Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics, today announced that it has crossed the midpoint to its first interim analysis in enrollment for its pivotal Phase 3 clinical trial (ARMADA 2000) of CPI-613® (devimistat) for relapsed or refractory acute myeloid leukemia (AML). The multicenter, open-label, randomized pivotal trial is evaluating the efficacy and safety of its lead compound devimistat in combination with high dose cytarabine and mitoxantrone (CHAM) in older patients.
The Food and Drug Administration (FDA) worked closely with Rafael to make changes to the protocol to help address enrollment challenges; the trial protocol modifications significantly increased patient enrollment.
"The majority of relapsed AML patients will pass away within five years of diagnosis," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "This enrollment milestone provides hope to these patients, as we work to deliver more effective treatments to patient populations that have few viable options. We are deeply appreciative of all of our principal investigators, patients and caregivers for their continued support of this trial."
The global study is currently active across more than 60 sites in countries including the United States, Canada, Europe, India, South Korea and Australia. While the COVID-19 pandemic has disrupted many clinical trials, Rafael has expanded safety protocols to continue enrollment, maintaining the safety and wellbeing of patients as the company's foremost priority.
Jorge Cortes, M.D., Director of the Georgia Cancer Center at Augusta University, is the principal investigator on this Phase 3 clinical trial. Dr. Cortes developed the rationale and design of the trial and is supervising the conduct of the ongoing clinical investigation.
"Actively and safely enrolling patients and reaching the midpoint to the first interim analysis is an achievement worth noting, especially given the challenges for clinical trial enrollment across the globe due to the coronavirus pandemic," said Dr. Cortes. "Relapsed or refractory AML remains a significant challenge for older patients, so it is encouraging to see enrollment continue to grow."
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.
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