Intellia Therapeutics(US:NTLA) Globenewswireยท2025-11-08 22:13Core Insights - Intellia Therapeutics presented positive clinical data for lonvo-z, a CRISPR-based therapy for hereditary angioedema (HAE), at the ACAAI 2025 Annual Scientific Meeting, indicating potential to redefine HAE treatment [1][2] - The pooled analysis from the Phase 1/2 trial showed that 97% of patients were attack-free and long-term prophylaxis (LTP)-free after a one-time 50 mg dose [4][6] - The ongoing Phase 3 HAELO clinical trial is expected to provide topline results by mid-2026 [2][7] Pooled Phase 1/2 Analysis - The analysis included 32 patients who received a one-time 50 mg treatment of lonvo-z, with a data cutoff on August 29, 2025 [3] - Among these patients, 15 received the 50 mg dose at study Day 1, while 17 were treated after unblinding [3] Efficacy Results - A mean reduction of 89% in plasma kallikrein levels was observed at month 24 [4] - 31 out of 32 patients (97%) were both attack-free and LTP-free, with 24 patients (75%) maintaining this status for at least seven months [4][6] - Among the 11 patients who initially received the 50 mg dose in Phase 2, 10 were attack-free and LTP-free [6] Safety Profile - The 50 mg dose demonstrated a well-tolerated safety profile with no long-term risks identified over three years of follow-up [5][6] - Common treatment-emergent adverse events included infusion-related reactions, fatigue, and headache [5] - No clinically significant shifts in liver enzymes or coagulation parameters were reported [5] Ongoing Clinical Trials - The global Phase 3 HAELO clinical trial, which completed enrollment in September 2025, is further evaluating the efficacy of a one-time 50 mg treatment of lonvo-z [7][8] - Lonvo-z is based on CRISPR/Cas9 technology and aims to prevent HAE attacks by inactivating the KLKB1 gene [9] Regulatory Designations - Lonvo-z has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA, and PRIME Designation from the European Medicines Agency [9]