Trovagene Gets FDA Orphan Drug Designation for AML Treatment

Mathew Shanley

Earlier today, Trovagene, Inc. was granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its drug PCM-075.

The drug, intended for the treatment of acute myeloid leukemia, is an oral, highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine Polo-like Kinase 1 (PLK1) enzyme, which is seemingly over-expressed in a variety of hematologic malignancies and solid tumors like breast, prostate, ovarian, gastric, and colon cancers.

AML, a cancer of the blood and bone marrow, progresses rapidly and is characterized by the almost immediate expansion of white blood cells, which obstructs the production of typical blood cells. Per the National Cancer Institute of the National Institutes of Health, approximately 21,380 people will be diagnosed with AML in 2017, and more than 10,000 will die as a result of it.

PCM-075 is orally bioavailable and has been studied in patients with advanced metastatic solid tumor cancers. Considering its significant advantages over previously evaluated PLK1 inhibitors, Trovagene is initiating a Phase 1b/2 clinical trial of the drug in AML at 10 research sites across the U.S. The study will assess the anti-leukemic activity of PCM-075 in patients with AML.

"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,” said Bill Welch, Chief Executive Officer of Trovagene. 

In the Phase 1b/2, hematologist Dr Jorge Cortes, Deputy Department Chair, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center will lead a team of researchers on the exploration of the usage of a correlative biomarker analysis to select patients most likely to respond to treatment.

The study is not yet recruiting, but will determine whether PCM-075 is safe and tolerable in adult patients with relapsed/refractory AML when administered orally for 5 consecutive days every 28 days. The study will also determine the maximum tolerated dose or recommended phase 2 dose of the drug in combination with decitabine and/or low-dose cytarabine, which are the current standards of care.

Primary outcome measures for the study include an evaluation of the number of participants with dose limiting toxicity (DLT), among others, while secondary outcome measures include overall survival and event-free survival.

The anticipated start date for the study is October 31, and it is expected to be completed in the second quarter of 2020.

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