Reporting the Intellia Therapeutics, Inc. operational highlights and financial results for the first quarter ended 31 March 2021, President and CEO John Leonard, M.D., says:
“Intellia continues to build momentum as we progress our full-spectrum pipeline towards key upcoming milestones this year. We plan on submitting first-in-human regulatory filings for NTLA-2002 for HAE.”
NTLA-2002 aims to prevent attacks for people living with HAE after treatment consisting of a single administration. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.
Intellia expects to submit a regulatory application for NTLA-2002 in the second half of 2021. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE.
In March, Intellia presented preclinical results confirming greater reductions in serum kallikrein protein levels and activity versus the current standard of care for HAE, sustained over 17 months following a single dose in an ongoing non-human primate study of its cyno-specific LNP formulation for NTLA-2002. Additionally, the Intellia presented data from a humanized KLKB1 mouse model of bradykinin-mediated vascular permeability, establishing that a single administration of NTLA-2002 prevented captopril-induced vascular leakage. These results, which affirm NTLA-2002’s therapeutic hypothesis of preventing HAE attacks, were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Annual Meeting.