Blarcamesine

Blarcamesine, an oral drug, significantly slowed Alzheimer's disease progression by 36.3% over 48 weeks, with stronger effects in patients with the common SIGMAR1 wild type gene. It activates SIGMAR1, enhancing autophagy to restore cellular homeostasis, offering a novel treatment with a good safety profile.

Clinical trials for Rett syndrome need better outcome measures to objectively confirm symptom improvement. Existing measures lack specificity for motor and autonomic functions. Acadia Pharmaceuticals' Daybue became the first FDA-approved treatment, while Anavex Life Sciences' ANAVEX2-73 failed in Phase III. Experts advocate for a holistic approach including behavioural and biomarker assessments. Video motor assessments and wearable biosensors are suggested for more objective evaluations.

Eisai and Biogen's Leqembi and Eli Lilly's Kisunla are first anti-amyloid antibodies to slow Alzheimer's cognitive decline. 32 therapeutics in Phase III trials target neuroprotection, neurotransmitters, neurogenesis, inflammation, and proteinopathies. Leqembi and Kisunla have limited efficacy and notable side effects, prompting diverse opinions on clinical benefit. Next-gen therapeutics aim for easier administration and multiple pathways to treat Alzheimer's.

Blarcamesine, a novel treatment for early-stage Alzheimer's disease, showed safety and significant slowing of clinical decline in a phase 2b/3 study. It demonstrated meaningful effects on biomarkers and reduced brain atrophy, with plans for a European regulatory submission in Q4 2024. Adverse events were mild and manageable.

Blarcamesine, an oral treatment for early Alzheimer's, significantly slowed clinical decline by 38.5% and 34.6% at 50 mg and 30 mg doses, respectively, over 48 weeks. It showed benefits on amyloid-beta and brain volume, with a good safety profile and no neuroimaging adverse events. EMA submission expected Q4 2024.

Anavex Life Sciences Corp. announced that blarcamesine (ANAVEX®2-73) significantly slowed cognitive decline in early Alzheimer's disease, with reductions in amyloid beta levels and brain atrophy. The Phase 2b/3 trial involved 508 participants across 52 centers, showing promising results with a good safety profile.