Paradigm Biopharmaceuticals Ltd (ASX:PAR) is poised to present data from its open-label Phase 2 study on pentosan polysulfate sodium (PPS) at the 2023 International Conference on Lysosomal Diseases (ICLD) meeting.
The presentation will examine PPS for mucopolysaccharidosis type I (MPS-I), and provide an update on the ongoing multi-centre double-blind randomised Phase 2 study comparing PPS to placebo in mucopolysaccharidosis type VI (MPS-VI) patients.
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 four subjects in Paradigm’s MPS-1 study have completed the full 48 weeks allotted, with preliminary data – which was presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney in 2022 – indicating favourable clinical responses and overall tolerance to PPS.
Paradigm welcomes “global recognition”
“Current MPS therapies are essential for MPS patients, however, they don’t provide relief from the daily pain and discomfort caused by their disorders,” Paradigm global head of Safety and head of the MPS program Dr Michael Imperiale said.
“We are very excited by the global recognition of Paradigm’s clinical development in this rare disease and the opportunity to present the exciting work we are undertaking at the International Conference on Lysosomal Diseases early next year.”
The second study being presented on – the MPS-VI study in Brazil – has reached 50% of planned subject recruitment for the 24-week study, which will compare PPS to placebo in a blinded, randomised and controlled trial.
The Safety Monitoring Physician for the MPS-VI study has given the go-ahead to assess PPS in subjects younger than 16 years old, and Paradigm is now scheduled to assess the treatment in two younger cohorts – nine to 16 years, followed by a safety review, and then five to nine years.
Strategic partnerships in the works
“Alongside our robust osteoarthritis clinical program, Paradigm is proud to work with specialists in the field of lysosomal storage diseases to potentially enable MPS sufferers to function more easily in their day-to-day activities,” Paradigm Biopharmaceutical CEO Marco Polizzi said.
“We are continuing discussions to progress the development of PPS for patients with MPS and believe that this data will contribute to planning and design for the registration of injectable PPS as an adjunctive therapeutic option for patients with MPS-I and MPS-VI.”
Paradigm saw ‘significant’ interest in its rare disease clinical development program during the Bio International Partnering conference in June 2022.
The company is now exploring strategic partnerships to progress current and future clinical studies designed to evaluate PPS as a treatment to address the critical unmet need in MPS patients.