Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial

Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam).

Scholar Rock also announced that it plans to present encore data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes in SMA after 36 months of treatment with apitegromab. Efficacy and safety data at 36 months, and an analysis of patient-reported outcome measures such as fatigue, mobility, and activities of daily living, will be presented during two poster presentations at the 28th Annual Congress of the World Muscle Society from October 3 – 7 in Charleston, South Carolina, USA.

Check out the full press release here.


Do you like what you're reading?

Help make a difference in the lives of people affected by spinal muscular atrophy.

Leave a Comment

Your email address will not be published. Required fields are marked *

Scroll to Top