Intellia Therapeutics Reports Positive Phase 2 Results for NTLA-2002 Treatment for Hereditary Angioedema

Intellia Therapeutics, Inc. (NASDAQ:NTLA) recently disclosed encouraging results from the Phase 2 study of NTLA-2002, an investigational in vivo CRISPR gene editing therapy aimed at treating Hereditary Angioedema (HAE). The company shared this information in an 8-K filing with the Securities and Exchange Commission on October 24, 2024.

According to the report, the Phase 2 data stemming from the ongoing Phase 1/2 study of NTLA-2002 in patients suffering from HAE displayed promising outcomes, suggesting that NTLA-2002 has the potential to prevent HAE attacks following a single infusion. HAE is a rare genetic disorder characterized by severe swelling episodes that can be life-threatening.

The study, which enrolled a total of 27 participants, involved random allocation to receive either one of two single doses of NTLA-2002 (25 mg or 50 mg) or a placebo through intravenous infusion. Data analysis up to April 4, 2024, indicated a substantial reduction in attack rates with the administration of NTLA-2002. Patients who received the 50 mg dosage showed an 86% mean reduction in kallikrein protein levels from baseline compared to a 55% reduction in the 25 mg group at week 16.

Moreover, the therapy demonstrated good tolerability at both dose levels, with common adverse events reported as headache, fatigue, and nasopharyngitis. No serious adverse events were recorded, and all events were categorized as Grade 1 or 2, except for one patient in the placebo group who experienced a Grade 4 edema of the tongue with breathing complications linked to underlying HAE.

Based on the favorable outcomes from the Phase 2 study, Intellia has decided to evaluate the 50 mg dose in the pivotal Phase 3 HAELO trial, which is actively screening potential participants. The Phase 3 study aims to further assess the efficacy, safety, and impact of NTLA-2002 in patients with HAE.

While highlighting these advancements, Intellia Therapeutics cautioned that forward-looking statements within the report are subject to various risks and uncertainties. The company emphasized the importance of considering potential factors that could lead to deviations in actual results from those anticipated in the forward-looking statements.

For interested parties, a press release detailing the Phase 2 results of NTLA-2002 was included as Exhibit 99.1 to the 8-K filing for reference.

This comprehensive update underscores Intellia Therapeutics’ commitment to advancing innovative treatments and addressing significant unmet medical needs in the healthcare landscape.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Intellia Therapeutics’s 8K filing here.

Intellia Therapeutics Company Profile

Intellia Therapeutics, Inc, a genome editing company, focuses on the development of curative therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; NTLA-2002 for the treatment of hereditary angioedema; and NTLA-3001 for alpha-1 antitrypsin deficiency associated lung disease.