The P-I study evaluates NTLA-2001 which showed sustained durability of serum TTR reduction with follow-up @12mos. in 0.1/0.3mg/kg & 6mos. in 0.7/1.0mg/kg
Pk modeling & simulation indicated that 80mg fixed-dose provides similar exposure to 1.0mg/kg. NTLA-2001 at 1.0mg/kg resulted in 93% mean & 98% maximum serum TTR reduction by 28 days in 6 patients while 3 patients reached 9mos. of follow-up with no loss in TTR reduction
In 0.7 & 0.3mg/kg dose cohort, 86% & 89% mean serum TTR reduction @28days & 12mos. & remained durable @6mos. In 0.1 & 0.3mg/kg, patients reached 12mos. of follow-up with durable response. The companies are expected the results from the cardiomyopathy in H2’22 with an expected enrollment completion in ATTRv-PN & ATTR-CM in 2022
Ref: GlobalNewswire | Image: Intellia
