aTyr Pharma, Inc. (Nasdaq: LIFE) (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 that it has dosed the first
patient in its Phase 2 EFZO-CONNECT™ study. The proof-of-concept
study will evaluate the efficacy, safety and tolerability of the
Company’s lead therapeutic candidate, efzofitimod, compared to
placebo in patients with systemic sclerosis (SSc, or
scleroderma)-related interstitial lung disease (ILD).
Efzofitimod is a first-in-class biologic
immunomodulator that selectively modulates activated myeloid cells
through neuropilin-2 (NRP2) to resolve inflammation without immune
suppression and potentially prevent the progression of fibrosis.
Efzofitimod has been granted U.S. Food and Drug Administration
(FDA) and European Union orphan drug and U.S. FDA Fast Track
designations for SSc.
“We are very pleased to begin patient dosing in
EFZO-CONNECT™, which is our second clinical study for efzofitimod
in ILD,” said Sanjay S. Shukla, M.D., M.S., President and Chief
Executive Officer of aTyr. “Efzofitimod has been shown
preclinically to reduce lung and skin fibrosis in models of SSc,
and NRP2, efzofitimod’s binding partner, is expressed in the skin
of SSc patients. We believe there is compelling rationale that
efzofitimod has the potential to target the underlying disease
pathology central to this form of ILD and positively impact lung
function and improve outcomes in these patients.”
“Patients with SSc-ILD have limited treatment
options and poor prognosis, with ILD being their leading cause of
death,” said Kristin Highland, M.D., Director, Rheumatic Lung
Disease Program at the Cleveland Clinic. “This study, which
evaluates a therapy that targets the inflammatory and fibrotic
characteristics of this disease, is an important step forward
towards developing a treatment that can potentially improve the
prognosis and quality of life for patients in need.”
The Phase 2 study is a randomized, double-blind,
placebo-controlled, proof-of-concept study to evaluate the
efficacy, safety and tolerability of efzofitimod in patients with
SSc-ILD. This is a 28-week study with three parallel cohorts
randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or
placebo dosed intravenously monthly for a total of 6 doses. The
study intends to enroll 25 patients at multiple centers in the
United States. The primary objective of the study is to evaluate
the efficacy of multiple doses of intravenous efzofitimod on
pulmonary, cutaneous and systemic manifestations in patients with
SSc-ILD. Secondary objectives include safety and tolerability.
More information on the EFZO-CONNECT™ study is
available at www.clinicaltrials.gov (NCT05892614).
About
SSc-ILD
Systemic sclerosis is a chronic, progressive,
autoimmune disease characterized by inflammation and fibrosis of
connective tissues throughout the body, including the skin and
other internal organs. SSc that occurs in the lungs is called
SSc-ILD. It is estimated that approximately 100,000 people in the
U.S. are affected by SSc and up to 80% may develop ILD. SSc-ILD
causes inflammation in the lungs and, if left untreated, can result
in scarring, or fibrosis, that causes permanent loss of lung
function. ILD is the primary cause of death in patients with SSc.
Current treatment options for SSc-ILD are limited, mainly focus on
slowing lung function decline and are associated with significant
toxicity.
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 “believes,” “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 enrollment of patients in the
EFZO-CONNECT™ study and the potential of efzofitimod to target the
underlying disease pathology, positively impact lung function and
improve outcomes in patients with SSc-ILD. 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
Report on Form 10-Q and in our subsequent 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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