Intellia (NTLA) Banks on Genome Editing Pipeline Amid Rivalry

Intellia Therapeutics, Inc. NTLA is developing its lead in vivo genome-editing candidate, NTLA-2001, for the treatment of transthyretin (ATTR) amyloidosis. The company is collaborating with Regeneron Pharmaceuticals REGN for the development of NTLA-2001.

NTLA-2001 is being studied for two indications: ATTR amyloidosis with polyneuropathy (ATTRv-PN) patients and ATTR amyloidosis with cardiomyopathy (ATTR-CM) patients. The phase III MAGNITUDE study evaluating NTLA-2001 to treat ATTR amyloidosis with cardiomyopathy is currently ongoing.

If the data from the MAGNITUDE study is found to be positive, it will enable global regulatory filings for NTLA-2001.

Intellia has received the FDA alignment for a pivotal phase III study design, which will support a biologics license application (BLA) filing for NTLA-2001 as a single-dose treatment in ATTRv-PN pending review of its investigational new drug application. The phase III study is likely to begin by 2024-end.

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.

The collaboration with Regeneron has given a boost to the company to develop its pipeline.

Shares of Intellia have declined 14.8% so far this year compared with the industry’s decrease of 5.1%.

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Intellia’s clinical-stage pipeline comprises another gene-editing candidate, NTLA-2002, which is currently being developed for treating hereditary angioedema (HAE). Earlier this month, the company reported encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for the given indication.

Per management, the long-term data has bolstered the candidate's potential as a groundbreaking treatment for HAE as patients experienced durable elimination of their HAE attacks in the ongoing phase I/II study.

The company has completed enrollment in the phase II portion of its study. The company plans to begin a pivotal phase III study on NTLA-2002 in the HAE indication in the second half of 2024.

This apart, NTLA submitted a clinical trial application to initiate a first-in-human phase I study of NTLA-3001 in December 2023. The company plans to begin patient dosing later in 2024.

Though Intellia is making good progress with its encouraging pipeline development, failure in ongoing studies will be a huge setback for the company.

Meanwhile, several companies are also using the CRISPR/Cas9 gene editing technology to address various diseases in specific areas. Companies like Editas Medicine, CRISPR Therapeutics and Precision BioSciences are developing their respective in-vivo and ex-vivo therapies. As a result, competition in the targeted space remains an overhang for Intellia.

Intellia Therapeutics, Inc. Price and Consensus

Intellia Therapeutics, Inc. Price and Consensus

Intellia Therapeutics, Inc. price-consensus-chart | Intellia Therapeutics, Inc. Quote

Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the healthcare sector are Acrivon Therapeutics, Inc. ACRV and Aligos Therapeutics, Inc. ALGS, 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 Acrivon Therapeutics’ 2024 loss per share have narrowed from $3.42 to $2.47. Loss per share estimates for 2025 have narrowed from $3.36 to $2.55. Year to date, shares of ACRV have surged 56.9%.

ACRV’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining one occasion, the average surprise being 3.56%.

In the past 60 days, estimates for Aligos Therapeutics’ 2024 loss per share have narrowed from 84 cents to 73 cents, while loss per share estimates for 2025 have narrowed from 82 cents to 71 cents. Year to date, shares of ALGS have declined 23.5%.

ALGS’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, the average surprise being 7.83%.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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