CPI-613 receives orphan designation for PTCL


The Food and Drug Administration has granted orphan drug designation to CPI-613 for the treatment of peripheral T-cell lymphoma (PTCL).

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CPI-613 is a novel lipoic acid analogue that inhibits multiple enzyme targets within the tricarboxylic acid cycle.

Rafael Pharmaceuticals is developing CPI-613 as a treatment for hematologic malignancies and solid tumors.

CPI-613 is currently under investigation in combination with bendamustine in a phase 1 study of patients with relapsed or refractory T-cell lymphoma or classical Hodgkin lymphoma, according to the press release from the company.

Results from this trial were presented at the 2016 annual meeting of the American Society of Hematology.

CPI-613 was given at escalating doses starting at 2,000 mg/m2 over 2 hours on days 1-4 as well as on days 8, 11, 15, and 18. Bendamustine was infused at 90 mg/m2 on days 4 and 5 of each 4-week treatment cycle. The treatment cycles were repeated for up to six cycles. There was no intrapatient dose escalation.

The ASH presentation included safety data on eight patients. The most common grade 3 or higher toxicities – lymphopenia and neutropenia – occurred in four patients.

A patient dosed at 2,750 mg/m2 had a dose-limiting toxicity of grade 3 acute kidney injury and grade 4 lactic acidosis. Because of this, the study protocol was amended to discontinue dose escalation at doses of 2,750 mg/m2 or higher and to expand the 2,500 mg/m2 cohort.

Six patients were evaluable for efficacy, and the overall response rate was 83% (5/6).

There were three complete responses in patients with PTCL not otherwise specified, a partial response in a patient with mycosis fungoides, and a partial response in a patient with angioimmunoblastic T-cell lymphoma.

One patient with T-cell acute lymphoblastic leukemia experienced progressive disease.

The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the United States.

The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.

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