Aquestive Therapeutics Announces FDA Confirmed 505(b)(2) Pathway for AQST-108 (Sublingual Film Formulation Delivering Systemi...
February 06 2020 - 7:00AM
Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty
pharmaceutical company focused on developing and commercializing
differentiated products that meet patients’ unmet needs and solve
therapeutic problems, announced today that it had a constructive
face-to-face pre-Investigational New Drug (IND) Application meeting
with the U.S. Food and Drug Administration (FDA) for its drug
candidate, AQST-108, a “first of its kind” oral sublingual film
formulation delivering systemic epinephrine that is in development
for the treatment of anaphylaxis using Aquestive’s proprietary
PharmFilm® technologies.
A pre-IND meeting provides an opportunity for an
open communication between a drug sponsor and the FDA to discuss
the sponsor’s IND development plan and to obtain the agency’s
guidance for clinical studies for the sponsor’s new drug candidate.
The FDA has confirmed that the clinical development for
AQST-108 will be reviewed under the 505(b)(2) pathway as proposed
by Aquestive and that no additional studies would be necessary
prior to opening the proposed IND Application. The FDA
indicated that there appears to be an unmet medical need among
patients who resist the standard of care use of intramuscular
injection in the treatment of anaphylaxis and that AQST-108 may
potentially address some of those unmet needs. Aquestive
plans to move forward with opening an IND and initiating its
pivotal pharmacokinetic (PK) clinical trials before the end of
2020.
“We are very pleased with the outcome of the
pre-IND meeting with the FDA. We look forward to preparing the IND
filing and commencing our pivotal PK study before year end,” said
Keith J. Kendall, Chief Executive Officer of Aquestive.
“Anaphylaxis is a serious condition that impacts a large patient
population for which there is a significant unmet need. The only
options currently available to patients require an injection and
AQST-108 can potentially bring meaningful innovation and positive
change for patients. We are focused on providing the first highly
portable, easy-to-administer and anxiety-free sublingual film
medication to treat this serious condition.”
About AQST-108AQST-108 is a
“first of its kind” oral sublingual film formulation delivering
systemic epinephrine that is in development for the treatment of
anaphylaxis using Aquestive’s proprietary PharmFilm®
technologies.
The data from the previously completed Phase 1
dose escalation study demonstrated that AQST-108 achieved similar
ranges of mean values of maximum concentration (Cmax) and time to
reach maximum concentration (Tmax) to that reported for injectables
EpiPen® and Auvi-Q®, provided a greater total exposure (AUC0-t;
area under the curve) than that reported for EpiPen and Auvi-Q, had
less interpatient variability when compared to degree of variation
(CV%) data reported for EpiPen and Auvi-Q, and was well tolerated,
with no study participants discontinuing participation due to an
adverse event.
Anaphylaxis is a potentially life-threatening
systemic allergic reaction, with an estimated incidence of 50 to
112 episodes per 100,000 people per year. The frequency of hospital
admissions for anaphylaxis has increased 500-700% in the last 10-15
years.1 The most common causes of reactions that can include
anaphylaxis are medications, foods (such as peanuts), and venom
from insect stings. Epinephrine injection is the current standard
of treatment intended to reverse the potentially severe
manifestation of anaphylaxis, which may include red rash, throat
swelling, respiratory problems, gastrointestinal distress and loss
of consciousness.
About Aquestive
TherapeuticsAquestive Therapeutics is a specialty
pharmaceutical company that applies innovative technology to solve
therapeutic problems and improve medicines for patients. Aquestive
is advancing a late-stage proprietary product pipeline to treat CNS
conditions and provide alternatives to invasively administered
standard of care therapies. The Company also collaborates with
other pharmaceutical companies to bring new molecules to market
using proprietary, best-in-class technologies, like PharmFilm®, and
has proven capabilities for drug development and
commercialization.
