Paradigm Biopharmaceuticals Ltd (ASX:PAR) has passed another key milestone by successfully completing a safety review for its ongoing multi-centre double-blind randomised phase 2 study comparing pentosan polysulfate sodium (PPS) to placebo in mucopolysaccharidosis type VI (MPS-VI) patients.
The safety monitoring physician for the trial being conducted in Brazil has confirmed that the safety review has been completed with no serious adverse events reported in the 9- to 16-year-old cohort.
This milestone now allows the inclusion of subjects aged 5 to 9 years to assess the safety and tolerability of PPS amongst this paediatric population.
Mucopolysaccharidosis type VI, also known as Maroteaux-Lamy syndrome, is an ultrarare autosomal recessive lysosomal storage disorder that affects between 0.36 and 1.30 of every 100,000 live births.
Important for commercial potential
Paradigm CEO Marco Polizzi said: “It is pleasing to have reached yet another milestone in this phase 2 MPS study.
“The ability to include the youngest patient cohort following a positive safety review is important for the commercial potential of PPS to treat the residual symptoms that impact the daily activities of MPS sufferers.
“Paradigm looks forward to further updates as we progress toward the completion of this study in calendar year 2023.”
Trial summary
Brazil has one of the largest populations of MPS-VI patients globally so researchers there are evaluating the use of Paradigm’s PPS to treat MPS-VI patients in a phase 2 study.
The study is randomised, double-blind and placebo-controlled to evaluate the safety and tolerability of PPS in patients with MPS-VI.
According to the study protocol, approximately 12 patients will be randomised 2:1 to receive PPS or placebo.
Participants are dosed weekly for 24 weeks with the primary endpoint being safety. The secondary endpoints are improvements in pain and function.
This study is the largest of its kind in the world and has attracted the interest of medical researchers and MPS patient advocacy groups globally.
Paradigm’s global head of Safety and head of the MPS program, Dr Michael Imperiale, said “We are working closely with our sites to provide comprehensive monitoring, and pleased to report that patients are tolerating the therapy well as we work to address this important unmet medical need in MPS VI patients.”
Business update
Meantime, Paradigm also confirms the release of the top-line PARA_OA_008 and preliminary analysis of PPS-treated canines that have completed the day-56 follow-up, during September.
Early interim observations in osteoarthritic dogs administered PPS subcutaneously at a dose of 3 mg/kg (human equivalent dose of 1.7 mg/kg) weekly for six weeks, will also be reported with the PARA_OA_008 human clinical trial data.
This will be an early examination of dogs that have completed the day 56 follow-up, with further data from the 20-week follow-up (3-year human equivalent) to be reported next year.