Paradigm Biopharmaceuticals Ltd. announced that it is scheduled to present data from the open-label phase 2 study of pentosan polysulfate sodium (PPS) for mucopolysaccharidosis type I (MPS-I), and an update on the ongoing multi-centre double- blind randomised phase 2 study comparing PPS to placebo in mucopolysaccharidosis type VI (MPS-VI) patients as an oral presentation at the 2023 ICLD meeting. The mucopolysaccharidoses and related disorders belong to a group of more than 40 inherited lysosomal storage diseases. Lysosomes are the recycling centres of all cells that break down excess or worn-out cell parts with their digestive enzymes.

Mucopolysaccharidoses disorders are due to errors with one of the enzymes that break down and recycle glycosaminoglycans (GAGs), previously known as mucopolysaccharides. As these waste products cannot be eliminated, they accumulate within the lysosomes of virtually every type of cell in the body, causing cells, tissues, and organs to function abnormally, leading to progressive damage. The heart, bones, joints, respiratory system, and central nervous system, including cognitive function, may eventually be affected.

In most cases, symptoms are not apparent at birth, but emerge gradually as a result of defective lysosomal storage and resulting cell damage over time (1,2). Eleven different types of mucopolysaccharidosis have been described, where each is the result of a deficiency in one of the enzymes in the glycosaminoglycan degradation pathway. Key Highlights: Paradigm has been invited to present results from the mucopolysaccharidosis (MPS) program, with an oral presentation at the XVII International Conference on Lysosomal Diseases (ICLD 2023), to be held in Sydney, Australia, February 20-21, 2023.

Dr Drago Bratkovic, Head of the Metabolic Clinic at the Adelaide Women's and Children's Hospital, will present the research entitled: Pentosan Polysulfate Sodium: A Potential Treatment to Improve Bone and Joint Manifestations of Mucopolysaccharidosis I. Three of the four subjects included in Paradigm's open-label Phase 2 trial of pentosan polysulfate sodium (PPS) in MPS-I have completed the 48-week study, with a 6-month treatment extension available. Preliminary data from the MPS-I study were previously presented at 14th International Congress of Inborn Errors of Metabolism in Sydney in 2022 and indicated favourable clinical responses and overall tolerance to PPS. In Paradigm's MPS-VI phase 2 trial based in Brazil, 50% of planned number of subjects have been recruited to the 24-week study comparing PPS to placebo in a blinded, randomised and controlled trial.

The Safety Monitoring Physician for the MPS-VI study confirmed successful evaluation of subjects aged 16 and above and the study is now scheduled to assess PPS in two younger cohorts (9 to 16 years, then following another safety review, 5 to 9 years). During the Bio International Partnering conference in June 2022, Paradigm saw significant interest in its rare disease clinical development program. Paradigm is currently exploring strategic partnerships to progress current and future clinical studies to further evaluate PPS as a treatment to address the critical unmet need in MPS patients.