Phase 3 trial of gene-editing therapy NTLA-2002 for HAE slated for 2024

NTLA-2002 designed to prevent HAE swelling attacks, ease treatment burden

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Enrollment for the Phase 2 portion of a clinical trial testing NTLA-2002, Intellia Therapeutics’ experimental gene-editing therapy for hereditary angioedema (HAE), is expected to be completed this year.

This is the result of “substantial interest from physicians and patients to participate in the NTLA-2002 clinical program,” the company stated in a recent corporate update.

“We are thrilled to see the excitement for this investigational therapy from the HAE community,” said John Leonard, MD, president and CEO of Intellia. “In just a handful of months, we were able to identify all patients required to fully enroll the ongoing Phase 2 study.”

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Phase 2 study completes recruitment outside US

While the company had previously announced regulatory clearance to open U.S. sites for the ongoing Phase 2 study, the pronounced interest in the program prompted the completion of recruitment at already active sites outside the U.S., including European countries and New Zealand.

Intellia is planning to launch a pivotal Phase 3 trial, including sites in the U.S., between July and September 2024, depending on regulatory feedback. A pivotal trial is a study whose data, if positive, can be used to support a future submission of an application seeking the therapy’s approval.

In HAE, swelling attacks are triggered by abnormally high levels of a signaling molecule called bradykinin, whose production is controlled by the kallikrein protein.

NTLA-2002 is a gene-editing therapy designed to lower bradykinin levels by disrupting the activity of the KLKB1 gene, which provides the instructions for making prekallikrein, a precursor of kallikrein.

By reducing the levels of kallikrein, and subsequently of bradykinin, the therapy is expected to prevent swelling attacks in HAE patients, as well as ease the treatment burden associated with currently available therapies.

In the Phase 1/2 trial (NCT05120830), researchers are testing the safety and efficacy of NTLA-2002 in adults with HAE.

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Phase 1 interim data show NTLA-2002 reduced monthly attack rate by 95%

Its Phase 1 portion tested three doses of the experimental therapy. Interim data showed that nine out of the 10 patients enrolled haven’t experienced any swelling attack for up to about one year after treatment. At the latest follow-up, the mean monthly attack rate was reduced by 95%.

All three doses were well-tolerated, with the majority of adverse events being mild in severity.

The trial’s Phase 2 portion, which began dosing in May, is testing two doses of NTLA-2002 against a placebo. Participants will be followed for up to about two years. Its main objective is to evaluate the treatment efficacy in lowering attack rates after 16 weeks (about four months).

Ahead of the Phase 3 trial and per request of the U.S. Food and Drug Administration upon clearance to include U.S. sites in the NTLA-2002 clinical program, Intellia will provide preclinical data about the inclusion of women of child-bearing age in the program. The data will complement findings from women in this age group who are participating in the ongoing Phase 1/2 trial.