Back to top

Image: Bigstock

NTLA Stock Down as Patient Faces Adverse Event in Gene Therapy Study

Read MoreHide Full Article

Shares of Intellia Therapeutics (NTLA - Free Report) were down 22.9% yesterday after it announced an update from the ongoing phase III study, evaluating the investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z), for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM).

Per the company, one of the patients in the phase III MAGNITUDE study, which evaluated nex-z for treating ATTR-CM, experienced grade 4 liver transaminase elevations, indicating a notable increase in liver enzymes. The patient was asymptomatic and identified during laboratory tests. The company also noted that this adverse event appeared to resolve without hospitalization or medical interference.

However, investors seem concerned about the safety of NTLA’s gene therapy candidate in the long run, which might have resulted in the stock declining on Thursday.

The phase III MAGNITUDE study is progressing and enrollment in the same is expected to be completed by 2027.

Year to date, shares of Intellia have plunged 36.1% compared with the industry’s decline of 5.4%.

Zacks Investment Research
Image Source: Zacks Investment Research

NTLA's Other Study on Nex-z & Pipeline Updates

Intellia has been developing nex-z for ATTR amyloidosis with polyneuropathy (ATTRv-PN).

The company dosed the first patient in the phase III MAGNITUDE 2 study evaluating nex-z for the treatment of patients with ATTRv-PN in April. Enrollment in this study is expected to be completed in 2026.

Upon successful completion of the MAGNITUDE 2 study, Intellia plans to submit a potential biologics license application for nex-z in ATTRv-PN by 2028.

Intellia has collaborated with Regeneron Pharmaceuticals (REGN - Free Report) 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.

Intellia has been developing another pipeline candidate, NTLA-2002, for the treatment of hereditary angioedema (“HAE”). The company dosed the first patient in the pivotal phase III HAELO study evaluating NTLA-2002 for treating HAE in January 2025. Enrollment in the same is likely to be completed in the third quarter of 2025.

The company plans to submit a potential biologics license application for NTLA-2002 in HAE in the second half of 2026.

While Intellia’s pipeline of innovative CRISPR-based therapies looks promising, the development of these remains a complex affair. Also, the recent adverse event observed in the MAGNITUDE study raises a significant concern regarding the safety of nex-z in the long run.

NTLA's Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the biotech sector are Lexicon Pharmaceuticals (LXRX - Free Report) and Chemomab Therapeutics (CMMB - Free Report) , 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 Lexicon’s loss per share have narrowed from 37 cents to 32 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 35 cents to 31 cents. Year to date, shares of LXRX have lost 10.7%.

LXRX’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, delivering an average surprise of 11.97%.

In the past 60 days, estimates for Chemomab Therapeutics’ loss per share have narrowed from 70 cents to 60 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 80 cents to 70 cents. Year to date, shares of CMMB have declined 24.8%.

CMMB’s earnings beat estimates in two of the trailing four quarters, matched once and missed the same on the remaining occasion, delivering an average surprise of 5.00%.

Published in