Tuesday, April 16, 2024

Cambritaxestat Receives Orphan Drug Status for Treatment of Highly Fibrotic Cancers

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Cambritaxestat after analyzing its chemical and biological properties, has been granted an Orphan Drug Designation by the US regulator. The drug has demonstrated a promising non-clinical safety and efficacy profile. This status offers various benefits that will aid in the development and marketing of the drug.

Cambritaxestat is being developed as a therapeutic option for highly fibrotic cancers. The World Health Organization has also proposed cambritaxestat as the International Nonproprietary Name (INN) following a separate application process. The oral therapy is intended to target highly fibrotic cancer indications that overexpress autotaxin. These include cancers such as pancreatic, liver, colorectal, ovarian, and breast cancers.

Novel Cancer Drug Cambritaxestat Targets Metastatic Pancreatic Cancer in Pioneering Phase I Study

Cambritaxestat has demonstrated preclinical efficacy in hindering the growth and proliferation of cancer cells. It also induces immune cell infiltration into tumors and prevents the development of fibrosis. The drug is currently undergoing testing in combination with standard chemotherapy agents nab-paclitaxel and gemcitabine. This is part of the Phase I AION-02 study for metastatic pancreatic cancer.

This study began in the fourth quarter of 2022 and represents a novel treatment strategy that offers a three-pronged attack on tumors. Translational research has highlighted the potential of cambritaxestat in various cancer models, particularly pancreatic cancer. Preclinical data revealed that it significantly reduces metastasis and tumor growth, alongside safe and tolerable dosing in healthy subjects.

In addition to cambritaxestat, iOnctura has advanced another candidate, Roginolisib, into mid-stage clinical trials. They also prepare their TGF-β pathway inhibitor, IOA-359, for first-in-human studies. There is an urgent need to develop new therapies for pancreatic cancer. This is currently the third largest cause of death by cancer in the US and the fourth in Europe.

Orphan Drug

Cambritaxestat Receives Orphan Drug Designation, Offering New Hope for Pancreatic Cancer Patients

Survival rates for patients with pancreatic cancer have improved in recent years. However, it still stands at just 13% after five years. The orphan drug designation will support the acceleration of cambritaxestat through the clinic to provide a new treatment option for patients with limited options.

In the battle against highly fibrotic cancers such as pancreatic, liver, colorectal, ovarian, and breast cancers, the development and marketing of cambritaxestat could prove crucial. Its potential to prevent fibrosis development and reduce metastasis and tumor growth, combined with its tolerable dosing, could offer new hope for patients with limited treatment options. The Orphan Drug Designation from the US regulator is a significant step forward in bringing this promising drug to the market.

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