Investors are racing to buy TrovaGene Inc (NASDAQ:TROV) shares after the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to PCM-075, Trovagene’s investigational Polo-like kinase 1 (PLK1) inhibitor, for the treatment of acute myeloid leukemia (AML). This information was posted to FDA’s website and can be accessed here.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the U.S. and provides certain incentives for medications intended for the treatment, diagnosis or prevention of rare diseases. At present, these incentives include seven years of marketing exclusivity for the orphan indication, certain federal grants, tax credits and waiver of certain FDA fees.
In August, Trovagene announced compelling results of preclinical research of PCM-075 with a Histone deacetylase (HDAC) inhibitor in Non-Hodgkin Lymphoma (NHL) cell lines. PCM-075 showed significant synergy in combination with a HDAC inhibitor of up to 80% in aggressive double-hit B-cell lymphoma (DLBCL) and mantle-cell lymphoma cell lines. DLBCL and mantle cell lymphomas within NHL represent challenging malignancies without a standard-of-care treatment and confer a poor prognosis for patients.
Additionally, PCM-075 synergy has been evaluated in combination with more than ten different chemotherapeutics, including cisplatin, cytarabine, doxorubicin, gemcitabine and paclitaxel, and targeted therapies, such as HDAC inhibitors, FLT3 inhibitors, and bortezomib. These therapeutics are used clinically for treatment of many solid and hematologic cancers, including Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Non-Hodgkin Lymphoma (NHL), Multiple Myeloma, Adrenocortical Carcinoma (ACC), Triple-Negative Breast Cancer (TNBC), Small-Cell Lung Cancer (SCLC), and Ovarian Cancer.
The consistent and significant synergistic effects observed in preclinical research on tumor cell death indicates that PCM-075 could be effective in combination therapies to address a broad range of tumor types, as well as the emergence of drug-resistant tumors.
Shares of TrovaGene are currently rising nearly 30% to $0.93. TROV has a 1-year high of $4.75 and a 1-year low of $0.61. The stock’s 50-day moving average is $0.73 and its 200-day moving average is $0.89.
On the ratings front, Maxim analyst Jason McCarthy reiterated a Buy rating on TROV, with a price target of $4, in a report issued on August 22. The current price target implies an upside of 463% from current levels. According to TipRanks.com, McCarthy has a yearly average return of 1.1%, a 40% success rate, and is ranked #2309 out of 4698 analysts.
Trovagene is a molecular diagnostic company, which focuses on the development and commercialization of a proprietary urine-based molecular diagnostic technology for use in disease detection and monitoring across a variety of medical disciplines.