Novartis on Monday announced positive topline results from the Phase III STEER study. According to the company’s statement, this pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2.
Efficacy and safety results for OAV101 IT were compared against a sham control, a procedure designed to mimic the administration of an investigational drug, without delivering any active treatment, the company stated.
The safety profile of OAV101 IT was favorable. The overall adverse events and serious adverse events were similar between arms. The most common adverse events were upper respiratory tract infection, pyrexia and vomiting, it added.
Novartis plans to share results with regulatory agencies in 2025, including the US Food and Drug Administration (FDA), with the aim to make OAV101 IT available to help patients with SMA in need. Data will be presented at an upcoming medical meeting in 2025.
“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,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “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.”
The results from the Phase III STEER study add to the clinical data and emerging real-world evidence for the use of one-time gene therapy to treat SMA, it added.
