– Launched Revuforj®
(revumenib) for treatment of R/R acute leukemia with a KMT2A
translocation in adult and pediatric patients one year and older
–
– Revumenib added to NCCN Clinical Practice
Guidelines in Oncology (NCCN Guidelines®)
for AML and ALL –
– Niktimvo™ (axatilimab-csfr) approved
by U.S. FDA for treatment of chronic GVHD after failure
of at least two prior lines of systemic therapy in adult and
pediatric patients weighing at least 40 kg –
– sNDA filing for revumenib in R/R mNPM1 AML
expected in 1H25 based on positive pivotal data from AUGMENT-101
trial –
WALTHAM,
Mass., Jan. 13, 2025 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX) will present at the 43rd
Annual J.P. Morgan Healthcare Conference on Tuesday, January 14th at 10:30 a.m. PT/1:30 p.m.
ET. Ahead of the presentation, Syndax highlighted its recent
accomplishments and anticipated 2025 milestones.
"Building on a transformative 2024 with the FDA approvals of
Revuforj® and Niktimvo™, we are focused on
executing two outstanding U.S. launches for these first-in-class,
practice-changing medicines," said Michael
A. Metzger, Chief Executive Officer. "Syndax is
well-positioned for continued success and long-term growth with two
approved drugs launching into multi-billion-dollar markets, a clear
strategy to expand into additional indications, and a strong cash
position expected to fund operations through profitability."
2024 Key Accomplishments
Revumenib:
- Launched Revuforj (revumenib), the first and only U.S. Food and
Drug Administration (FDA) approved menin inhibitor, in late
November 2024. Revuforj was approved
by the FDA under the Agency's Real-Time Oncology Review (RTOR)
program for the treatment of relapsed or refractory (R/R) acute
leukemia with a KMT2A translocation in adult and pediatric patients
one year and older.
- Revumenib was added to the latest NCCN Clinical Practice
Guidelines in Oncology (NCCN Guidelines®) for acute
myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) as a
category 2A recommendation for R/R acute leukemia with a KMT2A
rearrangement.1
- Announced that the primary endpoint was met in the
protocol-defined efficacy population of 64 adults with R/R mNPM1
AML in the Phase 2 cohort of the AUGMENT-101 trial of
revumenib.
- Reported additional positive results from a post-hoc efficacy
analysis of all 77 R/R mNPM1 AML patients who met the efficacy
evaluable criteria in the Phase 2 cohort of AUGMENT-101.
- Published data from the pivotal Phase 2 portion of the
AUGMENT-101 trial of revumenib in adult and pediatric patients with
R/R KMT2A-rearranged (KMT2Ar) acute leukemia in the Journal of
Clinical Oncology.
- Presented a larger data set with longer follow-up from the
pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in
R/R KMT2Ar acute leukemia at the 66th American Society
of Hematology (ASH) Annual Meeting.
- Presented data from multiple ongoing trials evaluating
revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment
landscape. These trials include:
- BEAT AML: A Phase 1 trial evaluating the combination of
revumenib with venetoclax and azacitidine in newly diagnosed mNPM1
or KMT2Ar AML patients. The trial is being conducted as part of the
Leukemia & Lymphoma Society's Beat AML® Master
Clinical Trial. Updated data from the trial showed an overall
response rate (ORR)2 of 100% (37/37) and a composite
complete remission (CRc) rate of 95% (35/37).
- SAVE: A Phase 1/2 trial evaluating an all-oral combination of
revumenib with venetoclax and decitabine/cedazuridine in pediatric
and adult patients with R/R AML or mixed-lineage acute leukemia
(MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The
trial is being conducted by investigators from MD Anderson Cancer
Center. Updated data that showed an ORR of 82% (27/33) and a CR/CRh
rate of 48% (16/33) were presented at the 66th ASH
Annual Meeting.
Axatilimab:
- Received U.S. Food and Drug Administration (FDA) approval for
Niktimvo (axatilimab-csfr) for the treatment of chronic
graft-versus-host disease (GVHD) after failure of at least two
prior lines of systemic therapy in adult and pediatric patients
weighing at least 40 kg (88.2 lbs).
- Announced axatilimab-csfr was added to the latest NCCN
Guidelines as a category 2A recommendation for the treatment of
GVHD after the failure of at least two prior lines of systemic
therapy in adult and pediatric patients weighing at least 40
kg.
- Published results from the pivotal Phase 2 AGAVE-201 trial of
axatilimab in adult and pediatric patients with
recurrent/refractory active chronic GVHD in the New England
Journal of Medicine.
