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Can Intellia's Pipeline Push Drive Long-Term Growth Amid Rivalry?
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Key Takeaways
Intellia advanced phase III studies for nex-z in ATTR amyloidosis after the FDA lifted clinical holds.
NTLA said the phase III HAELO study for lonvo-z met primary and key secondary endpoints.
Intellia plans a 2027 launch for lonvo-z, pending FDA approval after completing BLA submission.
Intellia Therapeutics (NTLA - Free Report) has been making decent progress with the advancement of its two pipeline candidates, nex-z (or, NTLA-2001) for transthyretin (ATTR) amyloidosis and lonvo-z (or NTLA-2002) for hereditary angioedema (HAE).
Intellia is developing nex-z in collaboration with Regeneron Pharmaceuticals (REGN - Free Report) . The candidate is being evaluated in two late-stage studies, MAGNITUDE and MAGNITUDE-2, for ATTR amyloidosis with cardiomyopathy (ATTR-CM) and ATTR amyloidosis with polyneuropathy (ATTRv-PN), respectively.
While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits. The company’s top line currently comprises only collaboration revenues from its partners, like Regeneron and others.
In March, the FDA lifted the clinical hold on the investigational new drug (IND) application for the phase III MAGNITUDE study evaluating nex-z in patients with ATTR-CM.
Earlier this year, the FDA lifted the clinical hold on the IND application for the phase III study, MAGNITUDE-2, evaluating nex-z in patients with ATTRv-PN. Enrollment in this study is expected to be completed in the second half of 2026.
Intellia is now focusing on completing patient enrollment in both late-stage studies as promptly as possible. The successful development of nex-z could provide impetus to the stock and create long-term growth visibility.
Meanwhile, last month, Intellia announced that the phase III HAELO study evaluating lonvo-z, an in vivo CRISPR gene editing therapy, for the treatment of HAE, has met its primary endpoint and all key secondary endpoints.
Simultaneously, NTLA initiated a rolling submission of a biologics license application (BLA) to the FDA seeking approval for lonvo-z for the treatment of HAE. The company expects to complete the BLA submission in the second half of 2026. Intellia plans to commercially launch lonvo-z in the first half of 2027, upon potential approval.
The successful development of its pipeline candidates will be a huge boost to Intellia. However, any regulatory or developmental setback related to ongoing studies will be a major setback. Growing competition in the target market also remains a worry.
NTLA's Competition in the Target Market
While Intellia’s pipeline of innovative CRISPR-based therapies appears promising, developing these candidates remains a complex and challenging process. Even if successfully developed and approved, the therapies are likely to face competition from other companies leveraging CRISPR/Cas9 gene-editing technology to target diseases across similar therapeutic areas.
CRISPR Therapeutics (CRSP - Free Report) is the first and only company in the world to market a CRISPR/Cas9-based therapy. CRSP’s one-shot gene therapy, Casgevy, was approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — sickle cell disease and transfusion-dependent beta-thalassemia.
CRSP has developed Casgevy in partnership with large biotech, Vertex Pharmaceuticals, which is responsible for the therapy’s global development and commercialization.
Beam Therapeutics (BEAM - Free Report) is developing its leading ex-vivo genome-editing candidate, risto-cel, in the phase I/II BEACON study for the treatment of patients with SCD. BEAM plans to submit a BLA for risto-cel by the end of 2026.
Beam Therapeutics is also expanding its genetic disease pipeline by developing BEAM-301 and BEAM-302 for the treatment of glycogen storage disease type 1a and alpha-1 antitrypsin deficiency, respectively.
NTLA's Price Performance, Valuation and Estimates
Year to date, shares of Intellia have rallied 41.1% against the industry’s decline of 2.2%. The stock has also outperformed the sector and the S&P 500 during the same time frame, as seen in the chart below.
Image Source: Zacks Investment Research
From a valuation standpoint, Intellia is trading at a discount to the industry. Going by the price/book ratio, the company’s shares currently trade at 2.46, lower than 3.13 for the industry. The stock is trading below its five-year mean of 2.58.
Image Source: Zacks Investment Research
The Zacks Consensus Estimate for Intellia’s 2026 loss per share has narrowed from $3.53 to $3.35 over the past 30 days. Loss per share estimates for 2027 have widened from $1.22 to $1.54 during the same time frame.
