Actinogen Medical (ACW) Status Update summary

Clinical and regulatory progress

• Pivotal phase II-B/III Alzheimer's study is on track, with screening closing and full enrollment expected by year-end, targeting around 240 patients.

• Recent FDA meeting resulted in agreement for a streamlined path to approval, requiring only one additional pivotal trial and open-label safety studies.

• Interim futility analysis is scheduled for late January, with top-line results anticipated mid-Q4 next year.

• Open-label extension phase will begin in Q1 next year, allowing long-term safety and efficacy data collection.

• Innovation Passport awarded in the UK, and EMA meeting planned for 2026 to align regulatory strategy.

Mechanism of action and clinical evidence

• Xanamem® targets brain cortisol by inhibiting 11β-HSD1, addressing a novel pathway in Alzheimer's and depression.

• Phase II trials demonstrated safety, tolerability, and promising disease-modifying activity in Alzheimer's and improvement in depressive symptoms.

• Target engagement confirmed via imaging, showing effective brain penetration and enzyme inhibition at clinical doses.

• Pilot study in biomarker-positive Alzheimer's patients showed clinically significant benefit over three months.

• Additional evidence of efficacy in treatment-resistant depression, especially in patients on SSRIs.

Manufacturing and intellectual property

• Large batch of drug substance manufactured at IC Chem in China, with scale-up for tablet production in the US.

• No tariff issues on import; process and formulation patent applications are in progress.

• FDA agreement on regulatory starting materials and confirmation that 10 mg tablets are suitable for once-daily dosing.

• Patent portfolio provides protection to 2036-2040s, with new patents potentially extending exclusivity.