Novel First-in-Class Anti-Cancer Agent in Development by Niiki Pharma Shows Promising Phase I Results

HOBOKEN, N.J. and PHILADELPHIA, June 1, 2011 /PRNewswire/ -- Niiki Pharma announced today that it will present interim data from the ongoing Phase I clinical study for its lead product, NKP-1339, at the 2011 American Society for Clinical Oncology meeting in Chicago, IL. NKP-1339 is a novel transferrin targeted ruthenium based anti-cancer compound. The intracellular targets of NKP-1339 include GRP78, a key regulator of mis-folded protein processing. In preclinical studies, in vivo and in vitro activity has been demonstrated against multiple tumor types, including those resistant to other anti-cancer agents.

The NKP-1339 Phase I trial is a dose-ascending trial and determines the safety, tolerability, maximum tolerated dose (MTD) and pharmacokinetics of NKP-1339. A total of 16 patients with metastatic solid tumors resistant to standard therapies have been treated to date at six different dose levels. NKP-1339 treatment has been generally well tolerated, with the most common drug related side effects of grade 1-2 fever and mild flu-like symptoms. MTD has not been reached and NKP-1339 dose escalation continues. Signs of anti-tumor activity (stable disease greater than or equal to four months; tumor regression) have been observed. A patient with a neuroendocrine tumor (NET) of the small intestine has been on NKP-1339 therapy for over 13 months with a continuing minor response. The patient remains on NKP-1339 therapy. Another patient with the metastatic gastrinoma NET exhibited six months stable disease, and two patients with metastatic non-small cell lung cancer exhibited four months stable disease.

The NKP-1339 Phase I trial is being led by Dr. Daniel Von Hoff, Translational Genomics Institute, Arizona, and Dr. Howard Burris, Sarah Canon Research Institute, Tennessee. Reflecting on NKP-1339's novel mechanism of action, Dr. Von Hoff noted "Resistance to anti-cancer therapy remains a major challenge in treatment of patients with metastatic cancer. First-

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