NEW YORK – Intellia Therapeutics on Monday said it has started the Phase III HAELO trial testing the activity of its in vivo CRISPR-based gene-editing therapy NTLA-2002 in patients with hereditary angioedema (HAE).
Patients with HAE, a rare genetic disease, experience sudden, painful, and sometimes life-threatening inflammatory attacks in different organs and tissues. NTLA-2002 is designed to prevent these HAE attacks by inactivating the KLKB1 gene, which encodes the kallikrein precursor protein prekallikrein and plays a critical role in the body's inflammatory responses.
The Cambridge, Massachusetts-based firm reported in August that a Phase II study of NTLA-2002 had met its primary and secondary efficacy endpoints, and after a successful end-of-Phase II meeting with the US Food and Drug Administration, it had confidence to move the program into Phase III.
Intellia has also reported positive readouts from the Phase I portion of the NTLA-2002 development program. "Data from the ongoing Phase I/II study showed great promise that a single-dose treatment can lead to a complete response — no more attacks and no further treatment required," Intellia President and CEO John Leonard said in a statement. The company will share detailed data from these earlier studies at an upcoming medical conference.
In the global, randomized, placebo-controlled HAELO study that Intellia has now launched, the firm is evaluating the efficacy and safety of NTLA-2002 in 60 adult patients with type I or type II HAE. Patients will be randomized to receive a single infusion of NTLA-2002 or placebo. Patients in the placebo arm will have the option to cross over to the treatment arm at week 28. The company is primarily interested in comparing the number of HAE attacks between the two arms from week five through week 28.
NTLA-2002 is Intellia's second in vivo gene-editing therapeutic candidate to enter late-stage clinical trials. In March, the firm dosed the first patient with transthyretin amyloidosis-associated cardiomyopathy with NTLA-2001, an investigational in vivo CRISPR gene-editing therapy, within a pivotal Phase III trial.