Paradigm Biopharmaceuticals Ltd (ASX:PAR) has outlined the results of 24 patient case studies with Osteoarthritis (OA) and Bone Marrow Edema Lesions (BMELs) that were treated with Pentosan Polysulfate Sodium (PPS) under the Therapeutic Goods Administration’s (TGA) Special Access Scheme.
PPS is a new multi-acting treatment.
In the 24 patients with a median age of 57.5 years (range 31 to 84 years), joint pain was reduced in 83% and knee function was improved in 80% of all cases treated.
These case studies are important to Paradigm as they provide Real World Evidence (RWE) data – i.e. the data from sources other than traditional clinical trials.
The United States Food and Drug Administration allows RWE data to be used in combination with Randomised Controlled Clinical Trials to support product registration for repurposed drugs such as PPS under the FDA 505(b)(2) regulatory pathway.
Next steps for Paradigm
In addition to the 24 case studies, Paradigm plans to generate an additional 75 case studies by Q4 CY2018.
These additional 75 case studies will be administered with increased doses compared to the first 24 cases.
This means by Q4 CY2018, Paradigm will have RWE data on 100 case studies in addition to the results of its Phase 2b randomised, double-blind, placebo-controlled clinical trial, which commenced in September 2017 and will enroll a total of 100 patients.
In the U.S. alone, the financial burden of OA has been estimated to be US$81 billion in medical costs and US$128 billion in total costs, given around 21 million people have OA associated limitations, while there are 36 million outpatient visits and 750,000 hospitalisations per year.
In Australia, arthritis affects around three million people or around 15% of the population.
OA is the leading cause of pain and disability among the elderly in Australia and the third-leading cause of life-years lost due to disability.