|Press Release from Rafael Pharmaceuticals: 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
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
April 12, 2018 11:00 ET | Source: Rafael Pharmaceutical Inc.
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 http://www.rafaelpharma.com/.
Safe Harbor Statement
This press release contains forward-looking statements. These statements relate to future events or the company’s future financial performance. In some cases, you can identify forward-looking statements by terminology such as "may", "will", "should", "expect", "plan", "anticipate", "believe", "estimate", "predict", "potential" or "continue", the negative of such terms, or other comparable terminology. These statements are only predictions. Actual events or results may differ materially from those in the forward-looking statements as a result of various important factors. Although we believe that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as a representation by the company, or any other person, that such forward-looking statements will be achieved. The business and operations of the company are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We undertake no duty to update any of the forward-looking statements, whether as a result of new information, future events or otherwise. In light of the foregoing, readers are cautioned not to place undue reliance on such forward-looking statements.
Chief Executive Officer, Rafael Pharmaceuticals, Inc.
Public Relations, Rafael Pharmaceuticals, Inc.
|Apr 13, 2018