Anavex Life Sciences Corp (AVXL)

[Doc328]

AVXL-273 is being tested only in late teens and adults with Rett in the current phase 2 studies for only 7 weeks while placebo controlled, so they are looking for symptomatic benefit not disease modifying effects. Theoretically, there could be a role for add on therapy at any point in life.

I would expect the gene therapy (which is disease modifying) to potentially only help those receiving the drug early in life, before severe permanent neurologic changes have occurred. Therefore, I would expect the initial study to be in infants and toddlers (Stage 1 ideally, maybe stage 1 and early stage 2 to allow more rapid enrollment)

We are in exciting times for genetic diseases.

From the Mayo Clinic:
Rett syndrome is commonly divided into four stages:

Stage I: early onset. Signs and symptoms are subtle and easily overlooked during the first stage, which starts between 6 and 18 months of age and can last for a few months or a year. Babies in this stage may show less eye contact and start to lose interest in toys. They may also have delays in sitting or crawling.

Stage II: rapid deterioration. Starting between 1 and 4 years of age, children lose the ability to perform skills they previously had. This loss can be rapid or more gradual, occurring over weeks or months. Symptoms of Rett syndrome occur, such as slowed head growth, abnormal hand movements, hyperventilating, screaming or crying for no apparent reason, problems with movement and coordination, and a loss of social interaction and communication.

Stage III: plateau. The third stage usually begins between the ages of 2 and 10 years and can last for many years. Although problems with movement continue, behavior may have limited improvement, with less crying and irritability, and some improvement in hand use and communication. Seizures may begin in this stage and generally don't occur before the age of 2.

Stage IV: late motor deterioration. This stage usually begins after the age of 10 and can last for years or decades. It's marked by reduced mobility, muscle weakness, joint contractures and scoliosis. Understanding, communication and hand skills generally remain stable or improve slightly, and seizures may occur less often.