Acasti Pharma Inc. (NASDAQ:ACST) is a specialty pharmaceutical company focused on developing and commercializing innovative drug delivery technologies for the treatment of rare and orphan diseases. The company's pipeline consists of three unique clinical-stage and multiple pre-clinical stage assets, supported by an intellectual property portfolio of more than 40 granted and pending patents in various jurisdictions worldwide.
Business Overview
Acasti Pharma is dedicated to applying new proprietary formulations to existing pharmaceutical compounds to achieve enhanced efficacy, faster onset of action, reduced side effects, more convenient drug delivery, and increased patient compliance - all of which could result in improved patient outcomes. The active pharmaceutical ingredients used in the drug candidates under development by Acasti may be already approved in a target indication or could be repurposed for use in new indications. This provides the opportunity to utilize the Section 505(b)(2) regulatory pathway, which may result in a potentially shorter path to regulatory approval.
The company's lead drug candidate, GTX-104, is a clinical-stage, novel, injectable formulation of nimodipine being developed for intravenous infusion in aneurysmal subarachnoid hemorrhage (aSAH) patients. GTX-104 aims to address significant unmet medical needs by providing a more convenient mode of administration, better control of blood pressure, and improved management of hypotension compared to the currently available oral nimodipine formulations.
Acasti's other pipeline drug candidates include GTX-102, an oral-mucosal betamethasone spray for the treatment of Ataxia Telangiectasia (A-T), and GTX-101, a topical bioadhesive film-forming bupivacaine spray for Postherpetic Neuralgia (PHN). Both GTX-102 and GTX-101 have received Orphan Drug Designation from the FDA, which provides potential benefits such as market exclusivity and tax credits.
Financials
For the fiscal year ended March 31, 2023, Acasti reported a net loss of $12,853,000, with no revenue generated. The company's annual operating cash flow was -$12,333,000, and its annual free cash flow was -$12,355,000.
In the latest quarter ended December 31, 2023, Acasti reported a net loss of $2,391,000. The company's quarterly operating cash flow was -$3,415,000, and its quarterly free cash flow was -$3,437,000.
Liquidity
As of December 31, 2023, Acasti had cash, cash equivalents, and short-term investments totaling $25,114,000, which the company believes will be sufficient to fund its operations into the second calendar quarter of 2026. The company will require additional capital to fund its daily operating needs beyond that time.
In September 2023, Acasti raised approximately $7,338,000 in net proceeds from a private placement offering with institutional and accredited investors. The company plans to use the net proceeds to fund the clinical trial expenses for the Phase 3 trial of GTX-104, pre-commercial planning, and other general corporate purposes.
Recent Developments
On October 23, 2023, Acasti enrolled the first patient in its pivotal Phase 3 STRIVE-ON safety trial evaluating GTX-104 in patients hospitalized for aSAH. The trial is expected to take approximately 18 months to complete and is the final clinical step required to seek FDA approval for GTX-104 under the 505(b)(2) regulatory pathway.
In May 2023, the company announced a strategic realignment plan to prioritize the development of GTX-104 and conserve resources. As part of this plan, Acasti has deprioritized the further development of GTX-102 and GTX-101, pending additional funding or the signing of strategic partnerships.
Outlook
Acasti has not provided any formal financial guidance for the upcoming fiscal year. However, the company's focus on advancing GTX-104 through the pivotal Phase 3 trial, while preserving cash resources, suggests that the near-term priority is to achieve regulatory approval and prepare for the potential commercialization of its lead drug candidate.
Risks and Challenges
Acasti faces several risks and challenges common to clinical-stage biopharmaceutical companies, including the successful completion of its ongoing and future clinical trials, obtaining regulatory approvals, and securing additional funding to support its operations and pipeline development. The company's heavy reliance on the success of its lead drug candidate, GTX-104, also presents a significant risk if the product fails to demonstrate the expected safety and efficacy profile.
Conclusion
Acasti Pharma is a promising specialty pharmaceutical company focused on developing innovative drug delivery technologies for the treatment of rare and orphan diseases. With its lead drug candidate, GTX-104, currently in a pivotal Phase 3 trial for aSAH, and a pipeline of other promising assets, Acasti is well-positioned to address significant unmet medical needs and create value for its shareholders. However, the company's success will depend on its ability to navigate the clinical development and regulatory approval process, as well as secure the necessary funding to support its operations and pipeline development.