Acasti Completes Patient Enrollment for Phase 3 GTX-104 Safety Trial

Acasti Pharma Inc., a biopharmaceutical company specializing in late-stage drug development, announced the completion of patient enrollment for its Phase 3 STRIVE-ON safety trial. This study evaluates GTX-104, a novel injectable formulation of nimodipine, aimed at addressing aneurysmal subarachnoid hemorrhage (aSAH). The STRIVE-ON trial is a prospective, open-label, randomized trial comparing GTX-104 with oral nimodipine in 100 patients across about 25 U.S. hospitals.

Acasti's CEO, Prashant Kohli, expressed his enthusiasm about this achievement, highlighting that it was reached ahead of the anticipated schedule. Kohli emphasized that GTX-104, if approved, could significantly improve the standard of care for aSAH patients by addressing the challenges associated with oral nimodipine. The company expects to release data from the trial in early 2025 and plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2025.

The primary focus of the STRIVE-ON trial is to assess safety, measured by comparing adverse events, such as hypotension, between the groups.

Aneurysmal subarachnoid hemorrhage (aSAH) involves bleeding over the brain's surface, typically caused by an aneurysm rupture. It is a severe condition with high mortality and morbidity rates. In the U.S., around 50,000 people are affected annually, with global estimates reaching 60,000 in the European Union and 150,000 in China.

GTX-104 is an innovative injectable nimodipine formulation designed for intravenous infusion in aSAH patients, utilizing unique nanoparticle technology to facilitate aqueous formulation. This IV delivery method could potentially eliminate the need for nasogastric tube administration in unconscious or dysphagic patients, reduce food effects and drug interactions, and minimize dosing errors. Over 150 healthy volunteers have already received GTX-104, showing good tolerance and significantly lower pharmacokinetic variability compared to oral nimodipine. The U.S. market for GTX-104 is estimated at approximately $300 million.

Acasti's portfolio also includes GTX-102 and GTX-101. GTX-102 is a concentrated oral-mucosal spray of betamethasone aimed at improving neurological symptoms of Ataxia-Telangiectasia (A-T), a condition with no current FDA-approved therapies. GTX-101 is a topical bupivacaine spray intended to relieve pain in patients with postherpetic neuralgia (PHN). Both GTX-102 and GTX-101 have been deprioritized to focus on GTX-104, with possibilities for licensing or selling these candidates.

Acasti is dedicated to developing treatments for rare and orphan diseases, leveraging novel drug delivery technologies to enhance drug performance by achieving quicker onset, better efficacy, fewer side effects, and more convenient administration. The company's leading clinical assets, including GTX-104, have received Orphan Drug Designation from the FDA, securing seven years of marketing exclusivity post-launch and additional intellectual property protections via over 40 granted and pending patents.

Acasti continues to progress in its mission to bring transformative treatments to patients with significant unmet medical needs, particularly in the realm of rare and life-threatening conditions like aSAH.