Rafael Pharmaceuticals to Present Plan for Phase 3 Trial (AVENGER 500) of devimistat (CPI-613®) in Combination with Modified FOLFIRINOX as a First Line Treatment for Metastatic Pancreatic Cancer at 2019 Gastrointestinal Cancers Symposium

Newark, New Jersey, UNITED STATES

Newark, NJ, Jan. 17, 2019 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that the study plan for a Phase 3 Trial (AVENGER 500) of devimistat in combination with modified FOLFIRINOX as a first-line treatment for patients with metastatic pancreatic cancer will be presented at the Annual Gastrointestinal Cancers Symposium, held by the American Society of Clinical Oncology (ASCO) in San Francisco, California on Friday, January 18, 2019.

Devimistat is Rafael’s lead altered metabolism directed (AMD) drug candidate, a first-in-class anticancer compound designed to disrupt the altered mitochondrial metabolism in cancer cells.

AVENGER 500 will compare the safety and efficacy of FOLFIRINOX (control arm) with devimistat in combination with modified FOLFIRINOX. Patients 18 – 75 years old of both sexes with metastatic (stage IV) pancreatic adenocarcinoma, not previously treated for metastatic disease and with ECOG performance status of 0 – 1 are eligible for enrollment in this study. This is a prospective, open label, multinational randomized trial and will be conducted in 9 different countries including the United States, six European Union countries, Israel and South Korea. Dr. Philip Agop Philip from Karmanos Cancer Institute of Wayne State University is the principal investigator of this trial. More information on the trial is available at www.clinicaltrials.gov (NCT03504423).

Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals, commented: “This is a multinational trial in ~100 sites. We are very excited to present the study design of this trial at the 2019 Annual Gastrointestinal Cancers Symposium.”

Philip A. Philip, MD, PhD, FRCP, the Principal Investigator of this study, commented: “Pancreatic cancer is the deadliest cancer worldwide with very limited treatment options. The current data on devimistat in combination with modified FOLFIRINOX in patients battling pancreatic cancer is immensely promising. I am, therefore, delighted to be involved in this important phase 3 trial.”

Timothy S. Pardee, MD, PhD, FACP, Chief Medical Officer of Rafael Pharmaceuticals, commented: “Devimistat targets tumors in a truly unique way and earlier clinical results suggest it leverages the metabolic vulnerability inherent in pancreatic cancer to improve outcomes in this deadly disease. I am very excited at the opportunity to discuss this important trial at such a preeminent meeting.”

About devimistat:
Devimistat is a first-in-class drug, developed based on the Altered Metabolism Directed (AMD) platform. Devimistat targets the altered regulation of metabolic processes specific to cancer cells. It is designed to be highly specific, simultaneously attack multiple targets, minimally toxic and have broad spectrum activity across a wide variety of cancers. Devimistat is being or has been evaluated in 19 ongoing or completed trials as a single agent, as well as in combination with standard drug therapy for hematological malignancies and solid tumors. To date, over 330 patients have received one or more doses of devimistat. In a phase I study in pancreatic cancer, devimistat in combination with modified FOLFIRINOX exhibited a 61% objective response rate (ORR), median overall survival (OS) of 19.9 months and median progression-free survival (PFS) of 9.9 months. In elderly patients with AML, devimistat in combination with high dose cytarabine and mitoxantrone exhibited 52% complete remission (CR) + CR with incomplete hematologic recovery (CRi) and 12.4 months median OS. In T-cell lymphoma, devimistat in combination with bendamustine exhibited a 75% objective response rate. Devimistat has been granted orphan drug designation by the U.S. FDA for pancreatic cancer, AML, MDS, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and AML.

About Pancreatic Cancer:
Pancreatic cancer is an extremely deadly disease , with more than 95% of patients affected dying of their disease1. Although its incidence in the United States is relatively low (estimated new cases in 2018 was 55,440)2, pancreatic cancer became the third leading cause of cancer-related deaths in 20163 and is expected to be the second cause of death after 20204 . There are a number of types of pancreatic cancer. The predominant one is adenocarcinoma, which accounts for approximately 95% of exocrine pancreatic cancers.5 Because the disease typically does not present with recognizable or distinctive symptoms in its early stages, when it is diagnosed, it is usually quite advanced and affords limited treatment options. Chemotherapy is the only treatment option for metastatic pancreatic cancer. National Comprehensive Cancer Network (NCCN) treatment guidelines recommend FOLFIRINOX or gemcitabine plus nab-paclitaxel for first-line treatments for patients who are healthy enough to tolerate them and have a support system for a relatively aggressive medical therapy, but efficacy response is limited (FOLFIRINOX6: ORR: 31.6%, median OS: 11.1 months, median PFS: 6.4 months; gemcitabine in combination with nab-paclitaxel7: ORR: 23%, median OS: 8.5 months, median PFS: 5.5 months). However, FOLFIRINOX is regarded as too toxic for use in elderly and poor performance status patients. For patients who wish to pursue cancer-directed therapy but cannot manage such aggressive treatments, gemcitabine alone or alternate choices are recommended. In general, clinical trials are highly recommended for patients with metastatic pancreatic cancer. Another option for patients who cannot tolerate the toxic effects of FOLFIRINOX is to be treated with a modified dosing regimen of FOLFIRINOX, which significantly decreases the adverse side effects (neuropathy, diarrhea, neutropenia) associated with FOLFIRINOX. The 5-year survival rate of patients with metastatic (stage IV) pancreatic cancer is only 1%.8 There is a great medical need for more effective first-line systemic therapies that are less toxic and more effective.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals, Inc. is a clinical-stage, metabolic oncology therapeutics company. Rafael’s primary objective is to develop innovative, highly selective, well tolerated and highly effective anti-cancer agents by selectively targeting altered metabolism in cancer cells. Rafael’s first-in-class clinical lead compound, CPI-613, is being evaluated in multiple Phase I, I/II, and III clinical studies. CPI-613 has been granted orphan drug designation for the treatment of pancreatic cancer, acute myeloid leukemia (AML), peripheral T-cell lymphoma (PTCL), Burkitt Lymphoma and myelodysplastic syndromes (MDS). Rafael Pharmaceuticals’ investors include Rafael Holdings, Inc. (NYSE American: RFL).
For more information, visit http://www.rafaelpharma.com/.


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  7. Hoff DV, Ervin T, Arena FP, et al. Increased survival in pancreatic cancer with nab-paclitaxel plus gemcitabine. New England Journal of Medicine. 2013;369:1691-703
  8. Cancer.org. (2018). Pancreatic Cancer Survival Rates, by Stage. [online] Available at: https://www.cancer.org/cancer/pancreatic-cancer/detection-diagnosis-staging/survival-rates.html [Accessed 7 Dec. 2018].

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.

Sanjeev Luther
President & CEO Rafael Pharmaceuticals, Inc. 

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.