Rafael Pharmaceuticals Announces Expansion Into France of Pivotal Phase 3 Trial

Rafael Pharmaceuticals, Inc. recently announced the expansion of its Phase 3 clinical trial for patients with metastatic pancreatic cancer (AVENGER 500) into France. AVENGER 500 is a global, multicenter, open-label, randomized pivotal trial that is evaluating the efficacy and safety of CPI-613 (devimistat), Rafael’s lead compound, in combination with modified FOLFIRINOX (mFFX) as a first-line therapy in patients with metastatic adenocarcinoma of the pancreas. This announcement comes on the heels of the trial’s expansion into Israel. The trial will open in 13 sites throughout France, with enrollment already underway in select locations.

“Pancreatic cancer claims the lives of more than 90,000 European citizens every year, and France is leading as one of the top countries with a rise in cases over the past 30 years,” said Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals. “Despite these outcomes, pancreatic cancer receives less than 2% of cancer research funding in the region,  making it an essential location for us to open our Phase 3 clinical trial in metastatic pancreatic cancer. We aim to bring hope to those patients with significant unmet needs in France, and throughout Europe.”

Dr. Michel Ducreux, MD, PhD, Head of the Gastrointestinal (GI) Oncology Unit, Department of Medical Oncology and Director of Medical Affairs at Gustave Roussy Cancer Campus, will be the lead investigator on the clinical trials opening in France.

“It’s an honor to be chosen as the lead investigator for Rafael’s devimistat clinical trials that are opening in France,” said Dr. Ducreux. “My site at Gustave Roussy is the first to open the trial, both in France and in Europe, and I feel that by providing pancreatic cancer patients with access to this trial, we are also providing them with hope.”

The clinical trials in France will begin opening at the following nine sites, with others to be announced this fall: Gustave Roussy, Institut Régional du Cancer de Montpellier, Hôpital Beaujon, CHU de Nantes – Hôpital Nord Laennec, CHRU Brest – Hôpital Morvan, Institut de Cancérologie de Lorraine, CHU Hopitaux de Bordeaux – Hôpital Saint-André, CHU de Poitiers and Hôpital Privé Jean Mermoz.

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.

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, visit www.rafaelpharma.com.