Rafael Pharmaceuticals Achieves Target Enrollment of 500 Patients in Pivotal Phase 3 Trial

Rafael Pharmaceuticals, Inc. recently announced it has reached its target enrollment of 500 patients in its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500), which is evaluating the efficacy and safety of Rafael’s lead compound CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy. The global study will remain open for a short while longer and continue to enroll additional patients.

“The speed at which we were able to fully enroll the Phase 3 trial, even amid the coronavirus pandemic, is demonstrative of pancreatic cancer patients’ needs for new and more effective treatment options for this devastating disease,” said Sanjeev Luther, President and CEO of Rafael. “This achievement is only possible because of the unwavering dedication and commitment of the Rafael and Covance teams, study investigators, hospital staff at the trial sites, patients and their families. We are also grateful to the FDA for working closely with us to ensure the continued safe enrollment of patients during the pandemic.”

AVENGER 500 is one of the largest Phase 3 clinical trials currently open in metastatic pancreatic cancer, taking place in 75 sites around the globe in countries including the United States, South Korea, Israel, Belgium, France, and Germany, among others.

“Devimistat has revived our hope in cancer metabolism,” said Philip A. Philip, MD, Professor of Oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University, and principal investigator of the trial. “Earlier phase trials of devimistat have demonstrated promising results. We are very hopeful for positive results, as we begin to compile and assess the Phase 3 data.”

“Metastatic pancreatic cancer is one of the most difficult diseases to treat,” said Timothy S. Pardee, MD, PhD, Co-Chief Medical Officer of the company. “Creating a trial to successfully evaluate the efficacy of devimistat while maintaining the highest level of patient safety was our top priority when designing the trial.”

“Indeed, this is a major milestone for our company,” said Howard Jonas, Chairman of Rafael. “But more importantly, we are hopeful that this may serve as a significant milestone for the thousands of pancreatic cancer patients worldwide who oftentimes have a poor prognosis with few treatment options currently available to them.”

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 US 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.

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: RFL). For more information, visit www.rafaelpharma.com.