On June 2, Intellia Therapeutics' (NTLA +4.98%) CEO John Leonard remarked on the biotech's progress with one of its early-stage clinical programs called NTLA-2002, uttering two very important words that are sure to make at least a few savvy investors salivate. The stock briefly rose 8% above where it was before the announcement.
But to the ears of other investors, the same two words imply a risk they'd rather not think about. Let's dive in and sort out what's going on, as well as which of these two perspectives is more relevant for those considering a purchase of this stock today.
NASDAQ: NTLA
Key Data Points
This is a good omen
Intellia makes gene therapies for genetic illnesses like hereditary angioedema (HAE). Some of those therapies, like NTLA-2002, one of its investigational medicines for HAE, entail editing the patient's genes directly so as to correct the inborn errors that cause illness. Such approaches are intended to be attempted only once per patient. But if they work as intended, the impact is potentially sufficient to make them symptom-free, potentially for the rest of their lives.
Such patients might still technically have the disorder, as it is currently impossible for 100% of the copies of their genes, which are distributed across the myriad cells of their body, to be fixed via editing. But for someone who is no longer experiencing any symptoms, which in the case of HAE means experiencing episodes of uncontrollable swelling of their body parts seemingly at random, the distinction between a functional cure and what the textbook might say counts as an actual cure is merely an academic question.
And that's why it's tremendously exciting that Intellia's CEO John Leonard said that based on some new phase 1/2 clinical trial data, NTLA-2002 could be a "functional cure" for HAE.
The data indicate that of the 10 patients enrolled in the study, eight experienced zero HAE attacks after treatment with NTLA-2002, even after 18 months. They appear to be functionally cured. Of the two patients who did still have swelling episodes caused by HAE, their number of attacks reduced by around 97% relative to before starting treatment, and neither had any severe attacks. None of the patients involved reported severe side effects.
Intellia now plans to initiate a phase 3 trial for NTLA-2002 sometime in the second half of this year, assuming regulators are on board. The program already has a handful of regulatory designations that'll move the process along, including the Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations. If the late-stage trial confirms the earlier results, NTLA-2002 will be headed for its shot at approval and commercialization shortly thereafter.
A few risks remain
Intellia now has some great data in hand, and it likely has what it takes to finish the development process for NTLA-2002. Management expects that it'll be competing in a global market for HAE medicines that will be worth roughly $6 billion annually by 2029.
As of the first quarter, it has $953 million in cash and marketable investments, and total operating expenses of close to $143 million for the three-month period. Its research and development (R&D) expenses were just under $112 million. So it isn't about to run out of money.
But it has no revenue outside its collaboration agreements right now. And regrettably, there is a possible downside to NTLA-2002's apparently high efficacy that'd put a ceiling on how much money it could bring in if it gets commercialized. It could theoretically cure so many patients that there won't be any remaining for treatment. Still, that risk won't be realized for years, if it ever is.
Is Intellia Therapeutics stock a buy based on this latest data?
Considering that NTLA-2002 isn't its only program, nor is it the most mature program it has, the biotech could have multiple medicines on the market before the end of the decade. Even a small amount of new revenue from sales of a therapy would make for significant growth.
So if you're the type that's usually comfortable investing in risky biotech stocks, this business is ready for a purchase today.
