Lonvo-z Phase 3 Enrollment and Imminent Readout
Completed enrollment of the HAELO Phase 3 trial with 80 patients in ~9 months; top-line data expected by mid-2026 and a planned BLA submission in H2 2026 — positioning Intellia for the world’s first pivotal readout for an in vivo CRISPR gene-editing therapy.
Strong Patient and Physician Demand for Lonvo-z
Market research: 99% of surveyed U.S. HAE patients were at least somewhat likely to take the gene-editing treatment (nearly two-thirds extremely/very likely); 92% of 151 U.S. HCPs said they could identify a patient to prescribe to — providers collectively manage >4,000 patients (≈60% of treated U.S. population) and indicated ~2,200 (54%) could be prescribed lonvo-z.
Durable Clinical Signals in Early Studies
With up to three years of follow-up in Phase 1/2 studies, no waning of serum kallikrein or TTR reductions was observed; pooled analysis showed 76% of patients ≥1 year beyond a 50 mg lonvo-z dose were free from attacks and ongoing therapy for ≥12 months.
Financial Improvements in Q4 2025
Q4 collaboration revenue rose to $23.0M from $12.9M (+~78% YoY); R&D expense decreased to $88.7M from $116.9M (≈-24% YoY); net loss narrowed to $95.8M from $128.9M (≈-26% YoY).
Cash Runway and Commercial Readiness
Cash, cash equivalents and marketable securities were $605.1M as of Dec 31, 2025 (down from $861.7M a year earlier, ≈-30%), which management expects is sufficient to reach into the second half of 2027 and through key milestones; CMC and commercial preparedness for lonvo-z described as largely complete, with commercial field teams and payer engagement underway.
Regulatory Progress on MAGNITUDE-2 (PN)
FDA lifted the clinical hold on MAGNITUDE-2 (polyneuropathy) in late January 2026 after agreeing to study modifications (additional early post-dose liver labs, short-term steroid regimen for LFT elevations, and modified screening criteria); target enrollment increased from 50 to ~60 and 47 patients were already enrolled.