BMN 250 is a new fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2).
The company has also started IND-enabling studies and intends to begin clinical trials with BMN 250 in mid-2015.
Discovered by BioMarin, BMN 250 is an enzyme replacement therapy that uses recombinant human NAGLU with an IGF2, or Glycosylation Independent Lysosomal Targeting (GILT) tag and it is delivered directly to the brain using the company’s patented technology.
The company has also issued patents which broadly cover delivery of lysosomal enzymes directly into the cerebrospinal fluid to treat lysosomal storage diseases.
BioMarin chief executive officer Jean-Jacques Bienaimé said the company is happy to add a new candidate to its pipeline that could be a potentially first-in-class therapy for Sanfilippo B patients who currently have no drug treatment options available.
"Developing BMN 250 for Sanfilippo B or MPS IIIB brings together the best of BioMarin’s scientific and clinical expertise," Bienaimé said.
"We are building upon a deep knowledge of MPS diseases, and experience developing fusion proteins and enzyme replacement therapies overall.
"Adding a fourth treatment for MPS complements our current franchise of two approved therapies for the treatment of MPS I and MPS VI and a third expected for MPS IVA."
The company intends to present the data on the NAGLU fusion protein at the Lysosomal Disease Network’s (LDN) 10th Annual WORLDSymposium being held February 11-13 in San Diego, California.
Image: BioMarin plans to start clinical trials with BMN 250 in mid-2015. Photo: courtesy of tiverylucky/ freedigitalphotos.net