Rafael Pharmaceuticals Enrolls More Than 75% of Patients Needed for Pivotal Phase 3 Trial

Rafael Pharmaceuticals, Inc. recently announced it has enrolled more than 75% of the 500 patients needed for 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.

“We are continuing to enroll patients ahead of schedule, as the patients in our trials suffer from life-threatening diseases and are in dire need of treatment; patient safety is our top priority,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “In order to support our clinical trial sites and enrolled patients during this unsettling and unprecedented time, we have established a COVID-19 task force to closely monitor our clinical trials and make changes as needed. We want to express our deepest gratitude to all of the principal investigators, nurses, hospital staff, patients, and their families for their continued support and cooperation.”

“To Save a Life is to Save a Universe” – Rafael’s motto – represents its dedication to patients who suffer from rare cancers globally, as well as to its employees and the communities it serves. As such, the company has implemented measures to help ensure the continuity of its clinical trials and programs, while maintaining the safety of patients, clinicians, and employees. The COVID-19 task force will closely monitor all clinical trials and collect data relevant to the patients’ study treatments and schedules.

Rafael is working closely with the clinical trial sites to preempt and promptly address any potential challenges and ensure that clinical supplies are available in sufficient quantities in order to support the continuity of care of all enrolled patients. Each clinical trial site has implemented their own safety measures based on guidelines from local authorities. If patients have questions about their clinical trial, they should reach out to the clinical trial coordinator at their medical center.

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 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. For more information, visit www.rafaelpharma.com.