Acasti Pharma Announces Initiation of Pharmacokinetic Study of GTX-102, Ataxia Telangiectasia Treatment Candidate, Canadian Business Journal

Laval, Quebec, September 13, 2022 (GLOBE NEWSWIRE) — Acasti Pharma Inc. (“Acasti” or the “Company”) (Nasdaq: ACST and TSX-V: ACST) today announced that it has ) Bridging Study Evaluating the Bioavailability, Pharmacokinetics, and Safety of Oral Betamethasone Spray, GTX-102 Compared to Intramuscular Betamethasone and Oral Betamethasone Solution in 48 Healthy Subjects .First subject, first dose he was dosed on September 13th^thThis PK study is the next step in the proposed 505(b)(2) regulatory pathway for GTX-102, with topline results expected by the end of the year.

GTX-102 is a novel proprietary oral mucosal concentrate of betamethasone intended to ameliorate the neurological symptoms of ataxia-telangiectasia (AT) in a pediatric population with no current FDA-approved therapy. It’s a metered spray. GTX-102 can be conveniently sprayed onto the tongue of AT patients who have frequent swallowing difficulties.

Jan D’Alvise, CEO of Acasti, said: with this rare genetic disease. GTX-102 is his third program to enter the clinic this year. We continue to exploit new drug delivery technologies that have the potential to improve the performance of currently marketed medicines by achieving faster onset of action, increased efficacy and reduced side effects. , more convenient drug delivery. We look forward to completing this PK trial later this year and, if positive, we plan to move rapidly to Phase 3 in the second half of 2023. There are currently no drugs approved for AT. Bring this exciting new treatment to children suffering from AT. “

This PK study was conducted in healthy male and female subjects to comparatively evaluate the bioavailability, pharmacokinetics and safety of GTX-102 administered as an oral spray compared to intramuscular injection of betamethasone. is a Phase 1, randomized, open-label, crossover study in is a reference product for submission purposes in the United States, a marketed oral solution of betamethasone in Europe, and a comparator product for bridging purposes under 505(b)(2) guidelines. The main purpose of this study is to evaluate and characterize the PK profile of his GTX-102 as an oral spray.

A total of 48 healthy adult male and female subjects will be enrolled in this single-center, 5-treatment, 2-period crossover study. Her four groups of subjects each receive her two treatments and are randomized to receive his three different doses of GTX-102 (low, medium, and high doses). Betamethasone IM injection.

Assuming the PK bridging study meets the primary endpoint, the final development step is to conduct a Phase 3 safety and efficacy study in AT patients. The company plans to request a Type B meeting with the FDA to confirm the design of the Phase 3 trial after the PK trial is completed, which he expects to begin in late 2023. Endpoint, followed by his NDA application for GTX-102 under Section 505(b)(2).

Changes in senior management

The Company also announces the appointment of Prashant Kohli as Chief Commercial Officer. Kohli previously served as Vice President of Commercial His Operations for Acasti, and held the same title at Grace Therapeutics before he was acquired by Acasti in August 2021.

About Ataxia Telangiectasia (AT)

AT is a progressive, inherited neurodegenerative disease that primarily affects young children and causes severe disability, immune system dysfunction, and increased susceptibility to infections and cancer. The hallmark symptoms of AT are cerebellar ataxia and other motor dysfunction, and blood vessel dilatation (telangiectasia) that occurs in the sclera of the eye. When children begin to walk, they begin to experience balance and coordination problems (in early childhood) and eventually become wheelchair-bound in their first 20 years of life. Before puberty (5-8 years), patients experience oculomotor apraxia, dysarthria, and dysphagia. They often have compromised immune systems and are at increased risk of developing respiratory tract infections and cancers (usually lymphoma and leukemia). The patient usually dies by the age of 25 from complications of lung disease or cancer.