- Presented a secondary analysis of overall and organ-specific
responses from the pivotal Phase 2 AGAVE-201 trial of axatilimab in
adult and pediatric patients with recurrent/refractory active
chronic GVHD who had received at least two prior lines of systemic
therapy at the 66th ASH Annual Meeting.
- Initiated enrollment in the MAXPIRe trial, a 26-week
randomized, double-blinded, placebo-controlled Phase 2 trial of
axatilimab on top of standard of care in patients with idiopathic
pulmonary fibrosis (IPF).
- The Company's partner, Incyte, initiated a Phase 2, open-label,
randomized, multicenter trial of axatilimab in combination with
ruxolitinib in patients ≥12 years of age with newly diagnosed
chronic GVHD.
- The Company's partner, Incyte, initiated a Phase 3, randomized,
double-blind, placebo-controlled, multi-center trial that will
investigate the use of axatilimab in combination with
corticosteroids as initial treatment for chronic GVHD.
Corporate:
- Announced a $350 million royalty
funding agreement with Royalty Pharma based on U.S. net sales of
Niktimvo. Syndax expects that its cash, cash equivalents and
marketable securities, together with the $350 million from the royalty funding agreement
and anticipated product revenue and interest income, will enable
the company to reach profitability.
Expected 2025 Key Milestones
Revumenib:
- Maximize U.S. adoption of Revuforj as the preferred menin
inhibitor, leveraging our first mover advantage and robust clinical
data.
- Submit a supplemental NDA (sNDA) filing for revumenib in R/R
mNPM1 AML in the first half of 2025, followed by a potential FDA
approval around year-end 2025.
- Publish pivotal data from AUGMENT-101 trial in R/R mNPM1 AML in
the first half of 2025.
- Initiate a pivotal trial of revumenib in combination with
venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute
leukemia patients unfit to receive intensive chemotherapy in the
first quarter of 2025.
- Report Phase 1 data from a trial evaluating the combination of
revumenib with intensive chemotherapy (7+3) followed by revumenib
maintenance treatment in newly diagnosed patients with mNPM1 or
KMT2Ar acute leukemias in the second half of 2025.
- Initiate multiple frontline trials evaluating revumenib in
combination with intensive chemotherapy, starting in 2025.
- Present additional data at medical congresses from ongoing
trials of revumenib in combination with standard-of-care
agents.
Axatilimab:
- Launch Niktimvo in the U.S. in early first quarter of 2025. In
the U.S., Niktimvo will be co-commercialized by Syndax and
Incyte.
- Complete enrollment in MAXPIRe Phase 2 IPF trial in 2025 with
topline data expected in 2026.
Presentation at the 43rd Annual J.P. Morgan
Healthcare Conference
- Syndax will webcast its presentation from the 43rd Annual J.P.
Morgan Healthcare Conference on Tuesday,
January 14, 2025 at 10:30 a.m.
PT (1:30 p.m. ET). A live
webcast of the fireside chat can be accessed from the Investor
section of the Company's website at www.syndax.com, where a replay
of the event will also be available for a limited time.
About Syndax
Syndax Pharmaceuticals is a commercial-stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. Highlights of the Company's pipeline include
Revuforj® (revumenib), an FDA-approved menin inhibitor,
and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal
antibody that blocks the colony stimulating factor 1 (CSF-1)
receptor. Fueled by our commitment to reimagining cancer care,
Syndax is working to unlock the full potential of its pipeline and
is conducting several clinical trials across the continuum of
treatment. For more information, please
visit www.syndax.com/ or follow the Company
on X 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, the acceptance of Syndax and its partners' products in
the marketplace, sales, marketing, manufacturing and distribution
requirements, 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 to Revuforj's commercial availability, 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.
References
1. NCCN Clinical Practice Guidelines in Oncology (NCCN
Guidelines®) for Acute Myeloid Leukemia (Version 1.2025
– December 20, 2024); NCCN Clinical
Practice Guidelines in Oncology (NCCN Guidelines®) for
Acute Lymphoblastic Leukemia (Version 3.2024 – December 20, 2024); NCCN Clinical Practice
Guidelines in Oncology (NCCN Guidelines®) for Pediatric
Acute Lymphoblastic Leukemia (Version 2.2025 – December 16, 2024). NCCN makes no warranties of
any kind whatsoever regarding their content, use or application and
disclaims any responsibility for their application or use in any
way.
2. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS,
and PR; Composite complete remission (CRc) includes CR, CRh, CRp,
and CRi.
CR = Complete remission
CRh = Complete remission with partial hematologic recovery
CRp = Complete remission with incomplete platelet recovery
CRi = Complete remission with incomplete count recovery
MLFS = Morphologic leukemia-free state
PR = Partial response
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
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SOURCE Syndax Pharmaceuticals, Inc.