The ASCEND study is a global clinical trial initiated by Biogen to evaluate if patients who have previously received treatment with risdiplam, an approved SMA drug also known as Evrysdi® (risdiplam), and may not have achieved optimal clinical outcomes (e.g., improvement or delay in disease progression), may benefit from receiving a higher dose of nusinersen, also known as Spinraza® (nusinersen).

On June 17th, Scholar Rock announced new data from the Phase 2 TOPAZ trial extension period evaluating outcomes after 24-months of treatment.

Apitegromab, or SRK-015, is a muscle-directed therapy, developed by Scholar Rock that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. The aim of the TOPAZ clinial trial, a phase 2 study, is to evaluate the efficacy and safety of apitegromab in people with later-onset SMA. 

Today, the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). This means that risdiplam is now approved in the US to treat SMA in children and adults of all ages. 

Following our request to receive important and timely updates on their SMA clinical programme, Roche has informed us that worldwide recruitment has completed for the RAINBOWFISH clinical trial, which investigates the efficacy and safety of risdiplam in infants with pre-symptomatic SMA Type 1. This milestone marks the enrolment of 26 participants from across the globe.