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6-K: Novartis Intrathecal Onasemnogene Abeparvovec Phase Iii Study Meets Primary Endpoint in Children and Young Adults with SMA

SEC ·  Dec 30, 2024 11:10

Summary by Moomoo AI

Novartis announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve SMA Type 2 patients aged 2-18 years. The study met its primary endpoint, demonstrating increased Hammersmith Functional Motor Scale - Expanded (HFMSE) scores compared to sham controls, indicating improved motor function in patients.The safety profile was favorable, with similar adverse events between treatment and control arms. The most common side effects included upper respiratory tract infection, pyrexia, and vomiting. The study involved over 100 patients who were able to sit but had never walked independently.Novartis plans to share results with regulatory agencies, including the FDA, in 2025 to pursue approval. The STEER results build on the previous Phase I/II STRONG study, which showed clinically meaningful HFMSE score increases in one year. This gene therapy represents a potential one-time treatment option for SMA patients who currently rely on chronic treatments.
Novartis announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve SMA Type 2 patients aged 2-18 years. The study met its primary endpoint, demonstrating increased Hammersmith Functional Motor Scale - Expanded (HFMSE) scores compared to sham controls, indicating improved motor function in patients.The safety profile was favorable, with similar adverse events between treatment and control arms. The most common side effects included upper respiratory tract infection, pyrexia, and vomiting. The study involved over 100 patients who were able to sit but had never walked independently.Novartis plans to share results with regulatory agencies, including the FDA, in 2025 to pursue approval. The STEER results build on the previous Phase I/II STRONG study, which showed clinically meaningful HFMSE score increases in one year. This gene therapy represents a potential one-time treatment option for SMA patients who currently rely on chronic treatments.
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