Intellia Therapeutics said early Friday it found a way to knock out the gene responsible for a rare and deadly swelling disorder — but NTLA stock sank on new safety concerns.
The study in patients with hereditary angioedema is key for CRISPR drug makers. It's Intellia's second test of a systemic and "in vivo" gene-editing drug in people. This means not only does the genetic edit take place inside a person, but the drug is infused in a patient's arm. From there, the gene-editing instructions are designed to pave the way to the proper organ. In this case, that's the liver.
But one patient experienced slightly elevated liver enzymes. Intellia also tested a gene-editing drug in patients with bad-actor protein that builds up on the nerves and heart. In the nerve group, one patient had severely elevated liver enzymes — more than 20 times normal levels — and one of the heart-disease patients experienced a serious infusion-site reaction, RBC Capital Markets analyst Luca Issi said in a report to clients.
In response, NTLA stock toppled 4.2% to 65.71. Also on the stock market today, shares of other CRISPR stocks Crispr Therapeutics and Editas Medicine toppled 6% and 10.4%, respectively.
NTLA Stock: Close To 'Blue Sky'
The results in the swelling disorder, known as hereditary angioedema, outperformed an approved drug from Takeda Pharmaceutical called Takhzyro. Intellia says its approach led to an average 65%-92% decrease in the level of the problematic protein at the center of the disease eight weeks after the infusion. Takeda's drug leads to a 60% decrease.
Patients with hereditary angioedema experience seemingly random swelling attacks. But patients who received a low dose of Intellia's gene-editing drug experienced a 91% drop in swelling attacks. Intellia Chief Executive John Leonard says the company hopes to make more effective treatments for patients with genetic diseases.
Patients with this disease "certainly take their medicine because they're fearful of what can happen," he told Investor's Business Daily. Taking away that uncertainty with a more effective drug could be "truly liberating for these patients."
Roughly four months after treatment, two out of three hereditary angioedema patients still hadn't experienced any swelling attacks. But RBC's Issi noted those patients had less severe disease before treatment.
NTLA Stock: A New Frontier In Medicine
In addition to its hereditary angioedema study, Intellia also offered an update for its CRISPR drug in people with a heart condition known as cardiomyopathy. In this condition, an abnormal protein called amyloid builds up on the heart, causing widespread problems.
After a single infusion, Intellia lowered the amount of the troublesome protein in patients' blood by an average 92%-93%, depending on the dosage.
The results add to an earlier release from June 2021 in which Intellia knocked down the same protein for patients with polyneuropathy. That release was the first in-human data from an "in vivo" and systemic CRISPR drug in people, and sent NTLA stock soaring 50% in a day.
Other advanced efforts in CRISPR differ.
Efforts From Other CRISPR Stocks
Editas is also testing an in vivo approach. But its eye-disease drug is infused directly into the eye. It's not systemic. Crispr and Vertex Pharmaceuticals are using CRISPR on cells outside the body before infusing their sickle cell disease treatment into patients. That approach is "ex vivo" — editing outside the body.
So, Intellia's updates offer an important view into the future of CRISPR technology. CEO Leonard says it gives the company a leadership position.
"We take the pioneering aspect of this very seriously," he told IBD. "We talk about the importance of being good stewards of the technology."
NTLA stock is trading in line with its 50-day moving average, according to MarketSmith.com. It's also consolidating with buy point at 76.55. Shares have a strong Relative Strength Rating of 86. This puts Intellia stock in the top 14% of all stocks in terms of 12-month performance, according to IBD Digital.
Rivaling Pfizer, Alnylam?
Intellia partners with Regeneron Pharmaceuticals on research in cardiomyopathy and polyneuropathy. Both are different versions of the same disease: transthyretin amyloidosis.
Success in this arena could put Intellia and Regeneron on track to rival Pfizer and Alnylam Pharmaceuticals.
Pfizer sells Vyndaqel to treat the heart aspect of the disease. Alnylam is working to expand to that patient group, and already makes Onpattro for the smaller pool of polyneuropathy patients.
In an early report Friday, RBC's Issi called the results close to a "blue sky scenario." In a follow-up after the company's conference call with investors, he noted the side effects slammed NTLA stock. But the move is taking place in an already choppy market.
"Nonetheless, when we step back and look at the bigger picture, we remain impressed by the data given the (superior effect) in hereditary angioedema vs. current standard of care should drive better outcomes for patients, the liver enzyme elevations were transient in nature with no bilirubin increases," he said.
He also applauded Intellia's decision to back off higher dosing in cardiomyopathy patients to retain the benefit without increasing the risk. Issi kept his outperform rating and 150 price target on NTLA stock.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