Forward-Looking StatementThis press release
includes 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 may include, but are not limited
to, statements regarding therapeutic benefits and plans and
objectives for regulatory approvals of the Company’s product
candidates; ability to obtain FDA approval and advance our product
candidates to the market; statements about our growth and future
financial and operating results and financial position, regulatory
approval and pathways, clinical trial timing and plans, our and our
competitors’ orphan drug approval and resulting drug exclusivity
for our products or products of our competitors, short-term and
long-term liquidity and cash requirements, cash funding and cash
burn, business strategies, market opportunities, and other
statements that are not historical facts.
These forward-looking statements are based on
our 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 our product
development activities and clinical trials and plans; risk of
delays in FDA approval of our drug candidate Libervant® (diazepam)
Buccal Film and our other drug candidates or failure to receive
approval; risk that a competitor obtains FDA orphan drug
exclusivity for a product with the same active moiety as the orphan
drug product for which we are seeking FDA approval and that such
earlier approved competitor orphan drug blocks our product in the
U.S. for seven years for the same indication; risk of our ability
to demonstrate to the FDA “clinical superiority” within the meaning
of FDA regulations of Libervant relative to the FDA-approved
Valtoco® (diazepam nasal spray) and Diastat® (diazepam rectal gel)
including by establishing a major contribution to patient care
within the meaning of FDA regulations relative to the approved
product and there can be no assurance that we will be successful;
risk inherent in commercializing a new product (including
technology risks, financial risks, market risks and implementation
risks and regulatory limitations); risk of development of our sales
and marketing capabilities; risk of legal costs associated with and
the outcome of our patent litigation challenging third party at
risk generic sale of our proprietary products; risk of sufficient
capital and cash resources, including access to available debt and
equity financing and revenues from operations, to satisfy all of
our short-term and longer term cash requirements and other cash
needs, at the times and in the amounts needed; risk of failure to
satisfy all financial and other debt covenants and of any default;
risk related to government claims against Indivior for which we
license, manufacture and sell Suboxone and which accounts for the
substantial part of our current operating revenues; risks
associated with Indivior’s announcement of its intention to cease
production of its authorized generic buprenorphine naloxone film
product, including the impact from loss of orders for the
authorized generic product and risk of eroding market share for
Suboxone and risk of sunsetting product; risks related to the
outsourcing of certain sales, marketing and other operational and
staff functions to third parties; risk of the rate and degree of
market acceptance of our products and product candidates; the
success of any competing products, including generics; risk of the
size and growth of our product markets; risk of compliance with all
FDA and other governmental and customer requirements for our
manufacturing facilities; risks associated with intellectual
property rights and infringement claims relating to the Company's
products; risk of unexpected patent developments; the impact of
existing and future legislation and regulatory provisions on
product exclusivity; legislation or regulatory action affecting
pharmaceutical product pricing, reimbursement or access; claims and
risks that may arise regarding the safety or efficacy of the
Company's products and product candidates; risk of loss of
significant customers; risks related to legal proceedings,
including patent infringement, investigative and antitrust
litigation matters; changes in governmental laws and regulations;
risk of product recalls and withdrawals; uncertainties related to
general economic, political, business, industry, regulatory and
market conditions and other unusual items; and other risks and
uncertainties affecting the Company including those described in
the “Risk Factors” section and in other sections included in the
Company's Annual Report on Form 10‑K filed with the SEC on March
14, 2019, in our quarterly reports on Form 10-Q, and in the Form
8-K filed on January 13, 2020. Given these 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 us or any person acting
on our 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.
PharmFilm® and the Aquestive logo are registered
trademarks of Aquestive Therapeutics, Inc. All other
registered trademarks referenced herein are the property of their
respective owners.
Media Inquiries: Mark
Corbaemark.corbae@icrinc.com858.213.7956
Investor Inquiries: Stephanie Carrington
stephanie.carrington@icrinc.com 646-277-1282
1 Epidemiology of anaphylaxis. Tejedor Alonso MA, Moro Moro M,
Mugica Garcia MC, Clin Exp Allergy. 45(6):1027-39, Jun
2015
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