Paradigm Biopharmaceuticals (ASX: PAR) has set its sights on applying to the US Food and Drug Administration (FDA) to begin a clinical trial for mucopolysaccharidoses (MPS) following a positive round of discussions with international leaders in the field.
The company provided a comprehensive update to shareholders today following a recent satellite symposium in Orlando, US, where it met with international key opinion leaders in the field of MPS.
The event, held in early February, included a series of panel discussions that addressed the unmet medical need in MPS types with musculoskeletal pain and dysfunction.
According to the company, the current standards of care for MPS are not adequate in treating pain associated with joint inflammation and musculoskeletal issues.
“The evidence presented from clinicians regarding the need for a product to assist with pain associated with musculoskeletal problems associated with MPS was very relevant to Paradigm’s proposed forthcoming MPS clinical trial,” the company said.
Paradigm highlighted some key findings presented at the symposium, bringing to light work by Dr Furujo.
According to the company, his findings further demonstrated that injectable pentosan polysulfate sodium (iPPS) could have a positive impact on pain and physical function in patients suffering from MPS while maintaining an excellent safety profile.
Importantly, Paradigm said the results from its phase 2a MPS clinical trial data were consisted with Dr Furujo’s findings.
Paradigm recently announced it would look to fast-track designation for its iPPS through engaging with US-based regulatory consultants.
Additional MPS trials in the pipeline
Paradigm is confident a successful phase 2/3 clinical trial will lead to iPPS being registered as a treatment for certain MPS indications, paving the way for commercial success in treating the disease.
The company’s clinical and regulatory team, in conjunction with the key opinion leaders, will now prepare a detailed study protocol and regulatory documents for the phase 2/3 trial.
The study will be a multi-national, multi-centre trial likely incorporating centres in Australia, US, UK and Germany.
Working in Paradigm’s favour is the fact that MPS is classified as an orphan indication in the US and EU, which it believes should help accelerate the regulatory approval process.
Paradigm expects to submit the trial documentation to the designated regulatory bodies in the second quarter of 2019.
As a progressive rare disease, MPS is caused by inherited defects in the catabolism of sulfated components of connective tissue known as glycosaminoglycans (GAGs).
The rate for all types of MPS is around 3.5 in 100,000 live births.
Paradigm noted that a successful clinical trial and drug registration would likely result in a “rapid take-up of the product once commercially available and attract a premium pricing as is normally the case with orphan indications”.
Big pharma currently has a growing interest in orphan indications, and the company said it would continue to engage with potential big pharma partners.
Paradigm’s shares jumped 1.6% higher in late morning trade to $1.215.