NEJM

PROPEL 3 met its primary endpoint, positioning infigratinib as a potential first oral rival to BioMarin's injectable Voxzogo. NDA filing is planned for Q3 2026.

Lonvoguran ziclumeran met its primary endpoint with an 87% reduction in monthly attacks, the first Phase 3 win for an in vivo CRISPR therapy.

Zenas BioPharma's obexelimab met its primary endpoint and all four key secondary endpoints in the Phase 3 INDIGO trial, with results published in NEJM and presented at EULAR 2026.

A single infusion of VERVE-102 reduced LDL-C by up to 62% and PCSK9 by up to 88% in a 35-patient trial, with effects sustained up to 18 months.

Brepocitinib, a first-in-class TYK2/JAK1 inhibitor, hit all primary and secondary endpoints in the largest-ever dermatomyositis trial, offering the first steroid-sparing treatment for a disease that has lacked targeted therapy for decades.