AT is diagnosed by a combination of clinical evaluation (especially neurological and oculomotor disorders), laboratory analysis, and genetic testing. There are no known treatments to slow disease progression, and the treatments used are either strictly symptomatic (e.g., physical, occupational, and speech therapy for neurological problems) or conditions secondary to the disease (e.g., for lung infections). antibiotics, chemotherapy, etc.). cancer, etc.).

A market research study commissioned by Acasti found that AT affects approximately 4,300 patients annually in the United States, with a potential total market size of $150 million, based on the number of treatable patients I understand.

About Akasti

Acasti is a specialty pharmaceutical company with drug delivery technology and drug candidates that address rare diseases and rare diseases. Acasti’s novel drug delivery technology has the potential to improve the performance of currently marketed drugs by providing faster onset of action, increased efficacy, reduced side effects and more convenient drug delivery. It’s hidden. All of these can help increase treatment compliance and improve patient outcomes.

Each of Acasti’s three major clinical assets has received orphan drug designation from the FDA, which gives the assets marketing exclusivity for seven years after launch in the United States, and more than 40 granted and pending Additional intellectual property protection is provided by patents. (i) GTX-104, intravenous infusion for subarachnoid hemorrhage (SAH), a rare and life-threatening medical emergency in which bleeding occurs on the surface of the brain in the subarachnoid space between the brains and the skull; ii) GTX-102 is an oral mucosal spray targeting ataxia telangiectasia (AT). AT is a progressive, neurodegenerative genetic disease that primarily affects children, causes severe disability, and currently has no cure. (iii) GTX-101 is a topical spray for postherpetic neuralgia (PHN), a persistent and often debilitating neuropathic pain caused by nerve damage caused by the varicella zoster virus (shingles); , which can persist for months or even years.

For more information, please visit https://www.acastipharma.com/en.

Forward-Looking Statements

Statements in this press release, which are not statements of historical or current fact, constitute “forward-looking information” within the meaning of the Canadian securities laws and are not “forward-looking information” within the meaning of the Private Securities Litigation Reform Act of 1995. constitute a description of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (collectively, “forward-looking statements”). Such forward-looking statements involve known and unknown risks, uncertainties and other unknown factors and Acasti’s actual results may not be implied by past results or forward-looking statements. or may differ materially from implied future results. In addition to statements that explicitly describe such risks and uncertainties, the reader “assumes,” “believes,” “believes,” “expects,” “intends,” “expects,” We encourage you to consider statements that include the terms “may” and “should”. , “may,” “will,” “plan,” “continue,” “targeted,” or other similar expressions indicate uncertain and forward-looking statements. Readers are cautioned not to place undue reliance on these forward-looking statements. These statements are made only as of the date of this press release. The forward-looking statements in this press release are based on Acasti’s current expectations and include assumptions that may never materialize or prove to be incorrect. Actual results and timing of events may differ materially from those anticipated in these forward-looking statements as a result of various risks and uncertainties. -102 and other preclinical and clinical trials of Acasti. (ii) regulatory requirements or developments; (iii) changes in clinical trial design and regulatory pathways; (iv) legislative, regulatory, political and economic developments; and (v) impact of COVID-19 on clinical programs and business operations. The foregoing list of significant factors that could cause actual events to differ from expectations should not be construed as exhaustive and is detailed in documents submitted or that may be submitted. should be read in conjunction with statements contained herein and elsewhere, including risk factors that From time to time by the Securities and Exchange Commission and Acasti. All forward-looking statements contained in this press release speak only as of the date they are made. Acasti undertakes no obligation to update any statement to reflect events occurring or circumstances existing after the date the statement was made, except as required by applicable securities laws. Neither NASDAQ, TSXV nor their regulated service providers (as those terms are defined in TSXV’s policy) are responsible for the adequacy or accuracy of this release.

Contact Acasti:

Jan Dalvise
Chief executive officer
Phone: 450-686-4555
Email:[email protected]
www.acastipharma.com

Investor Relations:
Robert Blum
Lytham Partners LLC
602-889-9700
[email protected]

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