Aquestive Therapeutics, Inc. (NASDAQ: AQST) (the “Company” or
“Aquestive”), a pharmaceutical company advancing medicines to solve
patients’ problems with current standards of care and provide
transformative products to improve their lives, today announced the
completion of its End-of-Phase 2 (EOP2) meeting with the U.S. Food
and Drug Administration (“FDA”) for AQST-109 (epinephrine
sublingual film) for the treatment of severe allergic reactions
including anaphylaxis.
The final EOP2 FDA meeting minutes provided
clarity to the Company as to the FDA’s expectations regarding key
program areas related to pharmacokinetics, administration and use
that should be addressed given the life-threatening condition that
may be treated with AQST-109, if approved.
The FDA provided clear guidance on its
expectation that AQST-109 PK performance be reasonably bracketed
between approved injectable epinephrine products with similarity to
epinephrine PK via auto-injectors, since autoinjectors are the most
commonly used device for self-treatment. From a reference listed
product perspective, the FDA urged the Company to utilize multiple
approved injectable products to create a bracketing of high and low
epinephrine plasma concentrations as well as mean maximum
concentration (Cmax) and partial area under the curve (AUC) within
30 minutes to 60 minutes after dosing (as determined by the
Company) with an emphasis on the early timepoints. Based on
existing data, Aquestive believes it can achieve this ‘bracketing’
standard in an adequately powered, well-controlled pivotal PK
study.
The FDA also provided comments on potential
issues the Company will need to address in regard to a sublingually
delivered product including (1) the impact of any product hold
time, (2) the potential for emesis (vomiting), and (3) the impact
of potential mouth conditions such as angioedema (swelling). At
this time, the Company believes it can provide sufficient data
related to administration and use conditions without overall delay
to the New Drug Application (“NDA”) submission timeline.
In addition, the FDA acknowledged that
pharmacodynamic parameters (PD) (vital signs) following AQST-109
are generally higher than following administration of injectable
products, and that the higher PD responses provided some supportive
evidence to the PK-driven development program. The FDA further
noted that use of these data as supportive evidence of acceptable
PK should be accompanied by further characterization to explain
similarities or any differences in mechanism of action.
“This positive interaction with the FDA marks a
major step forward for our AQST-109 program,” said Daniel Barber,
Chief Executive Officer of Aquestive. “We continue to educate the
FDA on our technology while also gaining important insights into
the review division’s expectations for a complete and thorough
submission. We remain excited to bring AQST-109 to market, if
approved by the FDA. We believe the appropriate data can be
generated to create a robust review package and we plan on doing
this as rapidly as possible.”
“After participating with Aquestive in its
recent FDA interaction, I remain excited by the potential benefits
that AQST-109 could provide to patients,” remarked David B.K.
Golden, MD, Associate Professor, Department of Allergy and Clinical
Immunology at Johns Hopkins. “Combining portability with an orally
delivered route of administration that delivers fast absorption
creates the potential for increasing the number of patients who
have their rescue medication with them when needed. I look forward
to continuing to work with Aquestive as it progresses this
important program.”
About AnaphylaxisAnaphylaxis is
a serious systemic hypersensitivity reaction with rapid onset and
potentially fatal. As many as 49 million people in the United
States are at chronic risk for anaphylaxis. Lifetime prevalence is
at least 5%, or more than 16 million people in the United States.
Direct costs of anaphylaxis have been estimated at $1.2 billion per
year, with direct expenditures of $294 million for epinephrine, and
indirect costs of $609 million. The frequency of hospital
admissions for anaphylaxis has increased 500–700% in the last 10–15
years. 52% of patients, who previously experienced anaphylaxis, had
never received an epinephrine autoinjector prescription, and 60%
did not have an autoinjector currently available. The most common
causes of anaphylaxis are foods (such as peanuts), venom from
insect stings, and medications. Epinephrine injection is the
current standard of treatment intended to reverse the severe
manifestation of anaphylaxis, which may include skin rash, throat
swelling, respiratory problems, gastrointestinal distress, and loss
of consciousness.
