Burkitt Lymphoma Treatment Granted Orphan Drug Designation

This morning, Rafael Pharmaceuticals, Inc announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to it product, CPI-613, for the treatment of Burkitt lymphoma.

In addition, Rafael Pharmaceuticals, Inc has initiated a phase 2 trial assessing CPI-613 in Burkitt lymphoma patients.

CPI-613 is a novel lipoic acid analogue with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells.

By attacking the TCA cycle, CPI-613 substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents, thus allowing for CPI-613 combination therapies with lower doses of these generally toxic drugs to be highly effective with diminished patient adverse effects.

“CPI 613 is the only investigational drug which has orphan designation to treat this rare disease,” shared Sanjeev Luther, Rafael Pharmaceutical’s President and Chief Executive Officer, in a recent statement. “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.”

Previous evidence for CPI-613 stands in a phase 1 clinical study that included a 19-year-old female with relapsed Burkitt lymphoma who was administered CPI-613 monotherapy (2940 mg/m²) once enrolled in CL-CPI-613-009. (This enrollment was preceded by a second relapse to the prior therapy.)

Over 51 weeks, the patient was administered 17 total cycles of therapy. After the third cycle, the patient achieved and maintained radiographic partial response (PR) status, and the treatment was observed to be well tolerated.

The patient discontinued treatment after 17 cycles to pursue a surgical resection of residual tumor, which revealed Burkitt lymphoma with extensive necrosis in the pathology of the surgical specimen. More than 36 months later, clinical follow-up on the patient indicated no evidence of disease.

Principal investigator, Ariela Noy, MD, medical oncologist of lymphoma and AIDS-associated cancers at Memorial Sloan-Kettering Cancer Center, and chair of the Lymphoma Working Group for the AIDS Malignancy Consortium spoke about the importance of this trial in the statement, sharing, “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.”

The phase 2 study will assess 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.