Intellia sees early evidence that new therapy may offer functional cure
At the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting, Intellia Therapeutics, Inc. announces updated data from an ongoing Phase 1/2 clinical study of NTLA-2002 for the treatment of HAE. The data are from 10 adult patients with HAE in the Phase 1, dose-escalation portion of the study, with a data cut-off date of 28 September 2022.
“We see early evidence that our one-time CRISPR-based investigational therapy may offer patients suffering from HAE a functional cure for their disease,” says Intellia President and CEO John Leonard, M.D. “Based on the extended data across multiple dose cohorts, we are strongly encouraged that all patients who received a single dose of NTLA-2002 subsequently became attack-free. In the patients with the longest follow-up to date, their attack-free interval has been maintained five to ten months from their last attack. Importantly, the safety data from all patients are highly encouraging, further supporting NTLA-2002’s potential to change the future HAE treatment paradigm. As the second clinical program from our in vivo pipeline to demonstrate deep and consistent protein reduction following a one-time administration, the latest interim data further reinforce the enormous potential of our modular CRISPR genome editing platform to treat a host of genetic diseases.”
At all 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 U.S. clinical sites, as part of the Phase 2 study.