Dr Roberto Giugliani Source: bipmed.org
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  • Paradigm Biopharmaceuticals (PAR) doses its first patient as part of the largest clinical trial ever conducted on Maroteaux-Lamy syndrome
  • The patient received a randomised dose as part of a Phase 2 clinical trial, which is looking to evaluate pentosan polysulfate sodium (PPS) as a treatment for the disease
  • Meanwhile, interim data from the company’s ongoing Australian Phase 2 study in MPS-I is set to be presented at the International ICIEM meeting in November 2021
  • Paradigm Biopharmaceuticals is trading in the grey at $2.17 at 1:40 pm AEST

Paradigm Biopharmaceuticals (PAR) has dosed its first patient as part of a Maroteaux-Lamy syndrome study in Brazil.

A patient with Maroteaux-Lamy syndrome (MPS-VI) has received a randomised dose as part of a Phase 2 clinical trial, which looks to evaluate pentosan polysulfate sodium (PPS) as a treatment for the disease.

MP-VI is a rare disorder caused by the deficiency of Nacetyl galactosamine 4–sulfatase, which can cause corneas, deafness, thickening of the dura (connective tissue that make up the meninges of the brain), and pain caused by compressed or traumatized nerves. In more severe cases, children can also develop a protruding abdomen and forward-curving spine. It can also cause learning difficulties, dementia and mild somatic abnormalities.  

The study is designed to enrol 12 patients with MPS-VI who are currently receiving enzyme replacement therapy (ERT) and exhibit pain and functional deficiency due to musculoskeletal symptoms. This will mark the largest clinical trial conducted using PPS in any MPS population.

Each subject in the trial will receive ERT throughout the study, along with PPS or a placebo, with the primary objective of evaluating its safety and tolerability.

On top of this, Paradigm will assess a number of key secondary and exploratory endpoints, including the effect of PPS on pain and function, mobility, urinary, mobility, quality of life, activities of daily living and pulmonary function.

“MPS-VI patients on Enzyme Replacement Therapy continue to experience stiffness, pain, inflammation, and heart and airway soft tissue manifestations,” principal investigator of the trial Roberto Giugliani said.

“Pentosan polysulfate sodium has been shown to reduce urinary GAGs and improve joint mobility and pain that impact Activities of Daily Living in MPS-I patients.

“Likewise, preclinical studies of PPS in Maroteaux-Lamy syndrome (MPSVI) have demonstrated similar results, which may translate to significant clinical benefit for this patient population. We strongly believe that PPS has the potential to address this unmet medical need in the MPS-VI patient population,” Dr Giugliani concluded.

Meanwhile, interim data from the company’s ongoing Australian Phase 2 study in MPS-I is set to be presented at the International ICIEM meeting in November 2021.

Paradigm Biopharmaceuticals was trading in the grey at $2.17 at 1:40 pm AEST.

PAR by the numbers
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