Company expects the first patient to be
enrolled during the second half of 2023
LAVAL,
QC, April 4, 2023 /PRNewswire/ -- Acasti
Pharma Inc. ("Acasti" or the "Company") (Nasdaq: ACST), a
late-stage, specialty pharma company advancing drug candidates for
rare and orphan diseases, today announced that it received a Type C
written meeting response and clarifying feedback from the United
States Food and Drug Administration (FDA) on Acasti's proposed
Phase 3 Safety Study for GTX-104. The FDA provided additional
comments on the Company's development plan that, pending submission
of the final clinical protocol and FDA approval of same, will allow
Acasti to proceed with the initiation of a Phase 3 safety clinical
trial in aneurysmal Subarachnoid Hemorrhage (aSAH) patients.
The FDA concurred with the suitability of the 505(b)(2)
regulatory pathway with the selected Reference Listed Drug (RLD)
Nimotop oral capsules (NDA 018869), and that Acasti's GTX-104-002
PK study may have met the criteria for a scientific bridge.
Based on FDA's proposed Phase 3 Study Design, the company will
target enrollment of aSAH patients (across all grades of severity)
in a 1:1 randomized trial with oral nimodipine, to be conducted in
an estimated 25-30 sites in the U.S.A. The FDA confirmed the use of the
Hunt and Hess scale to stratify patients based on
severity. The primary endpoint is safety, and it will be
measured as the percentage of significant adverse events of
hypotension related to study drugs in both arms.
Prashant Kohli, CEO of Acasti
Pharma, commented, "We expect to move quickly to submit the final
clinical protocol and all required study documentation to the FDA.
Once these documents are submitted and following any final feedback
and approval from the FDA, the Phase 3 safety study can be
initiated. If the Phase 3 study meets the primary endpoint, an NDA
filing for GTX-104 under Section 505(b)(2) is expected to
follow."
An application filed under Section 505(b)(2) is one that
contains full reports of investigations of safety and effectiveness
but where at least some of the information required for approval
comes from studies not conducted by or for the applicant and for
which the applicant has not obtained a right of reference.
About aneurysmal Subarachnoid Hemorrhage (aSAH)
aSAH is bleeding over the surface of the brain in the
subarachnoid space between the brain and the skull, which contains
blood vessels that supply the brain. A primary cause of such
bleeding is rupture of an aneurysm. The result is a relatively
uncommon type of stroke that accounts for about one-in-twenty (5%)
of all strokes and has an incidence of six per 100,000 person years
(Becske, 2018). In contrast to more common types of strokes in
elderly individuals, aSAH often occurs at a relatively young age,
with half the affected patients being younger than 60 years
(Becske, 2018). Particularly devastating for patients younger than
45, approximately 10% to 15% of aSAH patients die before reaching
the hospital (Rinkel, 2016), and those who survive the initial
hours post hemorrhage are admitted or transferred to
neurointensive care centers to manage the high risk of
complications, including rebleeding, vasospasm and delayed cerebral
ischemia (DCI). Systemic manifestations affecting cardiovascular,
pulmonary, and renal function are common, and often complicate the
management of DCI.
Approximately 70% of aSAH patients experience death or
dependence, and half die within one month after the hemorrhage. Of
those who survive the initial month, half remain permanently
dependent on someone else to maintain daily living (Becske,
2018).
About GTX-104
GTX-104 is a clinical stage, novel formulation of nimodipine
being developed for IV infusion in aSAH patients. It incorporates
surfactant micelles as the drug carrier to solubilize nimodipine.
This nimodipine injectable formulation is comprised of a nimodipine
base, an effective amount of a hydrophilic surfactant, and a
pharmaceutically acceptable carrier for injection. GTX-104 is an
aqueous solution substantially free of organic solvents, such that
the nimodipine is contained in a concentrated injection solution,
suspension, emulsion or complex as a micelle, a colloidal particle
or an inclusion complex, and the formulation is stable and clear.
