Sapphire Trial Results and Updates
Sapphire Trial Results and Updates
Last updated: 7th October 2024
2024
7th October: Apitegromab successfully meets primary endpoint in Phase 3 of the SAPPHIRE study.
There have been 188 trial participants aged 2-21 years who are unable to walk and live with Type 2 or Type 3 SMA. Enrolment for the SAPPHIRE trial was completed in September 2023.
Scholar Rock announced today that this trial of apitegromab demonstrated an overall statistically significant and clinically meaningful improvement.
Key findings include:
- 30.4% of patients treated with apitegromab saw a motor function improvement of more than 3 points, compared to 12.5% on a placebo.
- Early improvements were seen within 8 weeks, with benefits lasting through a year.
- The drug showed a favourable safety profile, consistent with earlier trials (Phase 2 TOPAZ).
Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study.
Scholar Rock plans to apply for European and US regulatory approval in early 2025.
These results will be presented during a poster presentation at the upcoming 29th Annual Congress of the World Muscle Society on Friday, October 11, 2024, being held in Prague.
2023
19th September: Scholar Rock announced the completion of patient enrolment for the SAPPHIRE Trial.
25th January (updated Sept 2023): Trial sites announced for the UK:
| Trial Centre | Current status / Contact | Consultant |
|---|---|---|
| Leeds | Activated | Dr. Childs |
| Oxford | Activated | Dr. Servais |
| Great Ormond Street | Activated | Dr. Baranello |
2022
20th September: Scholar announced the opening of the initial European clinical trial sites for the phase 3 SAPPHIRE study in Spain, with additional sites to be announced.
2021
2nd December: Scholar Rock announced the design of this Phase 3 trial.