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 the results of a post-hoc
analysis of data from its Phase 1b/2a study of efzofitimod in
patients with pulmonary sarcoidosis. The analysis was presented in
a poster at the European Respiratory Society (ERS) International
Congress 2023, which is taking place September 9 – 13, 2023, in
Milan, Italy. The poster is available on the Company’s website.
“This new data from a post-hoc analysis, which
pools efzofitimod 3.0 and 5.0 mg/kg and placebo and efzofitimod 1.0
mg/kg doses from the Phase 1b/2a study of efzofitimod in patients
with pulmonary sarcoidosis, is yet another indicator of the robust
efficacy demonstrated in this study,” said Sanjay S. Shukla, M.D.,
M.S., President and CEO of aTyr. “The statistically significant
difference in the relapse rate following steroid taper seen in the
two highest efzofitimod dose groups, combined with significantly
improved FVC and quality of life measures, suggests that
efzofitimod has the potential to be the first steroid-sparing and
disease-modifying treatment for sarcoidosis.”
“Oral corticosteroids remain the mainstay of
treatment for patients with pulmonary sarcoidosis, although
long-term treatment often comes with severe side effects and
toxicity. Steroid tapers in these patients are challenging, as
symptoms and FVC can worsen when steroid dose is reduced,” said
Robert P. Baughman, M.D., Professor of Medicine at the University
of Cincinnati Medical Center. “This analysis demonstrating a
relapse rate limited to 7.7% for the efzofitimod therapeutic group
is exciting, as we would normally expect to see a relapse rate as
high as approximately 50% over the course of 6 months, which is
what was observed in the subtherapeutic group, and the difference
may even be more evident in a longer study. This is one of the few
studies to demonstrate a steroid sparing effect of a drug
associated with a significant improvement in patient outcome. A
treatment such as efzofitimod that can reduce steroid burden is
greatly needed.”
Therapeutic Doses of Efzofitimod
Significantly Improve Multiple Pulmonary Sarcoidosis Efficacy
Measures
The poster presents findings from a pooled,
post-hoc analysis of data from a Phase 1b/2a randomized,
double-blind, placebo-controlled, multiple ascending dose (1.0, 3.0
and 5.0 mg/kg) 24-week study of efzofitimod in patients with
pulmonary sarcoidosis receiving oral corticosteroid (OCS) dose ≥
10.0 mg/day. Patients were randomized 1:2 (placebo:efzofitimod) and
underwent a forced steroid taper in the first 8 weeks of the study.
Dose dependent improvements in steroid burden, FVC and patient
reported outcomes (PRO) were noted, though the study was not
powered for efficacy.
In this pooled analysis, the 3.0 mg/kg (N=8) and
5.0 mg/kg (N=9) efzofitimod arms were considered therapeutic, and
pooled. The placebo (N=12) and 1.0 mg/kg (N=8) efzofitimod arm,
which was considered subtherapeutic, were pooled. Time to relapse
for steroid use (defined as dose of OCS increased after OCS taper
to 5.0 mg or less of prednisone or equivalent for at least five
consecutive days), rate of change for FVC and proportion of
patients with changes that are multiples of the minimally
clinically important difference (MCID) in PRO (Kings Sarcoidosis
Questionnaire-Lung, or KSQ-L) were compared. Additionally, a
responder endpoint was proposed (defined as reduction in OCS from
baseline without worsening in FVC or PRO) and an analysis was
performed. Key findings include:
- 7.7% of
patients in the therapeutic group relapsed for steroid use compared
to 54.4% of patients in the placebo/subtherapeutic group
(p=0.017);
- The rate of
change for FVC was significantly improved for the therapeutic group
compared to the placebo/subtherapeutic group (p=0.035);
- 52.9% of
patients in the therapeutic group showed an increase ≥12 for KSQ-L
(3 times MCID) compared with 15.0% in the placebo/subtherapeutic
group (p=0.032); and
- 64.7% of
patients in the therapeutic group achieved response compared to
20.0% in the placebo/subtherapeutic group (p=0.008).
aTyr is currently conducting EFZO-FIT™, a global
Phase 3 randomized, double-blind, placebo-controlled 52-week study
to evaluate the efficacy and safety of 3.0 mg/kg and 5.0 mg/kg of
efzofitimod in 264 patients with pulmonary sarcoidosis. 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.
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 potential of efzofitimod to provide a
differentiated approach to resolving inflammation and preventing
the progression of fibrosis to be the first steroid sparing and
disease-modifying treatment for sarcoidosis and to reduce steroid
burden and significantly improve multiple pulmonary sarcoidosis
efficacy measures, and the potential applications of efzofitimod.
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 Dunston |
|
Director, Investor Relations and
Public Affairs |
|
adunston@atyrpharma.com |
|
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