We have previously presented an early analysis of SUNFISH Part 1, which showed that RG7916 administration results in a dose-dependent increase in full-length SMN2 mRNA and a concomitant decrease in SMN2Δ7 mRNA. Recent analysis of SMN protein levels in whole blood showed that in patients with SMA, SMN protein increased in a dose-dependent manner up to median 2.5-fold. The safety, tolerability and PK/PD data from Part 1 informed the selection of a RG7916 dose level for SUNFISH Part 2 predicted to lead to clinically efficacious increases in SMN protein. To date, no drug-related adverse events leading to withdrawal have been observed.
SUNFISH (NCT02908685) — type 2 och 3 SMA