Rafael Pharmaceuticals Receives FDA Orphan Drug Designation for Treatment of Burkitt Lymphoma, and Initiates a Phase II Clinical Trial of CPI-613 for Patients with Relapsed or Refractory Burkitt Lymphoma/Leukemia

Newark, New Jersey, UNITED STATES

Newark, NJ, June 28, 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 Burkitt Lymphoma (BL).

BL is a highly aggressive hematologic B-cell malignancy classically characterized by the overexpression of c-Myc. Due to the rapid proliferation rate of these tumors, the mainstay of treatment includes aggressive chemotherapy and immunotherapy. As per NCCN guideline, there is no definitive second-line therapy for BL (NCCN Guideline, version 4.2018). There is a clear unmet medical need for additional treatment options for these patients.

CPI-613 is a novel lipoic acid analogue with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid cycle. In a phase I clinical study of CPI-613 in patients with advanced hematological malignancies (CL-CPI-613-009), a 19-year-old female with relapsed BL was treated with CPI-613 monotherapy. After second relapse to the prior therapy, the patient was enrolled in CL-CPI-613-009 and received CPI-613 monotherapy (2,940 mg/m2). The patient received 17 total cycles of therapy over 51 weeks, and achieved and maintained radiographic Partial Response (PR) status after the third cycle. The treatment was well tolerated. After 17 cycles, the patient decided to discontinue treatment and pursue a surgical resection of residual tumor. The pathology of the surgical specimen revealed BL with extensive necrosis. Clinical follow-up on the patient indicated no evidence of disease more than 36 months later.

Sanjeev Luther, Rafael Pharmaceutical’s President and Chief Executive Officer, said, “CPI 613 is the only investigational drug which has orphan designation to treat this rare disease. Our motto ‘To Save A Life Is to Save a Universe’ illustrates our desire to develop potential treatments for these patients and we are fortunate to launch this study with the renowned Dr. Noy as its Principal Investigator.”

Ariela Noy, M.D., Medical Oncologist for Lymphoma and AIDS-associated cancers, Memorial Sloan-Kettering Cancer Center, and Chair of the Lymphoma Working Group for the AIDS Malignancy Consortium is the Principal Investigator of this study. Dr. Noy commented, “We look forward to launching this study with CPI-613 for patients with relapsed/refractory Burkitt Lymphoma who do not have any viable treatment options. This could be a potentially life-saving therapy.”

This phase II study will look at the response rate of patients with relapsed or refractory Burkitt Lymphoma/Leukemia and high-grade B-cell Lymphoma with rearrangements of MYC and BCL2 and/or BCL6 (DHL/THL) treated with CPI-613.

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 AMERICAN: RFL). For more information, visit rafaelpharma.com.

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Sanjeev Luther
President & CEO Rafael Pharmaceuticals, Inc. 

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.