This is the fifth orphan drug designation CPI-613 has received, the others being for Burkitt’s lymphoma, pancreatic cancer, acute myeloid leukemia, and myelodysplastic syndromes.
Discovered by researchers at Stony Brook University in New York, CPI-613 is a small molecule with anti-cancer activity. It blocks the mitochondrial tricarboxylic acid cycle — a metabolic pathway widely used by cancer cells to survive and proliferate. By interfering with enzymes in this cycle, CPI-613 is intended to prevent cancer cells from being able to produce energy at the high rate they need, potentially killing them.
CPI-613 is also known to increase the sensitivity of the tumor cells to other chemotherapeutic agents, widening its scope to target multiple forms of cancers.
PTCL is an aggressive form of blood cancer that affects white blood cells and accounts for 10-15 percent of all lymphomas. There is an unmet therapeutic need among PTCL patients who have relapsed or do not respond to current treatments.
The FDA’s decision to grant orphan drug status to CPI-613 for PTCL treatment was based on interim results from an open-label Phase 1 clinical trial (NCT02168140), which is studying the side effects and optimal dose of CPI-613 when given together with bendamustine hydrochloride to patients with relapsed or refractory T-cell lymphoma.
To date, 10 patients have been dosed, and seven have been evaluated for effectiveness in the ongoing Phase 1 trial, which is still recruiting patients for a total of 14. Interim results revealed a complete response (absence of detectable cancer) in 43% of patients treated with CPI-613 in combination with bendamustine and a partial response in 43% of participants on the same regimen.
“This study of CPI-613 in combination with bendamustine in patients with relapsed or refractory T-cell Lymphoma showed a good safety profile and encouraging efficacy,” Zanetta Lamar, MD, lead investigator of the trial at Wake Forest Baptist Health, said in a press release.
“The orphan drug designation in this case provides hope to the poor-risk patient population, and inspires us to continue our tireless effort to bring these patients a legitimate treatment option,” Lamar said.