The study (NCT03793140) is planned to include 34 patients with Burkitt’s lymphoma/leukemia or high-grade B-cell lymphoma who carry high-risk genetic alterations in the MYC, BCL2, and/or BCL6 genes. Participants are being recruited at seven Memorial Sloan Kettering clinical centers in New York and New Jersey.
CPI-613 is a small molecule that blocks a metabolic pathway widely used by cancer cells to survive and proliferate. By interfering with enzymes in this pathway, CPI-613 prevents cancer cells from meeting their energy demands, potentially killing them. This new agent is also thought to increase the sensitivity of cancer cells to chemotherapeutic agents.
In a prior Phase 1 trial (NCT01034475) in people with advanced blood cancers, CPI-613 showed promising signs in a Burkitt’s lymphoma patient who had relapsed twice after prior therapies — chemotherapy and immunotherapy, followed by a stem cell transplant.
The patient, a 19-year-old woman, experienced a partial reduction of her tumor after only three cycles of the treatment. After 17 cycles, her tumor was surgically removed. Tumor samples revealed extensive cell death, and clinical follow-up continued to show no evidence of the disease more than three years later.
Now, the ongoing Phase 2 trial will continue to evaluate CPI-613 in Burkitt’s lymphoma patients who failed prior treatments and show genetic rearrangements that increase the risk for relapse.
CPI-613 will be given both as an induction and maintenance therapy. Patients will receive the treatment in the first five days of two two-week treatment cycles, then CPI-613 will be administered in three-week cycles, also in the first five days of each cycle.
Researchers will be evaluating patients’ response to the investigational therapy, including complete and partial responses, minor responses, and disease stabilization.
“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,” Sanjeev Luther, president and CEO of Rafael Pharmaceuticals, said in a press release.
The U.S. Food and Drug Administration granted orphan drug designation to CPI-613 for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndromes, and Burkitt’s lymphoma.