Catalyst Database

ENERGIZE and ENERGIZE-T studies. 6/3/24- ENERGIZE-T 48-wk study topline data, met primary & secondary endpoint. 6/15/24- Presented Ph3 data at EHA '24. 12/8/24- Filed sNDA. 1/13/25- PDUFA goal date of September 7, 2025.

11/7/24- Initiate trial by YE 2024. 2/6/25- Additional data in the summer of 2025.

9/11/23- Positive topline data from SAD. 1/4/24- Positive data from MAD. 12/20/24- Dosed first patient. 3/20/25- Data in summer of 2025 w/ larger long-term Ph2b study by YE 2025.

SHINE study. 9/25/24 Post hoc analysis at EADV '24. 2/28/25- Post-hoc efficacy data via poster at AAD'25 on March 7-11, 2025. 3/20/25- Topline data in summer 2025.

Big Mover: PATHFNDR-1 &2 studies. PATHFNDR-1 & 2 data w/ new long- term safety & efficacy and ACROBAT study at ENDO '24. 9/26/24- NDA submitted to FDA. 12/9/24- FDA accepted NDA submission w/ PDUFA action date of September 25, 2025.

01/24- OLE study initiated. 1/25- Initiated pediatric cohort. 3/24/25- Topline OLE data in September 2025. Initiate Ph3 study by mid-2025 and BLA submission by YE 2025.

SEQUOIA-HCM trial (oHCM). NDA rolling submission completed in Q3 2024. Data showed improvements in cardiac structure, function, & safety profile. 12/2/24- NDA accepted w/ PDUFA date set for September 26, 2025. 12/23/24- EMA validated MAA.

May 2022- Reported Ph3 top-line results. 2/29/24- Continued having constructive dialogue with FDA. 9/23/24 Positive topline results from BHV4157-206-RWE study of 3-year data. NDA in Q4 2024. 12/16/24- NDA submitted. 2/11/25- NDA acceptance and priority review. 3/3/25- NDA acceptance & priority review. PDUFA date in Q3 2025.

TROPION-Lung05 trial. w/ Daiichi Sankyo. 11/12/24- BLA submitted. 12/9/24- Breakthrough Therapy Designation granted based on trial data. 12/26/24- MAA in EU withdrawn. 1/13/25- Accepted & granted priority review.

1/21/25- Ph1 preliminary efficacy & safety data announced. New expansion cohort initiated w/ data in Q3 2025.