Intellia Therapeutics (NTLA 3.70%) is a biotech company that focuses on gene editing, a relatively new technology that could allow scientists to devise therapies for otherwise impossible-to-treat conditions. As with any new technology, those who invest in it early enough could profit immensely in the long run. The question for investors, though, is whether Intellia is the best company to invest in to benefit from this potentially lucrative market. Let's look at some of the company's most promising programs, as well as its current financial position, to determine whether Intellia's stock is worth buying today.
Intellia's pipeline
Intellia currently has about 10 products in its pipeline. The company is going after diseases including hemophilia, a rare condition characterized by abnormal blood clotting, and sickle cell disease (SCD), a disorder that leads to crescent-shaped red blood cells. However, most of Intellia's programs are still very early in their development. Intellia's most advanced pipeline candidate is OTQ923, its potential SCD treatment. Back in March, the U.S. Food and Drug Administration (FDA) approved Intellia's Investigational New Drug (IND) application for OTQ923.
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On the back of this win, the company expects to start a phase 1/2 clinical trial soon. Intellia will test OTQ923 in SCD patients with severe complications of the disease. Intellia's second most advanced pipeline candidate is NTLA-2001, a potential treatment for transthyretin amyloidosis. This rare genetic disorder can lead to nerve and tissue damage. The company should submit an IND application for NTLA-2001 soon.
It is worth noting that Intellia faces stiff competition for some of its programs from other gene-editing companies. For instance, several biotechs are currently looking to develop SCD treatments. Notably, CRISPR Therapeutics' CTX001 has already produced exciting results for patients with this condition. In a phase 1/2 clinical trial, one SCD patient treated with CTX001 was free of vaso-occlusive crises (VOCs) -- a common side effect of SCD characterized by acute pain -- nine months after the treatment. This patient experienced an average of seven VOCs per year before receiving the treatment. Other gene-editing companies going after SCD include bluebird bio, which recently reported encouraging results from a clinical trial as well.
Intellia's financial position
During the first quarter of 2020, Intellia reported total operating expenses of about $46 million and a net loss of $31.8 million. The company had $250.3 million in cash and cash equivalents. Intellia's healthy cash balance stems in part from its partnerships with larger pharmaceutical companies. For instance, Intellia teamed up with Regeneron in a licensing and collaboration agreement back in 2016.
The two entities recently expanded their partnership and penned a deal that would see Intellia receive $70 million in cash and a $30 million equity investment. For its part, Regeneron acquired the rights to co-develop and commercialize some of Intellia's pipeline candidates. Intellia also has a partnership with Novartis.
These deals don't make the company immune to dilutive forms of financing, however. In June, Intellia raised approximately $115 million (in gross proceeds) in a public offering of common stock. Still, the company now seems to have more than enough capital to fund its operations for at least a year without diluting its shareholders' positions.
A risky bet
Given that Intellia's pipeline products aren't even in clinical trials yet, it seems premature to bet on the company, especially as other gene-editing companies are further along in the development of their programs and arguably present better prospects at this point. Sure, Intellia could go on to market several blockbuster products in (at the very least) a few years. But it's crucial to remember that most newly developed drugs never make it to the market. Before even thinking about initiating a position in this biotech stock, I would recommend waiting for the company to show at least some success in human clinical trials. Until then, investors would do better to avoid Intellia's stock.
