Intellia Therapeutics, Inc. (NASDAQ:NTLA) has released positive new clinical data from the ongoing phase 1 trial of Nexiguran Ziclumeran (Nex-Z), also known as NTLA-2001, in patients with transthyretin (ATTR) amyloidosis. The data cut-off date for the new results was August 21, 2024.
For patients with ATTR-CM, a single dose of Nex-Z led to a mean serum TTR reduction of 90% at month 12, with all patients continuing to show a sustained response with no evidence of a waning effect over time. The consistently low levels of serum TTR are anticipated to reduce the rate of ongoing amyloid formation and potentially allow for amyloid clearance and improvement in cardiac function. Furthermore, evidence of disease stabilization or improvement at month 12 compared to baseline was observed across multiple markers of cardiac disease progression, with stability or improvement in NT-proBNP, high sensitivity troponin T (hs-Troponin T), and the 6-minute walk test (6MWT) in a high proportion of patients.
In the ATTRv-PN arm, patients who received a dose of 0.3 mg/kg or higher showed a mean serum TTR reduction of 91% at month 12, with all patients continuing to demonstrate a sustained response with no evidence of a waning effect over time. Favorable trends indicating stability or improvement were observed in patients with ATTRv-PN based on evaluation of multiple clinical measures, including neuropathy impairment score (NIS), modified neuropathy impairment score (mNIS+7), and modified BMI (mBMI).
The safety profile of Nex-Z was generally well tolerated across all patients, with the most commonly reported treatment-related adverse events being infusion-related reactions (IRRs), which were predominantly mild and moderate in severity and did not result in any discontinuations.
Following these announcements, the company's shares moved 1.9%, and are now trading at a price of $14.24. For the full picture, make sure to review Intellia Therapeutics's 8-K report.
