Rafael Pharmaceuticals Announces In Vivo Study to Evaluate the Effects of CPI-613® (Devimistat) in Combination with Azacitidine and Venetoclax on Acute Myeloid Leukemia Models

Company advances mission to combat AML, as this research comes on the heels of its Phase 3 trial, actively enrolling globally

Newark, New Jersey, UNITED STATES

Cranbury, NJ, May 07, 2020 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, today announced that it has entered into a research collaboration with a major medical institution and renowned researchers to evaluate the efficacy of CPI-613® (devimistat) in combination with chemotherapy agents azacitidine and venetoclax on acute myeloid leukemia (AML) models.

“With generally poor responses to current therapies and a five-year survival rate of just 28%, there are significant gaps in treatments for adult AML patients,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “Fighting AML has long been a goal of ours, and this research explores further options in treating AML, in addition to our current AML trial. Should these experiments show efficacy of devimistat additional AML trials in the upfront setting will be considered. Combined with Rafael’s myelodysplastic syndrome (MDS) trial in development, this will cover the major myeloid malignancies and bring hope to patients suffering from these diseases.”

Rafael also has a Phase 3 clinical trial in AML to evaluate the efficacy of devimistat in combination with chemotherapy agents cytarabine and mitoxantrone. The trial is currently enrolling patients in nearly 50 sites globally. 

Rafael’s additional trials in hematological malignancies include a Phase 2 trial in Burkitt’s lymphoma/leukemia, a Phase 1/2 trial in myelodysplastic syndrome, and a Phase 1 trial in T-cell lymphoma.

About CPI-613® (devimistat) 
CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals is a leader in the growing field of cancer metabolism. The company is developing a new, first-in-class category of metabolic oncology therapeutics that attack hard-to-treat cancers by targeting the metabolic processes the disease needs to survive, grow and proliferate. Rafael Pharmaceuticals’ lead compound, CPI-613® (devimistat), is a highly selective, well-tolerated and effective anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitt’s and peripheral T-cell lymphomas. The Company's investors include Rafael Holdings, Inc. (NYSE AMERICAN: RFL). For more information, please visit 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.


Rafael Media Contact:
Holly Dugan
(201) 465-8019