The U.S. Food and Drug Administration (FDA) has cleared Intellia Therapeutics, Inc.’s Investigational New Drug (IND) application for NTLA-2002 for the treatment of HAE, enabling the company to include the United States in the global Phase 2 portion of its ongoing Phase 1/2 study. NTLA-2002 is an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1), to permanently reduce plasma kallikrein protein activity and thus prevent HAE attacks after a single-dose treatment.
“The FDA’s acceptance of our IND application to initiate clinical evaluation of NTLA-2002 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with HAE,” says Intellia President and CEO John Leonard, M.D. “The NTLA-2002 IND clearance marks an important milestone for Intellia as we continue our track record of execution as the leader in the genome editing field. We are thrilled to advance the development of NTLA-2002 in the U.S. and are working to rapidly enroll patients in the Phase 2 portion of the study. We look forward to presenting additional data from the first-in-human, Phase 1 portion of the study later this year.”
Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. In 2022, Intellia reported positive interim results from the Phase 1 study demonstrating deep, dose-dependent reductions in plasma kallikrein and robust reductions in patient HAE attacks. The Phase 2 portion of the study has recently initiated patient screening outside of the U.S.