Rafael Pharmaceuticals Announces Published Data on CPI-613® (Devimistat) From Phase 2 Clinical Trial for Refractory Burkitt Lymphoma at the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition

Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a company focused on the growing field of cancer metabolism-based therapeutics, announced a publication on its Phase 2 clinical trial of CPI-613 ® (devimistat) for the treatment of relapsed or refractory Burkitt lymphoma/leukemia or high-grade B-cell lymphoma at the American Society of Hematology (ASH) Annual Meeting.

CRANBURY, N.J., Dec. 13, 2021 (GLOBE NEWSWIRE) --Rafael Pharmaceuticals (Formerly known as Cornerstone Pharmaceuticals, Inc.), (“Rafael” or the “Company”), a company focused on the growing field of cancer metabolism-based therapeutics, announced a publication on its Phase 2 clinical trial of CPI-613® (devimistat) for the treatment of relapsed or refractory Burkitt lymphoma/leukemia or high-grade B-cell lymphoma at the American Society of Hematology (ASH) Annual Meeting and Exposition.

Highlights from the published study, “Complete Remission with Devimistat (CPI-613) in Refractory Burkitt Lymphoma,” include a patient with four prior therapies, who had a near complete metabolic remission after four cycles of devimistat, and complete remission after cycle seven. As of July 2021, he was in cycle eleven. Typically, such patients have no viable treatment options.

The ongoing study is currently recruiting at MD Anderson Cancer Center, City of Hope, Memorial Sloan Kettering Cancer Center and Massachusetts General Hospital. Rafael plans to open four additional sites at key institutions across the United States in Q1 2022.

“We are honored that our research was selected for publication at this year’s ASH meeting and are grateful for the opportunity to continue to raise awareness around this highly aggressive disease, as we work towards developing viable therapies for those suffering from this and other rare cancers,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “Devimistat and cancer metabolism have the potential to open new doors of remission and survival for patients suffering from rare cancers and offer an opportunity to fill the significant gap in treatment, as our research demonstrates.”

The Annual ASH Meeting and Exposition is being held at the Georgia World Congress Center in Atlanta – and virtually – from December 11-14, 2021.

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 cellular stress and 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 side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients’ benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt’s lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt’s lymphoma.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals is focused on the growing field of cancer metabolism. The company is developing a new, first-in-class category of metabolic oncology therapeutic candidates that are designed to attack hard-to-treat cancers by targeting the metabolic processes that these cancers need to survive, grow and proliferate. Rafael Pharmaceuticals’ lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. The Company’s investors include Rafael Holdings, Inc. (NYSE: RFL). On June 21, 2021, we announced that we have entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. 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.


Media Contact: Morrena Villanueva rafael@antennagroup.com (609) 201-3408

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