Rafael Pharmaceuticals Reaches Milestone of 100 Patients Enrolled in Pivotal Phase 3 Trial (AVENGER 500) of CPI-613®️ (devimistat) for Patients with Metastatic Pancreatic Cancer

Cranbury, NJ, Oct. 08, 2019 (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 enrolled 100 patients in its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500). The multicenter, open-label, randomized trial is evaluating the efficacy and safety of Rafael’s lead compound CPI-613®️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy in patients with metastatic adenocarcinoma of the pancreas. The global study is active and still open for patient enrollment in sites across the United States, France, Israel and South Korea.

“Enrolling 100 patients in our pancreatic cancer clinical trial is an important milestone for Rafael,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “Pancreatic cancer is one of the deadliest cancers in the world and patients with metastatic pancreatic cancer, specifically, have a very low five-year survival rate of 3%. Reaching this milestone reflects our mission of improving the odds for patients with unmet medical needs, and is a major step towards the ultimate goal of enrolling 500 patients to the trial. We would like to extend our sincere gratitude to all the patients and doctors who have participated in the trail so far.”

Philip A. Philip, M.D., Professor of Oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University, is the principal investigator on Rafael’s Phase 3 clinical trial for patients with metastatic pancreatic cancer. Dr. Philip developed the rationale and design of the devimistat clinical trial and supervises the conduct of the ongoing clinical investigation.

“Reaching 100 patients is a monumental moment and brings the devimistat trial one step closer to conducting the interim analysis, which is the required first collection and analysis of data for this phase of the trial,” said Dr. Philip. “Pancreatic cancer has remained a difficult disease to treat, and I am hopeful that the data from this trial will demonstrate devimistat’s potential in treating metastatic pancreatic cancer.”

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 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
[email protected]
(201) 465-8019