IONS
Donidalorsen HAE PDUFA August 21, 2025; EU regulatory submission in process
ATYR
Enrollment completed in Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis; topline data expected in the third quarter of 2025.
COGT
Plan to report top-line results from registration-directed SUMMIT trial in NonAdvSM patients in July 2025
ZBIO
The Company expects to report the 12-week primary endpoint results for MoonStone by the third quarter of 2025.
AGIO
FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia; PDUFA Goal Date is September 7, 2025
INBX
The cohort is expected to enroll up to 50 patients, each with two to three prior lines of systemic therapy, and data are anticipated in Q3 2025
LENZ
New Drug Application (NDA) for LNZ100 for treatment of presbyopia accepted for review by the U.S. Food and Drug Administration (FDA); PDUFA target action date of August 8, 2025
VRNA
In November 2024, the Company completed enrollment in a Phase 2 dose-ranging trial with glycopyrrolate, a long-acting muscarinic antagonist (“LAMA”), supporting a fixed-dose combination program for the maintenance treatment of COPD via a nebulizer. Results will support initiation of a Phase 2b trial with a fixed dose combination of ensifentrine with glycopyrrolate in the third quarter of 2025.
