Anavex Life Sciences Corp (AVXL)

[ElsaSara]

https://www.fool.com/investing/2021/06/26/3-top-biotech-stocks-to-buy-right-now/

Anavex has a potential blockbuster in blarcamesine

Anavex's stock is up more than 376% this year, and it continued to climb on positive reports of the company's phase 2 trial of ANAVEX2-73 (blarcamesine) in adult female patients.

The small-molecule therapy targets Rett syndrome, a central nervous system disease caused by the mutation of the MECP2 gene. The trial found that ANAVEX 2-73 activated the sigma-1 receptor, helping to restore neural cell homeostasis (a stable, relatively constant internal environment) and promoting neuroplasticity, the ability of nerve networks to adapt through growth and reorganization. Rett syndrome affects the development of the brain, and according to the National Organization for Rare Disorders, Rett syndrome occurs almost exclusively in girls, affecting one in 10,000 by age 12. ANAVEX 2-73 has the potential to be the first therapy approved for Rett syndrome by the FDA. The drug has already been given Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug designation from the FDA for the treatment of Rett syndrome, so it could be considered for accelerated approval, the company said, meaning that it could come to the market later this year.

It is the company's leading drug, and it's also in late-stage studies as a therapy for Alzheimer's disease and Parkinson's disease dementia.

Anavex lost $8.2 million in the second quarter, but the company has $75.9 million in cash, enough, it said, to fund operations for three years.