Intellia Therapeutics (NTLA) said Sunday it received "positive" two-year follow-up data from an ongoing phase 1 trial of its investigational drug nexiguran ziclumeran for the potential treatment of hereditary transthyretin amyloidosis with polyneuropathy, a genetic disease associated with the liver.
The company said a one-time dose of the drug resulted in a reduction of the levels of the protein that cause the illness by a mean level of 90%.
It also said the study provided evidence that the drug could result in disease stability or "clinically meaningful improvement" based on the evaluation of certain clinical measures.
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