Avexis has been granted authorisation to start Phase 1 clinical trial of AVXS-101 for patients with SMA Type 2 by the U.S. Food and Drug Administration (FDA), based on review of data submitted. The company plans to initiate this trial immediately.
The open-label, dose-comparison, multi-center Phase 1 trial named STRONG, is designed to evaluate the safety, optimal dosing and proof of concept for efficacy of AVXS-101 in two distinct age groups of patients with SMA Type 2, using a one-time IT route of administration. The trial will enroll 27 infants and children with a genetic diagnosis consistent with SMA, including the bi-allelic deletion of SMN1 and three copies of SMN2 without the SMN2 genetic modifier, who are able to sit but have no historical or current ability to stand or walk.
Two dosage strengths will be evaluated and patients will be stratified into two age groups:
- patients less than 24 months
- patients at least 24 months but less than 60 months.
There will be at least a four-week interval between the dosing of the first three patients for each dose being studied and based on the available safety data, a decision will be made whether to proceed.
- Cohort 1 (Dose A) will receive a dose of 6.0 x 1013 vg of AVXS-101 and enroll three patients less than 24 months of age. If safety is established according to the Data Safety Monitoring Board (DSMB), the study will proceed to Cohort 2.
- Cohort 2 (Dose B) will receive a dose of 1.2 X 1014 vg of AVXS-101 and enroll three patients less than 60 months of age.
If safety is established, an additional 21 patients will be enrolled until there are a total of 12 patients less than 24 months and 12 patients at least 24 months but less than 60 months of age, who have received Dose B.