SAN DIEGO, Oct. 9, 2017 /PRNewswire/ -- Trovagene, Inc.
(NASDAQ: TROV), a precision medicine biotechnology company,
today announced that the U.S. Food and Drug Administration
(FDA) granted an orphan
drug designation to PCM-075 for the treatment of patients with
acute myeloid leukemia (AML) on
September 28, 2017. PCM-075 is
an oral, highly-selective adenosine triphosphate (ATP) competitive
inhibitor of the serine/threonine Polo-like Kinase 1 (PLK1) enzyme,
which appears to be over
expressed in several different hematologic malignancies and solid
tumor cancers.
"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."
Orphan drug designation is granted by the FDA to drugs that are intended treat rare
diseases or conditions for patients in the U.S. The orphan drug
designation allows the orphan drug indication for the
drug to be eligible for a seven-year period of U.S.
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 U.S., led by Hematologist Dr.
Jorge Cortes, Deputy Department
Chair, Department of Leukemia, Division of Cancer Medicine, The
University of Texas MD Anderson Cancer
Center.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a hematologic malignancy in
which myeloid lineage cells of the bone marrow cease to
differentiate appropriately, resulting in a marked increase in the
number of circulating immature blast cells. As a consequence, the
counts of mature red blood cells, platelets, and normal white blood
cells decline, causing fatigue, shortness of breath, bleeding, and
increased susceptibility to infection. The Surveillance,
Epidemiology and End Results (SEER) program estimates the annual
incidence rate of AML in the United
States (US) to be approximately 21,000 cases in 2017. Rates
of new AML cases have been rising an average of 3.1% each year over
the last 10 years. The median age of AML diagnosis is 68 years of
age, and approximately 45% of new diagnoses are among patients age
70 years or older.
About PCM-075
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. This study was accepted
by the National Library of Medicine (NLM) and is now publicly
viewable on www.clinicaltrials.gov. The NCT number assigned by
clinicaltrials.gov for this study is NCT03303339. PCM-075 has
demonstrated synergy in preclinical studies with over 10
chemotherapeutic and target agents used in hematologic and solid
tumor cancers, including FLT3 and HDAC inhibitors, taxanes, and
cytotoxins. Trovagene believes the combination of its targeted
PLK-1 inhibitor, PCM-075, with other compounds has the potential
for improved clinical efficacy in Acute Myeloid Leukemia (AML),
Castration-Resistant Prostate Cancer (CRPC), Non-Hodgkin Lymphoma
(NHL), Triple Negative Breast Cancer (TNBC) and Adrenocortical
Carcinoma (ACC).
About Trovagene, Inc.
Trovagene is a precision medicine biotechnology company
developing oncology therapeutics for improved cancer care by
leveraging its proprietary Precision Cancer Monitoring® (PCM)
technology in tumor genomics. Trovagene has broad intellectual
property and proprietary technology to measure circulating tumor
DNA (ctDNA) in urine and blood to identify and quantify clinically
actionable markers for predicting response to cancer therapies.
Trovagene offers its PCM technology at its CLIA/CAP – accredited
laboratory and plans to continue to vertically integrate its PCM
technology with precision cancer therapeutics. For more
information, please visit https://www.trovagene.com.
Third party trademarks used herein are trademarks of their
respective owners.
Forward-Looking Statements
Certain statements in this press release are forward-looking
within the meaning of the Private Securities Litigation Reform Act
of 1995. These statements may be identified by the use of words
such as "anticipate," "believe," "forecast," "estimated" and
"intend" or other similar terms or expressions that concern
Trovagene's expectations, strategy, plans or intentions. These
forward-looking statements are based on Trovagene's current
expectations and actual results could differ materially. There
are a number of factors that could cause actual events to differ
materially from those indicated by such forward-looking
statements. These factors include, but are not limited to, our
need for additional financing; our ability to continue as a going
concern; clinical trials involve a lengthy and expensive process
with an uncertain outcome, and results of earlier studies and
trials may not be predictive of future trial results; our clinical
trials may be suspended or discontinued due to unexpected side
effects or other safety risks that could preclude approval of our
product candidates; uncertainties of government or third party
payer reimbursement; dependence on key personnel; limited
experience in marketing and sales; substantial competition;
uncertainties of patent protection and litigation; dependence upon
third parties; our ability to develop tests, kits and systems and
the success of those products; regulatory, financial and business
risks related to our international expansion and risks related to
failure to obtain FDA clearances or approvals and noncompliance
with FDA regulations. There are no guarantees that any of our
technology or products will be utilized or prove to be commercially
successful, or that Trovagene's strategy to design its liquid
biopsy tests to report on clinically actionable cancer genes will
ultimately be successful or result in better reimbursement
outcomes. Additionally, there are no guarantees that future
clinical trials will be completed or successful or that any
precision medicine therapeutics will receive regulatory approval
for any indication or prove to be commercially successful.
Investors should read the risk factors set forth in Trovagene's
Form 10-K for the year ended December 31, 2016, and other
periodic reports filed with the Securities and Exchange Commission.
While the list of factors presented here is considered
representative, no such list should be considered to be a complete
statement of all potential risks and uncertainties. Unlisted
factors may present significant additional obstacles to the
realization of forward-looking statements. Forward-looking
statements included herein are made as of the date hereof, and
Trovagene does not undertake any obligation to update publicly such
statements to reflect subsequent events or circumstances.
Trovagene Contact:
Vicki
Kelemen
VP, Corporate Communications
858-952-7652
vkelemen@trovagene.com
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