Intellia Therapeutics Presents New Interim Data from First-in-Human Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting
- Robust reductions in plasma kallikrein levels and HAE attack rates observed at all doses tested
- All patients treated in the 25 mg and 75 mg cohorts have an ongoing attack-free interval through latest
- First three patients treated have an ongoing attack-free interval of 5.5 – 10.6 months after a single dose of NTLA-2002
- NTLA-2002 was generally well-tolerated at all dose levels
- Intellia to host investor event to discuss updated data from Phase 1/2 study of NTLA-2002, its second systemically administered in vivo CRISPR candidate, on
Monday, November 14, at 8:00 a.m. ET
The data presented are from 10 adult patients with
Plasma Kallikrein Reduction
|Cohort||Mean plasma kallikrein reduction
at latest follow-up
|25 mg (n=3)||64% (week 32)|
|50 mg (n=4)||81% (day 22)|
|75 mg (n=3)||92% (week 16)|
HAE Attack Rate Reduction
HAE attack rates are measured in the dose-escalation portion of the study, with the first analysis occurring at the end of the pre-specified 16-week primary observation period. To date, all patients in the 25 mg and 75 mg dose cohorts have reached the end of this initial observation period in ongoing follow-up as described below. Patients in the 50 mg cohort have not completed the primary 16-week observation period.
|Cohort||Baseline attack rate in screening period||Mean HAE attack rate reduction - week 1 to 16||Mean HAE attack rate reduction - week 5 to 16||Duration of ongoing attack-free interval|
|25 mg (n=3)||1.1 to 7.2 attacks / month||91%||89%||5.5 – 10.6 months|
|75 mg (n=3)||4.0 to 5.9 attacks / month||78%||89%||2.3 – 4.2 months|
“We see early evidence that our one-time CRISPR-based investigational therapy may offer patients suffering from hereditary angioedema a functional cure for their disease,” said Intellia President and Chief Executive Officer
At all three dose levels, NTLA-2002 was generally well-tolerated, and the majority of adverse events were mild in severity. The most frequent adverse events were infusion-related reactions, which were mostly Grade 1 and resolved within one day. There have been no dose-limiting toxicities, no serious adverse events and no adverse events of Grade 3 or higher observed to date. No clinically significant laboratory abnormalities were observed.
Intellia expects to select up to two doses to further evaluate NTLA-2002 in the Phase 2, placebo-controlled, dose-expansion portion of the study, which is expected to begin in the first half of 2023. Intellia anticipates expanding country and site participation, including
Intellia Therapeutics Investor Event and Webcast Information
Intellia will host a live webcast,
About the NTLA-2002 Clinical Program
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 hereditary angioedema (HAE). This includes the measurement of plasma 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.
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.
This press release contains “forward-looking statements” of
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to the successful enrollment of patients in the Phase 1/2 study for NTLA-2002 for the treatment of HAE; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to the authorization, initiation and conduct of studies and other development requirements, including manufacturing, for its in vivo and ex vivo product candidates, including NTLA-2002; the risk that any one or more of Intellia’s product candidates, including NTLA-2002, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies, including for NTLA-2002, will not be predictive of future results in connection with future studies; and the risk that Intellia will not be able to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies to develop additional product candidates, including to apply its proprietary CRISPR/Cas9 technology platform successfully to additional product candidates to treat other genetic diseases. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q filed with the
Senior Vice President, Investor Relations and Corporate Communications
Senior Director, Investor Relations and Corporate Communications
Source: Intellia Therapeutics, Inc.