European Commission Grants Orphan Drug Designation for aTyr Pharma’s Efzofitimod for Treatment of Systemic Sclerosis
22 Junio 2023 - 7:00AM
aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged
in the discovery and development of first-in-class medicines from
its proprietary tRNA synthetase platform, today announced that the
European Commission (EC) granted orphan drug designation for the
company’s lead therapeutic candidate, efzofitimod, for the
treatment of systemic sclerosis (SSc, or scleroderma) based on the
opinion of the European Medicines Agency (EMA) Committee for Orphan
Medicinal Products (COMP).
Efzofitimod is a first-in-class immunomodulator
that downregulates innate immune responses in uncontrolled
inflammatory disease states via selective modulation of
neuropilin-2. Efzofitimod is currently being investigated in a
global pivotal Phase 3 clinical trial in patients with pulmonary
sarcoidosis, a major form of interstitial lung disease (ILD), and a
Phase 2 proof-of-concept study in patients with SSc-ILD is expected
to begin in the third quarter of 2023.
“We are pleased that the EC recognizes the need
for new and impactful treatments for the nearly 100,000 people
living with systemic sclerosis in the European Union (EU),” said
Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “This
orphan drug designation takes into account more than just the
rarity of the disease. In addition to the clinical proof-of-concept
data generated for efzofitimod in pulmonary sarcoidosis, the COMP
considered that the non-clinical in vivo data for efzofitimod
in systemic sclerosis, which demonstrated a reduction in the
decline of lung function and skin fibrosis not achieved with an
authorized medicine in the EU, constitutes a potentially
clinically relevant advantage over existing therapies. We believe
efzofitimod presents a much-needed opportunity to meaningfully
advance treatment options for those whose lungs are affected by
systemic sclerosis, and we look forward to the expected initiation
of our Phase 2 clinical trial of efzofitimod in patients with
SSc-ILD in the third quarter of this year.”
The EMA grants orphan status to products
intended for the treatment, prevention or diagnosis of a disease
that is life-threatening or chronically debilitating
for which either no satisfactory method of diagnosis, prevention,
or treatment exists, or if such method exists, the medicine is of
significant benefit to those affected by such condition. To benefit
from such designation, either the prevalence of such condition must
not be more than five in 10,000 people in the EU, or, if more
prevalent, it must be unlikely that the marketing of the medicine
would generate sufficient returns to justify the investment needed
for its development. Additionally, the investigational product must
be of significant benefit to those affected by the condition. EMA
orphan drug designation provides certain benefits, including the
potential for 10 years of marketing exclusivity following
regulatory approval in the EU, reduction in regulatory fees and a
centralized EU approval process. Efzofitimod received orphan drug
and Fast Track designations for systemic sclerosis from the United
States Food and Drug Administration (FDA) in 2022.
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
EU 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 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 disease
progression and are associated with significant toxicity.
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 is currently conducting
EFZO-FIT™, a Phase 3 study of efzofitimod in pulmonary sarcoidosis
patients.
About aTyr
aTyr is a biotherapeutics company engaged in the
discovery and development of first-in-class 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 “expects,” “intends,” “may,”
“plans,” “forward,” “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 potential therapeutic benefits and applications of
efzofitimod; timelines and plans with respect to certain
development activities, including the timing of our Phase 2
proof-of-concept clinical trial in patients with SSc-ILD; and the
potential benefits of orphan drug designation. 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
geopolitical and macroeconomic events and any resulting delays in
clinical trials, 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 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 Corporate
Communicationsadunston@atyrpharma.com
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