Intellia Therapeutics, Inc. announced its strategic priorities and key anticipated 2025 milestones that support the company’s mission to transform the lives of patients and bring forth a new era in medicine.
“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,” said John Leonard, M.D., President and Chief Executive Officer of Intellia. “We understand the significant potential of our late-stage programs, and within a challenging market environment, have made a difficult decision to focus our resources predominantly on NTLA-2002 and nex-z where we have the greatest opportunity to create significant, near-term value.”
Recent Pipeline Advancement and Corporate Updates
- Strategic Reorganization Focused on Key Value Drivers:
- The pipeline prioritization is intended to focus resources on high value programs - NTLA-2002 and nex-z - to ensure efficient execution, achieve near-term clinical milestones, and prepare Intellia for commercial launch. As part of this prioritization, the Company discontinued development of NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency-associated lung disease and select research-stage programs. Over the course of 2025, the strategic reorganization will result in a net workforce reduction of approximately 27%.
- The Company expects to incur charges of approximately $8 million associated with the reorganization, which are anticipated to be incurred in the first quarter of 2025.
- Intellia ended the fourth quarter of 2024 with approximately $862 million in cash, cash equivalents and investments. The combination of its cash balance and the anticipated cost savings are expected to provide the Company with cash runway into the first half of 2027.
- Laura Sepp-Lorenzino, Ph.D., Intellia’s Chief Scientific Officer, has announced her retirement effective December 31, 2025 after more than 30 years of service in the biopharmaceutical industry and academia. Dr. Sepp-Lorenzino will transition from her role as CSO and continue as a Senior Scientific Advisor for the remainder of the year. “I would like to thank Laura for her service to Intellia. She has been a tireless innovator and advocate for the promise of gene editing, and we look forward to continuing the work and vision she championed,” said John Leonard M.D., President and Chief Executive Officer of Intellia.
- The Company also announced that Birgit Schultes, Ph.D., who has been leading Immunology and Cell Therapy since 2017, will be promoted to Executive Vice President and Chief Scientific Officer, effective January 13, 2025. Dr. Schultes has over 20 years of experience in drug development and biotechnology, including the clinical development of cell therapies and complex biologic products. Dr. Schultes received her M.S. in biology and Ph.D. in immunology from the University of Bonn, Germany, and completed the Advanced Management Development Program at the Boston University Questrom School of Business.
- NTLA-2002 for Hereditary Angioedema (HAE):
- Patients are actively enrolling in the pivotal Phase 3 HAELO study.
- Presented clinical data, in October at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting, from the Phase 2 study that demonstrated the potential of NTLA-2002 to end chronic, prophylaxis treatment and provide freedom from attacks following a one-time infusion.
- Nex-z for Transthyretin (ATTR) Amyloidosis:
- Strong enrollment and momentum continue in the Phase 3 MAGNITUDE study in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), tracking ahead of the Company’s target enrollment projections.
- Patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) are actively screening in the Phase 3 MAGNITUDE-2 study.
- Presented first clinical evidence, in November at the 2024 American Heart Association (AHA) Scientific Sessions, that consistent, rapid, deep and durable reductions in serum TTR achieved after a one-time treatment of nex-z may halt, and potentially reverse, disease progression.
Strategic Priorities and Anticipated 2025 Milestones
Intellia’s strategic priorities reflect the Company’s ongoing evolution from a late-stage development company to a commercial-ready organization by the end of 2026.
1. Drive focused clinical execution to complete or accelerate enrollment in the pivotal studies of NTLA-2002 and nex-z
- NTLA-2002 for HAE:Dose the first patient in the pivotal Phase 3 HAELO trial in 1Q25.
- Complete HAELO enrollment in second half of 2025.
- Present longer-term data from the Phase 1/2 study – data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study
- Nex-z for ATTR amyloidosis:
- Dose the first patient in the pivotal Phase 3 MAGNITUDE-2 trial for ATTRv-PN in 1Q25.
- Enroll at least 550 patients cumulatively within the MAGNITUDE trial for ATTR-CM.
- Present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study – data will include updated measures of clinical efficacy and safety.
2. Advance commercial readiness by implementing core commercialization and medical capabilities for initial launch in the U.S.
- Complete buildout of the commercial leadership team by second half of 2025.
- Expand the reach of medical education activities in HAE and ATTR amyloidosis in partnership with key medical societies and patient organizations.
- Initiate pre-approval information exchange to allow payers to begin planning for coverage and formulary decisions.
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