Intellia Therapeutics, Inc.‘s Clinical Trial Application (CTA) has been authorized by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with HAE. NTLA-2002 is a systemically administered single-dose CRISPR/Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to permanently reduce plasma kallikrein activity and thus prevent HAE attacks.

“We look forward to initiating this year our first-in-human study of NTLA-2002 for people living with HAE”, says Intellia President and CEO John Leonard, M.D.: “We believe NTLA-2002 has the potential to be a curative therapy for patients with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and eliminating the significant treatment burden associated with currently available HAE therapies. This study of NTLA-2002 leverages early insights from our ATTR amyloidosis program, where we established proof-of-concept for our modular in vivogenome editing platform with interim Phase 1 data earlier this year. The NTLA-2002 program represents the second systemic in vivo CRISPR genome editing therapy candidate to enter human clinical trials.”

The Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of kallikrein protein levels and 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 up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies.

Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development approach for NTLA-2002.
(Source: Intellia)