Catalyst Database

Two Ph3 trials are ongoing. Database lock in Q1 2024. 5/29/24 Interim analysis from 1st trial in Q2 '24 informed study continuation. Expect Ph3 Interim data analysis in H2 2024 & topline data in H1 2025.

sunRIZE trial. 2/12/25- Interim analysis of the primary endpoint, in early Q2 2025.

PCA001 Trial. 12/22- cleared IND & dosed first patient on 6/12/23. 10/1/24- Data update in H1 2025.

DREAM trial. H1 2023- Initiated. 5/23/24 Positive top-line data. Initiated 13-week dose titration trial. 9/9/24 Positive 13-week Ph1b data.Higher doses up to 26 mg & with 28 weeks of treatment has been added w/ topline data from part 2 in H1 2025.Ph2b planned in H1 '25.

Pediatrics trial. 1/10/24- First patient dosed. 6/12/24- Presentation first cohort data at IRSF '24. 6/18/24- Positive clinical data across adult & pediatric patients from low dose cohort. Dose first patient in cohort two in Q3 2024 with initial data in H1 2025. 9/26/24 Present clinical data up to 22 weeks at Rett syndrome congress in October.

REVEAL study. 6/12/24- Presentation first cohort data at IRSF '24. 6/18/24- Positive clinical data across adult & pediatric patients from low dose cohort. Dosed first patient in cohort two (high dose) in Q2 2024 with initial data in H1 2025. Present positive 52-week data at Rett syndrome congress in October.

EMERALD-1/2 Ph2a/b trials: 4/25/23- Positive Ph2a EMERALD-1 results. 2/22/24- Continued to enroll in EMERALD-2 w/ primary endpoint at 12-wks in H1 2025. 8/16/24- Company and asset acquired by LLY.

FORTITUDE trial: 6/12/24- Positive Ph1/2 data with favorable safety and tolerability profiles. 6/20/24- Presented initial findings at the Annual FSHD IRC. 10/30/24- Initiated biomarker cohort & complete enrollment in H1 '25. Initiate functional cohort in H1 '25. 1/13/25- Topline data in Q2 2025.

VOYAGENE, EMERGENE Studies (SRP-9003-301). 03/2022- Data from low- & high-dose cohorts. 05/02/23- Complete enrollment and pivotal trial in H2 2023. 01/16/24- Initiates patient screening. 12/18/24- Completed enrollment in EMERGENE w/ data expected in H1 2025.

LINKER-SMM1, LINKER-MM1 trials. 12/7/23- Updated data. Submitted BLA to FDA. 2/21/24- BLA accepted w/ PDUFA target date August 22, 2024. Ph3 confirmatory underway (LINKER-MM3). Presents Pivotal Ph1/2 data at AACR '24. 6/17/24- Presented 14-mt data. 8/21/24- Received CRL from FDA. 2/11/25- BLA accepted, PDUFA date July 10, 2025.