FDA Grants Orphan-Drug Designation to PCM-075 for AML

The FDA granted orphan-drug designation to PCM-075 for the treatment of acute myeloid leukemia (AML). PCM-075 is an oral, highly selective adenosine triphosphate competitive inhibitor of the serine/threonine Polo-like Kinase 1 enzyme.

The drug’s manufacturers are planning an open-label, phase Ib/II trial to evaluate the safety and anti-leukemic activity of PCM-075 in combination with standard of care for patients with AML at 10 U.S. research sites. The study, which is not yet recruiting, will determine whether PCM-075 is safe and tolerable in adult patients with relapsed/refractory AML when administered orally for five consecutive days every 28 days. The study will also determine the maximum tolerated or recommended dose of PCM-075 for phase II testing in combination with decitabine and/or low-dose cytarabine. The projected study completion date is mid-2020.

Source: Trovagene, Inc., press release, October 9, 2017.