Progress in Phase 3 Studies
Intellia dosed the first patient in their Phase 3 study for hereditary angioedema (HAE) and for hereditary ATTR with polyneuropathy. Enrollment in the global Phase 3 HAELO study for HAE is progressing ahead of schedule, and the Phase 3 MAGNITUDE study for ATTR with cardiomyopathy is also ahead of schedule with over 90 sites actively enrolling.
Regulatory Milestones
The FDA granted Intellia the RMAT designation for nexiguran ziclumeran (nex-z) for the treatment of ATTR with cardiomyopathy, adding to prior RMAT designations for ATTR with polyneuropathy and NTLA-2002 in HAE.
Strong Financial Position
Intellia maintains a solid balance sheet with cash, cash equivalents, and marketable securities totaling approximately $707.1 million as of March 31, 2025. The company expects a year-over-year decline in GAAP operating expenses of between 5% and 10% and has sufficient funds to operate into the first half of 2027.
Positive Clinical Data
Upcoming data presentations include two-year follow-up data from NTLA-2002 at the European Academy of Allergy and Clinical Immunology Congress and longer-term data from the Phase 1 study for ATTR amyloidosis, extending the durability window out to at least three years.