Intellia Therapeutics Reports Positive Phase 3 Results for HAE Treatment, Plans 2027 U.S. Launch

Intellia Therapeutics reported positive Phase 3 HAELO trial results for its hereditary angioedema treatment, lonvo-z, which reduced attacks by 87% compared to placebo. The company expects U.S. FDA acceptance of its biologics license application in the second half of 2026, targeting a commercial launch in the first half of 2027. Backed by $628.4 million in cash, which is projected to fund operations into 2028, Intellia is also advancing its Phase 3 trials for nex-z in transthyretin amyloidosis.

Lonvo-z Phase 3 HAE results

• ▪In the Phase 3 HAELO trial, a one-time infusion of lonvo-z reduced hereditary angioedema attacks by 87% compared to placebo during the six-month evaluation period.
• ▪All treatment-emergent adverse events reported in the lonvo-z arm of the Phase 3 HAELO trial were mild or moderate, with no serious adverse events observed.
• ▪In the Phase 3 HAELO trial, 62% of patients receiving lonvo-z were entirely attack-free and therapy-free, compared to 11% of patients in the placebo arm.

Lonvo-z regulatory timeline

• ▪Intellia Therapeutics expects the U.S. Food and Drug Administration to accept its biologics license application submission for lonvo-z in the second half of 2026.
• ▪Intellia Therapeutics plans to commercially launch the hereditary angioedema treatment lonvo-z in the United States in the first half of 2027, pending regulatory approval.

Lonvo-z commercialization readiness

• ▪Intellia Therapeutics finalized its field medical, reimbursement, and strategic accounts teams and engaged with hereditary angioedema treatment centers around the United States in mid-2026.
• ▪Intellia Therapeutics launched the website HAEreframed.com in the second quarter of 2026 to raise awareness of the disease burden of hereditary angioedema.

Nex-z ATTR trial enrollment

• ▪Intellia Therapeutics advanced enrollment in its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials of nex-z for transthyretin amyloidosis during the second quarter of 2026.
• ▪Intellia Therapeutics expects to complete patient enrollment in the MAGNITUDE-2 Phase 3 trial for hereditary transthyretin amyloidosis with polyneuropathy in the second half of 2026.

Nex-z genomic safety insights

• ▪Intellia Therapeutics will provide human leukocyte antigen genotyping results to investigators and patients enrolled or screening in ongoing nex-z Phase 3 clinical trials.
• ▪A genomic analysis of over 600 patient samples across nex-z trials revealed that the highest liver transaminase elevations occurred in patients carrying a specific human leukocyte antigen allele.

Cash position through 2028

• ▪Intellia Therapeutics expects its existing cash resources to fund operations at least into 2028, which is well beyond the planned 2027 U.S. launch of lonvo-z.
• ▪Intellia Therapeutics ended the second quarter on June 30, 2026, with approximately $628.4 million in cash, cash equivalents, and marketable securities.

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