Presents Data from its Phase 1b Program for Niemann-Pick Type B at the Lysosomal Disease Network’s WORLD Symposium 2015 Presented data from its Phase 1b clinical study at the Lysosomal Disease Networks WORLD Symposium 2015 in Orlando, Fla. detailing the investigational use of enzyme replacement therapy in the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type B), a lysosomal storage disease caused by genetic mutations that affect the metabolism of sphingomyelin. The Genzyme study evaluated the tolerability and safety of olipudase alfa (recombinant human acid sphingomyelinase) in five adult patients with ASMD.
Melissa P. Wasserstein, MD, Director of the Program for Inherited Metabolic Diseases; Medical Director of the International Center for Types A and B Niemann Pick Disease, Mount Sinai School of Medicine, presented: An open-label, multicenter, ascending-repeat-dose study of the tolerability and safety of recombinant human acid sphingomyelinase (rhASM) in patients with ASM deficiency (ASMD). In the trial, each patient received a starting dose of intravenous olipudase alfa at 0.1 mg/kg and escalated dosing every two weeks according to a predetermined schedule up to 3 mg/kg or their maximum tolerated dose. The secondary objective was to study the pharmacokinetics, pharmacodynamics, and exploratory efficacy of olipudase alfa administered every two weeks for 26 weeks. The study findings showed that the dose escalation regimen was well tolerated, with all patients reaching the maximum dose of 3 mg/kg. No serious or severe adverse events or deaths were reported. The data presented on the repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa support its continued development for the investigational use in non-neurological manifestations of ASMD. All five patients are participating in the Long-Term Study and will continue on therapy.
"Though a small number of patients, the response we have observed to date is an early indication that this ASM enzyme replacement therapy is promising for this therapeutic area, said Genzymes Acting Head of Rare Diseases, Richard Peters, M.D., Ph.D. We look forward to continuing this program and learning more as we work toward advancing a treatment option for patients that is both safe and well tolerated. Genzyme plans to begin enrolling patients in a Phase 2/3 program for Niemann-Pick Type B in 2015.