Intellia Therapeutics (NASDAQ:NTLA) announced a strategic reorganization to focus on its late-stage CRISPR-based therapies, NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis. The company aims to streamline operations and prepare for potential commercial launches, with key milestones anticipated in 2025.
The pipeline prioritization involves discontinuing development of NTLA-3001 for alpha-1 antitrypsin deficiency-associated lung disease and other early-stage research programs. This strategic shift will result in a net workforce reduction of approximately 27% in 2025, with the company expecting to incur approximately $8 million in reorganization charges during the first quarter of 2025.
Clinical Trial Updates and Milestones
NTLA-2002 for Hereditary Angioedema (HAE): The pivotal Phase 3 HAELO study is actively enrolling patients. Data presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting from the Phase 2 study demonstrated the potential of NTLA-2002 to eliminate the need for chronic prophylactic treatment, offering patients freedom from attacks after a single infusion. Intellia plans to complete enrollment for the HAELO study in the second half of 2025 and submit a Biologics License Application (BLA) in the second half of 2026.
Nex-z for Transthyretin (ATTR) Amyloidosis: Enrollment continues to progress in the Phase 3 MAGNITUDE study for ATTR amyloidosis with cardiomyopathy (ATTR-CM), exceeding the company’s internal projections. Screening is underway for patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) in the Phase 3 MAGNITUDE-2 study. Clinical evidence presented at the 2024 American Heart Association (AHA) Scientific Sessions indicated that nex-z achieved consistent, rapid, deep, and durable reductions in serum TTR levels, potentially halting or reversing disease progression.
Financial Position and Future Outlook
Intellia reported approximately $862 million in cash, cash equivalents, and investments at the close of 2024. The company anticipates that its current cash balance, combined with projected cost savings, will fund operations into the first half of 2027, supporting the advancement of its late-stage programs and preparations for its first commercial launch in the U.S.
John Leonard, M.D., President and Chief Executive Officer of Intellia, stated, "We have made significant progress and built strong momentum in 2024 with three actively enrolling, Phase 3, pivotal studies. Our early clinical data for both NTLA-2002 and nex-z support novel, highly differentiated product profiles that directly address the significant unmet needs of patients and prescribers in HAE and ATTR."
Leadership Transition
Intellia also announced that Laura Sepp-Lorenzino, Ph.D., Chief Scientific Officer, will retire effective December 31, 2025, transitioning to a Senior Scientific Advisor role for the remainder of the year. Birgit Schultes, Ph.D., currently leading Immunology and Cell Therapy, will be promoted to Executive Vice President and Chief Scientific Officer, effective January 13, 2025.
