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Intellia Crashes 60% in a Year: How Should You Play the Stock?

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Intellia Therapeutics, Inc. (NTLA - Free Report) has put up a dismal performance in 2024. Shares of the company have plunged 60.4% compared with the industry’s decline of 13.7%. The deterioration was more pronounced in the past five months. The stock has also underperformed the sector and the S&P 500 Index during this time frame.

Intellia is a leading clinical-stage gene editing company focused on developing innovative CRISPR-based therapies. While these innovative therapies have been in the spotlight following the FDA approval of a couple of these in late 2023, investors are disappointed by the recently released data from NTLA’s in vivo CRISPR-based therapies.

NTLA Underperforms Industry, Sector and S&P 500

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NTLA’s Promising Pipeline of CRISPR-based Therapies

Intellia’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body while its ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases.

The company has a promising pipeline with two of these therapies in late stage. Nexiguran ziclumeran (nex-z, also known as NTLA-2001) is an investigational in vivo CRISPR-based therapy designed to inactivate the transthyretin (“TTR”) gene in liver cells, thereby preventing the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis, a rare, progressive and fatal disease. Intellia has a collaboration agreement with Regeneron Pharmaceuticals (REGN - Free Report) for the development and commercialization of nex-z.

The FDA recently granted Regenerative Medicine Advanced Therapy designation to nex-z for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).

Intellia is also developing its wholly-owned NTLA-2002, an investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver and enable lifelong control of hereditary angioedema (HAE) attacks after just a single dose.

NTLA’s Recent Data Disappoints Investors

Data from NTLA’s ongoing phase I study of nex-z in patients with ATTR amyloidosis showed that a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction across all patients (n=36), regardless of baseline levels, through the latest follow-up.

The phase I study is evaluating the safety and activity of nex-z in patients with either ATTR amyloidosis with cardiomyopathy (ATTR-CM) or ATTRv-PN.  At month 12, the mean serum TTR reduction was 90% and the mean absolute residual serum TTR concentration was 17 µg/mL. Out of the total, 11 patients (who reached 24 months of follow-up) continued to show a sustained response with no evidence of a waning effect over time. The data support the hypothesis that greater TTR reduction may lead to a greater clinical benefit in ATTR amyloidosis.

The observed data also depicted favorable trends across multiple markers of cardiac disease progression at month 12 compared to baseline in an ATTR-CM population with a high proportion of advanced heart failure patients.

Data from the ATTRv-PN arm also showed stability or improvement of neuropathy as measured by multiple clinical measures of disease progression compared to baseline.

However, it seems investors weren’t impressed by the safety data from the study leading to a selloff.

In October 2024, NTLA announced positive data from the ongoing phase I/II study of NTLA-2002 in HAE patients. Single dose of NTLA-2002 led to deep attack rate reductions during the primary observation period. The mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1-16, and by 80% and 81% during weeks 5-16, respectively.

These data, however, didn’t seem to impress investors and the stock was hit hard following the announcement.

NTLA Strong Cash Balance: A Positive

NTLA has a strong cash balance, which should enable it to fund its ongoing studies. The company ended the third quarter with cash, cash equivalents and marketable securities of $944.7 million. The cash position is expected to fund operations into late 2026.

Intellia’s collaboration with biotech giant Regeneron is also a positive, given the influx of cash from the latter.

Valuation & Estimates

According to the price/book ratio, NTLA’s shares currently trade at 1.28x tangible book value, lower than the industry’s average of 3.47 and its mean of 3.26.

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The Zacks Consensus Estimate for its 2024 loss per share has narrowed 13 cents to $5.31 over the past 60 days. The loss per share estimate for 2025 has also narrowed 6 cents during the same time frame.

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Conclusion

The FDA approval of Vertex and CRISPR Therapeutics’ (CRSP - Free Report) CRISPR/Cas9 genome-edited cell therapy, Casgevy, for the treatment of sickle cell disease has put the spotlight on the gene editing space. 

While NTLA’s pipeline of innovative CRISPR-based therapies is promising, the development of these is a complex affair and their safety profiles need to be tested over a period of time. Clinching an FDA approval and commercializing the same can be a daunting task.

Investors’ skepticism about the recently released data on Nex-z and NTLA-2002 does not bode well for NTLA. Another biotech, Ionis Pharmaceuticals (IONS - Free Report) , is also developing a treatment for HAE.  The FDA recently accepted a new drug application for Ionis’ donidalorsen for prophylactic treatment of HAE.

NTLA is a risky bet as of now. We would advise investors to wait for additional data readouts. For those already owning the stock, staying invested as of now would be a prudent move. Any positive data announcement from the ongoing studies may provide an impetus to the stock.

NTLA currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

 


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