Anavex Life Sciences Corp. has presented promising results from its Phase 2b/3 study of blarcamesine (ANAVEX2-73), which significantly slowed clinical decline in early Alzheimer's disease during the 2024 Alzheimer’s Association International Conference.

The study reported a 38.5% reduction in clinical progression for the 50 mg group and a 34.6% reduction for the 30 mg group at 48 weeks, compared to placebo on the primary cognitive endpoint, ADAS-Cog13.

Additionally, the key secondary endpoint, CDR-SB, was significant for both dosages at Week 48, aligning with new FDA guidance that suggests it as an alternative primary endpoint for early Alzheimer's treatments.

While the functional co-primary endpoint, ADCS-ADL, showed positive trends, it did not reach statistical significance, likely due to its design being less sensitive for early Alzheimer's patients.

Concerns about safety arose as 30.7% of patients experienced treatment-related adverse effects that led to discontinuation of the drug.

Anavex plans to submit a full regulatory application for blarcamesine in Europe by the end of 2024, although the EMA's consideration will depend on further studies and evidence of efficacy relative to existing treatments.

The FDA's recent guidance allows for a single cognitive measure to serve as the primary endpoint for early Alzheimer's trials, which may facilitate the approval process for blarcamesine.

Blarcamesine targets the SIGMAR1 protein, which is crucial for cellular function and communication, potentially offering a unique mechanism of action compared to existing therapies.

In comparison to Eli Lilly's comparable drug, Kisunla, which showed a 35% reduction in clinical decline, blarcamesine demonstrated a 38.5% reduction, indicating superior efficacy.

Following the announcement of these results, Anavex's stock (AVXL) rose by 11.40% to $7.51 in premarket trading, reflecting positive investor sentiment.

Despite the positive study results, analysts remain divided on Anavex's market potential, with short interest in the stock exceeding 20% as of mid-July 2023.