Paradigm Biopharmaceuticals Ltd (ASX:PAR) has completed dosing of all ten patients with Zilosul® under the FDA’s Investigational New Drug Expanded Access Program (EAP) in the US.
All patients taking part in the study must complete regular evaluations with the treating physician, with the final evaluation taking place six weeks post the last injection received (day 83).
The EAP program will report on pain outcomes similar to those previously evaluated in the Therapeutic Goods Administration Special Access Scheme (TGA SAS) as well as those proposed as endpoints for the phase 3 clinical trial.
Earlier this month, Paradigm reported a 45% mean reduction in pain score across 34 patients with knee osteoarthritis (OA) using Zilosul as part of the TGA SAS.
Paradigm expects data for the EAP program’s total population (n=10) of patients to be available to the market during the third quarter of this calendar year.
Paradigm chief executive officer Paul Rennie said: “Paradigm is excited to have achieved this milestone, especially during the current health conditions created by COVID-19 and would like to thank all those involved in the Expanded Access Program for their continued diligence to the treatment program.”
Orphan designation granted
The company has also been advised by the FDA that its orphan designation request for MPS (mucopolysaccharidosis) Type-I has been granted, joining the previously granted designation for MPS Type-VI.
MPS-I is a rare inborn metabolic disorder caused by a genetic defect in the catabolism of two glycosaminoglycans (GAGs): heparan sulfate and dermatan sulfate.
US$1.4 billion market potential
Current treatment for MPS patients includes Enzyme Replacement Therapy (ERT) which acts to
reduce non-neurological symptoms and pain.
MPS patients undergoing approved ERT however, continue to report ongoing stiffness, pain, inflammation, and heart and airway soft tissue manifestations.
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, with BioMarin’s ERT treatments costing US$300,000 – US$600,000 per annum.
Paradigm believes iPPS (Zilosul) may be an effective adjunct/combination therapy with current ERT treatments.