Clinical TrialApr 27, 2026, 09:17 AM
NTLA Lonvo-z Phase 3 HAE Trial Positive; BLA Initiated
AI Summary
Intellia Therapeutics announced positive topline results from its global Phase 3 HAELO clinical trial of lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (HAE), marking a global first for in vivo gene editing. The trial met its primary and all key secondary endpoints, showing an 87% reduction in HAE attacks and a favorable safety profile. The company has initiated a rolling Biologics License Application (BLA) submission with the U.S. FDA, anticipating a potential U.S. launch in the first half of 2027.
Key Highlights
- Phase 3 HAELO trial of lonvo-z met primary and all key secondary endpoints.
- Lonvo-z reduced HAE attacks by 87% vs. placebo (mean monthly attack rate 0.26 vs. 2.10).
- 62% of lonvo-z patients were attack-free and therapy-free for six months.
- Favorable safety and tolerability observed; no serious adverse events in lonvo-z arm.
- Rolling Biologics License Application (BLA) submission initiated with U.S. FDA.
- Anticipate U.S. launch in the first half of 2027, if approved.
- All patients who received lonvo-z remained long-term prophylaxis (LTP) free.