Swiss pharmaceutical company Novartis Pharma AG (SIX:NOVN) (NYSE:NVS) on Monday announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2.

The study met its primary endpoint, demonstrating an increase in Hammersmith Functional Motor Scale - Expanded (HFMSE) scores in patients treated with OAV101 IT compared to a sham control.

OAV101 IT had a favourable safety profile with adverse events similar across treatment groups.

Novartis plans to submit the STEER study results to regulatory agencies in 2025 with the aim of making OAV101 IT available to patients with SMA.

These findings build upon the positive results from the Phase I/II STRONG study, further supporting the potential of one-time gene therapy for the treatment of SMA.