– IDMC recommendation to advance based on
favorable safety profile observed in Phase 1a portion of trial
–
WALTHAM,
Mass., June 6, 2024 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical
company developing an innovative pipeline of cancer therapies,
today announced that it has advanced into the Phase 1b portion of its Phase 1/2 proof-of-concept
trial of revumenib, the Company's highly selective, oral menin
inhibitor, as a monotherapy in patients with relapsed or refractory
(R/R) metastatic microsatellite stable (MSS) colorectal cancer
(CRC). The Company's decision was supported by the trial's
Independent Data Monitoring Committee (IDMC) following its recent
pre-planned review of initial data from the Phase 1a portion of the
trial.
"In our first clinical trial exploring expansion
of revumenib beyond hematological malignancies, initial
results from the Phase 1a portion of the trial are encouraging,"
said Neil Gallagher, M.D., Ph.D., President, Head of Research
and Development at Syndax. "We are particularly pleased to observe
a compelling safety profile consistent with our existing revumenib
dataset, as well as efficacy signals that include a 33% stable
disease rate at 16 weeks as a monotherapy, which compares favorably
with current standard-of-care and supports advancement into the
Phase 1b portion. As we continue to
focus on preparations for the potential launches of revumenib and
axatilimab later this year, we look forward to continuing to
explore the role revumenib could play in the treatment of R/R
metastatic MSS CRC."
The Phase 1/2 trial (NCT05731947) is designed to assess the
safety, tolerability, and anti-tumor activity of revumenib in
patients with relapsed or refractory metastatic MSS CRC. The Phase
1a dose escalation portion of the trial enrolled a total of 19
patients who had a median of four prior therapies across three dose
cohorts, including 163 mg, 226 mg, and 276 mg three times a day
(TID). Revumenib was well-tolerated at all dose levels tested
and the safety profile was consistent with the Company's previously
reported data. No Grade 3 or greater treatment-related adverse
events (TRAEs) were observed and the most common TRAEs were
decreased appetite, dysgeusia, nausea, and fatigue. In
addition, the initial efficacy results provide early clinical
support that revumenib may be able to impact disease progression in
R/R patients with metastatic MSS CRC. At doses believed to achieve
full target saturation, dose levels 2 and 3, 44% (4/9) of patients
had stable disease at 8 weeks, and 33% (3/9) of patients had stable
disease at 16 weeks. One patient with prolonged stable
disease remained on study for 32 weeks. Based on the initial data,
276 mg TID was selected as the go-forward dose in the Phase
1b portion.
About Metastatic MSS CRC
Metastatic microsatellite stable (MSS) colorectal cancer (CRC)
represents the second leading cause of cancer death in the U.S.
with an estimated incidence in the relapsed or refractory (R/R)
setting of over 55,000 patients per year. Activation of the
Wnt/β-catenin signaling pathway is believed to be a key initiating
step and growth driver for the majority of CRC tumors. The
menin-MLL1 protein complex has been shown to regulate β-catenin
activity and disrupting this complex through menin inhibition
blocks growth of Wnt/β-catenin driven CRC tumors in preclinical
models.
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 (KMT2Ar), also known as mixed lineage
leukemia rearranged or MLLr, acute leukemias including ALL and AML,
and mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML).
Positive topline results from the pivotal 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. Pivotal data from the AUGMENT-101
trial in R/R NPM1 AML patients are expected in the fourth quarter
of 2024. 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 by the FDA for the treatment of adult and
pediatric patients with R/R acute leukemia harboring a KMT2A
rearrangement. The NDA filing for revumenib in R/R KMT2Ar
acute leukemia is currently under Priority Review by the FDA under
RTOR with a PDUFA action date of September 26, 2024.
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 menin inhibitor, and axatilimab, a monoclonal antibody
that blocks the 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.