WALTHAM, Mass., June 28, 2021 /PRNewswire/ -- Syndax
Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:
SNDX), a clinical stage biopharmaceutical company developing an
innovative pipeline of cancer therapies, today announced that the
U.S. Food and Drug Administration (FDA) has granted Fast Track
Designation (FTD) to SNDX-5613 for the treatment of adult and
pediatric patients with relapsed or refractory acute leukemias
harboring a mixed lineage leukemia rearranged (MLLr) or
nucleophosmin (NPM1) mutation. SNDX-5613 is the Company's highly
selective, oral menin inhibitor.
"Genetically-defined acute leukemias represent an underserved
area marked by particularly poor prognosis and limited therapeutic
options," said Briggs W. Morrison,
M.D., Chief Executive Officer of Syndax. "As we move toward
initiating our pivotal study, receipt of FTD from the FDA
underscores SNDX-5613's potential to meaningfully improve outcomes
for patients with MLLr and NPM1 mutant acute leukemias."
About Fast Track Designation
Fast Track Designation is designed to facilitate the development
and expedite the review of drugs to treat serious conditions and
fulfill an unmet medical need, enabling drugs to reach patients
earlier. The FDA created this process to help deliver important new
drugs to patients earlier and it covers a broad range of serious
illnesses. These clinical programs may also be eligible to apply
for Accelerated Approval and Priority Review if relevant criteria
are met.
About SNDX-5613
SNDX-5613 is a potent, selective, small molecule inhibitor of
the menin-MLL binding interaction that is being developed for the
treatment of mixed lineage leukemia rearranged (MLLr) acute
leukemias including acute lymphoblastic leukemia (ALL) and acute
myeloid leukemia (AML), and NPM1 mutant AML. In preclinical models
of MLLr acute leukemias, SNDX-5613 demonstrated robust,
dose-dependent inhibition of tumor growth, resulting in a marked
survival benefit. Menin-MLL interaction inhibitors have also
demonstrated robust treatment benefit in multiple preclinical
models of NPM1 mutant AML, which represents the most frequent
genetic abnormality in adult AML. SNDX-5613 is currently being
evaluated in the Company's AUGMENT-101 Phase 1/2 open-label
clinical trial for the treatment of relapsed/refractory (R/R) acute
leukemias. SNDX-5613 was granted Orphan Drug Designation by the
U.S. Food and Drug Administration for the treatment of patients
with AML, and Fast Track designation for the treatment of adult and
pediatric patients with relapsed or refractory acute leukemias
harboring a mixed lineage leukemia rearranged MLLr or NPM1
mutation.
About Mixed Lineage Leukemia Rearranged Acute
Leukemias
Rearrangements of the MLL gene give rise to mixed lineage
leukemia rearranged (MLLr) acute leukemias known to have a poor
prognosis, with less than 25% of adult patients surviving past five
years. MLL rearrangements produce fusion proteins that require
interaction with the protein called menin to drive leukemic cancer
growth. Disruption of the menin-MLLr interaction has been shown to
halt the growth of MLLr leukemic cells. MLLr leukemias, which are
routinely diagnosed through currently available cytogenetic or
molecular diagnostic techniques, occur in approximately 80% of
infant acute leukemias and up to 10% of all acute leukemias. There
are currently no approved therapies indicated for MLLr
leukemias.
About NPM1 Mutant Acute Myeloid Leukemia
NPM1 mutant acute myeloid leukemia (AML), which is distinguished
by point mutations in the NPM1 gene that drive the leukemic
phenotype, is the most common type of cytogenetically normal adult
AML and represents approximately 30% of all adult AML cases. This
subtype of AML has a five year overall survival rate of
approximately 50%. Similar to mixed lineage leukemia rearranged
(MLLr) leukemias, NPM1 mutant AML is highly dependent on the
expression of specific developmental genes shown to be negatively
impacted by inhibitors of the menin-MLL interaction. NPM1 mutant
AML is routinely diagnosed through currently available screening
techniques. There are currently no approved therapies indicated for
NPM1 mutant AML.
About Syndax Pharmaceuticals, Inc.
Syndax Pharmaceuticals is a clinical stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. The Company's pipeline includes SNDX-5613, a
highly selective inhibitor of the Menin–MLL binding interaction,
axatilimab, a monoclonal antibody that blocks the colony
stimulating factor 1 (CSF-1) receptor, and entinostat, a class I
HDAC inhibitor. For more information, please
visit www.syndax.com or follow the Company
on Twitter and LinkedIn.
Syndax's Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Words such as "may," "will," "expect," "plan," "anticipate,"
"estimate," "intend," "believe" and similar expressions (as well as
other words or expressions referencing future events, conditions or
circumstances) are intended to identify forward-looking statements.
These forward-looking statements are based on Syndax's expectations
and assumptions as of the date of this press release. Each of these
forward-looking statements involves risks and uncertainties. Actual
results may differ materially from these forward-looking
statements. Forward-looking statements contained in this press
release include, but are not limited to, statements about the
design, progress, timing, clinical development and scope of
clinical trials, plans for initiating future clinical trials,
reporting of clinical data for Syndax's product candidates, the
association of data with treatment outcomes, and the potential use
of our product candidates to treat various cancer indications and
fibrotic diseases. Many factors may cause differences between
current expectations and actual results including unexpected safety
or efficacy data observed during preclinical or clinical trials,
clinical trial site activation or enrollment rates that are lower
than expected, changes in expected or existing competition, changes
in the regulatory environment, the COVID-19 pandemic may disrupt
our business and that of the third parties on which we depend,
including delaying or otherwise disrupting our clinical trials and
preclinical studies, manufacturing and supply chain, or impairing
employee productivity, failure of Syndax's collaborators to support
or advance collaborations or product candidates and unexpected
litigation or other disputes. Other factors that may cause Syndax's
actual results to differ from those expressed or implied in the
forward-looking statements in this press release are discussed in
Syndax's filings with the U.S. Securities and Exchange
Commission, including the "Risk Factors" sections contained
therein. Except as required by law, Syndax assumes no obligation to
update any forward-looking statements contained herein to reflect
any change in expectations, even as new information becomes
available.
Syndax Contacts
Investor Contact
Melissa Forst
Argot Partners
melissa@argotpartners.com
Tel 212.600.1902
Media Contact
Ted Held
ted.held@gcihealth.com
Tel 212.798.9842
SNDX-G
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SOURCE Syndax Pharmaceuticals, Inc.