- Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN have announced positive interim data from an ongoing Phase 1 study of NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
- Interim results come from the first six patients for the in vivo CRISPR/Cas9 genome editing candidate that reduced serum levels of transthyretin, a key biomarker for the disease, by 87% in patients who received a higher dose.
- Mean reductions of 52% among the three patients in the 0.1 mg/kg dose group and 87% among the three patients in the 0.3 mg/kg dose group, including one patient with a 96% reduction.
- The results bested the standard of care therapy, which typically reduces transthyretin by 80%.
- NTLA-2001 was generally well-tolerated at both dose levels, with no serious adverse events and no liver findings by day 28.
- The candidate is continuing to be evaluated in the dose-escalation portion of the study.
- After identifying a recommended dose, Intellia expects to begin a single-dose expansion cohort in Part 2 of the Phase 1 trial later this year.
- After completing the Phase 1 trial, the company plans to move to pivotal studies for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.
- Intellia will host an investor event today at 8:00 a.m. E.T.
- Price Action: NTLA shares are up 31.1% at $116.49 during the premarket session on the last check Monday, while REGN stock closed at $550.55 on Friday.
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