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Rafael Pharmaceuticals Receives FDA Orphan Drug Designation of CPI-613 for the Treatment of Peripheral T-Cell Lymphoma (PTCL)

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Newark, NJ, Aug. 16, 2018 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CPI-613, the Company's lead Altered Energy Metabolism Directed (AEMD) drug candidate, for the treatment of Peripheral T-Cell Lymphoma (PTCL).

T-Cell Lymphomas account for 10-15% of lymphoid malignancies and display significant heterogeneity. T-Cell Lymphomas have a worse prognosis than most B-Cell Lymphomas. There is no standard treatment for relapsed or refractory disease, and median progression free and overall survival rates have been reported as 3.7 and 6.5 months respectively. There is clear unmet clinical need for relapsed or refractory T-Cell Lymphoma treatments.

CPI-613 is a novel lipoic acid analogue with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid cycle. This drug is currently under investigation in combination with bendamustine in patients with relapsed or refractory T-Cell Lymphoma. 10 patients were dosed to date and 7 were evaluated for efficacy. The interim results of CPI-613 in combination with bendamustine in patients with T-Cell Lymphoma exhibited a very good signal of efficacy with an 86% Objective Response Rate (43% Complete Response and 43% Partial Response). The results from this study were first presented at the American Society of Hematology (ASH) Annual Meeting in 2016.

Zanetta Lamar, MD, the Principal Investigator of this trial at Wake Forest Baptist commented: "This study of CPI-613 in combination with bendamustine in patients with relapsed or refractory T-Cell Lymphoma showed a good safety profile and encouraging efficacy. The orphan drug designation in this case provides hope to the poor-risk patient population, and inspires us to continue our tireless effort to bring these patients a legitimate treatment option."

Sanjeev Luther, Rafael Pharmaceutical's President and Chief Executive Officer, said, "Rafael is now the only oncology company in the U.S. with five orphan drug designations. These orphan drug designations include: Acute Myeloid Leukemia, Myelodysplastic Syndrome, Pancreatic Cancer, Burkitt's Lymphoma, and now Peripheral T-cell Lymphoma, another patient population with severe unmet clinical need."

The primary objective of the current study is to determine the Maximum Tolerated Dose (MTD) and safety profile of CPI-613 when used in combination with bendamustine to treat patients with relapsed or refractory T-Cell Lymphoma, or classic Hodgkin Lymphoma. Rafael recently announced the activation of a second clinical trial site for this study at the Abramson Cancer Center of the University of Pennsylvania.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals, Inc. is a privately held 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 II 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:RFL). For more information, visit http://www.rafaelpharma.com/.

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.

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

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

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