Anavex Life Sciences
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Anavex Life Sciences (AVXL) investor relations material

Anavex Life Sciences 44th Annual J.P. Morgan Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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44th Annual J.P. Morgan Healthcare Conference summary15 Jan, 2026

Strategic vision and therapeutic approach

  • Focus on upstream intervention in CNS diseases by restoring autophagy, aiming for patient-oriented, personalized medicine in neurodegeneration.

  • Lead asset blarcamesine is a once-daily oral small molecule targeting sigma-1 activation to restore cellular homeostasis and autophagy.

  • Emphasis on convenience and patient preference for oral therapies, reducing the need for hospital visits and complex procedures.

  • Financially stable with over three years of cash and no debt, supported by non-dilutive funding and strong IP protection through 2040.

  • Expanding pipeline includes additional CNS indications such as Parkinson’s disease, Rett syndrome, Fragile X syndrome, schizophrenia, and others.

Clinical data and efficacy highlights

  • Phase IIb/III trial of blarcamesine showed significant slowing of cognitive decline, with up to 49.8% benefit in pre-specified populations at 48 weeks.

  • Demonstrated strong safety profile with no neuroimaging adverse events or deaths, and manageable side effects.

  • Biomarker data showed significant slowing of brain atrophy and improvement in Aβ42 plasma ratio.

  • Long-term extension study indicated up to 18 months of sustained patient benefit in maintaining functionality and independence.

  • Quality of life measures improved above baseline for key genetic subgroups, with net improvement after 48 weeks.

Mechanism of action and precision medicine

  • Blarcamesine activates sigma-1 receptors, restoring autophagy and reducing cellular stress, confirmed in vivo and in vitro.

  • Stronger clinical response observed in patients with wild-type sigma-1 gene, representing about 70% of the population.

  • Combination of wild-type sigma-1 and COL24A1 genes led to up to 84.7% clinical benefit compared to placebo.

  • Precision medicine approach aims to match treatment response to normal cognitive decline in healthy aging.

  • Mechanistic focus on upstream intervention differentiates from failed downstream approaches in Alzheimer’s trials.

Detail blarcamesine's EU regulatory path
How will precision medicine impact market access?
Prioritize non-Alzheimer's pipeline milestones
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Q1 202611 Feb, 2026
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Frequently asked questions

Anavex Life Sciences Corp., a clinical stage biopharmaceutical company, engages in the development of drug candidates for the treatment of central nervous system (CNS) diseases. The company's lead drug candidate is 2-73 that has completed phase II clinical trial for the treatment of Alzheimer's disease. Its programs also includes ANAVEX 3-71, which is in pre-clinical testing for the treatment of amyotrophic lateral sclerosis disease; and ANAVEX 1-41, a pre-clinical stage small molecule compound for the treatment of epilepsy.

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