NEWARK, NJ, USA I January 08, 2019 I Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced the initiation of patient enrollment for a phase II clinical trial of CPI-613 for patients with relapsed or refractory Burkitt Lymphoma/Leukemia. In June 2018, CPI-613 received orphan drug designation for treatment of Burkitt Lymphoma from the US FDA. 

Burkitt Lymphoma 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. No definitive second-line therapy currently exists (NCCN Guideline, version 1.2019). There is a clear unmet medical need for additional treatment options for Burkitt Lymphoma patients.

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

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) following treatment with CPI-613.

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. After receiving orphan drug designation earlier this year, this trial marks another positive step toward realizing our vision.”

About CPI-613:
CPI-613 is a first-in-class drug developed through Rafael’s Altered Metabolism Directed (AMD) platform. CPI-613 targets altered regulation of metabolic processes specific to cancer cells. It is highly specific, simultaneously attacks multiple targets, minimally toxic and has demonstrated 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 therapy for hematological malignancies and solid tumors. To date, over 300 patients have received one or more doses of CPI-613. The drug has consistently 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 a 52% CR+CRi and 12.4 months median overall survival. In both trials, the efficacy of the CPI-613 combination was substantially higher than the standard therapy. 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 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.  The EMA has granted CPI-613 orphan drug designation for pancreatic cancer and AML.

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).

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SOURCE: Rafael Pharmaceuticals