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Intellia Therapeutics NTLA announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001), for a new indication.
The regulatory body has now granted the RMAT designation to nex-z for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
The RMAT designation provides the candidate with increased opportunities to meet FDA officials, as well as arrange early meetings to discuss potential surrogate or intermediate endpoints. It opens up early interactions between the FDA and sponsors to facilitate accelerated approval and potential priority review of a product’s biologics license application.
The FDA previously granted the RMAT designation to nex-z for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) in November 2024.
According to management, treatment with a single dose of nex-z demonstrated unprecedented, rapid, durable, and consistent reductions in serum TTR, which positively impacted markers of disease progression in both polyneuropathy and cardiomyopathy.
Shares of Intellia have plunged 28.7% year to date against the industry’s increase of 4.3%.
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NTLA's Ongoing Nex-Z Developments
Intellia 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.
Two separate phase III studies are evaluating nex-z for treating ATTR-CM and ATTRv-PN.
The phase III MAGNITUDE study is evaluating the safety and efficacy of nex-z in patients with ATTR-CM. Enrollment is currently ongoing in the study.
The phase III MAGNITUDE 2 study is actively screening patients with ATTRv-PN, with the first patient expected to be dosed shortly. The FDA cleared the investigational new drug application seeking to initiate the pivotal phase III MAGNITUDE-2 study evaluating nex-z for treating ATTRv-PN in November 2024.
NTLA's Zacks Rank & Stocks to Consider
Intellia currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Jazz Pharmaceuticals, Inc. JAZZ and Krystal Biotech, Inc. KRYS, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Jazz Pharmaceuticals’ earnings per share have increased from $22.06 to $23.33 for 2025. During the same time, earnings per share estimates for 2026 have increased from $23.13 to $23.35. Year to date, shares of JAZZ have risen 6.3%.