Core Insights - Intellia Therapeutics announced promising three-year follow-up data from the Phase 1 portion of its ongoing Phase 1/2 study for lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE) [1][2] - All 10 patients in the Phase 1 study were attack-free and treatment-free for a median of nearly two years, demonstrating the potential of lonvo-z as a one-time therapy [2][6] - The global Phase 3 HAELO trial has completed screening ahead of schedule, with over half of the patients screened in the United States [4][6] Clinical Results - A single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% compared to pre-treatment baseline [2][6] - Patients showed deep, dose-dependent, and durable reductions in plasma kallikrein protein levels throughout the study [2][6] - The treatment was well tolerated, with the most frequent adverse events being infusion-related reactions, which were mostly Grade 1 and resolved without complications [3][6] Development Plans - The ongoing Phase 3 HAELO trial is randomized, double-blind, and placebo-controlled, assessing the safety and efficacy of lonvo-z at the 50 mg dosage [4][5] - Intellia plans to submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027 [4][8] - New data from the Phase 2 portion of the ongoing Phase 1/2 study is expected to be presented in the second half of 2025 [4][10] About Lonvo-z - Lonvo-z is based on CRISPR/Cas9 technology and aims to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene [8] - The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA [8][9] - Intellia is focused on leveraging gene editing technology to develop novel therapies that address unmet medical needs [9]

Verve Therapeutics (VERV) 2025 Conference June 04, 2025 09:20 AM ET Speaker0 Okay. Welcome everyone to twenty twenty five Jefferies Global Healthcare Conference. It it is my my name is Roger Song, one of the senior analysts covers MidCap biotech in US. And then it's my pleasure to have the next five star chat with our company, Zach, from the so excuse me. So from the Verve Therapeutics. So it it is a it is it is a very exciting timing for Verve because they just reported a very exciting data from the one tw ...

Core Insights - Intellia Therapeutics announced positive two-year follow-up data from the Phase 1 trial of investigational nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) [1][2] - The data presented at the 2025 Peripheral Nerve Society Annual Meeting indicates that a single dose of nex-z leads to significant reductions in serum TTR levels and improvements in neuropathic impairment measures [2][5] ATTRv-PN Results - In the dose-escalation portion (N=15), the mean Neuropathy Impairment Score (NIS) improved by -2.0 at Month 12 and -4.5 at Month 24 [4] - In the dose expansion portion (N=21), the mean NIS improved by -2.1 at Month 12 and -5.2 at Month 24 [4] - The overall mean change in modified NIS +7 (mNIS+7) was -0.6 at Month 12 and -8.5 at Month 24 [4] - Among patients previously on patisiran, the mean mNIS+7 change was -6.3 at Month 12 and -6.5 at Month 24 [4] - The Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) score improved by -3.5 at Month 12 and -8.5 at Month 24 [4] - Neurofilament light chain (NfL) showed a reduction of -8.6% at Month 12 [4] Safety and Tolerability - Nex-z demonstrated generally favorable safety and tolerability with no new drug-related adverse events reported during the follow-up period [5][7] - The most common treatment-related adverse events were mild to moderate infusion-related reactions, which did not lead to discontinuations [7] Clinical Program and Future Prospects - The ongoing Phase 1 trial is a multi-center study evaluating nex-z in adults with hereditary ATTR amyloidosis [8] - The Phase 3 MAGNITUDE-2 trial is designed to measure clinical outcomes and evaluate the efficacy of a single dose of nex-z, with a potential biologics license application submission by 2028 [5][9] - Nex-z is based on CRISPR technology and aims to be the first one-time treatment for ATTR amyloidosis by inactivating the TTR gene [10]