Intellia's CRISPR Therapy Cuts HAE Attacks 87%, Targets 2027 Launch
Intellia's HAELO Phase 3 trial cut hereditary angioedema attacks by 87% versus placebo over six months, with treated patients averaging 0.26 monthly attacks versus 2.10. The company has begun rolling BLA submission for lonvo-z and targets a U.S. launch in first half 2027 pending approval.
1. Phase 3 HAELO Trial Results
The HAELO trial enrolled 80 hereditary angioedema patients, randomizing 52 to a single dose of lonvoguran ziclumeran and 28 to placebo. Treatment met its primary and all key secondary endpoints, reducing mean monthly attack rates to 0.26 versus 2.10 on placebo—an 87% decrease over six months.
2. Regulatory Submission and Launch Timeline
Intellia has initiated a rolling biologics license application with the FDA for lonvo-z and expects to complete the full BLA submission in the second half of 2026. The company is targeting a potential U.S. launch in the first half of 2027, subject to priority review and approval.
3. Safety and Tolerability
Lonvo-z demonstrated a favorable safety profile, with the most common treatment-emergent adverse events being infusion-related reactions, headache and fatigue. All reported events were mild to moderate and no serious adverse events or liver toxicities were observed as of the data cutoff.
4. Stock Reaction and Competitive Context
Shares of Intellia slipped 3% following the data release despite robust results, reflecting short-term profit taking. If approved, lonvo-z would become the first in vivo CRISPR therapy in the U.S., competing against ex vivo product Casgevy.