Day: May 18, 2025

 Two New Analyses from SEQUOIA-HCM on Effect of Aficamten Between Patientswith Mild and Moderate-to-Severe Symptoms, and Across Geographic Regions HEOR Analyses of Real-World Data Reveal Disparities in Outcomes In Females and Older Patients with Non-Obstructive HCM SOUTH SAN FRANCISCO, Calif., May 18, 2025 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that additional data arising from two analyses from SEQUOIA-HCM, (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM), and results from a real-world analysis related to non-obstructive HCM were presented at the European Society of Cardiology Heart Failure 2025 Congress. The results from an...

 Slowing of progression of disease observed via MRI at 24 months Improvements in outcomes and reduction in sorbitol maintained through 24 months Statistically significant correlation between absolute reduction in sorbitol and change in 10MWRT and CMT-FOM Lower Limb domain at 12 months, though the primary endpoint of 10MWRT at 12 months was not statistically significant (the measure has since been removed from the CMT-FOM clinical outcomes assessment based upon the results of the ACT-CMT study*) Statistically significant improvement in CMT-HI and statistically significant correlation between percent change in sorbitol and change in CMT-HI at 12 months Treatment with govorestat in CMT-SORD-Null rats demonstrated a highly statistically significant 37% (p=0.005) reduction of sorbitol in the sciatic nerve, the tissue of interest, after treatment...

Deep, durable and consistent reductions in TTR were sustained at two years, following a one-time dose of nex-z Clinically meaningful improvements in ATTRv-PN related outcomes observed at 24 months compared to baseline, including in patients who were previously progressing on patisiran Continue to observe generally favorable safety and tolerability data in the full Phase 1 cohort with no new drug-related adverse events within the follow-up period Enrollment continues to progress well in MAGNITUDE-2, which is designed to measure clinical outcomes (including mNIS+7) and evaluate how a single dose of nex-z can lead to reduction in serum TTR, to potentially support a BLA submission by 2028CAMBRIDGE, Mass., May 18, 2025 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused...