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Novartis NVS announced positive top-line results from the late-stage STEER study on experimental candidate intrathecal onasemnogene abeparvovec (OAV101IT).
Intrathecal onasemnogene abeparvovec (OAV101IT) is an investigational, one-time gene therapy for patients with spinal muscular atrophy (SMA).
The STEER study is a phase III randomized, double-blind, sham-controlled study evaluating the clinical efficacy, safety and tolerability of a one-time dose of intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment naïve patients with SMA type 2, aged two to less than 18 years who were able to sit, but never walked independently.
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More on NVS’ STEER Study
The efficacy and safety results for OAV101IT in the STEER study were compared against a sham control, a procedure designed to mimic the administration of an investigational drug without delivering any active treatment.
Results showed that the study met its primary endpoint. Data from the study showed an increase from baseline across the study population in total Hammersmith Functional Motor Scale - Expanded (“HFMSE”) scores.
HFMSE is a standard for SMA-specific assessment of motor ability and disease progression.
The increase was primarily observed in patients treated with OAV101IT compared to sham controls, indicating better motor function in patients with SMA.
SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, results in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.
In addition, the safety profile of OAV101IT was favorable as the overall adverse and serious adverse events were similar between arms.
Novartis plans to share STEER study results with regulatory agencies in 2025.
Apart from the STEER study, OAV101IT was evaluated in two clinical studies, including phase I/II STRONG and phase IIIb STRENGTH. The STRONG study was an open-label, dose ranging study evaluating the safety and efficacy of OAV101IT in patients with SMA with three copies of SMN2 aged 6 months to less than 60 months. The STRENGTH study was an open-label, single arm, multi-center study evaluating the safety, tolerability and efficacy of OAV101IT in patients with SMA who had discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY Evrysdi (risdiplam).
The OAV101 IT clinical development program was studied in a broad population of approximately 170 patients with SMA and follow-up was conducted for up to 6.4 years.