Business
Intellia Therapeutics Announces Fourth Quarter and Full-Year 2024 Financial Results and Highlights Recent Company Progress
Dosed first patient in global Phase 3 HAELO study evaluating NTLA-2002 for hereditary angioedema (HAE)Expect to complete enrollment of the HAELO study in the
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Dosed first patient in global Phase 3 HAELO study evaluating NTLA-2002 for hereditary angioedema (HAE)Expect to complete enrollment of the HAELO study in the second half of 2025 and submit a Biologics License Application in the second half of 2026 to support plans for U.S. launch in 2027Enrollment in the pivotal Phase 3 MAGNITUDE trial of nexiguran ziclumeran (nex-z) in patients with transthyretin amyloidosis (ATTR) with cardiomyopathy continues to track ahead of projections; more than 550 total patients expected to be enrolled by year endActively screening for the Phase 3 MAGNITUDE-2 trial for nex-z in hereditary ATTR amyloidosis with polyneuropathy; on track to dose first patient in 1Q25Ended 2024 with approximately $862 million in cash, cash equivalents and marketable securities CAMBRIDGE, Mass., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2024. “We are off to an excellent start in 2025 with renewed focus and strong operational execution across our three, pivotal Phase 3 studies,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are excited by the clinical data presented during the fourth quarter. Our Phase 1/2 results in HAE suggest that NTLA-2002 could represent a functional cure for patients with HAE – for the first time a patient has the potential to be both free from attacks and free from chronic therapy. Similarly, the rapid, deep and durable reductions in serum TTR demonstrated to date in our Phase 1 study of nex-z in ATTR represent a highly differentiated profile that may offer patients an opportunity to stabilize or improve their clinical readouts in an otherwise unrelenting, progressive disease.” Fourth Quarter 2024 and Recent Operational Highlights Hereditary Angioedema (HAE) NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose. In January, Intellia announced the first patient was dosed with NTLA-2002 in the global Phase 3 HAELO study. The Company expects to complete enrollment in the secon...