aTyr Pharma Announces Phase 3 Study of Efzofitimod (ATYR1923) in Pulmonary Sarcoidosis
May 16 2022 - 8:00AM
aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged
in the discovery and development of innovative medicines from its
proprietary tRNA synthetase platform, today announced a Phase 3
study evaluating the efficacy and safety of its lead therapeutic
candidate, efzofitimod (ATYR1923), in patients with pulmonary
sarcoidosis. The study, which will be known as EFZO-FIT™, is
expected to initiate in the third quarter of 2022.
The EFZO-FIT™ study 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 will be 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 intends to enroll 264 subjects
with pulmonary sarcoidosis at multiple centers in North America,
Europe and Japan. The trial design will incorporate a forced
steroid taper. The primary endpoint of the study is steroid
reduction. Secondary endpoints include measures of lung function
and sarcoidosis symptoms.
Efzofitimod is a first-in-class immunomodulator
that downregulates innate and adaptive immune responses in
uncontrolled inflammatory diseases states via selective modulation
of neuropilin-2 (NRP2). Clinical proof-of-concept was recently
established for efzofitimod in a Phase 1b/2a study in patients with
pulmonary sarcoidosis, a major form of interstitial lung disease
(ILD).
“We are very pleased with the input we received
from the FDA regarding the design of this important study of
efzofitimod in pulmonary sarcoidosis patients,” said Sanjay S.
Shukla, M.D., M.S., President and CEO of aTyr. “We aligned with the
FDA on the prioritization of efficacy endpoints, with a primary
focus on steroid reduction, which is clinically meaningful to
patients and providers. This late-stage study is a major milestone
for aTyr and the sarcoidosis community, and we look forward to the
expected initiation of the study in the third quarter of this
year.”
“This study is a major step forward in
developing a new treatment for patients with sarcoidosis,” said
Robert P. Baughman, M.D., Emeritus Profess of Medicine at the
University of Cincinnati. “Treatment options for patients with
sarcoidosis are limited. Prednisone toxicity is the most common
complaint of patients on therapy. The steroid sparing primary
endpoint prioritized by the FDA highlights the need for new,
disease modifying treatment options that can improve clinical
outcomes while reducing steroid toxicity with the goal of truly
improving quality of life for patients.”
About Pulmonary Sarcoidosis
Pulmonary sarcoidosis is an inflammatory disease
characterized by the formulation of granulomas, clumps of
inflammatory cells, in one or more organs of the body.
Approximately 200,000 Americans live with pulmonary sarcoidosis
and the prognosis ranges from benign and self-limiting to
chronic, debilitating disease, permanent loss of lung function and
death. Current treatment options include corticosteroids and other
immunosuppressive therapies, which have limited efficacy and are
associated with serious side-effects that many patients cannot
tolerate long-term.
About
Efzofitimod
aTyr is developing efzofitimod as a potential
therapeutic for patients with fibrotic lung disease. Efzofitimod, a
fusion protein comprised of the immuno-modulatory domain of
histidyl-tRNA synthetase fused to the FC region of a human
antibody, is a selective modulator of neuropilin-2 that
downregulates innate and adaptive immune response in inflammatory
disease states. aTyr’s lead indication for efzofitimod is pulmonary
sarcoidosis, a major form of interstitial lung disease. Clinical
proof-of-concept for efzofitimod was recently established in a
Phase 1b/2a multiple-ascending dose, placebo-controlled study of
efzofitimod in patients with pulmonary sarcoidosis, which
demonstrated safety and a consistent dose response and trends of
benefit of efzofitimod compared to placebo on key efficacy
endpoints, including steroid reduction, lung function, clinical
symptoms and inflammatory biomarkers. aTyr intends to initiate
EFZO-FIT™, a Phase 3 study of efzofitimod in pulmonary sarcoidosis
patients, in the third quarter of 2022.
About aTyr
aTyr is a biotherapeutics company engaged in the
discovery and development of innovative medicines from its
proprietary tRNA synthetase platform. aTyr’s research and
development efforts are concentrated on a newly discovered area of
biology, the extracellular functionality and signaling pathways of
tRNA synthetases. aTyr has built a global intellectual property
estate directed to a potential pipeline of protein compositions
derived from 20 tRNA synthetase genes and their extracellular
targets. aTyr’s primary focus is efzofitimod, a clinical-stage
product candidate which binds to the neuropilin-2 receptor and is
designed to downregulate immune engagement in fibrotic lung
disease. 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 “anticipates,” “believes,”
“estimates,” “expects,” “intends,” “may,” “plans,” “projects,”
“seeks,” “should,” “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 statements regarding potential therapeutic benefits and
applications of efzofitimod; timelines and plans with respect to
certain development activities, including the timing of clinical
trials; and certain development goals. 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 or
strategies 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 the
COVID-19 pandemic, risks associated with the discovery, development
and regulation of our product candidates, the risk that we or our
partners may cease or delay preclinical or clinical development
activities for any of our existing or future product candidates 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 Quarterly Report on Form 10-Q for the
quarter ended March 31, 2022 filed with the SEC on May 10, 2022,
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 Dunston |
Director, Investor Relations and
Corporate Communications |
adunston@atyrpharma.com |
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