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US FDA Grants Orphan Drug Designation to iOnctura’s Autotaxin Cancer Therapy

iOnctura, a prominent clinical-stage biotechnology company dedicated to advancing revolutionary cancer therapies, has achieved a significant milestone with the recent announcement of the US Food and Drug Administration (FDA) granting Orphan Drug Designation (ODD) for its groundbreaking autotaxin inhibitor, cambritaxestat, intended for the treatment of pancreatic cancer. This designation underscores the exceptional potential of cambritaxestat and provides crucial support and incentives during its development and eventual commercialization process.

The autotaxin inhibitor, also known as IOA-289, has garnered attention for its innovative chemical and biological properties, coupled with a favorable non-clinical safety and efficacy profile. Its designation as an orphan drug highlights its promising prospects in addressing the unmet medical needs of patients grappling with pancreatic cancer, a disease notorious for its high mortality rates.

Cambritaxestat, proposed with the International Nonproprietary Name (INN), is currently undergoing evaluation in the Phase I AION-02 trial, where it is being administered in combination with standard chemotherapy, specifically nab-paclitaxel and gemcitabine, for metastatic pancreatic cancer. This trial represents a crucial step in assessing the efficacy and safety of cambritaxestat in a clinical setting, potentially offering a new therapeutic avenue for patients facing this challenging condition.

The unique mechanism of action of cambritaxestat as an autotaxin inhibitor presents a multifaceted approach to combating tumors, encompassing direct inhibition of cancer cells, stimulation of immune effector cells, and suppression of fibrotic processes. This comprehensive strategy holds promise for enhancing treatment outcomes by facilitating better access of drugs and immune cells to the tumor microenvironment.

Recent translational research published in esteemed journals such as ESMO’s Immuno-Oncology and Technology (IOTECH), Cancer Research, the Journal of Experimental & Clinical Cancer Research, and Cancers has underscored the considerable potential of cambritaxestat across various cancer models, including pancreatic cancer. Preclinical studies have demonstrated significant reductions in metastasis and tumor outgrowth, alongside favorable safety and tolerability profiles observed in healthy volunteers.

Catherine Pickering, the Chief Executive Officer of iOnctura, emphasized the urgent need for novel therapies in pancreatic cancer, a disease with alarmingly low survival rates despite recent advancements in treatment. With Orphan Drug Designation in hand, iOnctura is poised to expedite the clinical development of cambritaxestat, with the ultimate aim of delivering a much-needed treatment option to patients grappling with limited therapeutic choices.


Joan David-Leonhard

Joan David Leonhard is a recent Pharm.D graduate with a strong passion for the pharmaceutical industry and a particular interest in pharmaceutical media and communication. Her brief internship experience includes roles in pharmacy where she built strong patient-pharmacist relationships and a pharmaceutical media internship where she actively contributed to drug information articles, blog posts, social media engagement, and various media projects.
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