UK Gives Regulatory Support to NTLA-2002 as Patient Trial Advances

Innovation passport awarded gene-editing treatment of hereditary angioedema

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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NTLA-2002, an experimental gene-editing therapy for hereditary angioedema (HAE) being developed by Intellia Therapeutics, has been awarded an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), the company announced.

This designation is the first step in the U.K.’s Innovative Licensing and Access Pathway (ILAP), a program that aims to shorten the time it takes for potential therapies to be approved and bought to the market for patients in that country. NTLA-2002 is being evaluated in adults HAE patients in an ongoing clinical trial.

“With the high treatment burden of currently available chronic therapies for hereditary angioedema, we are pleased to receive the ILAP designation, which will enable Intellia to further accelerate the clinical development of NTLA-2002,” John Leonard, MD, Intellia’s president and CEO, said in a company press release.

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Trial of potential hereditary angioedema treatment to enter next phase

The innovation passport is given to therapies in the early stages of clinical development that have the potential to address substantial health needs for the public or for individual patients. One of the ILAP program’s benefits is access to a target development profile — a roadmap through the developmental process that outlines the steps necessary to secure regulatory approval.

ILAP is delivered by the MHRA in partnership with other U.K. agencies, namely the All Wales Therapeutics and Toxicology Centre, the National Institute for Health and Care Excellence, and the Scottish Medicines Consortium.

In HAE, swelling attacks are triggered by spikes in the production of the signaling molecule bradykinin. Production of bradykinin is controlled by a protein called kallikrein.

NTLA-2002 is designed to reduce kallikrein levels, thereby lowering bradykinin production and preventing swelling. The gene-editing therapy uses the CRISPR-Cas9 editing tool to interfere with the gene that provides instructions for making a kallikrein precursor, reducing how much of the protein is made by cells.

Intellia is currently sponsoring a Phase 1/2 trial (NCT05120830) to investigate the safety and pharmacological properties of NTLA-2002 in adults with HAE types 1 and 2. Interim data from the first 10 patients, presented late last year, showed the single-infusion therapy was generally well-tolerated and reduced swelling attack rates.

The trial’s Phase 2 portion, which will compare NTLA-2002-treated patients with those given a placebo, is expected to start in the next few months, Intellia reported. In addition to safety and tolerability, a primary study goal is changes in the number of monthly HAE attacks at 16 weeks, about four months, post-treatment.

“We expect to begin the Phase 2 portion of the NTLA-2002 clinical study in the first half of this year, and we look forward to working with the U.K. and other regulatory agencies to bring this investigational single-dose genome editing treatment to patients as quickly as possible,” Leonard said.