aTyr Pharma Announces Third Positive DSMB Review for Efzofitimod in Phase 3 EFZO-FIT™ Study in Pulmonary Sarcoidosis
December 10 2024 - 7:00AM
aTyr Pharma, Inc. (Nasdaq: ATYR) (“aTyr” or the “Company”), a
clinical stage biotechnology company engaged in the discovery and
development of first-in-class medicines from its proprietary tRNA
synthetase platform, today announced the outcome of a third,
pre-planned interim safety analysis conducted by an independent
data and safety monitoring board (DSMB) for the ongoing Phase 3
EFZO-FIT™ study of the Company’s lead therapeutic candidate,
efzofitimod, in patients with pulmonary sarcoidosis. The DSMB
recommended that the study continue without any modifications.
“We are pleased to report yet another positive
safety review for efzofitimod, which includes all 268 patients that
have been enrolled in our global pivotal Phase 3 EFZO-FIT™ study,”
said Sanjay S. Shukla, M.D., M.S., President and Chief Executive
Officer of aTyr. “Safety is paramount when looking to provide a
disease modifying treatment for a chronic condition such as
pulmonary sarcoidosis, where reducing or replacing a toxic standard
of care such as oral corticosteroids could be highly meaningful and
improve quality of life for patients.”
EFZO-FIT™ is a global Phase 3 randomized,
double-blind, placebo-controlled study to evaluate the efficacy and
safety of efzofitimod in patients with pulmonary sarcoidosis. This
is a 52-week study consisting of three parallel cohorts randomized
equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo
dosed intravenously once a month for a total of 12 doses. The study
enrolled 268 subjects with pulmonary sarcoidosis at multiple
centers in the United States, Europe, Japan and Brazil. The trial
design incorporates a forced steroid taper. The primary endpoint of
the study is steroid reduction. Secondary endpoints include
measures of lung function and sarcoidosis symptoms. More
information on the EFZO-FIT™ study is available at
www.clinicaltrials.gov (NCT05415137).
About
Efzofitimod
Efzofitimod is a first-in-class biologic
immunomodulator in clinical development for the treatment of
interstitial lung disease (ILD), a group of immune-mediated
disorders that can cause inflammation and fibrosis, or scarring, of
the lungs. Efzofitimod is a tRNA synthetase derived therapy that
selectively modulates activated myeloid cells through neuropilin-2
to resolve inflammation without immune suppression and potentially
prevent the progression of fibrosis. aTyr is currently
investigating efzofitimod in the global Phase 3 EFZO-FIT™ study in
patients with pulmonary sarcoidosis, a major form of ILD, and in
the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis
(SSc, or scleroderma)-related ILD. These forms of ILD have limited
therapeutic options and there is a need for safer and more
effective, disease-modifying treatments that improve outcomes.
About aTyr
aTyr is a clinical stage biotechnology company
leveraging evolutionary intelligence to translate tRNA synthetase
biology into new therapies for fibrosis and inflammation. tRNA
synthetases are ancient, essential proteins that have evolved novel
domains that regulate diverse pathways extracellularly in humans.
aTyr’s discovery platform is focused on unlocking hidden
therapeutic intervention points by uncovering signaling pathways
driven by its proprietary library of domains derived from all 20
tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod,
a first-in-class biologic immunomodulator in clinical development
for the treatment of interstitial lung disease, a group of
immune-mediated disorders that can cause inflammation and
progressive fibrosis, or scarring, of the lungs. For more
information, please visit www.atyrpharma.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements are usually
identified by the use of words such as "anticipate," “believes,”
“designed,” “could” “can,” “expects,” “intends,” “may,” “plans,”
“potential,” “will,” and variations of such words or similar
expressions. We intend these forward-looking statements to be
covered by such safe harbor provisions for forward-looking
statements and are making this statement for purposes of complying
with those safe harbor provisions. These forward-looking statements
include, among others, statements regarding the clinical
development for efzofitimod, including the potential of efzofitimod
to be an improved treatment for pulmonary sarcoidosis over the
standard of care and our expectations with respect to the conduct,
timing and results of EFZO-FIT™. These forward-looking statements
also reflect our current views about our plans, intentions,
expectations, strategies and prospects, which are based on the
information currently available to us and on assumptions we have
made. Although we believe that our plans, intentions, expectations,
strategies and prospects, as reflected in or suggested by these
forward-looking statements, are reasonable, we can give no
assurance that the plans, intentions, expectations, strategies or
prospects will be attained or achieved. All forward-looking
statements are based on estimates and assumptions by our management
that, although we believe to be reasonable, are inherently
uncertain. Furthermore, actual results may differ materially from
those described in these forward-looking statements and will be
affected by a variety of risks and factors that are beyond our
control including, without limitation, uncertainty regarding
geopolitical and macroeconomic events, risks associated with the
discovery, development and regulation of efzofitimod, the risk that
we or our partners may cease or delay preclinical or clinical
development activities for efzofitimod for a variety of reasons
(including difficulties or delays in patient enrollment in planned
clinical trials), the possibility that existing collaborations
could be terminated early, and the risk that we may not be able to
raise the additional funding required for our business and product
development plans, as well as those risks set forth in our most
recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q
and in our other SEC filings. Except as required by law, we assume
no obligation to update publicly any forward-looking statements,
whether as a result of new information, future events or
otherwise.
Contact:Ashlee DunstonDirector,
Investor Relations and Public Affairsadunston@atyrpharma.com
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