June 20, 2017
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FDA grants orphan drug designation to IT-139 for pancreatic cancer

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The FDA granted orphan drug designation to IT-139 for the treatment of pancreatic cancer, according to the drug’s manufacturer.

“IT-139 (Intezyne Technologies) downregulates the stress-induction of GRP78 in cancer cells, a critical cell survival protein that is associated with both drug resistance and tumor proliferation,” Suzanne Bakewell, PhD, vice president and program manager for IT-139, said in a company-issued press release. “GRP78 expression is not elevated in the same way in normal cells, leaving them unaffected.”

Results of a phase 1 trial showed IT-139 monotherapy demonstrated antitumor activity in numerous tumor types. The agent appeared well tolerated and side effects were manageable.

“Our receipt of an orphan drug designation for IT-139 in pancreatic cancer is an exciting regulatory milestone for Intezyne and a critical step toward clinical advancement of this promising first-in-class therapy addressing an unmet need,” Carolyn Paradise, MD, chief medical officer of Intezyne, said in the release.

Pancreatic cancer is the fourth most common cancer in the United States. Most of the approximately 54,000 new cases diagnosed each year are detected after the cancer has spread locally or metastasized to other organs.

“Sadly, pancreatic cancer has proven extremely difficult to treat, with 1-year and 5-year survival rates of only 20% and 8%, respectively — rates which we hope to improve with IT-139,” Paradise said.

The company plans to investigate IT-139 in at least one combination phase 1b/2a study next year.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.