Kura Oncology’s Menin-MLL Inhibitor KO-539 Receives Orphan Drug Designation from FDA for Treatment of Acute Myeloid Leukemi...
July 24 2019 - 6:30AM
Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage
biopharmaceutical company focused on the development of precision
medicines for oncology, today announced that the U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation to the
Company’s menin-mixed lineage leukemia (menin-MLL) inhibitor KO-539
for the treatment of acute myeloid leukemia (AML).
“Orphan Drug Designation for AML represents a significant
milestone in the development of KO-539,” said Troy Wilson, Ph.D.,
J.D., President and Chief Executive Officer of Kura Oncology.
“This decision by the FDA follows the clearance of our
investigational new drug (IND) application in March 2019 and
recognizes the potential for KO-539 to address a high unmet need
for patients suffering from AML. We are very encouraged by our
preclinical data in genetically defined subsets, such as tumors
with MLL fusions and rearrangements and NPM1 mutations, and we are
in the final stages of study startup for our Phase 1 clinical trial
in relapsed or refractory AML.”
The FDA's Orphan Drug Designation program provides orphan status
to drugs defined as those intended for the safe and effective
treatment, diagnosis or prevention of rare diseases that affect
fewer than 200,000 people in the United States. Orphan Drug
Designation qualifies the sponsor of the drug for certain
development incentives, including tax credits for qualified
clinical testing, prescription drug user fee exemptions and
seven-year marketing exclusivity upon FDA approval.
About KO-539
KO-539 is a potent and selective small molecule inhibitor of the
menin-MLL protein-protein interaction. MLL-rearranged leukemias are
characterized by chromosomal translocations of the MLL gene that
are primarily found in patients with AML and acute lymphoblastic
leukemia (ALL). These translocations form oncogenes encoding MLL
fusion proteins, which play a causative role in the onset,
development and progression of MLL-rearranged leukemias. The target
genes of the MLL fusion proteins are also found to be overexpressed
in a broader subset of AMLs characterized by oncogenic driver
mutations in genes such as NPM1. These mutations also appear to be
dependent on the interaction between menin and MLL, suggesting that
the menin-MLL complex is a central node in epigenetic dysregulation
driven by distinct oncogenic driver mutations known to be important
in AML and other hematologic malignancies. In preclinical studies,
KO-539 has demonstrated potent and selective inhibition of the
proliferation of MLL-rearranged leukemia cell lines. Kura has also
generated preclinical data showing robust and durable efficacy in
multiple in vivo models of AML characterized by MLL-rearrangements
or oncogenic driver mutations in genes such as NPM1.
About Kura Oncology
Kura Oncology is a clinical-stage biopharmaceutical company
committed to realizing the promise of precision medicines for the
treatment of cancer. The Company’s pipeline consists of small
molecule drug candidates that target cancer signaling pathways
where there is a strong scientific and clinical rationale to
improve outcomes by identifying those patients most likely to
benefit from treatment. Kura’s lead drug candidate is tipifarnib, a
farnesyl transferase inhibitor, for which the Company is conducting
a registration-directed trial of tipifarnib in recurrent or
metastatic patients with HRAS mutant HNSCC. In addition, tipifarnib
is being evaluated in multiple other Phase 2 clinical trials in
solid tumor and hematologic indications. Kura’s pipeline also
includes KO-947, an ERK inhibitor, currently in a Phase 1
dose-escalation trial, and KO-539, a menin-MLL inhibitor, which is
anticipated to enter into a Phase 1 clinical trial shortly. For
additional information about Kura, please visit the Company’s
website at www.kuraoncology.com.
Forward-Looking Statements
This news release contains certain forward-looking statements
that involve risks and uncertainties that could cause actual
results to be materially different from historical results or from
any future results expressed or implied by such forward-looking
statements. Such forward-looking statements include statements
regarding, among other things, the efficacy, safety and therapeutic
potential of Kura’s product candidate KO-539, the progress and
expected timing of Kura’s drug development programs and clinical
trials and the potential benefits of Orphan Drug Designation.
Factors that may cause actual results to differ materially include
the risk that compounds that appeared promising in early research
or clinical trials do not demonstrate safety and/or efficacy in
later preclinical studies or clinical trials, the risk that Kura
may not obtain approval to market its product candidates,
uncertainties associated with performing clinical trials,
regulatory filings and applications, risks associated with reliance
on third parties to successfully conduct clinical trials, the risks
associated with reliance on outside financing to meet capital
requirements, risks that the actual benefits of Orphan Drug
Designation will not be as expected and other risks associated with
the process of discovering, developing and commercializing drugs
that are safe and effective for use as human therapeutics, and in
the endeavor of building a business around such drugs. You are
urged to consider statements that include the words "may," "will,"
"would," "could," "should," "believes," "estimates," "projects,"
"promise," "potential," "expects," "plans," "anticipates,"
"intends," "continues," "designed," "goal," or the negative of
those words or other comparable words to be uncertain and
forward-looking. For a further list and description of the risks
and uncertainties the Company faces, please refer to the Company's
periodic and other filings with the Securities and Exchange
Commission, which are available at www.sec.gov. Such
forward-looking statements are current only as of the date they are
made, and Kura assumes no obligation to update any forward-looking
statements, whether as a result of new information, future events
or otherwise.
Contacts
Company:Pete De SpainVice President, Investor Relations
&Corporate Communications(858)
500-8803pete@kuraoncology.com
Investors:Robert H. UhlManaging DirectorWestwicke Partners,
LLC(858) 356-5932robert.uhl@westwicke.com
Media:Jason SparkManaging DirectorCanale Communications(619)
849-6005jason@canalecomm.com
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