Novartis is preparing to present the trial findings to regulatory bodies in 2025. Credit: Novartis AG.

Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, intrathecal onasemnogene abeparvovec (OAV101 IT) in treating individuals with spinal muscular atrophy (SMA) Type 2.

The randomised, sham-controlled study assessed the efficacy and safety of the gene therapy in treatment-naïve individuals aged two to less than 18 years who can sit but have never walked independently.

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It randomised over 100 subjects to receive either gene therapy by intrathecal injection or a sham procedure.

The study’s primary endpoint was met, indicating an increase in total Hammersmith Functional Motor Scale – Expanded (HFMSE) scores from baseline. This finding indicated ‘improved’ motor function in subjects treated with gene therapy compared to sham controls.

Furthermore, the gene therapy’s safety profile was reported as ‘favourable’, with the incidence of adverse events and serious adverse events being similar between the treatment and control arms.

Novartis Development president and chief medical officer Shreeram Aradhye said: “Many patients with SMA currently rely on chronic treatments to manage their disease. These positive topline results from the STEER trial underscore the efficacy, safety, and tolerability of OAV101 IT in patients with SMA aged two and above.

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“The totality of evidence clearly supports a positive risk benefit profile of OAV101 which is expected to support registration covering a broad range of SMA patients. We remain committed to leading innovation in SMA treatment through our one-time gene therapies, uniquely designed to replace the function of the missing or defective SMN1 gene.”

Assessing the therapy’s efficacy and safety using the Revised Upper Limb Module (RULM) scale were the secondary objectives of the trial.

The company is preparing to present these findings to regulatory bodies in 2025, including the US Food and Drug Administration (FDA), aiming to make the therapy available for SMA patients.

In August this year, the company reported that its Phase III V-MONO trial of Leqvio for treating individuals at low or moderate risk of atherosclerotic cardiovascular disease (ASCVD), met its primary endpoints.

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