Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical
company advancing medicines to solve patients’ problems with
current standards of care and provide transformative products to
improve their lives, today announced positive topline results from
the first three arms of Part 3 of the EPIPHAST study for its
AQST-109 epinephrine oral film.
The purpose of Part 3 was to continue to study
the administration of the film under a variety of conditions and
further characterize its pharmacokinetics, pharmacodynamics, and
safety. The first three arms were designed to assess the impact of
holding the film under the tongue and limiting swallowing for
different periods of time. These time periods were (1) the target
holding time of 4 minutes, (2) a 50% reduction in hold time to 2
minutes and (3) no hold time, or 0 minutes. The remaining two arms
of the study for which data are not yet available include (4)
dosing the film 2 minutes after eating a peanut butter sandwich and
(5) swallowing the film immediately with 240 mL of water.
In the first three arms of Part 3, AQST-109 12
mg continued to show rapid absorption with favorable
pharmacokinetics across a variety of key metrics at the target hold
time as follows:
- The median time to maximum
concentration (Tmax) was observed to be 12 minutes for AQST-109
compared to 50 minutes for the epinephrine 0.3mg intra-muscular
(IM) injection from Part 2 of the EPIPHAST study.
- The Area Under the Curve (AUC)
within the clinically relevant periods of 10 minutes, 20 minutes,
and 30 minutes in each of three arms were comparable for both
AQST-109 and the 0.3mg IM injection.
- The median time to reach 100 pg/mL,
which has been suggested to be the threshold for the onset of
hemodynamic effects, was 8 minutes for AQST-109 and 10 minutes for
the 0.3mg IM injection as reported in Part 2.
- Part 3 demonstrated maximum plasma
concentration (Cmax) values that were consistent with the Part 2
findings, 0.3mg IM Injection, as well as those previously reported
for approved injectable epinephrine devices such as EpiPen®.
A chart accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/4f56f5f2-7a84-41df-b527-3fe63fd52ddb
Study Results |
AQST-10912mg 4-minute
hold time(Target)(N=22
doses) |
AQST-10912mg 2-minute
hold time(N=23 doses) |
AQST-10912mg 0-minute
hold time(N=21 doses) |
AQST-10912 mg (from Part
2)(N=48 doses) |
Epinephrine IMInjection 0.3 mg(from Part
2)(N=48 doses) |
Arithmetic Mean Cmax (pg/mL) |
678.4 |
663.9 |
359.8 |
426.1 |
396.7 |
Geometric Cmax (pg/mL) |
350.4 |
303.9 |
211.2 |
274.3 |
350.6 |
AUC 0-10 minutes (hr*pg/mL) |
12.8 |
9.5 |
9.4 |
7.9 |
9.4 |
AUC 0-20 minutes (hr*pg/mL) |
51.2 |
45.7 |
30.9 |
33.1 |
23.0 |
AUC 0-30 minutes (hr*pg/mL) |
79.1 |
75.1 |
49.8 |
56.7 |
47.5 |
Median Tmax (minutes) |
12 |
15 |
15 |
15 |
50 |
“The latest study results from first three arms
of Part 3 of the EPIPHAST study confirm, once again, our ability to
deliver significant levels of epinephrine through the oral mucosa.
We have achieved a Tmax of 15 minutes or faster in multiple
studies, including last year’s Proof-of-Concept Study and now in
Parts 1, 2, and 3 of the EPIPHAST study,” said Dan Barber, Chief
Executive Officer of Aquestive. “We are anxious to share this data
with the FDA following the completion of the upcoming head-to-head
study with EpiPen. We continue to believe AQST-109 has the
potential to transform how patients and caregivers treat
anaphylaxis.”
“These results build on prior Phase 1 trials
showing the promise of AQST-109 - as a sublingually administered
medicine for Type I allergic reactions, including anaphylaxis - to
improve upon the current standard of care, namely epinephrine
auto-injectors,” said David Golden, M.D., Allergy
Division Chief at Medstar Franklin Square Hospital in
Baltimore. “The data from this trial demonstrate that AQST-109 has
the potential to fill an unmet medical need by providing an
epinephrine treatment that patients can more easily carry and more
quickly administer in a life-threatening emergency situation. I
look forward to the next phase of clinical trials and the continued
development of AQST-109.”
EPIPHAST is a randomized, open-label, three-part
adaptive design, crossover study in healthy adult subjects
comparing the pharmacokinetics and pharmacodynamics of epinephrine
delivered via Aquestive’s AQST-109 oral film compared to
intramuscular injection of epinephrine. The study is being
conducted pursuant to clearance from Health Canada.
