After disclosing data from a Phase 2 trial for its CRISpen-based medication, NTLA-2002, covering hereditary angioedema (HAE), Intellia Therapeutics Inc. (NTLA, Financial) saw its stock tumble almost 9% in pre-market trading. Based on an April 4 cut-off, the data included 25 patients and indicated that single doses of 25 mg and 50 mg lowered monthly HAE attacks by 75% and 77% respectively in the first 16 weeks. The cuts between weeks five and sixteen came out to be 80% and 81%.
While no patients in the placebo group showed complete remission, eight of 11 patients in the 50 mg group and four of 10 in the 25 mg group reported complete responses according to Intellia. Generally speaking, the side effects—which included headache and tiredness—were mild.
Based on these findings, Intellia chose the 50 mg dose for their Phase 3 research, HAELO, which is now under enrollment globally. As the company works toward regulatory approval, the study seeks to validate even more the effectiveness and safety of the therapy.
