Intellia Therapeutics has reported positive results from its ongoing Phase I/II trial of NTLA-2002, a CRISPR-based gene editing therapy for hereditary angioedema (HAE).
NTLA-2002, which leverages the CRISPR/Cas9 technology, is expected to provide a one-time treatment by inactivating the kallikrein B1 (KLKB1) gene responsible for producing prekallikrein, a precursor protein linked to the condition.
HAE is a rare genetic condition that causes potentially life-threatening swelling attacks.
The Phase II double-blind, randomised, placebo-controlled trial is designed to assess the safety, efficacy, pharmacodynamics and pharmacokinetics of the therapy.
In this trial, 27 participants were randomised to receive either a 25mg or 50mg dose of NTLA-2002 or a placebo through intravenous infusion.
As of the data cut-off date on 4 April 2024, a significant reduction in mean monthly attack rates was observed in patients treated with NTLA-2002 compared to placebo.
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By GlobalDataIntellia president and CEO John Leonard said: “These positive NTLA-2002 Phase II results underscore the tremendous potential of our in vivo CRISPR gene editing therapy to be a functional cure and redefine the treatment paradigm for HAE.
“We are highly encouraged by these results, which we believe sets NTLA-2002 apart from other prophylaxis treatments.”
The 25mg and 50mg doses resulted in a 75% and 77% reduction in attack rates during the first 16 weeks, improving to 80% and 81% during weeks five to 16, respectively.
In the 50mg cohort, eight out of 11 patients achieved a complete response, with no attacks throughout the 16-week observation period.
In comparison, the 25mg cohort saw four out of ten patients experiencing a complete response, while no patients in the placebo group did.
Patients receiving the 50mg dose also exhibited a greater reduction in kallikrein protein levels, with an 86% mean reduction from baseline at week 16, as opposed to a 55% reduction in the 25mg group.
The therapy was observed to be well tolerated at both dosage levels.
Based on these positive outcomes, Intellia has chosen the 50mg dose for further evaluation in the global, pivotal Phase III HAELO study, initiated earlier this month, which is currently screening patients.
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