2023 ACMG Annual Clinical Genetics Meeting Digital Edition: Poster Gallery-Results of an Open-label, Single-Center, Clinical Study Evaluating the Safety and Tolerability of Pentosan Polysulfate Sodium in Mucopolysaccharidosis I Subjects

Results of an Open-label, Single-Center, Clinical Study Evaluating the Safety and Tolerability of Pentosan Polysulfate Sodium in Mucopolysaccharidosis I Subjects

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A phase 2 clinical trial investigated the therapeutic effects of Pentosan Polysulfate Sodium (PPS) on knee osteoarthritis. The trial aimed to assess the safety and tolerability of PPS and explore its efficacy in reducing pain, inflammation, and promoting cartilage protection. The study included physical assessments of mobility and dexterity such as the 2-minute walk test, 6-minute walk test, and 9-hole peg test. Patient-reported outcomes were measured using various forms of the Patient-Reported Outcomes Measurement Information System (PROMIS) and the Patient Global Impression of Change (PGIC). Safety assessments included monitoring treatment-emergent adverse events and changes in clinical laboratory data. Four subjects with MPS I-Hurler, both male and female, were enrolled in the study.<br /><br />The results showed that PPS was well tolerated at doses of 0.75mg/kg or 1.5mg/kg over 48 weeks of dosing. Subjects reported improved mobility and function based on the physical assessments. Patient-reported outcomes indicated improvements in pain and quality of life. The study also highlighted the mechanisms of action of PPS in reducing inflammation, pain, and preserving tissue integrity. The authors concluded that PPS shows promise in improving pain and function issues related to MPS I.<br /><br />Mucopolysaccharidosis I (MPS I) is a rare genetic disorder caused by a deficiency of the enzyme α-L-iduronidase, leading to the accumulation of glycosaminoglycans (GAGs) and resulting in multiorgan dysfunction. The current treatment approaches for MPS I include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT). PPS, a polysulfated semi-synthetic non-biologic, has shown potential therapeutic effects in reducing pain and inflammation in osteoarthritis and may provide beneficial effects in MPS I.<br /><br />The study was funded by Paradigm Biopharmaceuticals Ltd. PPS was found to be safe and well-tolerated, with no serious adverse events reported. PPS treatment showed improvements in pain and physical function in patients with MPS I. The authors acknowledged the contributions of other researchers and the funding support of Paradigm Biopharmaceuticals Ltd.

Asset Subtitle

Co-Author - Curtis Gravance, PhD; Co-Author - Drago Bratkovic, MD MBBS FRACP; Co-Author - David Ketteridge, MD; Presenting Author - Michael Imperiale, MD; Co-Author - Ravi Krishnan, PhD; Co-Author - Divya Navuru, BPharm, CCDM;

Meta Tag

Therapy

Lysosomal Diseases

Enzyme Replacement Therapy

Co-Author Divya Navuru, BPharm, CCDM

Co-Author Ravi Krishnan, PhD

Co-Author David Ketteridge, MD

Co-Author Drago Bratkovic, MD MBBS FRACP

Co-Author Curtis Gravance, PhD

Presenting Author Michael Imperiale, MD

Keywords

Pentosan Polysulfate Sodium

PPS

knee osteoarthritis

therapeutic effects

safety and tolerability

pain reduction

inflammation reduction

cartilage protection

physical assessments

patient-reported outcomes