Intellia's Gene-Editing Candidate Shows Durability In ATTR Amyloidosis

Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN announced additional interim data from the Phase 1 study of NTLA-2001, in vivo genome editing candidate for transthyretin (ATTR) amyloidosis.
Intellia had said that at the highest dose level (1.0mg/kg), six patients had shown a 93% mean reduction in the toxic protein. In the new data, three patients reached the nine-month follow-up point and maintained that level of TTR reduction.
Related: Intellia Shares Updated Data On Gene Editing Durability In Rare Disease.
The mean reduction for the three patients at the second-highest dose (0.7mg/kg) was 86%. For the longest-tracked group, dose level two at 0.3mg/kg, the three patients had a mean reduction of 89%.
Headaches, infusion-related reactions, back pain, rash, and nausea were the most frequent adverse events across all four dose levels.
Eleven of the 15 patients reported a maximal adverse event of grade 1. The most serious adverse event, which Intellia said was “possibly related” to its treatment, was Grade 3 vomiting in a patient with a medical history of gastroparesis.
Price Action: NTLA shares are down 1.87% at $49.81, and REGN shares are down 0.39% at $605.00 during the market session on the last check Friday.