Intellia Therapeutics announces fourth quarter and full-year 2023 financial results and highlights recent company progress
Intellia Therapeutics today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2023.
Intellia President and Chief Executive Officer, John Leonard MD, said: “We’re off to a very strong start in 2024 as we execute against our strategic priorities to realize the full potential of CRISPR-based gene editing. We also remain on track to begin the Phase 3 trial for our second in-vivo CRISPR-based therapy, NTLA-2002 for hereditary angioedema, later in the year.”
Recent Operational Highlights
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.
Intellia plans to initiate the global pivotal Phase 3 study, including US patients, in the second half of 2024, subject to regulatory feedback.
As previously announced in January, Intellia completed enrollment and dosing in the Phase 2 portion of the Phase 1/2 study in adults with HAE. The Company plans to present updated data from the Phase 1 and new data from the Phase 2 portion in 2024.
In January, the Company announced that positive interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published in the New England Journal of Medicine (NEJM). The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. NTLA-2002 was well tolerated at all dose levels. The most frequent adverse events reported were mild, transient infusion-related reactions and fatigue.
During the fourth quarter of 2023, Intellia received Priority Medicines (PRIME) designation from the European Medicines Agency and orphan drug designation from the European Commission for NTLA-2002.
(Source: Intellia)
