Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical
company focused on developing life-changing therapies for allergic
and immunologic diseases, today announced that navenibart
(STAR-0215) has been granted Orphan Drug Designation for the
treatment of hereditary angioedema (HAE) by the U.S. Food and Drug
Administration (FDA). Navenibart is a monoclonal antibody inhibitor
of plasma kallikrein designed to provide long-acting attack
prevention for HAE. Initial results from the Phase 1b/2 ALPHA-STAR
clinical trial of navenibart have demonstrated a favorable safety
and tolerability profile and a reduction of monthly attack rates by
90-96% when dosed once or twice over six months.
“Receiving orphan drug designation for navenibart is an
important affirmation of our belief that there is a significant
unmet need for people living with HAE,” said Jill C. Milne, Ph.D.,
Chief Executive Officer. “We believe navenibart has the potential
to be the market-leading HAE treatment because of its trusted
mechanism and modality, efficacy observed to date, and low
treatment burden with infrequent dosing, and think that navenibart
could change the way that people live with their HAE. We expect to
share additional results from the ALPHA-STAR trial in Q4 and to
progress navenibart into a Phase 3 trial initiating in the first
quarter of 2025.”
The FDA’s Orphan Drug Designation program grants orphan status
to medicines intended for the safe and effective prevention,
diagnosis, or treatment of rare diseases or disorders that affect
fewer than 200,000 people in the United States. Orphan status
provides sponsors with development and commercial incentives for
designated compounds and medicines.
About Navenibart (STAR-0215):
Navenibart is a monoclonal antibody inhibitor of plasma
kallikrein in development for the treatment of HAE. Our goal with
navenibart is to provide rapid and sustained HAE attack prevention
with a validated mechanism and trusted modality administered every
3 and 6 months. The FDA has granted navenibart Orphan Drug and Fast
Track Designations. For more information on navenibart, please
visit www.astriatx.com.
About Astria Therapeutics:
Astria Therapeutics is a biopharmaceutical company, and our
mission is to bring life-changing therapies to patients and
families affected by allergic and immunologic diseases. Our lead
program, navenibart (STAR-0215), is a monoclonal antibody inhibitor
of plasma kallikrein in clinical development for the treatment of
hereditary angioedema. Our second program, STAR-0310, is a
monoclonal antibody OX40 antagonist in preclinical development for
the treatment of atopic dermatitis. Learn more about our company on
our website, www.astriatx.com, or follow us on X and Instagram
@AstriaTx and on Facebook and LinkedIn.
Forward-Looking Statements:
This press release contains forward-looking statements within
the meaning of applicable securities laws and regulations
including, but not limited to, statements regarding: our
expectations about the potential significance of the initial
results from the Phase 1b/2 ALPHA-STAR clinical trial of
navenibart; the expected timing of the release of additional data
from the ALPHA-STAR trial; the expected timing of initiation of the
planned navenibart Phase 3 trial and the receipt of topline results
from such trial, subject to regulatory feedback; the potential for
navenibart in the HAE market, including the potential to be the
market leading treatment, the potential therapeutic benefits of
navenibart as a treatment for HAE, the administration timing of
navenibart, and our overall vision and goals for the navenibart
program; the potential unmet medical need for HAE; and the goal of
bringing life changing therapies to patients and families affected
by allergic and immunologic diseases and to become a leading
allergy and immunology company. The use of words such as, but not
limited to, “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “goals,” “intend,” “may,” “might,” “plan,”
“potential,” “predict,” “project,” “should,” “target,” “will,”
“would,” or "vision," and similar words expressions are intended to
identify forward-looking statements. Forward-looking statements are
neither historical facts nor assurances of future performance.
Instead, they are based on Astria’s current beliefs, expectations
and assumptions regarding the future of its business, future plans
and strategies, future financial performance, results of
pre-clinical and clinical results of the Astria’s product
candidates and other future conditions. Actual results may differ
materially from those indicated by such forward-looking statements
as a result of various important factors, including the following
risks and uncertainties: changes in applicable laws or regulations;
the possibility that we may be adversely affected by other
economic, business, and/or competitive factors; risks inherent in
pharmaceutical research and development, such as: adverse results
in our drug discovery, preclinical and clinical development
activities, the risk that the results of preclinical studies,
including of navenibart and STAR-0310, may not be replicated in
clinical trials, that the preliminary or interim results from
clinical trials may not be indicative of the final results, that
the results of early stage clinical trials, such as the results
from the navenibart Phase 1a clinical trial and the initial results
from the ALPHA-STAR trial, may not be replicated in later stage
clinical trials, including additional and final results from the
ALPHA-STAR trial or the planned navenibart Phase 3 development
program, the risk that we may not be able to enroll sufficient
patients in our clinical trials on a timely basis, and the risk
that any of our clinical trials may not commence, continue or be
completed on time, or at all; decisions made by, and feedback
received from, the U.S. Food and Drug Administration and other
regulatory authorities on our regulatory and clinical trial
submissions and other feedback from potential clinical trial sites,
including investigational review boards at such sites, and other
review bodies with respect to navenibart, STAR-0310, and any other
future development candidates, and devices for such product
candidates; our ability to manufacture sufficient quantities of
drug substance and drug product for navenibart, STAR-0310, and any
other future product candidates, and devices for such product
candidates, on a cost-effective and timely basis, and to develop
dosages and formulation for navenibart, STAR-0310, and any other
future product candidates that are patient-friendly and
competitive; our ability to develop biomarker and other assays,
along with the testing protocols therefore; our ability to obtain,
maintain and enforce intellectual property rights for navenibart,
STAR-0310, and any other future product candidates; our potential
dependence on collaboration partners; competition with respect to
navenibart, STAR-0310, or any of our other future product
candidates; the risk that survey results and market research may
not be accurate predictors of the commercial landscape for HAE, the
ability of navenibart to compete in HAE and the anticipated
position and attributes of navenibart in HAE based on clinical data
to date, its preclinical profile, pharmacokinetic modeling, market
research and other data; risks with respect to the ability of
STAR-0310 to compete in AD and the anticipated position and
attributes of STAR-0310 in AD based on its preclinical profile; our
ability to manage our cash usage and the possibility of unexpected
cash expenditures; our ability to obtain necessary financing to
conduct our planned activities and to manage unplanned cash
requirements; the risks and uncertainties related to our ability to
recognize the benefits of any additional acquisitions, licenses or
similar transactions; and general economic and market conditions;
as well as the risks and uncertainties discussed in the “Risk
Factors” section of our Annual Report on Form 10-K for the period
ended December 31, 2023 and in other filings that we may make with
the Securities and Exchange Commission.
New risks and uncertainties may emerge from time to time, and it
is not possible to predict all risks and uncertainties. Astria may
not actually achieve the forecasts or expectations disclosed in our
forward-looking statements, and investors and potential investors
should not place undue reliance on Astria’s forward-looking
statements. Neither Astria, nor its affiliates, advisors or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as result of new
information, future events or otherwise, except as required by law.
These forward-looking statements should not be relied upon as
representing Astria’s views as of any date subsequent to the date
hereof.
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version on businesswire.com: https://www.businesswire.com/news/home/20240930717552/en/
Astria: Investor Relations and
Media: Elizabeth Higgins investors@astriatx.com
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