Actinogen Medical (ACW) Status Update summary

Program milestones and trial status

• Final patient enrolled and treated in the pivotal XanaMIA Phase IIb trial, reaching 246 participants for a 36-week treatment period.

• Full enrollment achieved in the US and Australia, with an exemplary safety profile observed over 36 weeks and no serious adverse events related to the drug.

• Interim safety and futility analysis scheduled for late January, with final results expected in November next year.

• Open-label extension to begin at the end of Q1 next year, allowing all participants to receive active drug and providing long-term safety and efficacy data.

• Funding secured beyond mid-next year, with additional non-dilutive options and potential regional partnerships under consideration.

Scientific rationale and clinical evidence

• Xanamem targets brain cortisol by inhibiting 11-beta-HSD1, a unique mechanism with no direct competition in Alzheimer's treatment.

• PET data confirm brain penetration and target engagement at low doses, with published studies showing robust slowing of disease progression in p-Tau positive patients.

• Phase II trials in depression demonstrated significant improvement in treatment-resistant patients, supporting the cortisol hypothesis.

• Elevated p-Tau is used as a biomarker for trial inclusion, aligning with FDA guidance and ensuring rapid disease progression in the study population.

• The primary endpoint is the CDR Sum of Boxes, a sensitive and universally accepted measure for Alzheimer's trials.

Competitive landscape and commercial outlook

• Current anti-amyloid and anti-tau therapies offer only modest benefits and have safety concerns, while Xanamem is positioned as a safe, effective, once-daily oral therapy.

• Market research with neurologists indicates strong interest, with 80% likely to prescribe Xanamem within six months of launch.

• Xanamem can be combined with existing Alzheimer's medications and is expected to move rapidly into first-line therapy if approved.

• Multiple global partners are reviewing data, with regional deals considered only if terms are favorable.

• Final results could enable expedited regulatory approval and attract significant partnership interest.