Paradigm Biopharmaceuticals Ltd (ASX:PAR) has welcomed the acceptance of an abstract on the company’s Phase 2 clinical trial evaluating injectable Pentosan Polysulfate Sodium (iPPS) against placebo in mucopolysaccharidosis type VI (MPS-VI) for a poster presentation to the WORLDSymposium in 2023.
The WORLDSymposium is the annual research conference focused on new therapies for lysosomal storage diseases, including MPS-VI, which is an inherited metabolic disease caused by the absence or malfunction of vital enzymes within the body.
Dr Roberto Giugliani MD, PhD, MSc, the principal investigator of Paradigm’s phase 2 clinical trial, will be presenting the poster, which provides an update on the enrolment and baseline characteristics of MPS-VI participants in the trial.
Encouraged by global support and interest
“This study is evaluating a potential treatment for the residual musculoskeletal symptoms suffered by MPS patients despite being on enzyme replacement therapy (ERT) for one year or more,” Paradigm Pharmaceuticals global head of safety and head of the MPS program, Dr Michael Imperiale said.
“It is pleasing to see continued global support and interest from medical researchers and MPS patient advocacy groups in the work Paradigm is undertaking.”
IPPS is a non-opioid subcutaneous injectable with the potential to treat residual musculoskeletal symptoms in MPS as an adjunct therapy to current standards of care.
Previous studies have shown iPPS improves pain and function in patients with MPS-I, MPS-II and MPS-VI.
“The data from this trial will be invaluable for further development for potential registration of iPPS for this indication,” Paradigm Pharmaceuticals CEO Marco Polizzi said.
“We hope that iPPS can address the unmet need of patients with lysosomal storage diseases as a key adjunct therapy that provides relief from painful and inflamed joints to improve their quality of life.”