NTLA stock rose Monday after Intellia Therapeutics said its CRISPR-based gene-editing drug almost completely eliminated attacks in a rare swelling disease.
The company tested its drug in 10 patients with hereditary angioedema, or HAE. Patients with this disorder experience severe, recurring and unpredictable inflammatory attacks in various organs and tissues across the body.
After a single dose of Intellia's gene-editing drug, NTLA-2002, patients experienced an average 95% reduction in monthly swelling attacks. Intellia has followed the patients for 5.6 months to 14.1 months. All but one patient have been attack-free since 16 weeks following treatment.
"We are thrilled to see that the earliest-dosed patients are attack-free for approximately a year or longer, with NTLA-2002 demonstrating a very favorable safety profile," Intellia Chief Executive John Leonard said in a written statement. "These remarkable attack rate reductions have been consistent, even in patients with the most severe symptoms."
On today's stock market, NTLA stock rose 0.9% to close at 43.61, paring back bigger gains. That briefly helped prop up shares of other gene-editing companies. But Crispr Therapeutics lost 2.2% while Editas Medicine gained 2.7% at the closing bell. Verve Therapeutics advanced 1.5% and Beam Therapeutics popped 2.4%.
NTLA Stock: What's The Future For CRISPR?
Intellia's journey could be telling for the future of CRISPR drugs. Intellia is the first with an in vivo treatment that's also systemic. That means the actual gene-editing takes place inside the body. Further, the drug is infused and then must find its way to the right organ. In this case, that's the liver.
NTLA-2002 focuses on lowering the level of an inflammatory molecule called plasma kallikrein. Due to a genetic defect, patients with hereditary angioedema make too much plasma kallikrein.
The company tested three dose levels. Intellia believes a 60% reduction in plasma kallikrein would help patients with hereditary angioedema. All nine patients who met that mark have been attack-free since 16 weeks following treatment. One patient went from about 17 swelling attacks monthly to zero.
Just one patient who received the low dose of 25 milligrams didn't reach the 60% reduction in plasma kallikrein. That patient experienced a swelling attack after 12.3 months free of episodes. The event followed a sports injury to the hand, and was mild enough not to require any treatment.
SVB Securities analyst Mani Foroohar says he's now focused on the mid-level 50-milligram dose.
"While investors will dissect patient-level data in this small dataset at length, we continue to see the key debates for this program as commercial/competitive positioning and patient propensity to switch, as we view durability/safety as fairly well characterized at this point," he said in a report.
Bullishly for NTLA stock, the six patients who were taking preventative HAE medicine have been able to drop those regimens. They haven't experienced any subsequent swelling episodes.
Side Effects Were Mostly Mild
The treatment also was well tolerated. Most side effects were mild in severity and included infusion-related reactions and fatigue. The lion's share of those resolved within two days. Intellia says there have been no serious side effects.
Intellia is now working on a Phase 2 study of NTLA-2002 and expects to wrap patient enrollment in the second half of the year.
NTLA stock briefly topped its 200-day moving average for the first time since late 2021, according to MarketSmith.com. But shares closed down below that mark.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
