(By Balaseshan) Coronado Biosciences Inc. (NASDAQ:CNDO) said it has received orphan-drug designation from the U.S. Food and Drug Administration (FDA) for CNDO-109-activated allogeneic natural killer cells for the treatment of acute myeloid leukemia (AML).
The Burlington, Massachusetts-based biopharmaceutical company said CNDO-109 is a biologic that activates the immune system's natural killer cells to seek and destroy cancer cells.
Preclinical studies of CNDO-109 have demonstrated activity in multiple myeloma, breast cancer, prostate cancer and ovarian cancer and, based on data obtained from a Phase 1 investigator-sponsored clinical trial in patients with AML, the company believes early efficacy was observed.
Coronado currently intends to initiate a Phase 1/2 dose escalation trial in AML patients in the second half of 2012.
Acute myeloid leukemia is cancer that starts inside bone marrow, the soft tissue inside bones that helps form blood cells. The cancer, which develops quickly, grows from cells that would normally turn into white blood cells.
Orphan-drug designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S.
The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication.
It also provides tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
CNDO is trading up 3.14% at $5.58 on Tuesday. The stock has been trading between $4.95 and $11 for the past 52 weeks.