FDA accepts investigational new drug application for PCM-075 to treat acute myeloid leukemia

The FDA accepted the investigational new drug application for PCM-075 for the treatment of patients with acute myeloid leukemia, according to the drug’s manufacturer.

An open-label phase 1b/phase 2 trial will evaluate the safety and antileukemic activity of PCM-075 (Trovagene), a polo-like kinase 1 (PLK1) inhibitor, in combination with decitabine in approximately 60 patients with AML.

“The FDA's timely acceptance of our investigational new drug application and phase 1b/phase 2 protocol is an important milestone for Trovagene,” Bill Welch, CEO of Trovagene, said in a company-issue release. “We have already received positive indications of interest from numerous key investigators and clinical institutions as we make preparations to initiate our trial in the U.S. Also, we are fortunate to have Jorge Eduardo Cortes, MD, as the principal investigator on our trial given his vast experience in leukemia and his prior work with PLK1 inhibitors.”

The phase 1b dose escalation trial will include patients who have relapsed or were resistant to three or fewer prior therapeutic regimens. The goal of the trial is to evaluate safety, tolerability, dose and scheduling of PCM-075.

Researchers will increase the initial phase 1b dose level of 12 mg/m² PCM-075 by 50% increments to assess pharmacokinetics and correlative biomarker activity. Results will determine a recommended clinical treatment dose for the phase 2 continuation trial.

An open-label phase 2 trial will include patients who have received no more than one prior treatment regimen, have either relapsed or refractory disease, and are not considered candidates for reinduction therapy.

In both phases of the trial, doses of PCM-075 will be administered orally each day for 5 consecutive days in a 28-day cycle.