The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Innovation Passport for Intellia Therapeutics, Inc.’s NTLA-2002, an in vivo genome editing candidate being developed for the treatment of HAE. The Innovation Passport is the point of entry into the U.K.’s Innovative Licensing and Access Pathway (ILAP), which is designed to accelerate time to market and facilitate patient access to innovative medicines.

“With the high treatment burden of currently available chronic therapies for HAE, we are pleased to receive the ILAP designation, which will enable us to further accelerate the clinical development of NTLA-2002,” says Intellia President and CEO John Leonard, M.D. “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.”

The Phase 1/2 study evaluating NTLA-2002 in adults with Type I or Type II HAE is currently ongoing, with the Phase 2 portion expected to initiate in the first half of 2023.

Delivered in partnership by the All Wales Therapeutics and Toxicology Centre (AWTTC), the Medicines and Healthcare products Regulatory Agency (MHRA), the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC), the Innovation Passport prioritizes innovative medicines in early clinical development stages for the treatment of diseases with a significant patient or public health need. Benefits of the ILAP include access to a Target Development Profile, which defines key regulatory and development features and creates a roadmap for advancement through regulatory approval.

Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of plasma kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies.
(Source: Intellia)