Date: 2016-04-21
Type of information: Recruitment of the first patient
phase: 1-2
Announcement: recruitment of the first patient
Company: Biomarin Pharmaceutical (USA - CA)
Product: BMN 250 - chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulinlike growth factor 2
Action
mechanism: enzyme replacement therapy. This fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2) is an enzyme replacement therapy. BMRN 250 is delivered directly to the brain using BioMarin's patented technology.
Disease: mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial
details: The BMN 250 development program consists of two independent and complementary, multicenter, international studies. BMN 250-901 is an observational study of the progression of MPS IIIB over time in children. BMN 250-201 is a Phase 1/2 treatment study conducted in two parts, with Part 1 focused on safety and dose escalation. Part 2 consists of eligible patients rolling over from the BMN 250-901 observational study in addition to continued treatment of the patients from Part 1 of the study. Efficacy will be assessed by comparing changes in disease progression in the observational BMN 250-901 study vs. changes observed in Part 2 of the BMN 250-201 Phase 1/2 treatment study.
Latest
news: * On April 21, 2016, BioMarin Pharmaceutical announced that it has enrolled the first patient in a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB). Discovered by BioMarin, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with MPS IIIB receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via intracerebroventricular (ICV) infusion.
BioMarin has three approved therapies to treat different forms of MPS. BMN 250 is a potential fourth therapy in development for the treatment of an MPS disorder.
