases/us-fda-grants-orphan-drug-designation-for-ioncturas-first-in-class-autotaxin-cancer-therapy-302082016.html
