– BLA submitted for axatilimab in chronic
graft-versus-host disease; Syndax exercised option to
co-commercialize axatilimab in the U.S. with Incyte
–
– NDA submitted for revumenib in R/R KMT2Ar
acute leukemia under U.S. FDA's RTOR program –
– Completion of enrollment in AUGMENT-101
mNPM1 patient cohort expected in late 1Q/early
2Q with topline data anticipated in 4Q24 –
– Completion of $230
million follow-on offering in December extends runway
through 2026 –
WALTHAM,
Mass., Jan. 2, 2024 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical
company developing an innovative pipeline of cancer therapies,
today highlighted recent updates and anticipated 2024
milestones.
"With positive pivotal data readouts for both
revumenib and axatilimab and subsequent presentations at the
American Society of Hematology Annual Meeting, 2023 was truly a
landmark year for Syndax," said Michael A.
Metzger, Chief Executive Officer. "We are working with the
FDA during their review of our regulatory submissions while
diligently preparing for the potential launch of two first- and
best-in-class products in the U.S. in 2024. We look forward to
continuing to broadly expand revumenib and axatilimab beyond
their first approvals and into additional indications which have
the potential to deliver meaningful clinical benefit."
Recent Company Updates and Planned 2024
Milestones
Revumenib:
- The Company announced the submission of a New Drug Application
(NDA) under the U.S. Food and Drug Administration (FDA) Real-time
Oncology Review (RTOR) program to the FDA for revumenib, a
first-in-class menin inhibitor, for the treatment of adult and
pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar)
acute leukemia on December 29, 2023.
As per RTOR guidance, the submission is not deemed complete
until the FDA issues a PDUFA date, which Syndax expects to receive
in the first quarter. RTOR allows for close engagement between the
sponsor and the FDA throughout the submission process and enables
the FDA to review individual modules of a drug application rather
than requiring a complete application prior to initiating its
review.
- The Company expects to complete enrollment in the AUGMENT-101
pivotal trial cohort of patients with R/R mutant nucleophosmin
(mNPM1) acute myeloid leukemia (AML) later this quarter or early in
the second quarter. The Company plans to report topline data in the
fourth quarter of 2024, which could support a regulatory filing for
revumenib in an additional indication of R/R mNPM1 AML.
- At the 65th American Society of Hematology (ASH) Annual Meeting
in December 2023, the Company
highlighted positive results in multiple presentations from the
Phase 1 and 2 portions of the AUGMENT-101 trial, including
the pivotal AUGMENT-101 results that were featured as a
late-breaking oral presentation.
- Investigators presented data from multiple Phase 1
combination trials of revumenib in mNPM1 and KMT2Ar acute
leukemia across the treatment landscape at the ASH Annual Meeting
and the Company's corporate event. The trials are expanding to
validate the recommended Phase 2 dose and the Company expects to
have additional data on the trials in the second half of 2024.
These include:
- BEAT AML: Evaluating the combination of revumenib with
venetoclax and azacitidine in front-line AML patients. This trial
is being conducted as part of the Leukemia & Lymphoma Society's
Beat AML® Master Clinical Trial.
- SAVE: Evaluating the all-oral combination of revumenib
with venetoclax and decitabine/cedazuridine in R/R AML or mixed
phenotype acute leukemias. The trial is being conducted by
investigators from the MD Anderson Cancer Center and continues to
accrue patients.
- AUGMENT-102: Evaluating the combination of revumenib with
fludarabine and cytarabine in patients with R/R acute
leukemias.
- The Company plans to initiate a trial of revumenib in
combination with 7+3 cytarabine and daunorubicin chemotherapy
followed by maintenance treatment in newly diagnosed patients with
mNPM1 or KMT2Ar acute leukemias this quarter.
- The Company plans to initiate a pivotal trial of revumenib
in combination with venetoclax and azacitidine in newly diagnosed
patients with mNPM1 or KMT2Ar acute leukemias by the end of
2024.
- Enrollment is ongoing in a Phase 1 proof-of-concept clinical
trial of revumenib in patients with unresectable metastatic
microsatellite stable colorectal cancer. The Company expects to
provide an update on the trial in the first half of 2024.
Axatilimab:
- The Biologics License Application (BLA) for axatilimab, an
anti-CSF-1R antibody, in adult and pediatric patients six years or
older with chronic graft-versus-host disease (cGVHD) after failure
of at least two prior lines of systemic therapy was submitted to
the FDA on December 28, 2023.
- Results from the pivotal Phase 2 AGAVE-201 trial were
featured in the Plenary Scientific Session at the 65th ASH Annual
Meeting in December 2023.
- Syndax announced today that it has exercised its option under
the Company's 2021 collaboration agreement with Incyte to
co-commercialize axatilimab in the U.S.
- Syndax announced today the randomized, double-blind and
placebo-controlled Phase 2 trial to assess the efficacy, safety and
tolerability of axatilimab in patients with idiopathic pulmonary
fibrosis (IPF) is open for enrollment.
- Additionally, Incyte plans to initiate two combination
trials with axatilimab in cGVHD in mid-2024, including a Phase 2
combination trial with ruxolitinib and a Phase 3 combination trial
with steroids.
Corporate
- In the fourth quarter of 2023, Syndax issued 12,432,431 shares
of its common stock at $18.50 per
share. Additionally in the quarter, the Company sold 2,719,744
shares from its ATM facility. Aggregate net proceeds from these
offerings were approximately $258.1
million after deducting underwriting discounts and
commissions and estimated offering expenses payable by Syndax.
With these proceeds, the Company now believes that it has
sufficient cash runway to fund its clinical and commercial
operations through 2026.
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
recently 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 BTD by the FDA for the treatment of adult and
pediatric patients with R/R acute leukemia harboring a KMT2A
rearrangement.
About Axatilimab
Axatilimab is an investigational monoclonal
antibody that targets colony stimulating factor-1 receptor, or
CSF-1R, a cell surface protein thought to control the survival and
function of monocytes and macrophages. In pre-clinical models,
inhibition of signaling through the CSF-1 receptor has been shown
to reduce the number of disease-mediating macrophages along with
their monocyte precursors, which has been shown to play a key role
in the fibrotic disease process underlying diseases such as chronic
graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis
(IPF). Positive topline results from the Phase 2 AGAVE-201 trial
showing the trial met its primary endpoint were
recently presented at the 65th American Society of
Hematology Annual Meeting and Phase 1/2 data of axatilimab in
chronic GVHD were published in the Journal of
Clinical Oncology. Axatilimab was granted Orphan Drug
Designation by the U.S. Food and Drug Administration for
the treatment of patients with chronic GVHD and IPF.
In September 2021, Syndax and Incyte entered into an
exclusive worldwide co-development and co-commercialization license
agreement for axatilimab. Axatilimab is being developed under an
exclusive worldwide license from UCB entered into between Syndax
and UCB in 2016.
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 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 "may," "will," "expect," "plan,"
"anticipate," "estimate," "intend," "could," "believe" and similar
expressions such as "look forward" (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 sufficiency of the Company's cash runway, 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;
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.