Intellia Therapeutics, Inc. NTLA announced that it has initiated the pivotal phase III HAELO study evaluating NTLA-2002 for the treatment of hereditary angioedema (HAE).

The global double-blind, placebo-controlled study will evaluate the safety and efficacy of NTLA-2002 in adults with Type I or Type II HAE. The primary endpoint of the study is to see the change in number of HAE attacks from week five through week 28 of treatment.

Patient screening is currently ongoing in the study.

Year to date, shares of Intellia have plunged 41.5% compared with the industry’s decrease of 2.8%.

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More on NTLA's Gene-Editing Candidate NTLA-2002

NTLA-2002 is Intellia’s investigational single-dose CRISPR/Cas9 gene-editing therapy, which is being developed as a one-time cure for HAE. It is a rare genetic condition marked by severe inflammatory attacks in various body organs and tissues, which can be painful and even life-threatening.

Despite the current treatment options that require chronic administration for disease control, HAE attacks still occur. NTLA-2002 aims to prevent HAE attacks by suppressing the plasma kallikrein activity.

Previously, data from the ongoing phase I/II study showed that treatment with a single-dose of NTLA-2002 can lead to a complete response – no more attacks as well as no further treatment required.

In June, the company announced encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for treating HAE.

Studies on NTLA's Another Candidate NTLA-2001 Progressing Well

Intellia is collaborating with Regeneron Pharmaceuticals REGN for the development of its investigational in vivo genome-editing candidate, NTLA-2001.

NTLA-2001 is part of the company’s co-development and co-promotion agreement with Regeneron. While NTLA is the lead party in the deal for NTLA-2001, REGN shares 25% of the development costs and commercial profits.

NTLA-2001 is being studied for two indications, transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) and ATTR amyloidosis with cardiomyopathy (ATTR-CM).

The ongoing phase III MAGNITUDE study is evaluating NTLA-2001 to treat ATTR amyloidosis with cardiomyopathy. If the data from the MAGNITUDE study is found to be positive, it will enable global regulatory filings for NTLA-2001.

Intellia also plans to initiate a pivotal phase III study evaluating NTLA-2001 as a single-dose treatment in ATTRv-PN by 2024-end.

NTLA's Zacks Rank & Stocks to Consider

Intellia currently carries a Zacks Rank #3 (Hold).