About AQST-109AQST-109 is a
polymer matrix-based epinephrine prodrug administered as a
sublingual film that is applied under the tongue for the rapid
delivery of epinephrine. The product candidate is similar in size
to a postage stamp, weighs less than an ounce, and begins to
dissolve on contact. No water or swallowing is required for
administration. The packaging for AQST-109 is thinner and smaller
than an average credit card, can be carried in a pocket, and is
designed to withstand weather excursions such as exposure to rain
and/or sunlight.
About Aquestive
TherapeuticsAquestive Therapeutics, Inc. (NASDAQ: AQST) is
a pharmaceutical company advancing medicines to solve patients’
problems with current standards of care and provide transformative
products to improve their lives. We are developing orally
administered products to deliver complex molecules, providing novel
alternatives to invasive and inconvenient standard of care
therapies. Aquestive has five commercialized products, marketed by
our licensees in the U.S. and around the world. The Company also
collaborates with pharmaceutical companies to bring new molecules
to market using proprietary, best-in-class technologies, like
PharmFilm®, and has proven drug development and commercialization
capabilities. Aquestive is advancing a late-stage proprietary
product pipeline focused on treating diseases of the central
nervous system and an earlier stage pipeline for the treatment of
severe allergic reactions, including anaphylaxis.
Forward-Looking Statements
Certain statements in this press release include
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995. Words such as “believe,”
“anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,”
“will,” or the negative of those terms, and similar expressions,
are intended to identify forward-looking statements. These
forward-looking statements include, but are not limited to,
statements regarding not needing efficacy studies in the
comparability submission for AQST-109 FDA approval, the timing of
the AQST-109 product launch and NDA submission, the Company’s
ability to provide sufficient data to address the FDA’s concerns,
the potential benefits AQST-109 could bring to patients, and other
statements that are not historical facts. These forward-looking
statements are subject to the uncertain impact of the COVID-19
global pandemic on the Company’s business including with respect to
its clinical trials including site initiation, enrollment and
timing and adequacy of clinical trials; on regulatory submissions
and regulatory reviews and approval of AQST-109; pharmaceutical
ingredient and other raw materials supply chain, manufacture, and
distribution; and ongoing availability of an appropriate labor
force and skilled professionals.
These forward-looking statements are based on
the Company’s current expectations and beliefs and are subject to a
number of risks and uncertainties that could cause actual results
to differ materially from those described in the forward-looking
statements. Such risks and uncertainties include, but are not
limited to, risks associated with the Company’s development work,
including any delays or changes to the timing, cost and success of
its product development activities and clinical trials for
AQST-109; risk of the Company’s failure to generate sufficient data
in its PK/PD comparability submission for FDA approval of AQST-109;
risk of the Company’s failure to address the concerns identified in
the FDA E0P2 meeting for AQST-109; risk of delays in or the failure
to receive FDA approval of AQST-109; risk of insufficient capital
and cash resources, including insufficient access to available debt
and equity financing and revenues from operations, to satisfy all
of the Company’s short-term and longer term liquidity and cash
requirements and other cash needs, at the times and in the amounts
needed; uncertainties related to general economic, political,
business, industry, regulatory and market conditions and other
unusual items; and other risks and uncertainties affecting the
Company described in the “Risk Factors” section and in other
sections included in its Annual Report on Form 10-K, in its
Quarterly Reports on Form 10-Q, and in its Current Reports on Form
8-K filed with the Securities and Exchange Commission. Given those
uncertainties, you should not place undue reliance on these
forward-looking statements, which speak only as of the date made.
All subsequent forward-looking statements attributable to the
Company or any person acting on its behalf are expressly qualified
in their entirety by this cautionary statement. The Company assumes
no obligation to update forward-looking statements or outlook or
guidance after the date of this press release whether as a result
of new information, future events or otherwise, except as may be
required by applicable law.
Investor Inquiries:ICR
WestwickeStephanie
Carringtonstephanie.carrington@westwicke.com646-277-1282
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