Shots:

• The P-II/III ASCEND trial involves assessing olipudase alfa 3 mg/kg (IV- q2w) vs PBO in 36 adult patients with ASMD type B to evaluate treatment impact on pulmonary function & spleen & liver volume for 52wks. The study demonstrated improvement in lung function (22% vs 3%)- reduction in spleen volume (39.5% vs 0.5%)- reduction in liver volume (31.7% vs 1.4%); improvement in platelet counts (16.8% vs 2.5%)
• The P-II ASCEND-Peds trial involves assessing olipudase alfa 3 mg/kg (IV) q2w for 64 wks- in 20 pediatric patients with ASMD without acute or rapidly progressive neurological abnormalities. The study demonstrated 33% increment in predicted DLCO- reduction in spleen volume & liver volume (49% & 41%); 34% increment in platelet count
• Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM- allowing for the breakdown of sphingomyelin- currently being investigated to treat non-CNS manifestations of ASMD. The therapy has received FDA’s BT designation- EMA’s PRIME Designation and MHLW’s SAKIGAKE designation

 ­ Ref: Sanofi | Image: Evaluate Pharma