Genentech presents data from FIREFISH and SUNFISH studies
Genentech, a member of the Roche Group, announced new data from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy achieved key motor milestones after one year of treatment with investigational risdiplam. Among the infants who received the dose selected for the confirmatory Part 2 of the study, seven were able to sit without support for at least 5 seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development - Third Edition. In addition, 11 infants were able to sit (with or without support) while nine achieved upright head control after 12 months of treatment as assessed by the Hammersmith Infant Neurological Examination Module 2. Finally, one infant achieved the milestone of standing by this 12-month time point. The confirmatory Part 2 portions of the SUNFISH and FIREFISH studies have completed enrollment and will conduct their primary efficacy analyses in Q4 2019 and Q1 2020, respectively.