$RFL EMA Grants Orphan Drug Designation to devimistat (CPI-613) in Pancreatic Cancer

Newark, NJ, Jan. 10, 2019 — Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to devimistat (CPI-613®), the Company’s lead Altered Metabolism Directed (AMD) platform drug candidate, for the treatment of Metastatic Pancreatic Cancer.

Pancreatic cancer is a deadly disease, with a mortality rate of more than 95% within five years. Because pancreatic cancer does not present with recognizable symptoms in its early stages, most patients are diagnosed with advanced disease that affords limited treatment options. Chemotherapy is currently 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 who have a support system for these relatively aggressive medical therapies.

Devimistat is a first-in-class drug developed based on Rafael’s Altered Metabolism Directed (AMD) platform. Devimistat targets the altered regulation of metabolic processes specific to cancer cells. It is highly specific, simultaneously attacking multiple targets, minimally toxic and has broad spectrum activity across a wide variety of cancers. Devimistat is currently being evaluated in 7 trials as a single agent, as well as in combination with standard drug therapies for hematological malignancies and solid tumors. To date, over 300 subjects have received one or more doses of devimistat with encouraging response rates and durations of response in several tumor types. In pancreatic cancer, devimistat 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 – all substantially higher than current therapy. Devimistat also exhibited a very good safety profile both as a single agent and in combination with other standard-of-care drugs. Devimistat has previously been granted orphan drug designation for pancreatic cancer, AML, MDS, peripheral T-cell lymphoma and Burkitt lymphoma by the US FDA.

Sanjeev Luther, Rafael Pharma’s President and Chief Executive Officer, said, “We are glad that devimistat has received orphan designation to treat this dreaded disease. Our motto ‘To Save A Life Is to Save a Universe’ illustrates our desire to develop treatments for patients with limited treatment options.”

Dr. Timothy Pardee, Chief Medical Officer of Rafael Pharmaceuticals, said, “The orphan drug designation given by the EMA is very encouraging and an important step in the development of devimistat for patients who suffer from this terrible disease.”

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, devimistat, is being evaluated in multiple Phase I, I/II, and III clinical studies. Devimistat 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) by the US FDA. The EMA has granted devimistat orphan drug designation for pancreatic cancer and AML. The Company’s investors include Rafael Holdings, Inc. (NYSE American: RFL). For more information, visit http://www.rafaelpharma.com/.

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Contact
Sanjeev Luther
President & CEO Rafael Pharmaceuticals, Inc.
sanjeev.luther@rafaelpharma.com

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.
jacob.jonas@rafaelpharma.com