Novartis Shares Positive Phase III Data for Spinal Muscular Atrophy Therapy
Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a statistically significant improvement in HFMSE in the Phase III STEER study.
Novartis has announced positive results from its Phase III clinical trial program evaluating intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, in patients aged two to <18 years with spinal muscular atrophy (SMA).1
In the Phase III STEER study, the primary endpoint was met with OAV101 IT treatment demonstrating a statistically significant change from baseline to 52 weeks in Hammersmith Functional Motor Scale Expanded (HFMSE) score. Treatment with OAV101 IT achieved a 2.39-point improvement in HFMSE score, compared to 0.51 points in the sham group. At the end of the 52-week period, all 126 patients in the study received both OAV101 IT and the sham procedure.
In a press release, Crystal Proud, MD, pediatric neurologist and a principal investigator at Children's Hospital of the King's Daughters, said: “In the STEER study evaluating treatment-naïve patients, OAV101 IT demonstrated a statistically significant improvement in motor function across a broad SMA population. These results—paired with those in the STRENGTH study—support the potential for OAV101 IT to be a meaningful treatment option for people living with SMA with a goal of maintaining or improving motor function through a one-time therapy.”
Novartis also shared data from the Phase IIIb STRENGTH trial, which evaluated OAV101 IT in patients with SMA aged two to <18 years who had discontinued treatment with nusinersen or risdiplam. Over 52 weeks, OAV101 IT demonstrated stabilization for the overall patient population. In this study, 27 patients were enrolled.
“The data presented today from our OAV101 IT program reinforce our belief in this therapy, which has the potential to have a meaningful impact on a broad range of people with SMA through its continuous benefit via a one-time dose,” Shreeram Aradhye, MD, president, development and chief medical officer, Novartis, said in the press release. “Together with patients, caregivers and healthcare professionals, we are committed to continuing to advance our mission to lead innovation in SMA treatment and broaden therapy options with our gene replacement therapies.”
In December 2024, Novartis shared initial results from STEER. At the time, the company stated that OAV101 IT became “the first investigational gene therapy to provide clinical benefit in treatment-naïve patients with SMA aged two and older with a positive risk benefit profile.”2
In an earlier press release, 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. 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.”
References
1. New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA. News release. Novartis. March 19, 2025. Accessed March 20, 2025. https://www.novartis.com/news/media-releases/new-novartis-phase-iii-data-demonstrate-meaningful-efficacy-and-safety-results-intrathecal-onasemnogene-abeparvovec-broad-patient-population-sma
2. Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA. News release. Novartis. December 30, 2024. Accessed March 20, 2025. https://www.novartis.com/news/media-releases/novartis-intrathecal-onasemnogene-abeparvovec-phase-iii-study-meets-primary-endpoint-children-and-young-adults-sma
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