Actinogen Accelerates Alzheimer's Trial with $22.9M Cash Boost

Progress in Alzheimer's Clinical Trial

Actinogen Medical (ASX: ACW) has reported solid progress in its late-stage clinical development program for Xanamem (emestedastat), its novel brain cortisol synthesis inhibitor targeting Alzheimer's disease (AD) and major depressive disorder (MDD). The company’s flagship XanaMIA phase 2b/3 trial is actively enrolling 220 participants with elevated blood biomarker pTau181, a marker linked to disease progression in AD. This biomarker-driven approach aims to enrich the trial population with patients most likely to demonstrate measurable treatment benefits over the 36-week treatment period.

Recruitment momentum has picked up following the holiday season lull, with the first US patient randomized in early December 2024 and the tenth US site now actively recruiting alongside 15 established Australian sites. Actinogen maintains a robust recruitment funnel with over 300 patients pre-screened, approximately 40 randomized and treated, and more than 20 in intermediate screening stages. An interim analysis is planned once around 100 patients complete 24 weeks of treatment, expected in Q4 2025, while final trial results remain on track for the second half of 2026.

Commercial Readiness and Regulatory Engagement

Beyond clinical progress, Actinogen is laying the groundwork for eventual commercialization. The company has demonstrated clinical proof of concept in both AD and MDD indications, reinforcing the validity of its cortisol hypothesis, the control of brain cortisol production as a therapeutic mechanism. Regulatory planning for marketing approvals is underway, with multiple meetings scheduled to clarify registrational requirements for both indications.

In parallel, Actinogen is actively pursuing partnering discussions with potential regional and global collaborators, leveraging recent engagements at high-profile events such as the Sachs Neuroscience Innovation Forum and the JPM Healthcare Conference. Manufacturing scale-up has commenced, producing batches of drug substance to support ongoing and future trials, while also demonstrating readiness for commercial-scale production.

Intellectual Property and Scientific Validation

Intellectual property protection remains a strategic focus. Xanamem benefits from granted patents covering its chemistry and therapeutic applications, supplemented by data exclusivity provisions that shield clinical data from generic competition for several years across key markets. The World Health Organization recently granted the International Nonproprietary Name (INN) 'emestedastat' to Xanamem, marking a significant regulatory milestone and underscoring its unique mechanism as a brain tissue cortisol synthesis inhibitor.

Scientific validation continues with peer-reviewed publications in the Journal of Alzheimer's Disease and upcoming manuscripts detailing clinical pharmacology and depression trial results. Presentations by Actinogen’s leadership at international conferences have highlighted the drug’s mechanism and clinical potential, reinforcing investor and partner confidence.

Financial Position and Outlook

Financially, Actinogen reported a strong quarter with research and development expenditure steady at $2.3 million, focused on accelerating trial recruitment. The company benefited from a $9.0 million R&D tax incentive inflow, contributing to a positive operating cash flow of $5.3 million for the quarter. Combined with proceeds from the final tranche of a recent $11.1 million capital raise, Actinogen’s cash position improved by $9.3 million to a healthy $22.9 million as of 31 December 2024.

This cash runway is sufficient to fund operations through mid to late 2026, aligning with the anticipated timeline for pivotal trial results and regulatory milestones. The company also issued 59.5 million loan shares to employees and contractors under its incentive plan, reflecting ongoing commitment to talent retention and motivation.

CEO Dr. Steven Gourlay expressed confidence in the company’s trajectory, emphasizing the stepped-up recruitment and comprehensive preparations for regulatory submissions and commercialization. The coming months will be critical as interim data readouts and regulatory dialogues shape the path forward for Xanamem.