– PDUFA action date set for September 26, 2024 –
– NDA being reviewed under FDA's RTOR
program –
WALTHAM,
Mass., March 26, 2024
/PRNewswire/ -- Syndax Pharmaceuticals (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 Priority Review
for its New Drug Application (NDA) for revumenib, the Company's
first-in-class menin inhibitor, for the treatment of adult and
pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar)
acute leukemia. The NDA filing is being reviewed under the FDA's
Real-Time Oncology Review Program (RTOR) and has been assigned a
Prescription Drug User Fee Act (PDUFA) target action date of
September 26, 2024. RTOR allows for a
more efficient review and close engagement between the sponsor and
the FDA throughout the submission process, which historically has
led to earlier approvals.
"The receipt of Priority Review for the revumenib
NDA filing is a significant milestone as we transition to a leading
commercial-stage oncology company with the planned launches of two
first- and best-in class drugs in 2024," said Michael A. Metzger, Chief Executive Officer.
"With two regulatory filings now under FDA Priority Review, our
team is focused on commercial preparations to enable Syndax's
continued success as we enter this next stage of growth."
The NDA submission is supported by positive data
from the pivotal AUGMENT-101 trial of revumenib in adult and
pediatric patients with KMT2Ar acute myeloid leukemia (AML) and
acute lymphoid leukemia (ALL). As previously reported, the trial
met its primary endpoint at the protocol-defined interim analysis
with a complete remission (CR) or a CR with partial hematological
recovery (CRh) rate of 23% (13/57; 95% confidence interval [CI]:
[12.7, 35.8, one-sided p-value = 0.0036]) among the 57 efficacy
evaluable patients in the pooled KMT2Ar acute leukemia population.
70% of patients who achieved a CR/CRh and were assessed for minimal
residual disease (MRD) were MRD negative. Additionally, 63%
(36/57) of the efficacy-evaluable patients achieved an overall
response, 39% (14/36) of whom underwent hematopoietic stem cell
transplant (HSCT), with 50% (7/14) restarting revumenib as
post-transplant maintenance at the time of the data cutoff.
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 Real-Time Oncology Review (RTOR)
RTOR provides a more efficient review process for
oncology drugs to ensure that safe and effective treatments are
available to patients as early as possible, while improving review
quality and engaging in early iterative communication with the
applicant. Specifically, it allows for close engagement between the
sponsor and the FDA throughout the submission process and it
enables the FDA to review individual sections of modules of a drug
application rather than requiring the submission of complete
modules or a complete application prior to initiating review.
Additional information about RTOR can be found
at: https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program
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 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.