EAACI – Intellia pushes forward in crowded rare swelling disease

Intellia’s in vivo edited Crispr project NTLA-2002 has already moved into phase 2 in hereditary angioedema, so the latest cut of data from the phase 1 portion of that trial make for encouraging reading. Across 10 patients, one dose of NTLA-2002 produced a 95% mean reduction in monthly attack rate, according to the latest data cut presented at the European Academy of Allergy & Clinical Immunology meeting this weekend. A dose response on plasma kallikrein reduction was also evident across the three doses tested, small patient numbers notwithstanding. With no concerning toxicity signals these results raise hopes for placebo-controlled data from the phase 2 stage, which Intellia expects to be fully enrolled by year end. Importantly, this will include US patients, after the FDA cleared Intellia’s IND in March. Perhaps a bigger question is where NTLA-2002 will fit in a crowded HAE field, where a number of effective therapies already exist, and other novel prophylactic projects are in the pipeline. Most notably, readout of Ionis’s pivotal donidalorsen trial, Oasis-HAE, is due in the first half of next year; the antisense project is given once a month.