Anavex Life Sciences (AVXL) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

2024 highlights and regulatory progress

• Submitted Marketing Authorization Application (MAA) to EMA for Alzheimer's disease, based on phase II-B/III and long-term open-label studies.

• Peer-reviewed publication of pivotal Alzheimer's study forthcoming; open-label ATTENTION-AD study ongoing with nearly three years of data.

• Over 70 patients have received blarcamesine for up to nine years, including compassionate use extensions.

• EMA submission supported by randomized, placebo-controlled and open-label studies, plus real-world evidence.

• Four years of cash runway and no debt; multiple regulatory and clinical catalysts expected in 2025.

Clinical development and mechanistic insights

• Blarcamesine acts primarily as a sigma-1 receptor agonist, with muscarinic activity less central to its effect.

• Demonstrated restoration of impaired autophagy, potentially intervening earlier in Alzheimer's pathology.

• Efficacy in phase II-B/III trial improved with longer titration; adverse events mostly mild dizziness.

• No significant confounding from background Alzheimer's therapies; subgroup analysis showed wild-type sigma-1 gene carriers responded best.

• Both wild-type and variant sigma-1 carriers benefited, with overall significant results in the intent-to-treat population.

Pipeline updates and future plans

• Parkinson's pivotal program design pending regulatory feedback; phase II dementia study showed cognitive and motor improvements.

• New biomarkers, such as alpha-synuclein, will inform Parkinson's trial design; regulatory buy-in prioritized before launch.

• Anavex 371, a pure sigma-1/M1 agonist, completed phase I and began phase II in schizophrenia; early data show target engagement and good safety.

• Schizophrenia phase II trial is U.S.-based, with part two recently started and readout expected in first half of next year.

• Additional pipeline indications may advance after Alzheimer's regulatory focus; updates expected on EMA progress, Parkinson's, and open-label Alzheimer's data.