LL schreef op 4 november 2022 18:56:
Intellia Therapeutics Q3 HAE highlightsPresented interim data from the ongoing Phase 1/2 study of NTLA-2002, demonstrating that a single dose of NTLA-2002 resulted in significant, dose-dependent and durable reductions in plasma kallikrein and a 91% reduction in attack rate in the 25 mg cohort through week 16; additional interim data to be presented at ACAAI on November 12.
Expects to initiate the Phase 2 portion of NTLA-2002 study for the treatment of hereditary angioedema (HAE) in 1H 2023
Hereditary Angioedema (HAE)
NTLA-2002: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous reduction of plasma kallikrein activity, following a single dose, and to eliminate the significant treatment burden associated with currently available HAE therapies. NTLA-2002 is being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE.
Intellia announced in September positive interim results from an ongoing Phase 1/2 clinical study of NTLA-2002, its second in vivo genome editing candidate, at the 2022 Bradykinin Symposium held in Berlin, Germany. Administration of single doses of NTLA-2002 led to dose-dependent reductions in plasma kallikrein, with mean reductions of 65% and 92% in the 25 mg (n=3) and 75 mg (n=3) dose cohorts, respectively, by week eight. In addition to plasma kallikrein levels, HAE attack rates are also being measured in the study, with the first analysis occurring at the end of the pre-specified 16-week primary observation period. A single dose of 25 mg of NTLA-2002 resulted in a 91% mean reduction in HAE attacks through the 16-week observation period. Additionally, two of the three patients have not had a single HAE attack since treatment, and all three patients have been attack-free since week 10 (based on follow-up through weeks 24 – 32). Patients in the 75 mg cohort had not completed the primary 16-week observation period. At both dose levels, NTLA-2002 was generally well-tolerated, and the majority of adverse events were mild in severity.
Intellia will be presenting additional data at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place November 10 – 14 in Louisville, Kentucky. The new presentation is expected to include interim safety and kallikrein reduction data from the 50 mg dose cohort, and additional safety, kallikrein reduction and attack rate data from the 25 mg and 75 mg dose cohorts from the dose-escalation portion of the study.
Intellia expects to select up to two doses to further evaluate in the Phase 2, placebo-controlled, dose-expansion portion of the study, slated to begin in the first half of 2023. Intellia anticipates expanding country and site participation, including U.S. clinical sites, as part of the Phase 2 study.
bron:
ir.intelliatx.com/news-releases/news-...