– Topline data expected in 4Q24 could
support sNDA filing in 1H25 –
– Revumenib has the potential to
address $2 billion mNPM1 and KMT2Ar
R/R acute leukemia
U.S. market opportunity –
WALTHAM,
Mass., March 28, 2024 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical
company developing an innovative pipeline of cancer therapies,
today announced completion of enrollment in the AUGMENT-101 pivotal
trial cohort of patients with relapsed/refractory (R/R) mutant
nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is
expected in the fourth quarter of 2024 and could support a
supplemental New Drug Application (sNDA) filing for revumenib in
R/R mNPM1 AML in the first half of 2025.
"We are thrilled to announce that we are one step closer to
potentially expanding the therapeutic reach of revumenib in
genetically defined acute leukemias," said Neil Gallagher, M.D., Ph.D., President, Head of
Research and Development at Syndax. "We look forward to reporting
topline data for this pivotal cohort in the fourth quarter of this
year, which will follow closely behind a potential first approval
of KMT2A acute leukemia in the third quarter."
Michael A. Metzger, Chief
Executive Officer, added, "With revumenib and axatilimab, two
first-and best-in-class drugs, expected to launch in 2024 and the
potential to expand beyond first approvals and into additional
indications, Syndax is well positioned to deliver on its mission to
improve the lives of cancer patients and create meaningful
long-term value for shareholders."
Sixty-four (64) adult and up to 20 pediatric patients with mNPM1
AML have been enrolled into the pivotal portion of AUGMENT-101, a
pivotal trial designed to evaluate the safety, tolerability,
pharmacokinetics, and efficacy of orally administered revumenib.
The primary endpoint for the trial is efficacy as measured by
complete remission (CR) or a CR with partial hematological recovery
(CRh) rate (CR + CRh) per protocol, with secondary endpoints
including duration of response (DOR) and overall survival (OS).
A new drug application (NDA) is under review by the U.S. Food
and Drug Administration (FDA) for revumenib in R/R KMT2Ar acute
leukemia with a Prescription Drug User Fee Act (PDUFA) action date
of September 26, 2024. The NDA
submission is supported by positive data from the AUGMENT-101
pivotal trial cohort of revumenib in adult and pediatric patients
with KMT2Ar AML and acute lymphoblastic leukemia (ALL).
About Revumenib
Revumenib is a potent, selective, small molecule inhibitor of
the menin-KMT2A binding interaction that is being developed for the
treatment of KMT2A-rearranged, also known as mixed lineage leukemia
rearranged or MLLr, acute leukemias including ALL and AML, and
NPM1-mutant AML. Positive topline results from the Phase 2
AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial
met its primary endpoint were presented at the
65th American Society of Hematology Annual Meeting and
data from the Phase 1 portion of AUGMENT-101 in acute leukemia
was published in Nature. Revumenib was granted
Orphan Drug Designation by the FDA and European
Commission for the treatment of patients with AML, and Fast
Track designation by the FDA for the treatment of adult and
pediatric patients with R/R acute leukemias harboring a KMT2A
rearrangement or NPM1 mutation. Revumenib was granted Breakthrough
Therapy Designation (BTD) by the FDA for the treatment of adult and
pediatric patients with R/R acute leukemia harboring a KMT2A
rearrangement.
About NPM1-Mutant Acute Myeloid Leukemia
NPM1-mutant AML, which is distinguished by 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 KMT2A-rearranged
acute leukemia, NPM1-mutant AML is highly dependent on the
expression of specific developmental genes shown to be negatively
impacted by inhibitors of the menin-KMT2A 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
Syndax Pharmaceuticals is a clinical stage biopharmaceutical
company developing an innovative pipeline of cancer therapies.
Highlights of the Company's pipeline include revumenib, a highly
selective inhibitor of the menin–KMT2A binding interaction, and
axatilimab, a monoclonal antibody that blocks the colony
stimulating factor 1 (CSF-1) receptor. For more information, please
visit. For more information, please visit www.syndax.com or
follow the Company on X (formerly
Twitter) and LinkedIn.
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 "anticipate," "believe," "could," "estimate,"
"expects," "intend," "may," "plan," "potential," "predict,"
"project," "should," "will," "would" or the negative or plural of
those terms, 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
progress, timing, clinical development and scope of clinical
trials, the reporting of clinical data for Syndax's product
candidates, and the potential use of its 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; 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 Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
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SOURCE Syndax Pharmaceuticals, Inc.