On Thursday, Intellia Therapeutics, Inc. (NASDAQ:NTLA) released Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE).

NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for HAE, a rare genetic condition that results in recurrent attacks of severe swelling.

The data cut-off date for the analysis was April 4, when the 25th patient completed the 16-week primary observation period.

A single dose of NTLA-2002 showed mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1–16 and by 80% and 81% during weeks 5–16, respectively.

In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months), and no subsequent treatment has been required.

Four of the ten patients in the 25 mg arm experienced a complete response, and zero patients in the placebo arm.

Patients who received the 50 mg dose achieved a greater kallikrein protein reduction, with an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16.

At both dose levels, NTLA-2002 was well tolerated.

Based on these results, Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, actively screening patients.

The Phase 3 study is expected to conclude by September 2027.

When in June, Intellia Therapeutics released data from the Phase 1 portion of the ongoing Phase 1/2 study, a single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients.

The trial, which administered a single dose of NTLA-2002 to 10 patients, tracked a 90% reduction in attacks from weeks 1 to 16, which increased to 92% for weeks 5-16.

Price Action: NTLA stock is down 18.3% at $16.28 at last check Thursday.

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