Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Key Inclusion criteria:

1. Diagnostic confirmation during screening period of 5q SMA
2. The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
3. Onset of clinical signs and symptoms at ≥ 6 months of age
4. A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility
5. Able to sit independently at screening, but has never had the ability to walk independently.

Key Exclusion criteria:

1. Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to > 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab.
2. Infectious process (e.g. viral, bacterial) or febrile illness prior to start of screening, and up to OAV101 treatment or sham procedure
3. Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), > upper limit of normal (ULN).
4. Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours during a 24-hour period, noninvasive ventilation for > 12 hours during a 24-hour period or requiring tracheostomy
5. Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle > 40 in a sitting position
6. Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks
7. Clinically significant sensory abnormalities in the neurological examination at Screening