Intellia Therapeutics announced additional positive results from its global Phase 3 HAELO clinical trial for lonvoguran ziclumeran (lonvo-z), a single-dose gene editing treatment for hereditary angioedema (HAE). The data demonstrated statistically significant reductions in HAE attacks and a favorable safety profile, supporting the company's ongoing regulatory submission.
Key Details
- High Efficacy: The trial showed an 89% reduction in monthly attacks requiring on-demand treatment and a 91% reduction in moderate/severe attacks compared to placebo (p<0.0001). Additionally, 62% of patients treated with lonvo-z were completely attack-free for the six-month evaluation period versus 11% for placebo.
- Safety Profile: Lonvo-z demonstrated favorable safety and tolerability. All reported treatment-emergent adverse events in the lonvo-z arm were mild or moderate, with no serious adverse events observed.
- Regulatory Timeline: Intellia initiated a rolling Biologics License Application (BLA) submission with the U.S. FDA in April 2026 and anticipates a potential U.S. launch in the first half of 2027.