Paradigm’s study could give options to those with this rare incurable disease

Clinical stage biopharmaceutical company Paradigm Biopharmaceuticals (ASX: PAR) has been given the green light to go ahead with a Phase 2 study of their flagship drug, pentosan polysulfate sodium (PPS) in patients with Mucopolysaccharidosis type VI (MPS VI). A big word to digest on a Friday morning, I know.

The inherited disease is caused by a recessive gene and is characterised by severe bone dysplasia, joint inflammation and musculoskeletal issues. Whilst there is no cure, current treatment options include enzyme replacement therapy and pain management.

Paradigm’s latest clinical trial seeks to address pain associated with joint inflammation with PPS. The trial design has been approved by ethics and regulatory bodies in Brazil.

The Principal Investigator will be Dr. Roberto Giugliani, a professor at the Department of Genetics of the Federal University of Rio Grande do Sul and Chief of the Medical Genetics Service at the Hospital de Clinicas de Porto Alegre.

The trial will assess both safety and tolerability of PPS as well as the effect on pain and mobility. The study will also take into consideration pulmonary function, quality of life and subject/parent impressions of responses to therapy.

PPS is currently used in the treatment of bladder pain caused by cystitis however, Paradigm is focused on re-purposing the drug for indication in musculoskeletal disorders. The Company has applied for Investigational New Drug Status with the FDA for PPS as a treatment for osteoarthritis. As of last month the application is still pending.

Paradigm’s CEO, Paul Rennie said: “We are delighted to achieve this regulatory milestone during a busy period with the IND application process with the US FDA for our lead indication in Osteoarthritis. Paradigm will be monitoring the current COVID situation in Brazil to plan the trial commencement and will update the market once a timeline is determined.”

A successful trial outcome could lead to partnering discussions for further studies of PPS use in conjunction with enzyme replacement therapy for MPS VI patients. Enzyme replacement therapy costs between USD $180,000 and USD $540,000 per year.