Intellia Therapeutics’ CRISPR-based treatment for a rare genetic disease called hereditary angioedema reduced the bouts of swelling associated with the disease in a mid-stage test, the company announced Thursday as part of its second-quarter earnings.
Intellia plans to start a Phase 3 trial in the second half of the year.
Both the 25 mg and 50 mg doses of the therapy, known as NTLA-2002, led to “deep reductions in attacks” at 16 weeks, according to Intellia. However, the gene editing company is moving forward with a 50 mg dose for the pivotal trial based on the “greater number of patients with complete attack elimination,” as well as greater reduction in kallikrein, a key protein associated with the disease.
Intellia said the treatment also met all secondary endpoints in the study.
In June, Intellia reported updated results from a Phase 1 study, showing that the treatment eliminated swelling attacks by 98% in 10 people. At the time, Intellia’s CEO John Leonard said the treatment could potentially serve as a “functional cure” for the disease.
In developing a gene editing therapy for hereditary angioedema, Intellia faces competition with a number of treatments, including Takeda’s antibody drug Takhzyro. Also, Ionis Pharmaceuticals’ RNA drug donidalorsen succeeded in cutting swelling attacks in a Phase 3 study earlier this year.
“We’re trying to reset what is the current therapeutic regimen that’s available to patients, and that starts with having a deep understanding of what patients are actually looking for,” said Leonard on a Thursday morning earnings call.
“They’re not looking for minor improvements in prophylactic therapy, they’re not looking for primarily extensions in dosing intervals,” he said. “What they’re looking for is to be as normal as possible, and that means not having to take any medicines.”
Intellia plans to present the detailed Phase 2 data at a medical conference later this year.
The company is also developing a gene editing treatment for ATTR amyloidosis where it has started a Phase 3 trial and plans to begin a second one later this year. In July, Intellia won clearance from the UK to start testing a gene insertion treatment for lung disease due to alpha-1 antitrypsin deficiency.