WALTHAM, Mass., April 8, 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 idiopathic pulmonary fibrosis
(IPF).
"IPF is a serious, life-limiting orphan disease, and today's
Orphan Drug Designation validates axatilimab's potential to serve
as an effective therapeutic option for the currently-underserved
patients living with this rare disease," said Briggs W. Morrison, M.D., Chief Executive
Officer of Syndax. "Building on promising data demonstrating
meaningful multiorgan clinical benefit in patients with chronic
graft versus host disease, we are actively evaluating options to
expand the axatilimab franchise into additional areas of high unmet
need where the monocyte-macrophage lineage plays a key role in the
fibrotic disease process. We look forward to providing updates on
these plans in the coming months."
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.
About Idiopathic Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis (IPF) is a serious, life-limiting
chronic lung disease characterized by fibrosis and scarring of lung
tissue with a median survival of 3-5 years after diagnosis.
Patients with IPF experience debilitating symptoms including
progressive shortness of breath, particularly with exertion,
chronic cough, fatigue, weakness, and chest discomfort. Currently
approved drugs slow but do not halt disease progression and the
only curative therapy is lung transplant, which is an option for
less than 5% of patients. Estimates indicate that IPF could affect
approximately 150,000 patients in the U.S. and approximately
260,000 patients across the seven major pharmaceutical markets (US,
Japan, UK, Spain, Germany, Italy, and France).1
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 (cGVHD) and idiopathic pulmonary fibrosis. Axatilimab
data has demonstrated deep, durable responses and multiorgan
clinical benefit in patients with cGVHD refractory to multiple
therapeutic agents, and is currently being evaluated in the
pivotal Phase 2 AGAVE-201 trial in patients with cGVHD. Axatilimab
was granted Orphan Drug Designation by the U.S. Food and Drug
Administration for the treatment of 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 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, 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 SMARTImmunology Insights. "Idiopathic Pulmonary
Fibrosis." Presentation, March
2020.
View original
content:http://www.prnewswire.com/news-releases/syndax-announces-orphan-drug-designation-granted-to-axatilimab-for-treatment-of-idiopathic-pulmonary-fibrosis-301265461.html
SOURCE Syndax Pharmaceuticals, Inc.