Benzinga | Dec 13, 2021 07:35AM EST

Intellia Therapeutics Announces First Patient Dosed In Phase 1/2 Clinical Trial Of NTLA-2002 For The Treatment Of Hereditary Angioedema

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced that the first patient has been dosed with NTLA-2002, the company's in vivo CRISPR/Cas9 genome editing candidate being developed as a single-dose therapy to prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a systemically administered therapy designed to inactivate the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity and thus prevent HAE attacks.



"HAE is a genetic disorder that can cause painful and life-threatening inflammatory attacks, and currently available chronic therapies have a high treatment burden," said Intellia President and Chief Executive Officer John Leonard, M.D. "With the progress of our first-in-human clinical study evaluating NTLA-2002 for people living with HAE, we look forward to beginning clinical testing as we aim to develop a single-dose treatment for these patients."

About the NTLA-2002 Clinical Program

Intellia's multi-national Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (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. Visit clinicaltrials.gov (NCT05120830) for more details.

About NTLA-2002

Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia's second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. Intellia's proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which together carry out the precision editing.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.