Update on Scholar Rock's Apitegromab Trials

Update on Scholar Rock's Apitegromab Trials

We are pleased to share that Scholar Rock has announced the completion of enrollment for their Phase 3 SAPPHIRE trial. This trial is significant as it evaluates the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are on SMN therapy (like nusinersen or risdiplam).

Scholar Rock is set to share encore data from the Phase 2 TOPAZ trial at the 28th Annual Congress of the World Muscle Society this October. This includes insights on patient outcomes after 36 months of treatment with apitegromab. The data focuses on factors crucial to our community - fatigue, mobility, and daily activities. The relevant presentations will be made available on Scholar Rock’s website.

What Makes Apitegromab Special?

Apitegromab stands out as it's the only muscle-targeted therapy candidate with clinical proof of concept in SMA. By targeting myostatin in skeletal muscles, it aims to bring about significant improvements in motor functions for SMA patients. The drug has already earned recognitions like Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. FDA, indicating its potential.

Scholar Rock looks forward to reporting the topline results from this study in Q4 2024.

For more detailed scientific and trial data, please visit Scholar Rock’s official announcement here.

Leave a comment