Orphan Drug Designation Granted to Peripheral T-Cell Lymphoma Treatment, CPI-614

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Rafael Pharmaceuticals, Inc.’s CPI-613 for the treatment of peripheral T-Cell lymphoma (PTCL).

CPI-613 is a novel lipoic acid analogue with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid cycle. Currently, 10 patients with relapsed or refractory T-Cell lymphoma are under investigation in a phase 1 trial with CPI-614 in combination with bendamustine. Of the 10 patients dosed, 7 were evaluated for efficacy.

“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,” said Zanetta Lamar, MD, the principal investigator of the trial at Wake Forest Baptist, in a recent statement.

According to interim results presented at the American Society of Hematology (ASH) Annual Meeting in 2016 regarding CPI-613 in combination with bendamustine in 7 of the 10 dosed patients, an 86% objective response rate (43% complete response and 43% partial response) was observed.

"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,” added Dr. Lamar.

Determination of the maximum tolerated dose (MTD) and safety profile of CPI-613 when used in combination with bendamustine to treat patients with relapsed or refractory T-Cell Lymphoma or classic Hodgkin Lymphoma is the primary objective of the current study.

Activation of a second clinical trial site for this study at the Abramson Cancer Center of the University of Pennsylvania was also recently announced by Rafael.