This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below!
Scholar Rock presents two posters featuring additional analyses from the TOPAZ phase 2 trial of Apitegromab:
Poster: Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (LBP.10)
- This late breaking poster presentation features multiple efficacy endpoints, including Hammersmith scales and Revised Upper Limb Module (RULM) function, from the 12-month TOPAZ Phase 2 trial evaluating apitegromab in patients with Type 2 and 3 Spinal Muscular Atrophy (SMA).
- An additional exploratory analysis evaluating time to achieving various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores further support the dose response in clinical efficacy.
- Increases in RULM were also observed in both non-ambulatory cohorts.
- Greater increases in HFMSE (non-ambulatory) and Revised Hammersmith Scale (ambulatory) scores were seen in patients who were not limited by scoliosis or joint contractures.
Poster: Insights into the Potential Pharmacological Effects of Apitegromab in Health and Disease: Data from Preclinical and Clinical Studies (EP.277)
- The poster showcases insights into the target engagement and exposure levels of apitegromab in the TOPAZ trial as well as from Phase 1 healthy volunteer and preclinical studies.
Roche presents new data from the RAINBOWFISH Trial: 
Oral Presentation: RAINBOWFISH: A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA)
- Data from the ongoing open label RAINBOWFISH study were presented. Four out of five of those treated with Evrysdi for at least 12 months achieved standing and walking independently within the World Health Organization windows for healthy children. In addition, all five babies maintained the ability to swallow and were able to feed exclusively orally after 12 months of treatment.
- Previously reported results showed that babies treated with Evrysdi for at least 12 months achieved Hammersmith Infant Neurological Examination (HINE-2) motor milestones, with 100% (n=5) able to maintain head control, sitting upright, rolling and crawling.
- These data further add to the growing body of evidence supporting Evrysdi’s efficacy in a broad patient population. More than 4,000 patients have been treated with Evrysdi in clinical trials, compassionate use, and real-world settings.