The addressable market in the United
States for GTX-104 is estimated to be about $300 million based on market research conducted
by Fletcher Spaght.
About Acasti
Acasti is a late-stage specialty pharma company with drug
delivery technologies and drug candidates addressing rare and
orphan diseases. Acasti's novel drug delivery technologies have the
potential to improve the performance of currently marketed drugs by
achieving faster onset of action, enhanced efficacy, reduced side
effects, and more convenient drug delivery—all which could help to
increase treatment compliance and improve patient outcomes.
Acasti's three lead clinical assets have each been granted Orphan
Drug Designation by the FDA, which provide the assets with seven
years of marketing exclusivity post-launch in the United States, and additional intellectual
property protection with over 40 granted and pending patents.
Acasti's lead clinical assets target underserved orphan diseases:
(i) GTX-104, an intravenous infusion targeting aneurysmal
Subarachnoid Hemorrhage (aSAH), a rare and life threatening medical
emergency in which bleeding occurs over the surface of the brain in
the subarachnoid space between the brain and skull; (ii) GTX-102,
an oral mucosal spray targeting Ataxia-telangiectasia (A-T), a
progressive, neurodegenerative genetic disease that primarily
affects children, causing severe disability, and for which no
treatment currently exists; and (iii) GTX-101, a topical spray
targeting Postherpetic Neuralgia (PHN), a persistent and often
debilitating neuropathic pain caused by nerve damage from the
varicella zoster virus (shingles), which may persist for months and
even years.
For more information, please visit: https://www.acasti.com.
Forward-Looking Statements
Statements in this press release that are not statements of
historical or current fact constitute "forward-looking statements"
within the meaning of the U.S. Private Securities Litigation Reform
Act of 1995, as amended, Section 27A of the Securities Act of 1933,
as amended, and Section 21E of the Securities Exchange Act of 1934,
as amended, and "forward-looking information" within the meaning of
Canadian securities laws (collectively, "forward-looking
statements"). Such forward looking statements involve known and
unknown risks, uncertainties, and other factors that could cause
the actual results of Acasti to be materially different from
historical results or from any future results expressed or implied
by such forward-looking statements. In addition to statements which
explicitly describe such risks and uncertainties, readers are urged
to consider statements containing the terms "believes," "belief,"
"expects," "intends," "anticipates," "estimates", "potential,"
"should," "may," "will," "plans," "continue", "targeted" or other
similar expressions to be uncertain and forward-looking. Readers
are cautioned not to place undue reliance on these forward-looking
statements, which speak only as of the date of this press release.
The forward-looking statements in this press release are based upon
Acasti's current expectations and involve assumptions that may
never materialize or may prove to be incorrect. Actual results and
the timing of events could differ materially from those anticipated
in such forward-looking statements as a result of various risks and
uncertainties, including, without limitation: (i) the success and
timing of regulatory submissions of the planned Phase 3 safety
study for GTX-104; (ii) regulatory requirements or developments and
the outcome and timing of the proposed IND application for GTX-104;
(iii) changes to clinical trial designs and regulatory pathways;
(iv) legislative, regulatory, political and economic developments;
and (v) actual costs associated with Acasti's clinical trials as
compared to management's current expectations. The foregoing list
of important factors that could cause actual events to differ from
expectations should not be construed as exhaustive and should be
read in conjunction with statements that are included herein and
elsewhere, including the risk factors detailed in documents that
have been and are filed by Acasti from time to time with the
Securities and Exchange Commission and Canadian securities
regulators. All forward-looking statements contained in this press
release speak only as of the date on which they were made. Acasti
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made, except as required by applicable securities
laws. The NASDAQ does not accept responsibility for the adequacy or
accuracy of this release.
Acasti Contact:
Prashant Kohli
Chief Executive Officer
Tel: 450-686-4555
Email: info@acasti.com
www.acasti.com
Investor Relations:
Robert
Blum
Lytham Partners, LLC
602-889-9700
ACST@lythampartners.com
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SOURCE Acasti Pharma Inc.