Image: Bigstock
Can Intellia's Pipeline Push Drive Long-Term Growth Amid Rivalry?
Key Takeaways
Intellia Therapeutics (NTLA - Free Report) has been making decent progress with the advancement of its two pipeline candidates, nex-z (or, NTLA-2001) for transthyretin (ATTR) amyloidosis and lonvo-z (or NTLA-2002) for hereditary angioedema (HAE).
Intellia is developing nex-z in collaboration with Regeneron Pharmaceuticals (REGN - Free Report) . The candidate is being evaluated in two late-stage studies, MAGNITUDE and MAGNITUDE-2, for ATTR amyloidosis with cardiomyopathy (ATTR-CM) and ATTR amyloidosis with polyneuropathy (ATTRv-PN), respectively.
While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits. The company’s top line currently comprises only collaboration revenues from its partners, like Regeneron and others.
In March, the FDA lifted the clinical hold on the investigational new drug (IND) application for the phase III MAGNITUDE study evaluating nex-z in patients with ATTR-CM.
Earlier this year, the FDA lifted the clinical hold on the IND application for the phase III study, MAGNITUDE-2, evaluating nex-z in patients with ATTRv-PN. Enrollment in this study is expected to be completed in the second half of 2026.
Intellia is now focusing on completing patient enrollment in both late-stage studies as promptly as possible. The successful development of nex-z could provide impetus to the stock and create long-term growth visibility.
Meanwhile, last month, Intellia announced that the phase III HAELO study evaluating lonvo-z, an in vivo CRISPR gene editing therapy, for the treatment of HAE, has met its primary endpoint and all key secondary endpoints.
Simultaneously, NTLA initiated a rolling submission of a biologics license application (BLA) to the FDA seeking approval for lonvo-z for the treatment of HAE. The company expects to complete the BLA submission in the second half of 2026. Intellia plans to commercially launch lonvo-z in the first half of 2027, upon potential approval.
The successful development of its pipeline candidates will be a huge boost to Intellia. However, any regulatory or developmental setback related to ongoing studies will be a major setback. Growing competition in the target market also remains a worry.
NTLA's Competition in the Target Market
While Intellia’s pipeline of innovative CRISPR-based therapies appears promising, developing these candidates remains a complex and challenging process. Even if successfully developed and approved, the therapies are likely to face competition from other companies leveraging CRISPR/Cas9 gene-editing technology to target diseases across similar therapeutic areas.
CRISPR Therapeutics (CRSP - Free Report) is the first and only company in the world to market a CRISPR/Cas9-based therapy. CRSP’s one-shot gene therapy, Casgevy, was approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — sickle cell disease and transfusion-dependent beta-thalassemia.
CRSP has developed Casgevy in partnership with large biotech, Vertex Pharmaceuticals, which is responsible for the therapy’s global development and commercialization.
Beam Therapeutics (BEAM - Free Report) is developing its leading ex-vivo genome-editing candidate, risto-cel, in the phase I/II BEACON study for the treatment of patients with SCD. BEAM plans to submit a BLA for risto-cel by the end of 2026.
Beam Therapeutics is also expanding its genetic disease pipeline by developing BEAM-301 and BEAM-302 for the treatment of glycogen storage disease type 1a and alpha-1 antitrypsin deficiency, respectively.
NTLA's Price Performance, Valuation and Estimates
Year to date, shares of Intellia have rallied 41.1% against the industry’s decline of 2.2%. The stock has also outperformed the sector and the S&P 500 during the same time frame, as seen in the chart below.
Image Source: Zacks Investment Research
From a valuation standpoint, Intellia is trading at a discount to the industry. Going by the price/book ratio, the company’s shares currently trade at 2.46, lower than 3.13 for the industry. The stock is trading below its five-year mean of 2.58.
Image Source: Zacks Investment Research
The Zacks Consensus Estimate for Intellia’s 2026 loss per share has narrowed from $3.53 to $3.35 over the past 30 days. Loss per share estimates for 2027 have widened from $1.22 to $1.54 during the same time frame.
Image Source: Zacks Investment Research
NTLA's Zacks Rank
Intellia currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.