$AVXL - Alzheimer’s Drug that Actually Works - Pending EMA Approval

1. Introduction to Blarcamesine

Blarcamesine, also known as ANAVEX®2-73, is an investigational oral therapy developed by Anavex Life Sciences. It functions as an agonist of the sigma-1 (SIGMAR1) receptor and muscarinic receptors, aiming to restore cellular homeostasis disrupted in neurodegenerative diseases like Alzheimer’s.

**Additional reading:** https://www.sotcanalytics.com/

2. Clinical Trial Insights

A pivotal Phase 2b/3 randomized clinical trial evaluated blarcamesine’s efficacy in early Alzheimer’s disease (AD) patients over 48 weeks. The results were promising:
• Cognitive Benefits: Blarcamesine significantly slowed clinical progression by 36.3% at 48 weeks, indicating a notable preservation of cognitive function. 
• Safety Profile: The therapy demonstrated a favorable safety profile, with no significant neuroimaging adverse events reported. 
• SIGMAR1 Wild-Type Subgroup Analysis: In a prespecified analysis focusing on patients with the common SIGMAR1 wild-type gene (excluding carriers of the mutated SIGMAR1 rs1800866 variant), blarcamesine exhibited an even greater clinical benefit. These patients experienced a 49.8% reduction in clinical progression at 48 weeks on the primary cognitive endpoint ADAS-Cog13. **This represents over 70% of the patient population.**

These results were published in a peer reviewed journal article over 50 doctors/researchers representing 42 external organizations: https://www.sciencedirect.com/science/article/pii/S2274580724006083

3. Market Potential

The Alzheimer’s therapeutics market is poised for substantial growth:
• Current and Projected Market Size: Valued at approximately $4.05 billion in 2022, the market is expected to expand at a compound annual growth rate (CAGR) of 19.99%, reaching around $15.19 billion by 2030.
• Valuation of Alzheimer’s Drugs: An FDA-approved Alzheimer’s drug can carry a market value of approximately $43.4 billion. For instance, Biogen’s Aducanumab was valued at this figure upon approval. Similarly, drugs in Phase 3 trials, like Eli Lilly’s Donanemab, have been valued around $20.2 billion, with potential increases upon receiving Breakthrough Therapy Designation.

**AVXL inexplicably has a market cap under $1B with over 25% of the float short**. If they even sniff the valuations argued here, we’re looking at a triple digit share price.

https://ipwatchdog.com/2021/10/26/assessing-much-alzheimers-drug-worth/id=139154/

4. Investment Considerations

Investors should monitor:
• Regulatory Milestones: Blarcamesine has received EMA filing acceptance for the treatment of Alzheimer’s disease, marking a significant step toward potential market approval. **A decision should be made by the end of 2025**

• Upcoming conference on April 5th which Anavex is expected to reveal additional subgroup analysis of their trial.

5. Conclusion

Blarcamesine represents a compelling candidate in the Alzheimer’s therapeutics sector. Its innovative mechanism of action, coupled with favorable clinical trial results, positions it as a potential frontrunner in addressing a significant unmet medical need. Given the substantial market valuations observed with similar drugs, blarcamesine’s successful development and approval could yield considerable financial returns.

Corporate presentation: https://www.anavex.com/_files/ugd/79bcf7_da38b4e0ad4b4ff888363403e3962ef2.pdf

P.S. - yes I know Martin Shkreli is short. Yes, I watched his “analysis”. Yes I know people will say it’s SAVA 2.0. **Clearly I disagree based on my long position.**

Disclaimer: This information is for educational purposes and should not be construed as financial advice. Always conduct your own research before making investment decisions.