Intellia Therapeutics, Inc. NTLA lost 21.6% in a week as investors were disappointed with the company’s plans for portfolio reorganization.
Intellia is a clinical-stage gene editing company focused on developing innovative CRISPR-based therapies. As part of the recently announced portfolio reorganization, NTLA is planning to prioritize the development of its late-stage programs — NTLA-2002 and nexiguran ziclumeran (nex-z). The company will also reduce its workforce by 27% in 2025.
NTLA Underperforms Industry, Sector and S&P 500
Zacks Investment Research
Image Source: Zacks Investment Research
More on NTLA’s Objectives for 2025
Nexiguran ziclumeran 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 for the development and commercialization of nex-z.
Nexiguran ziclumeran is being studied for two indications — ATTR amyloidosis with polyneuropathy (ATTRv-PN) and ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The phase III MAGNITUDE study is evaluating the safety and efficacy of nex-z in patients with ATTR amyloidosis with cardiomyopathy. Enrollment is currently ongoing in the study.
The phase III MAGNITUDE 2 study is actively screening patients with hereditary ATTR amyloidosis with polyneuropathy, with the first patient expected to be dosed later in the first quarter of 2025.
The FDA had earlier granted Regenerative Medicine Advanced Therapy designation to nex-z for the treatment of 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.
The phase III HAELO study is actively enrolling patients to evaluate NTLA-2002 for treating HAE. Intellia plans to dose the first patient in this pivotal study later in the first quarter of 2025. Enrollment is likely to be completed in the second half of 2025.
Intellia is gearing up to transition from a late-stage development company to a commercial-ready organization by the end of 2026.
Owing to the latest portfolio reorganization, Intellia has decided to stop the development of its in vivo gene insertion candidate, NTLA-3001, for the treatment of alpha-1 antitrypsin deficiency-associated lung disease.
NTLA to Cut Workforce
As part of this portfolio reorganization, NTLA plans to reduce its current workforce by almost 27% by 2025.
The company expects to incur around $8 million in charges related to the reorganization, expected to be recorded in the first quarter of 2025.
NTLA had approximately $862 million in cash, cash equivalents and investments at the end of the fourth quarter of 2024.
Per the company, the existing cash balance and the anticipated cost savings from the above-mentioned strategic reorganization are expected to provide the cash runway into the first half of 2027.
Valuation & Estimates
According to the price/book ratio, NTLA’s shares currently trade at 1x tangible book value, lower than the industry’s average of 3.41 and its mean of 3.26.
Zacks Investment Research
Image Source: Zacks Investment Research
The Zacks Consensus Estimate for its 2024 loss per share has narrowed 5 cents to $5.28 over the past 60 days. The loss per share estimate for 2025 has also narrowed 37 cents during the same time frame.
Zacks Investment Research
Image Source: Zacks Investment Research
Conclusion
NTLA's stock was hammered in 2024 as data from an ongoing phase I study of Nex-z in patients with ATTR amyloidosis failed to impress investors.
In October 2024, NTLA announced positive data from the ongoing phase I/II study of NTLA-2002 in HAE patients. These data, however, didn’t seem to impress investors and the stock was hit hard following the announcement.
The FDA approval of Vertex and CRISPR Therapeutics’ CRSP 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. 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 company’s restructuring plans does not bode well for NTLA. Another biotech, Ionis Pharmaceuticals IONS, 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.
Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
