- Paradigm Biopharmaceuticals (PAR) says it has received positive interim data from a phase 2 pilot study of pentosan polysulfate sodium (PPS) for the treatment of mucopolysaccharidosis type I (MPS-I)
- Paradigm’s study enrolled three patients with the disease who are now over halfway through the 48-week treatment regime
- The trial found the use of PPS was well tolerated and may address the unmet medical needs for patients who continue to experience pain that affect function following treatment
- The data is set to be presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney
- Paradigm Biopharmaceuticals last traded at $2.19 on November 22
Paradigm Biopharmaceuticals (PAR) said it has received positive interim data from a phase two rare disease trial.
The data comes from a phase 2 pilot study of pentosan polysulfate sodium (PPS) for the treatment of mucopolysaccharidosis type I (MPS-I).
MPS-I is a rare disease caused by reduced levels of an enzyme responsible for the breakdown of glycosaminoglycans (GAG), resulting in the accumulation of GAG in the tissues.
The disorder can cause problems with neurological, skeletal and cardiovascular development, which sees children with a severe form of the disease often not surviving beyond ten years of age. Current standard treatments include bone marrow transplant and enzyme replacement therapy to address the underlying cause of the disease.
Paradigm’s study at the Adelaide Women’s and Children’s Hospital in South Australia enrolled three patients who are now over halfway through the 48-week treatment regime.
The trial found the use of PPS was well tolerated and may address the unmet medical needs for patients who continue to experience pain that affect function following treatment.
After being administered with PPS, patients saw improvements in walk tests, range of motion and other standard tests of activities important to daily function of the patients.
The company also noted changes in the profile of biomarkers suggested PPS has the potential to modulate the inflammatory and joint degenerating biomarkers associated with arthralgia in MPS-I patients.
“We are very encouraged by the overall preliminary study data which supports our clinical development strategy for PPS as a viable treatment for children with residual musculoskeletal symptoms despite standard of care therapy in this difficult to treat disease,” Paradigm interim Chief Executive Officer, Dr Donna Skerrett said.
The data is set to be presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney.
Meanwhile, the company has been preparing a phase 2 open-label study in up to ten patients with MPS-I, which was first initiated in September 2020. The primary aim of the study is to evaluate safety and tolerability of PPS over an initial 48-week period, with a 6-month treatment extension available, in patients treated with the current standard of care.
The open-label study remains ongoing, with additional patient recruitment expected in 2022.
Paradigm Biopharmaceuticals last traded at $2.19 on November 22.