The Food and Drug Administration (FDA) has granted orphan drug designation to PCM-075 (Trovagene) for the treatment of patients with acute myeloid leukemia (AML).

PCM-075 is an oral, highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine Polo-like Kinase 1 (PLK1) enzyme. This enzyme appears to be overexpressed in several different hematologic malignancies and solid tumor cancers.

A Phase 1b/2 open-label study is being planned to assess the safety and efficacy of PCM-075 in combination with standard-of-care in patients with AML. A Phase 1 open-label, dose escalation safety study has already been completed in patients with advanced metastatic solid tumor cancers.

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“AML is an aggressive cancer of the blood and bone marrow with approximately 20,000 new cases in the U.S each year,” said Bill Welch, Chief Executive Officer of Trovagene.  “We see the FDA’s granting of orphan drug designation for PCM-075 as underscoring the medical need for new therapies for patients with AML and an important step forward in our clinical development program.”

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