Clinical Cancer Research Publishes Phase I Data from a Study of Rafael Pharmaceuticals' Lead CPI-613 Compound in Combination with High-Dose Cytarabine and Mitoxantrone for Relapsed or Refractory Acute Myeloid Leukemia

Newark, New Jersey, UNITED STATES

Newark, NJ, April 12, 2018 (GLOBE NEWSWIRE) --

Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that data from the Phase I clinical trial evaluating CPI-613 in Combination with High-Dose Cytarabine and Mitoxantrone for Relapsed or Refractory Acute Myeloid Leukemia (AML) has been published in the recent online issue of AACR’s Clinical Cancer Research. CPI-613 is the Company’s lead drug candidate, a first-in-class anticancer compound designed to disrupt the altered energy production pathways in cancer cells by targeting mitochondrial metabolism.

This Phase I study explored the role of mitochondrial metabolism in chemotherapy response and determined the MTD, efficacy, and safety of CPI-613 combined with high-dose cytarabine and mitoxantrone in patients with relapsed or refractory AML. The study found that CPI-613 sensitized AML cells to chemotherapy, indicating that mitochondrial metabolism is a source of resistance.

The study enrolled 67 patients and 62 were evaluable for response. The overall response rate was 50% (26CR+5CRi/62). Median survival was 6.7 months. In patients over 60 years old, the CR/CRi rate was 47% (15/32) with a median survival of 6.9 months. The response rate for patients with poor-risk cytogenetics was also encouraging, with 46% (11/24 patients) achieving a CR or CRi. The data supports the conclusion that the addition of CPI-613 to chemotherapy is a promising approach in older patients and those with poor-risk cytogenetics. Click for full article.

“These data are very encouraging especially for patients 60 years of age or older who have historically done very poorly with this disease. We look forward to the opening of our randomized study to further investigate this promising approach, said Timothy Pardee, M.D., Ph.D., Chief Medical Officer of Rafael Pharmaceuticals, and Associate Professor of Medicine and Director of leukemia translational research at Wake Forest Baptist Medical Center.

Dr. Pardee is currently running a Leukemia & Lymphoma Society (LLS) Man & Woman of the Year fundraising campaign. To donate now, please visit Dr. Pardee’s fundraising campaign homepage.

About Rafael Pharmaceuticals, Inc.

Rafael Pharmaceuticals, Inc. is a clinical-stage, oncology-focused pharmaceutical company committed to the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. Rafael’s primary objective is to develop highly selective and effective agents with minimal toxic effects on normal cells and tissues. Rafael’s first-in-class clinical lead compound, CPI-613 is being evaluated in multiple Phase I, I/II, and II clinical studies. CPI-613 has been granted orphan drug designation for the treatment of pancreatic cancer, AML, myelodysplastic syndromes (MDS), Burkitt’s lymphoma, and T-cell lymphoma. For more information, visit

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Sanjeev Luther
Chief Executive Officer, Rafael Pharmaceuticals, Inc.

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.