Rafael Pharmaceuticals to Present Phase 1 Data on CPI-613® (Devimistat) in Patients with Locally Advanced or Metastatic Panc...

 Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, today announced that the positive results of a single-arm, open-label, Phase 1 study of CPI-613

®

 (devimistat) with gemcitabine and nab-paclitaxel in patients with locally advanced or metastatic pancreatic cancer, will be presented in a poster presentation at the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program. The results will be presented by Dr. Angela Tatiana Alistar, M.D., Medical Director of Gastrointestinal (GI) Medical Oncology at Morristown Medical Center and principal investigator on the trial.  

 Rafafel_Pharma_PHP_Graphic_Updated_V3The presentation titled, “Abstract #4635: A single-arm, open-label, Phase 1 study of CPI-613 (devimistat) in combination with gemcitabine and nab-paclitaxel for patients with locally advanced or metastatic pancreatic adenocarcinoma,” includes the results of a two-year Phase 1 study consisting of gemcitabine and nab-paclitaxel in 26 patients with locally advanced or metastatic pancreatic cancer to determine maximum tolerated dose (MTD), safety, and preliminary efficacy of devimistat in combination with chemotherapy. Overall, the treatment was well-tolerated and demonstrated positive results that show devimistat can be safely administered with gemcitabine and nab-paclitaxel at doses up to 1,500 m/g2.

“We’re proud to share our findings at ASCO 2020,  to announce the latest positive Phase 1 results of our lead compound devimistat in the treatment of patients with pancreatic cancer,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “These findings give us a glimpse of the promising work underway, as we continue the momentum of inspiring hope for patients with hard-to-treat cancers, including pancreatic cancer.”

“There is a significant need to enhance our current standard of care options in pancreatic cancer,” said Dr. Alistar. “When we started this trial, I anticipated good tolerance of devimistat in combination with the gemcitabine/nab-paclitaxel regimen and am thrilled to see that the results met this expectation in full.”

Presentation Details:Title:  Abstract #4635: A single-arm, open-label, Phase 1 study of CPI-613 (devimistat) in combination with gemcitabine and nab-paclitaxel for patients with locally advanced or metastatic pancreatic adenocarcinomaDate: May 29, 2020Time: 8:00 a.m. ETLocation: ASCO Meeting Library

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 represent 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 StatementThis 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.

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