Companies focusing on developing gene editing therapies have made significant progress over the past year. However, that has done little to keep Intellia Therapeutics (NTLA 3.70%) afloat. The biotech's stock has substantially lagged the broader market over the trailing 12 months. Still, Intellia Therapeutics has several promising candidates in the pipeline. If the company can record solid clinical and regulatory progress from here on out, its stock could rebound. Does Intellia Therapeutics have what it takes to make a comeback, or is it too late to get in on the stock?
Intellia Therapeutics' leading programs
Intellia Therapeutics is a clinical-stage company that generates no profit. However, the company has at least one highly promising candidate in the pipeline. Intellia is developing NTLA-2001 for the treatment of transthyretin amyloidosis. This disease is caused by a dangerous protein buildup, typically in patients' hearts. It can generate a range of issues, including progressive muscle weakness.
There are two broad kinds of transthyretin amyloidosis. First, there's the hereditary version, which affects about 50,000 people worldwide. The non-hereditary kind affects between 200,000 and 500,000 patients globally.
There are no cures for this illness, but if all goes according to plan, NTLA-2001 could be the first, or at least one of the first, as other companies are on the trail. NTLA-2001 reported encouraging results in early stage trials. In a phase 1 study, the gene editing therapy significantly reduced the disease-causing protein in patients.
The company plans to start a phase 3 study for NTLA-2001 in the first quarter. Importantly, Intellia Therapeutics has some help with this program. The company is partnering with a biotech giant: Regeneron. That substantially reduces the risk that Intellia won't have the funds to sustain the program until approval.
Having the backing of a much more successful drugmaker also helps smaller drugmakers in navigating the sometimes tricky and highly regulated biotech industry. Intellia Therapeutics does have several other pipeline candidates. The company's NTLA-2002 is currently undergoing the phase 2 portion of a phase 1/2 study for hereditary angioedema, a disease that causes severe swelling.
Intellia plans to announce data from this trial this year while starting a late-stage study by year's end. The biotech also plans to begin a phase 1 study for NTLA-3001 in treating alpha-1 antitrypsin deficiency-associated lung disease this year.
Plenty of potential with plenty of risk
In the past two years, investors have grown tired of speculative and unprofitable stocks. Intellia Therapeutics fits that bill, partly explaining its recent poor performance on the stock market. Can the biotech bounce back? That will depend on whether it can execute its strategy while making meaningful clinical and regulatory progress.
Funding shouldn't be an issue. The company ended 2023 with roughly $1 billion in cash, which management thinks can last until mid-2026. And with the cash-rich Regeneron in its corner, investors shouldn't worry too much about that side of things. However, whether Intellia Therapeutics will deliver the kinds of results investors hope for in clinical trials is anyone's guess.
Yes, the company's candidates have so far delivered encouraging results. But that doesn't mean they will also do so in late-stage studies. Positive results could send the stock soaring, but negative ones will do the opposite. What does that mean for investors? Intellia Therapeutics looks like a high-risk, high-reward play at the moment. Only investors comfortable with heightened volatility should consider investing in the company.
And for those investors, it may still be pretty early in the game. If Intellia Therapeutics can show solid clinical progress, it could deliver outsize returns for many years to come. For everyone else, there are safer biotech stocks out there to buy. However, keeping a close watch on Intellia Therapeutics is worth it: It could be a much better, less risky option in just a few years.
