Lonvo-z Phase 3 HAELO meets all endpoints, cutting HAE attacks 87% vs placebo; rolling BLA begins

Intellia Therapeutics/Lonvo-z (NTLA-2002)

In the 80-patient HAELO Phase 3 trial, a single 50 mg infusion of lonvo-z cut hereditary angioedema attacks 87 percent versus placebo over six months, and 62 percent of treated patients were free of attacks and other therapy. Intellia began a rolling Biologics License Application submission, marking the first Phase 3 success for in vivo gene editing with a potential US launch in H1 2027 if approved.

• 80 patients; single 50 mg infusion reduced attacks 87% versus placebo over 6 months
• 62% of lonvo-z patients attack-free and therapy-free for evaluation period versus 11% placebo
• Rolling BLA submission initiated; potential US launch H1 2027 if approved
• First Phase 3 success for in vivo gene editing; in vivo KLKB1 inactivation in liver