MPS is a progressive rare disease caused by the absence or malfunctioning of lysosomal enzymes and has a severe unmet need.
Paradigm is focused on repurposing the drug pentosan polysulfate sodium in its injectable form (iPPS), a US Food and Drug Administration (FDA) approved drug that has a long track record of safely treating inflammation.
The company conducted a satellite meeting on February 3-4th at the “World Symposium 2019, 15th Annual Research Meeting on Lysosomal Disease Research” in Orlando, USA to discuss plans for its upcoming multinational phase 2/3 MPS clinical trial.
The clinical trial discussion panel included world-renowned KOLs in lysosomal storage disease and MPS from USA, UK, Germany and Japan, members of Paradigm’s clinical team and the manufacturer of PPS - bene pharmaChem.
Furthermore, Paradigm’s recently acquired MPS clinical data was further validated at the World Symposium by a paper presented by Dr Furujo that demonstrated iPPS has a positive impact on pain and physical function in patients with MPS types I, II and VI.
Upcoming phase 2/3 clinical trial
Paradigm is confident that a single successful phase 2/3 clinical trial will enable iPPS to be registered as a treatment for certain MPS indications.
MPS is classified as an orphan indication/designation in the US and EU and this should help accelerate the regulatory approval process and ultimately the time it takes for the treatment to enter the market as well as providing for a minimum 7 years (10 for EU) market exclusivity.
The panel concurred that the trial design will likely involve patients receiving ERT or BMT as standard of care and that PPS treatment be used as an adjunct therapy (i.e. therapy that is given in addition to the primary or initial therapy to maximize its effectiveness).
Paradigm’s clinical and regulatory team in conjunction with the KOLs will be preparing a detailed study protocol and regulatory documents for the phase 2/3, which will be a multi-national, multi-centre trial likely incorporating centres in Australia, USA, UK and Germany.
It is anticipated these documents will be submitted to the regulatory agencies in Q2 CY2019
The current standards of care are not adequate in treating pain associated with joint inflammation and musculoskeletal issues and these drugs currently equate to a market size of around US$1.4 billion per annum.
Big Pharma have a growing interest in orphan indications given the exclusivity from regulators and strong demand due to the critical unmet needs and attractive margins.
Paradigm will continue to actively engage with potential big pharma partners on this blockbuster indication.