tiprankstipranks
Trending News
More News >
Advertisement
Advertisement

Genentech presents two-year data from RAINBOWFISH study at WMS

Genentech, a member of Roche, presented two-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society, or WMS, Congress, October 8-12, assessing the efficacy and safety of Evrysdi in children with SMA who were treated pre-symptomatically as infants before six weeks of age. The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation. All of the children treated with Evrysdi who had three or more SMN2 copies, achieved standing and walking milestones as assessed by Bayley Scales of Infant and Toddler Development, Third Edition and Hammersmith Infant Neurological Examination, Module 2, or HINE-2, with most achieving these milestones within World Health Organization, or WHO, windows of typical child development. Among the children with two SMN2 copies, all could sit and most could stand and walk independently after two years of treatment. After two years of treatment, all children were able to swallow and feed orally and none required permanent ventilation. Natural history studies indicate that without disease-modifying treatment, children with Type 1 SMA would not be able to reach such milestones, nor typically live past the age of two. After 2 years of Evrysdi treatment, children in the study showed cognitive skills typical of children without SMA, as assessed by the BSID-III Cognitive Scale. This study was the first clinical trial in SMA to assess cognition as an exploratory endpoint using a standardized scale. To assess outcomes of early treatment initiation before the onset of symptoms, children in the study started treatment with Evrysdi before 6 weeks of age (median age of first dose was 25 days). The study analyzed outcomes against the number of copies of the SMN2 gene each child had. Generally, fewer SMN2 copy numbers are associated with more severe SMA. There were no deaths or adverse events, or AEs, leading to withdrawal or treatment discontinuation. The most common AEs were teething, gastroenteritis, diarrhea, eczema and pyrexia. The AEs observed in the year-two analysis are generally consistent with those AEs seen in other Evrysdi trials in SMA. AEs were more reflective of age than underlying SMA. The majority of AEs were not considered treatment-related and resolved over time. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Elevate Your Investing Strategy:

  • Take advantage of TipRanks Premium at 55% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.

Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>

Disclaimer & DisclosureReport an Issue

1