The Food and Drug Administration gave Intellia Therapeutics the go-ahead to begin a pivotal study of its gene-editing treatment for a deadly heart disease, but NTLA stock reversed its premarket gains.
Intellia is testing a CRISPR-based approach to gene-editing in patients with transthyretin amyloidosis with cardiomyopathy, a disease in which abnormal protein builds up on the heart. The condition is fatal if left untreated. Intellia expects to begin its Phase 3 study before the end of the year.
Wedbush analyst David Nierengarten noted the landscape in cardiomyopathy is becoming competitive. Pfizer sells an approved treatment, Vyndaqel. AstraZeneca and Alnylam Pharmaceuticals are working on drugs that could "pose substantial headwinds."
On today's stock market, NTLA stock skidded 3.8%, reversing from a premarket climb and closing at 27.96. Shares of Regeneron Pharmaceuticals, which is collaborating with Intellia on the drug, fell 1.3% to 826.03.
A First In CRISPR Could Be Promising For NTLA Stock
RBC Capital Markets analyst Luca Issi says the update is "great news for Intellia and the broader field of gene editing."
"Despite (a use) where there is another therapeutic option like (Vyndaqel), the FDA is clearly comfortable green-lighting a technology that will permanently edit (liver cells) in hundreds of Americans," he said in a report.
If successful, Intellia would snap up a first-ever for the CRISPR landscape. Later this year, the FDA will consider a drug from Crispr Therapeutics and Vertex Pharmaceuticals that uses CRISPR gene-editing on cells outside the body. The cells are then reinfused into patients with two blood diseases for therapeutic effect. The process is called ex-vivo gene editing.
But Intellia is testing out an in-vivo gene-editing approach, in which genetic edits take place inside a patient's body. The approach is also systemic, meaning the drug is infused and then must find its way to the right organ. The company is also testing an in vivo, systemic CRISPR drug in patients with a genetic swelling disease.
"Gene editing has clearly gone out of favor, but we remain buyers given we think Intellia is making material progress, the company remains best positioned for in-vivo gene editing and ahead of key catalysts," he said.
He kept his outperform rating and 80 price target on NTLA stock.
Analysts Didn't Expect The Stock Dive
Leerink Partners analyst Mani Foroohar says Intellia "continues to execute" and expected shares to trade up in the mid, single-digit percentage range on the update. Instead, NTLA stock touched its lowest point since November 2020, MarketSmith.com shows.
Wedbush's Nierengarten acknowledged Intellia's Phase 1 test results were promising in patients with cardiomyopathy. Roughly a month after receiving one dose, patients' serum showed 92% to 94% average reductions in abnormal protein. That was sustained out to four to six months.
But he lowered his price target on NTLA stock to 45 from 48, and kept his neutral rating. NTLA stock has trended lower since mid-July.
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