Aquestive received a written response from the
U.S. Food and Drug Administration (FDA) in December 2021 to its
Pre-Investigational New Drug Application (IND) meeting submission
confirming that the development of AQST-109 for the treatment of
anaphylaxis under the 505(b)(2) approval pathway is acceptable.
Aquestive opened the IND for AQST-109 after receiving FDA clearance
in February 2022. AQST-109 met the regulatory criteria for Fast
Track designation as announced in March 2022.
Aquestive plans to conduct a repeat dosing
comparative study of AQST-109 and 0.3 mg EpiPen during the third
quarter 2022. This data, along with the complete EPIPHAST study
data, will be the basis for the End-of-Phase 2 meeting with the FDA
that the Company plans to request in the fourth quarter 2022.
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 acute anaphylactic episodes.
Lifetime prevalence may be higher than 5%. Chronic allergic illness
costs the US healthcare system more than $18 billion annually. The
frequency of hospital admissions for anaphylaxis has increased
500-700% in the last 10-15 years. 52% of patients, who had
previously experienced anaphylaxis, had never received an
epinephrine autoinjector prescription, and 60% did not have an
autoinjector currently available. 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 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 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 on
the U.S. market, four licensed products and one stand-alone
proprietary product to date, Sympazan® (clobazam) oral film for the
treatment of seizures associated with Lennox-Gastaut syndrome. Our
licensees market their products 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, or CNS, and an
earlier stage pipeline for the treatment of severe allergic
reactions, including anaphylaxis. For more information,
visit Aquestive.com and follow us on LinkedIn.
Forward-Looking
StatementCertain 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 the advancement and related timing of AQST-109
through the regulatory and development pipeline and clinical and
business strategies, market opportunities, 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
our business including with respect to our clinical trials
including site initiation, patient enrollment and timing and
adequacy of clinical trials; on regulatory submissions and
regulatory reviews and approvals of our product candidates;
pharmaceutical ingredient and other raw materials supply chain,
manufacture, and distribution; sale of and demand for our products;
our liquidity and availability of capital resources; customer
demand for our products and services; customers’ ability to pay for
goods and services; and ongoing availability of an appropriate
labor force and skilled professionals. Given these uncertainties,
the Company is unable to provide assurance that operations can be
maintained as planned prior to the COVID-19 pandemic.
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 for AQST-109 and our
other product candidates; risk of delays in FDA approval of
AQST-109, Libervant™ (diazepam) Buccal Film and our other drug
candidates or failure to receive FDA approval; ability to address
the concerns identified in the FDA’s Complete Response Letter dated
September 25, 2020 regarding the New Drug Application for
Libervant; risk of our ability to demonstrate to the FDA “clinical
superiority” within the meaning of the FDA regulations of Libervant
relative to FDA-approved diazepam rectal gel and nasal spray
products including by establishing a major contribution to patient
care within the meaning of FDA regulations relative to the approved
products as well as risks related to other potential pathways or
positions which are or may in the future be advanced to the FDA to
overcome the seven year orphan drug exclusivity granted by the FDA
for the approved nasal spray product of a competitor in the U.S.
and there can be no assurance that we will be successful; risk that
a competitor obtains FDA orphan drug exclusivity for a product with
the same active moiety as any of our other drug products for which
we are seeking FDA approval and that such earlier approved
competitor orphan drug blocks such other product candidates in the
U.S. for seven years for the same indication; risk in obtaining
market access for other reasons; 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
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 liquidity and cash
requirements and other cash needs, at the times and in the amounts
needed; risks related to the outsourcing of certain marketing and
other operational and staff functions to third parties; risk of the
rate and degree of market acceptance of our product and product
candidates; the success of any competing products, including
generics; risk of the size and growth of our product markets; risks
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
actions 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
and associated costs, including patent infringement, investigative
and antitrust litigation matters; changes in government 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
uncertainties affecting the Company described in the “Risk Factors”
section and in other sections included in our Annual Report on Form
10-K, in our Quarterly Reports on Form 10-Q, and in our 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 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®, Sympazan® and the Aquestive logo are
registered trademarks of Aquestive Therapeutics, Inc. All other
registered trademarks referenced herein are the property of their
respective owners.
Investor InquiriesICR WestwickeStephanie
CarringtonStephanie.carrington@westwicke.com646-277-1282
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