Intellia: Shares Tank as Data From NTLA-2002 Study Worries Investors

No-moat Intellia Therapeutics released detailed phase 2 results from a study of a gene-editing therapy for hereditary angioedema, which drew mixed reactions from investors and medical experts and sent the stock down 20% on Oct. 24. The pipeline candidate, NTLA-2002, is designed to inactivate a gene involved in potentially life-threatening inflammatory attacks for people suffering from this rare disorder. In the ongoing phase 2 study of 27 patients, researchers found either a 75% or 77% reduction in attacks in the 16-week period after receiving therapy, depending on whether patients received the 25 mg or 50 mg dosages. Some of the data looks very promising, as 8 of the 11 patients in the higher-dose arm were completely free of attacks after a one-time infusion, and all these patients continued to be attack-free through the latest follow-up, which was a median of eight months.