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• First-of-its-kind Intellia data suggest CRISPR drug could be given more than once

Dive Brief:

• Intellia Therapeutics has begun a second Phase 3 study testing one of its CRISPR gene editing medicines, announcing Monday the initiation of a trial that will assess its treatment for a rare swelling disorder known as hereditary angioedema.

• The trial, which Intellia has titled “HAELO,” will involve 60 adults with hereditary angioedema, or HAE, a condition that involves recurring inflammatory attacks on the body’s organs and tissues. An estimated 1 in 50,000 people have HAE, which is currently treated with a number of on-demand and preventive drugs.

• Called NTLA-2002, Intellia’s drug uses CRISPR to knock out a gene encoding for a protein associated with blood clotting and with HAE. The treatment does its editing inside the body — or in vivo — and is designed to be a one-time fix for the condition.

Dive Insight:

Intellia has ambitions to play spoiler to the looming three-way competition between Pfizer, BridgeBio Pharma and Alnylam Pharmaceuticals to offer an effective treatment for another rare disease, transthyretin amyloidosis with cardiomyopathy.

The CRISPR company launched a Phase 3 trial of its treatment, “NTLA-2001,” nearly one year ago. At the time, the study had the distinction of being the first late-stage trial initiated of an in vivo CRISPR medicine.

Intellia, together with its partner Regeneron Pharmaceuticals, continues to enroll that study across nearly three dozen sites globally.

Now, it’s moving into late-stage testing with NTLA-2002, following positive Phase 2 results that showed treatment significantly reduced HAE attacks as well as lowered levels of the kallikrein protein it targets.

HAELO will randomize participants 2-to-1 to receive either NTLA-2002 or a placebo, and assess the change in HAE attacks from week 5 of the study through week 28. At that point, participants who were randomized to receive the placebo can opt to go onto NTLA-2002 in a “crossover” phase of the trial.

“We are working urgently to bring forward NTLA-2002 to address the real-world needs of people suffering from this disease and, ultimately, believe it will bring significant value to patients, physicians and payors,” said Intellia CEO John Leonard in a statement.

Shares in Intellia have declined by more than 40% this year amid heightened investor scrutiny of genetic medicines developers, as well as broader challenges in the market for biotechnology companies. Intellia is currently worth about $1.8 billion.