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Intellia Therapeutics NTLA announced that it has dosed the first patient in the phase III HAELO study evaluating NTLA-2002, its novel in vivo CRISPR gene editing therapy for the treatment of hereditary angioedema (HAE).
Shares of the company were up 10.8% on Jan. 22 following the announcement of the news.
The global double-blind, placebo-controlled HAELO study will evaluate the safety and efficacy of NTLA-2002 in adults with Type I or Type II HAE. The key endpoint of the study is to see the change in the number of HAE attacks from week five through week 28 of treatment.
Enrollment of patients in the study is expected to be completed in the second half of 2025. Intellia plans to submit a biologics license application for NTLA-2002 in HAE in 2026. Upon potential approval, the company plans to launch NTLA-2002 in 2027 in the United States.
In October 2024, NTLA initiated the HAELO study on NTLA-2002 for treating HAE.
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.
In the past year, shares of Intellia have plunged 56.1% compared with the industry’s decline of 12%.
Image Source: Zacks Investment Research
NTLA Developing Another Candidate NTLA-2001
Besides NTLA-2002, Intellia is developing its investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001). The company has collaborated with Regeneron Pharmaceuticals REGN for the development of nex-z.
Nex-z is part of the company’s co-development and co-promotion agreement with Regeneron. While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits.
Earlier this month, the company announced a strategic reorganization of its portfolio wherein it prioritized the development of nex-z, which is being studied for two indications, ATTR amyloidosis with polyneuropathy (ATTRv-PN) and ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The phase III MAGNITUDE study is evaluating the safety and efficacy of nex-z in patients with ATTR amyloidosis with cardiomyopathy. Enrollment is currently ongoing in the study.
The phase III MAGNITUDE 2 study is actively screening patients with hereditary ATTR amyloidosis with polyneuropathy, with the first patient expected to be dosed later in the first quarter of 2025.
NTLA's Zacks Rank & Stocks to Consider
Intellia currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Voyager Therapeutics, Inc. VYGR and Castle Biosciences, Inc. CSTL, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.