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US FDA grants Orphan Drug Designation for iOnctura’s Autotaxin cancer therapy

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iOnctura’s autotaxin inhibitor cambritaxestat for the treatment of pancreatic cancer gets Orphan Drug Designation (ODD) by the US FDA.


IOnctura is a pioneering clinical-stage biotechnology company developing transformative cancer therapies.

After assessing its novel chemical and biological properties including an attractive non-clinical safety and efficacy profile, the US Food and Drug Administration (FDA) has granted Orphan Drug Status, conferring certain benefits during development and commercialisation.

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

Cambritaxestat is being developed as a first-in-class therapy for highly fibrotic cancer indications. The drug’s lead indication is metastatic pancreatic cancer where it is being combined with standard of care nab-paclitaxel and gemcitabine in the Phase I AION-02 study.

Inhibition of autotaxin is a novel treatment strategy that offers a three-pronged attack on the tumour through direct cancer cell inhibition, immune effector stimulation and inhibition of fibrotic processes, giving drugs and immune cells better access to the tumour.

Catherine Pickering, Chief Executive Officer, iOnctura, said: “There is an urgent need to develop new therapies for pancreatic cancer, which is currently the third-largest cause of death by cancer in the U.S., and the fourth in Europe. Although survival of patients with pancreatic cancer has improved in recent years, it still stands at just 13% after five years. This Orphan Drug Designation will support our goal to accelerate cambritaxestat through the clinic to provide a new treatment to patients with limited options.”



Source: press release