- Intellia Therapeutics (NTLA, Financial) showcased a 90% reduction in serum TTR maintained for 24 months in the Phase 1 study of nexiguran ziclumeran (nex-z).
- 14 out of 18 patients showed clinically meaningful improvement in mNIS+7 scores after two years.
- Enrollment in the MAGNITUDE-2 trial continues, with a potential BLA submission set for 2028.
Intellia Therapeutics, Inc. (NTLA) announced encouraging two-year follow-up data from a Phase 1 study of its investigational one-time gene editing therapy, nexiguran ziclumeran (nex-z), during the Peripheral Nerve Society Annual Meeting. This gene therapy, aimed at treating hereditary transthyretin (ATTR) amyloidosis with polyneuropathy, demonstrated sustained efficacy in patients over 24 months.
Key trial outcomes revealed that patients receiving a single dose of 0.3 mg/kg or higher exhibited a 90% mean reduction in serum transthyretin (TTR) levels by Day 28, with levels remaining unchanged for at least 24 months. Notably, 14 out of 18 evaluated participants showed clinically significant improvements, marked by a ?4 point increase in mNIS+7 scores, with half of the previously progressing patients on patisiran experiencing noticeable clinical improvements.
The study also reported a favorable safety profile, with only mild to moderate infusion-related reactions observed. Importantly, liver enzyme abnormalities were not deemed serious and resolved without intervention, affirming the safety of the treatment.
Intellia has set a target for Biologics License Application (BLA) submission by 2028, contingent on the successful completion of the ongoing MAGNITUDE-2 trial. This trial is designed to further evaluate the efficacy of nex-z, potentially positioning it as a transformative treatment for ATTR amyloidosis that offers a one-time, lifelong benefit. The ongoing trial reflects Intellia's strategic push toward addressing the root causes of hereditary amyloidosis with its gene-editing technology.
