Trovagene gets US FDA orphan drug status for PCM-075 to treat AML

"AML is an aggressive cancer of the blood and bone marrow with approximately 20,000 new cases in the US 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." 

Orphan drug designation is granted by the FDA to drugs that are intended treat rare diseases or conditions for patients in the US The orphan drug designation allows the orphan drug indication for the drug to be eligible for a seven-year period of US marketing exclusivity upon approval of the drug, as well as other development assistance and financial incentives. 

Trovagene is initiating a phase 1b/2 open-label trial to evaluate the safety and anti-leukemic activity of PCM-075 in combination with standard-of-care in patients with AML, at ten research sites across the US, led by Hematologist Dr. Jorge Cortes, Deputy Department Chair, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. 

PCM-075 is a highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine polo-like-kinase 1 (PLK 1) enzyme, which is over-expressed in multiple hematologic and solid tumor cancers. Studies have shown that inhibition of polo-like-kinases can lead to tumor cell death, including a phase 2 study in Acute Myeloid Leukemia (AML) where response rates up to 31% were observed when used in conjunction with a standard therapy for AML (low-dose cytarabine-LDAC) versus treatment with LDAC alone with a 13.3% response rate. A phase 1 open-label, dose escalation safety study of PCM-075 has been completed in patients with advanced metastatic solid tumor cancers, and published in Investigational New Drugs. Trovagene plans to initiate a phase 1b/2 clinical trial with PCM-075 in AML, since it has shown significant advantages over prior PLK1 inhibitors evaluated in this indication, including a higher selectivity, greater potency, oral bioavailability and shorter half-life.

Trovagen, us fda orphan drug status, pcm, aml