Kymera Therapeutics Receives FDA Orphan Drug Designation for KT-253, a Novel, Highly Potent and Selective MDM2 Degrader for the Treatment of Acute Myeloid Leukemia
22 Junho 2023 - 9:30AM
Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage
biopharmaceutical company advancing targeted protein degradation to
deliver novel small molecule protein degrader medicines, today
announced that the U.S. Food and Drug Administration (FDA) has
granted orphan drug designation to KT-253 for the treatment of
Acute Myeloid Leukemia (AML).
KT-253 is a highly potent and selective degrader that targets
MDM2, the crucial regulator of the most common tumor suppressor,
p53. p53 remains intact (wild type) in close to 50% of cancers,
meaning that it retains its ability to modulate cancer cell growth.
While small molecule inhibitors have been developed to stabilize
and upregulate p53 expression, they have been found to induce a
feedback loop that increases MDM2 protein levels, which can repress
p53 and limit their efficacy. In preclinical studies, KT-253 has
shown the ability to overcome the MDM2 feedback loop and rapidly
induce cancer cell death, even with brief exposures. Given MDM2
overexpression and amplification is implicated in AML, KT-253 is
being explored in this cancer, as well as other liquid and solid
tumors.
“This orphan drug designation reinforces the potential of KT-253
to advance the treatment of AML by targeting MDM2, a protein that
has been challenging to effectively drug with conventional
medicines,” said Nello Mainolfi, Founder, President and CEO, Kymera
Therapeutics. “We have a significant opportunity to deliver an
important new medicine that acts on this common cancer mechanism,
and we look forward to rapidly advancing KT-253 in AML and
exploring its potential in other hematological and solid
tumors.”
The Phase 1 study initiated in March 2023 will evaluate the
safety, tolerability, pharmacokinetics/pharmacodynamics, and
clinical activity of KT-253 in patients with relapsed or refractory
high grade myeloid malignancies, including AML, acute lymphocytic
leukemia (ALL), lymphoma and solid tumors. Patients in the KT-253
Phase 1a dose escalation study will receive IV doses of KT-253
administered once every 3 weeks. The open-label study is intended
to identify the recommended Phase 2 dose for KT-253, and is
comprised of two arms, with ascending doses of KT-253 in each arm.
The first arm will consist of patients with lymphomas and advanced
solid tumors and the second arm will consist of patients with high
grade myeloid malignancies and ALL.
The FDA's Orphan Drug Designation program provides
orphan status to drugs defined as those intended for the 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 Acute Myeloid Leukemia (AML)AML is a
rapidly growing cancer of the blood and bone marrow characterized
by a broad spectrum of cytogenetic and molecular abnormalities
resulting in uncontrolled clonal expansion of myeloid blasts cells.
In 2019, approximately 70,000 patients had been diagnosed with AML
in the United States.
About Kymera TherapeuticsKymera is a
biopharmaceutical company pioneering the field of targeted protein
degradation, a transformative approach to address disease targets
and pathways inaccessible with conventional therapeutics. Kymera’s
Pegasus platform is a powerful drug discovery engine, advancing
novel small molecule programs designed to harness the body’s innate
protein recycling machinery to degrade dysregulated,
disease-causing proteins. With a focus on undrugged nodes in
validated pathways, Kymera is advancing a pipeline of novel
therapeutic candidates designed to address the most promising
targets and provide patients with more effective treatments.
Kymera’s initial programs target IRAK4, IRAKIMiD, and STAT3 within
the IL-1R/TLR or JAK/STAT pathways, and the MDM2 oncoprotein,
providing the opportunity to treat patients with a broad range of
immune-inflammatory diseases, hematologic malignancies, and solid
tumors.
Founded in 2016, Kymera is headquartered in Watertown, Mass.
Kymera has been named a “Fierce 15” company by Fierce Biotech and
has been recognized by both the Boston Globe and the Boston
Business Journal as one of Boston’s top workplaces. For more
information about our people, science, and pipeline, please visit
www.kymeratx.com or follow us on Twitter or LinkedIn.
Cautionary Note Regarding Forward-Looking
Statements This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended, including, without limitation,
implied and express statements by Kymera Therapeutics regarding
its: strategy, business plans and objectives for the IRAKIMiD,
STAT3, and MDM2 degrader programs; plans and timelines for the
preclinical and clinical development of its product candidates,
including the therapeutic potential, clinical benefits and safety
thereof; expectations regarding timing, success and data
announcements of current ongoing preclinical and clinical trials.
The words "may," "might," "will," "could," "would," "should,"
"expect," "plan," "anticipate," "intend," "believe," "expect,"
"estimate," "seek," "predict," "future," "project," "potential,"
"continue," "target" and similar words or expressions are intended
to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Any
forward-looking statements in this press release are based on
management's current expectations and beliefs and are subject to a
number of risks, uncertainties and important factors that may cause
actual events or results to differ materially from those expressed
or implied by any forward-looking statements contained in this
press release, including, without limitation, risks associated
with: the impact of COVID-19 on countries or regions in which we
have operations or do business, as well as on the timing and
anticipated results of our current and future preclinical studies
and clinical trials, supply chain, strategy and future operations;
the delay of any current and future preclinical studies or clinical
trials or the development of Kymera Therapeutics' drug candidates;
the risk that the results of current preclinical studies and
clinical trials may not be predictive of future results in
connection with current or future preclinical and clinical trials,
including those for KT-474, KT-333, KT-413 and KT-253; Kymera
Therapeutics' ability to successfully demonstrate the safety and
efficacy of its drug candidates; the timing and outcome of the
Kymera Therapeutics' planned interactions with regulatory
authorities; obtaining, maintaining and protecting its intellectual
property; and Kymera Therapeutics' relationships with its existing
and future collaboration partners. These and other risks and
uncertainties are described in greater detail in the section
entitled "Risk Factors" in the Quarterly Report on Form 10-Q for
the quarter ended March 31, 2023 filed on May 4, 2023, as well as
discussions of potential risks, uncertainties, and other important
factors in Kymera Therapeutics' subsequent filings with the
Securities and Exchange Commission. In addition, any
forward-looking statements represent Kymera Therapeutics' views
only as of today and should not be relied upon as representing its
views as of any subsequent date. Kymera Therapeutics explicitly
disclaims any obligation to update any forward-looking statements.
No representations or warranties (expressed or implied) are made
about the accuracy of any such forward-looking statements.
Investor Contact: Bruce
Jacobs Chief Financial
Officer investors@kymeratx.com 857-285-5300 Chris
Brinzey Managing Director,
Westwicke chris.brinzey@westwicke.com 339-970-2843 |
Media Contact: Todd
Cooper Senior Vice President, Corporate
Affairs media@kymeratx.com 857-285-5300 |
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