Intellia Therapeutics Announces Anticipated 2025 Milestones and Strategic Reorganization to Prioritize the Advancement of its Late-Stage Programs, NTLA-2002 and Nexiguran Ziclumeran (nex-z)

Strategic Priorities and Milestones - Intellia Therapeutics announced its strategic priorities and key anticipated 2025 milestones, focusing on transforming patient lives through CRISPR-based therapies [1] - The company aims to transition from a late-stage development company to a commercial-ready organization by the end of 2026 [7] - Key priorities include driving clinical execution for NTLA-2002 and nex-z, advancing commercial readiness, and preparing for the first U S commercial launch [8][9] Pipeline Advancements - NTLA-2002 for Hereditary Angioedema (HAE): Phase 3 HAELO study enrollment to complete in the second half of 2025, with a Biologics License Application submission planned for the second half of 2026 [5] - Nex-z for Transthyretin (ATTR) Amyloidosis: Over 550 patients expected to be enrolled in the Phase 3 MAGNITUDE study by year-end 2025, with strong enrollment momentum [5][12] - NTLA-3001 discontinued to focus resources on NTLA-2002 and nex-z, resulting in a net workforce reduction of approximately 27% in 2025 [5][6] Financial and Operational Updates - Intellia ended Q4 2024 with approximately $862 million in cash, cash equivalents, and investments, providing a cash runway into the first half of 2027 [6] - The company expects to incur$8 million in charges related to the strategic reorganization in Q1 2025 [6] - Cost savings from workforce reduction and pipeline prioritization will support operations through the anticipated first commercial launch in the U S [5][6] Leadership Changes - Laura Sepp-Lorenzino, Ph D , Chief Scientific Officer, announced her retirement effective December 31, 2025, transitioning to a Senior Scientific Advisor role [6] - Birgit Schultes, Ph D , promoted to Executive Vice President and Chief Scientific Officer, effective January 13, 2025, bringing over 20 years of experience in drug development and biotechnology [6] Clinical Data and Presentations - Phase 2 data for NTLA-2002 presented at the 2024 ACAAI Scientific Meeting demonstrated potential to end chronic prophylaxis treatment with a one-time infusion [6] - Phase 1/2 data for NTLA-2002 and Phase 1 data for nex-z to be presented in 2025, including longer-term efficacy and safety measures [13] - First clinical evidence for nex-z presented at the 2024 AHA Scientific Sessions showed rapid, deep, and durable reductions in serum TTR, potentially halting or reversing disease progression [12] Commercial Readiness - Intellia plans to complete the buildout of its commercial leadership team by the second half of 2025 [13] - The company will expand medical education activities in HAE and ATTR amyloidosis and initiate pre-approval information exchange with payers in 2025 [13] - John Leonard, M D , President and CEO, will present a company overview at the 43rd Annual J P Morgan Healthcare Conference on January 13, 2025 [9]