As several clinical trials continue in SMN1-related proximal spinal muscular atrophy, new results have been shared.
- DEVOTE trial, testing higher doses (50/28 mg) of Spinraza than currently available (12 mg): clinical benefits of higher doses in patients already treated or treatment naïve. Motor neuron degeneration, which is reflected in the reduction of neurofilaments in plasma, appears to be slower.
- STEER trial, evaluating intrathecal Zolgensma in more than 100 patients with type II SMA, aged between 2 and 18, able to sit but not to walk independently, and naïve to any treatment: increase in HFMSE score and good tolerance of the product after one year of treatment.
- SMART trial, analysing the effects of intravenous Zolgensma in symptomatic children weighing between 8.5 and 21 kg, some of whom had already been treated with another innovative therapy: after one year, the treatment was well tolerated and effective in stabilising or improving their motor function.
- RAINBOWFISH trial, evaluating Evrysdi in 23 presymptomatic infants aged under 6 weeks: after two years of treatment, all were able to sit up; 100% of those with 3 copies of SMN2 and 60% with 2 copies were able to walk independently. All were able to eat orally and swallow. None required permanent ventilation.
- JEWELFISH trial, testing the safety, tolerability and pharmacokinetics of Evrysdi in patients already treated with Spinraza, Zolgensma, olesoxime or RG7800: drug well tolerated and presence of SMN protein in the blood after two years of treatment.
- RESILIENT trial, analysing the combination of taldefgrobep alfa in SMA patients treated with one of three innovative therapies: at the end of a one-year follow-up, the trial’s primary endpoint (increase in MFM-32 score compared with the placebo group) was not met.
- SAPHIRRE trial, testing the combination of apitegromab with Spinraza or Evrysdi in type II or III SMA: after one year of treatment, improvement in motor function versus placebo.
New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA. Biogen. 8 October 2024 press release.
Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA. Novartis.30 December 2024 press release.
Majority of children with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate. Roche. 14 October 2024 press release.
Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy and Obesity. Biohaven. 25 November 2024 press release.
Scholar Rock Reports Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA). Scholar Rock. 7 October 2024 press release.
