Alport Syndrome

Sanofi ACT - Alport Syndrome

Drug name: Lademirsen (SAR339375)

• Phase 2, double-blind, placebo-controlled
• Mechanism: inhibition of microRNS-21 to prevent fibrosis and disease progression

Key inclusion criteria:

• Alport syndrome
• GFR 35 – 90 at screening
• Males 18-23 years old with eGRF < 90 OR ACE inhibitor/ARB dosing stable for 30 days prior to screening OR Prior eGFR Slope Criteria is defined as a decline in eGFR of ≥4 mL/min/1.73m2/year(eGFR slope ≤-4 mL/min/1.73m2/year)based on a linear regression slope analysis of≥4eGFR measurements within 3 years prior to the study and with a minimum of 2-yeartime span

Exclusion:

• Significant/unstable cardiac disease in last 6 months