Phase II Burkitt’s Lymphoma and Leukemia Trial


Rafael Pharmaceuticals has Initiated a Phase II Clinical Trial of CPI-613® (devimistat) for Patients with Relapsed or Refractory Burkitt Lymphoma/Leukemia

Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, has initiated patient enrollment for a phase II clinical trial of CPI-613® (devimistat) 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. Ariela Noy, MD, will lead the trial at Memorial Sloan Kettering Cancer Center. Collaborating sites include City of Hope Medical Center and Massachusetts General Hospital.

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®.

FOR ADDITIONAL INFORMATION ABOUT THE TRIAL:

Please contact Sanjeev.Luther@rafaelpharma.com or Timothy.Pardee@rafaelpharma.com.

More information on this trial is available at www.clinicaltrials.gov (NCT03793140).


Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals: “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® (devimistat):

CPI-613® (devimistat) 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.


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

Timothy Pardee
Chief Medical Officer, Rafael Pharmaceuticals, Inc.
timothy.pardee@rafaelpharma.com