- Paradigm Biopharmaceuticals’ (PAR) phase two clinical trial is well on its way, with the first participant dosed with injectable pentosan polysulfate sodium
- The study primarily assesses the safety and tolerability of the drug in patients with Mucopolysaccharidosis type I (MPS-I) and secondly, its ability to alleviate pain and symptoms
- MPS-I is a metabolic disorder which can cause abnormal growth and bone development, cardiac and respiratory problems and cognitive impairment
- The clinical trial will enrol 10 people, aged five or above, whose symptoms persist despite receiving other treatments
- Earlier this year, the drug was granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency
- Shares have been trading 2.6 per cent higher at $3.16
Paradigm Biopharmaceuticals’ (PAR) phase two clinical trial is well on its way, with the first participant dosed with injectable pentosan polysulfate sodium.
The study primarily assesses the safety and tolerability of the drug in patients with Mucopolysaccharidosis type I (MPS-I) and secondly, its ability to alleviate pain and symptoms.
MPS-I is a metabolic disorder which can cause abnormal growth and bone development, cardiac and respiratory problems and cognitive impairment.
The clinical trial will enrol 10 people, aged five or above, who have received Enzyme Replacement Therapy (ERT) or Hematopoietic Stem Cell Therapy, yet musculoskeletal symptoms, such as pain, persist.
CEO Paul Rennie said the trial will shed light on whether pentosan polysulfate sodium (PPS) could work alongside existing treatments.
“The data collected from the Phase 2 trial will be vital to support Paradigm’s future regulatory filings and applications for the development of PPS as a potential adjunctive therapy to Enzyme Replacement Therapy treatments,” Paul stated.
“Our MPS programs will treat subjects as adjunct to ERT as well as previously bone marrow transplanted patients who may or may not remain on ERT,” he explained.
PPS has previously been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency, which provides for seven years of regulatory exclusivity.
The late-stage drug development company also wants to commercialise PPS for the treatment of pain in other musculoskeletal disorders, such as osteoarthritis.
Shares have been trading 2.6 per cent higher at $3.16 at 10:10 am AEDT.