WALTHAM, Mass., March 31, 2021 /PRNewswire/ -- Syndax
Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (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 Orphan Drug
Designation to axatilimab, its anti-CSF-1R monoclonal antibody, for
the treatment of patients with chronic graft versus host disease
(cGVHD).
"Receipt of Orphan Drug Designation underscores axatilimab's
potential to serve as a safe and effective intervention for
patients with cGVHD," said Briggs W.
Morrison, M.D., Chief Executive Officer of Syndax. "Through
its ability to inhibit monocyte derived macrophages, which play a
key role in the fibrotic disease process, we believe axatilimab
could represent a meaningful therapeutic approach for cGVHD, as
well as other fibrotic diseases. As previously announced, our
pivotal Phase 2 AGAVE-201 trial is now underway in patients with
cGVHD, with topline results expected in 2023."
The FDA's Office of Orphan Drug Products grants Orphan Drug
Designation to support drug candidates in development for
underserved patient populations or rare disorders that affect fewer
than 200,000 people in the U.S. Orphan Drug Designation qualifies a
candidate for various development incentives, including tax credits
for eligible clinical trials, waiver of application fees, and
market exclusivity for seven years upon FDA approval.
At the 62nd American Society of Hematology (ASH)
Annual Meeting and Exposition in December 2020,
Syndax reported updated data from its Phase 1 trial of
axatilimab in patients with cGVHD
which demonstrated deep, durable responses and
multiorgan clinical benefit in patients refractory to multiple
therapeutic agents. The Company recently announced that the pivotal
Phase 2 AGAVE-201 trial, which will evaluate the safety and
efficacy of three doses and schedules of axatilimab in patients
with cGVHD, is now underway. The primary endpoint will assess
objective response rate based on the 2014 NIH consensus criteria
for GVHD, with key secondary endpoints including duration of
response and improvement in modified Lee Symptom Scale score. The
Company expects to report topline data in 2023.
About Chronic Graft Versus Host Disease
Chronic graft versus host disease (cGVHD), an immune response of
the donor-derived hematopoietic cells against recipient tissues, is
a serious, potentially life-threatening complication of allogeneic
hematopoietic stem cell transplantation (HSCT) which can last for
years. cGVHD is estimated to develop in approximately 40% of
transplant recipients, and affects approximately 14,000 patients in
the U.S.1,2 cGVHD typically manifests across
multiple organ systems, with skin and mucosa being commonly
involved, and is characterized by the development of fibrotic
tissue.3
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, and block the development of cutaneous and pulmonary
cGVHD. Axatilimab data has demonstrated deep, durable responses and
multiorgan clinical benefit in patients refractory to multiple
therapeutic agents to date, and is currently being evaluated
in a pivotal Phase 2 AGAVE-201 trial in patients with cGVHD.
About Syndax Pharmaceuticals, Inc.
Syndax Pharmaceuticals is a clinical stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. The Company's pipeline includes SNDX-5613, a
highly selective inhibitor of the Menin–MLL binding interaction,
axatilimab, a monoclonal antibody that blocks the colony
stimulating factor 1 (CSF-1) receptor, and entinostat, a class I
HDAC inhibitor.
Syndax's Cautionary Note on 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," "believe" 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
and the reporting of clinical data for Syndax's product candidates,
and the potential use of our product candidates to treat various
cancer indications 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, the COVID-19 pandemic may disrupt
our business and that of the third parties on which we depend,
including delaying or otherwise disrupting our clinical trials and
preclinical studies, manufacturing and supply chain, or impairing
employee productivity, 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 Contacts
Investor Contact
Melissa Forst
Argot Partners
melissa@argotpartners.com
Tel 212.600.1902
Media Contact
Ted Held
ted.held@gcihealth.com
Tel 212.798.9842
SNDX-G
1 SmartAnalyst 2020 SmartImmunology Insights
chronic GVHD report.
2 Bachier, CR. et al. ASH annual meeting 2019;
abstract #2109 Epidemiology and Real-World Treatment of Chronic
Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell
Transplantation: A U.S. Claims Analysis
3 Kantar 2020 GVHD Expert Interviews N=32
interviews
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SOURCE Syndax Pharmaceuticals, Inc.