M6P Therapeutics Presented Data on M041, A Recombinant Enzyme Therapy, for the Treatment of Sanfilippo B Syndrome at MPS 2021

 M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced pre-clinical proof-of-concept data on M041 in a poster presentation by Patricia Dickson, M.D., Centennial Professor of Pediatrics and Chief, Division of Genetics and Genomic Medicine, Washington University of St. Louis, titled “Recombinant human NAGLU with improved mannose 6-phosphorylation for Sanfilippo B syndrome” at the 16^th Annual International Symposium of MPS and Related Diseases (“MPS 2021”). These data illustrate M041’s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.

“There are currently no approved therapies for Sanfilippo B syndrome, also known as MPS IIIB. As the study suggests, M6PT’s M041 recombinant enzyme therapy (NAGLU^M6P) employs an efficient and well-trafficked lysosomal enzyme as the key to developing a potentially effective therapy to treat this rare pediatric disorder,” said Patricia Dickson, M.D., Centennial Professor of Pediatrics and Chief, Division of Genetics and Genomic Medicine, Washington University of St. Louis. “I anticipate that M6PT’s platform technology will lead to advancements in therapies for other LSDs.”

Sanfilippo B syndrome is characterized by a defect in the NAGLU gene providing instructions for producing the enzyme alpha-N-acetylglucosaminidase. This enzyme is located in lysosomes, which are compartments within cells that process and recycle different types of molecules. Alpha-N-acetylglucosaminidase is involved in the stepwise breakdown of large molecules called glycosaminoglycans (GAGs).

“These preclinical data further demonstrate the utility of M6PT’s proprietary S1S3 bicistronic expression system as a platform technology with potential to address multiple LSDs. As such, we remain committed to the research and development of best-in-class recombinant enzyme and gene therapies for LSDs,” said Pawel Krysiak, president and chief executive officer of M6P Therapeutics.

Presentation highlights from the poster include:

• M6PT’s ability to express NAGLU with enhanced phosphorylation levels.
• Evidence that M041, phosphorylated alpha-N-acetylglucosaminidase, has improved trafficking and binding to M6P receptors.
• M041 has markedly improved cellular uptake in patient fibroblasts when compared to wild type alpha-N-acetylglucosaminidase.

The study’s conclusion: co-expression of S1S3 phosphotransferase (Ptase) is an effective way to improve the mannose 6-phosphate content of rhNAGLU, making it more suitable for use as a potential recombinant enzyme therapy.