Rafael Pharmaceuticals Announces Initiation of Phase II Trial of CPI-613 in Combination with Modified FOLFIRINOX in Locally Advanced Pancreatic Cancer

Newark, New Jersey, UNITED STATES

Newark, NJ, Jan. 09, 2019 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced the initiation of a phase II open-label trial in collaboration with University Hospitals Cleveland Medical Center to evaluate the efficacy and safety of investigational drug CPI-613 in combination with modified FOLFIRINOX (mFFX) in patients with localized borderline and unresectable pancreatic cancer.

The primary objective of this study is to determine the potential increase in overall survival (OS) when CPI-613 is administered in combination with mFFX and to further assess the safety of the CPI-613+ mFFX combination. Pancreatic adenocarcinoma patients 19 – 80 years old of both sexes with ECOG performance status 0 – 1 are eligible for enrollment. More information on the trial is available at www.clinicaltrials.gov (NCT03699319).

The safety, tolerability and efficacy of CPI-613 in combination with mFFX was previously evaluated in patients with first line metastatic pancreatic cancer in a single-center, open-label, dose-escalation study (CCCWFU 57112, NCT01835041). A total of 20 patients were dosed in this study. The maximum tolerated dose of CPI-613 was 500 mg/m2. Two patients enrolled at a higher dose of 1000 mg/m2 and both experienced dose-limiting toxicity. Overall, the treatment was well tolerated. No deaths due to adverse events were reported and no patients died while on active treatment. Of the 18 patients given the maximum tolerated dose, 61% achieved an objective response (ORR) with a median overall survival (OS) of 19.9 months and median progression free survival (PFS) of 9.9 months. This efficacy is substantially higher than the current standard of care (FOLFIRINOX1: ORR: 31.6%, OS: 11.1, PFS: 6.4; gemcitabine in combination with nab-paclitaxel2: ORR: 24%, OS: 8.5, PFS: 5.5). Given the favorable safety and efficacy profiles of CPI-613 in combination with mFFX, further evaluation of CPI-613 in pancreatic cancer was warranted.

Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals, commented: “Our motto, ‘To Save A Life Is To Save A Universe,’ illustrates our desire to develop potential treatments for patients with significant unmet clinical need. Initiation of this trial in collaboration with University Hospitals Cleveland Medical Center is a significant milestone in that direction.”

Jeffery Hardacre, MD, a pancreatic surgeon and lead investigator for the study, commented, “University Hospitals is committed to leading the way in the fight against pancreatic cancer. Through this trial, we hope to identify a strategy to extend the lives of patients with pancreatic cancer, and increase the patient eligibility for surgery for those with locally advanced disease.”

About CPI-613:
CPI-613 is a first-in-class drug developed through Rafael Pharmaceutical’s Altered Metabolism Directed (AMD) platform. CPI-613 targets the altered regulation of metabolic processes specific to cancer cells. It is highly specific, simultaneously attacks multiple targets, minimally toxic and exhibits a broad spectrum activity across a wide variety of cancers. CPI-613 has been evaluated in 18 ongoing or completed trials as a single agent, as well as in combination with standard drug therapies for hematological malignancies and solid tumors. To date, over 300 patients have received one or more doses of CPI-613. The drug has exhibited very good signals of efficacy with excellent response rates and extended durations of response in several tumor types. In pancreatic cancer, CPI-613 in combination with modified FOLFIRINOX exhibited an objective response rate of 61%, median overall survival of 19.9 months and median progression free survival of 9.9 months. In elderly patients with AML, CPI-613 in combination with high dose cytarabine and mitoxantrone exhibited 52% CR+CRi and 12.4 months median overall survival. In both these trials, CPI-613 combinations were substantially more efficacious than standard therapies. In T-cell lymphoma, CPI-613 in combination with Bendamustine exhibited a 75% objective response rate. CPI-613 also exhibited a very good safety profile both as a single agent and in combination with other standard-of-care drugs. CPI-613 has been granted orphan drug designation by the U.S. FDA for pancreatic cancer, AML, MDS, peripheral T-cell lymphoma and Burkitt Lymphoma, and has been granted orphan drug designation by the EMA for pancreatic cancer and AML.

About Pancreatic Cancer:
Pancreatic cancer is an extremely deadly disease, with a mortality rate of more than 95%. Although its prevalence in the USA is relatively low (53,070 individuals per year), pancreatic cancer became the third leading cause of cancer-related deaths in 2016 and is expected to become the second leading cause of death by 2030.3 There are a number of types of pancreatic cancer.  The predominant one is adenocarcinoma, which accounts for approximately 95% of cases. Because the disease typically does not present with recognizable/distinctive symptoms in its early stages, when it is diagnosed, the disease is usually quite advanced and affords only limited treatment options.4-5

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals, Inc. is a clinical-stage, metabolic oncology therapeutics company committed to the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. 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). The Company's investors include Rafael Holdings, Inc. (NYSE American: RFL). For more information, visit http://www.rafaelpharma.com/.


  1. Conroy T, Desseigne F, Ychou M, et al. FOLFIRINOX versus Gemcitabine for Metastatic Pancreatic Cancer. New England Journal of Medicine. 2011;364(19):1817–1825. doi:10.1056/nejmoa1011923.
  2. 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
  3. Surveillance Epidemiology and End Results Program: Pancreas Cancer. 2016; https://seer.cancer.gov/statfacts/html/pancreas.html.
  4. Cascinu S, Falconi M, Valentini V, Jelic S. Pancreatic Cancer: ESMO Clinical Practice Guidelines for Diagnosis, Treatment and Follow-Up. Annals of Oncology: Official Journal of the European Society for Medical Oncology. 2010;21 Suppl 5:v55-58.
  5. Vaccaro V, Sperduti I, Vari S, et al. Metastatic Pancreatic Cancer: Is There a Light at the End of the Tunnel? World Journal of Gastroenterology. 2015;21(16):4788-4801.

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Sanjeev Luther
President & CEO Rafael Pharmaceuticals, Inc. 

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.