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FDA Grants Orphan Drug Designation for iOnctura's Innovative Autotaxin Cancer Treatment

Friday, March 08, 2024

IOnctura, a pioneering biotechnology company focused on developing innovative cancer therapies, has announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its autotaxin inhibitor, cambritaxestat, for the treatment of pancreatic cancer.

After assessing its unique chemical and biological properties, alongside its promising non-clinical safety and efficacy profile, the US FDA has awarded Orphan Drug Status to cambritaxestat, providing certain benefits during its development and commercialization.

Following a separate submission process, the World Health Organization has proposed the International Nonproprietary Name (INN) for cambritaxestat.

Cambritaxestat is being developed as a first-in-class therapy for highly fibrotic cancer indications, with its lead indication being metastatic pancreatic cancer. It is being investigated in combination with the standard of care drugs nab-paclitaxel and gemcitabine in the Phase I AION-02 study.

The inhibition of autotaxin offers a novel treatment approach, targeting tumors through direct cancer cell inhibition, immune effector stimulation, and inhibition of fibrotic processes, thereby improving drug and immune cell access to the tumor.

Recent translational research, published in respected journals such as Immuno-Oncology and Technology (IOTECH), Cancer Research, the Journal of Experimental & Clinical Cancer Research, and Cancers, has demonstrated the potential of cambritaxestat in various cancer models, including pancreatic cancer. Across these studies, cambritaxestat showed significant reductions in metastasis and tumor growth in preclinical models, with safe and well-tolerated dosing in healthy volunteers.

Catherine Pickering, CEO of iOnctura, stated, "The urgent need for new therapies for pancreatic cancer, currently the third leading cause of cancer-related deaths in the US and the fourth in Europe, is undeniable. While there has been some improvement in survival rates for patients with pancreatic cancer in recent years, the five-year survival rate remains at a mere 13%. This Orphan Drug Designation will support our efforts to accelerate cambritaxestat through clinical development and provide a new treatment option for patients with limited alternatives."



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