Drug developer Paradigm Biopharmaceuticals (ASX:PAR) has achieved the primary safety endpoint in its phase two multi-centre Maroteaux-Lamy syndrome randomised controlled clinical trial.
The $110.62 million market capitalisation company designed the phase two trial to evaluate the use of its injectable pentosan polysulfate sodium (iPPS) drug compared to a placebo in 13 participants with MPS VI.
Maroteaux-Lamy syndrome (MPS VI) is a progressive condition that causes many tissues and organs to enlarge, become inflamed or scarred, and eventually waste away.
The company reports that an analysis of its study demonstrates that iPPS is a safe adjunctive therapy to enzyme replacement therapy (ERT) for the continual joint pain, stiffness, and functional disability associated with MPS VI.
Paradigm Biopharma Managing Director Paul Rennie says to achieve the primary endpoint of the study, trial participants were dosed with iPPS weekly for 24 weeks, and the treatment was found to be safe and well tolerated in MPS VI patients 5 years old and over.
“Current MPS therapies such as ERT are essential for MPS patients. However, they don’t provide relief from the daily pain and discomfort caused by their disorders.
Alongside our robust osteoarthritis clinical program, Paradigm believes the data produced in both the MPS I and in this phase two study with MPS VI participants will form important data packages for discussions with our key opinion leaders and ongoing commercial activities, to progress the planning and design for the registration of injectable PPS as an adjunctive therapeutic option for patients with MPS I and MPS VI.”
“Current MPS therapies such as ERT are essential for MPS patients. However, they don’t provide relief from the daily pain and discomfort caused by their disorders.”
All participants in the study were on ERT for over one year and at a stable dose for 3 months prior to baseline. Participants included 6 males and 7 females, with 8 participants aged 5 to 16 years of age and 5 participants over 16 years of age.
This is the first placebo-controlled phase two study evaluating the safety and tolerability of iPPS in participants with MPS VI.
The data uncovered from this trial has been presented today (9 February) as a moderated poster presentation at the 20th annual WORLDSymposim — which is an international scientific meeting dedicated to advancing lysosomal disease research.