Intellia and Regeneron update data for CRISPR candidate in ATTR amyloidosis
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On Monday, Intellia Therapeutics (NASDAQ:NTLA) and Regeneron Pharmaceuticals (NASDAQ:REGN) issued updated interim data for CRISPR gene editing candidate NTLA-2001 from a Phase 1 trial in transthyretin (ATTR) amyloidosis, a rare but fatal disease that can cause complications in multiple tissues.
The companies said that the single-dose therapy led to a sustained effect in reducing the disease-causing protein called transthyretin (TTR) in blood.
The 15 patients in the trial had received single doses of 0.1 mg/kg, 0.3 mg/kg, 0.7 mg/kg, and 1.0 mg/kg of NTLA-2001 intravenously.
The changes in their baseline values of serum TTR protein indicated that the experimental therapy has led to dose-dependent reductions of the compound with the maximum effect at Day 28.
Among the three patients each in the 0.1 mg/kg, 0.3 mg/kg, and 0.7 mg/kg dose groups, the mean reductions stood at 52%, 87%, and 86%, respectively, and among six patients in the 1.0 mg/kg dose group, the decline reached 93%.
The reductions seen on Day 28 lasted through the time the last measurement was taken for the six patients at the 1.0 mg/kg dose level, indicating a duration of two to six months.
âThese data suggest that treatment with a one-time, systemically delivered CRISPR-based investigational therapy has the potential to substantially reduce levels of a disease-causing protein,â CEO of Intellia (NTLA) John Leonard remarked.
NTLA-2001 was generally well tolerated, the companies said, adding that 73% (n = 11) of patients experienced side effects at Grade 1 severity. In the 1.0 mg/kg dose group, there was a single related serious adverse event of vomiting (Grade 3) in a patient who previously had gastroparesis. In addition, in the 0.7 mg/kg dose group, there was a single unrelated event of COVID-19 pneumonia.
Intellia (NTLA) has lost ~9% in the post-market, while Regeneron (REGN) is trading flat.
In June 2021, the two companies reported interim findings from the trial, indicating the potential of in-vivo CRISPR genome editing for the first time in humans.