Paradigm Biopharmaceuticals Ltd (ASX:PAR) has drawn attention in the medical community with a presentation from medical investigators introducing the company’s Phase 2 trial for the treatment of mucopolysaccharidosis type-VI (MPS VI) with Paradigm’s Pentosan Polysulfate Sodium (PPS) treatment.
The Phase 2 study design was presented to delegates at the WORLD Symposium in San Diego, California by Dr Roberto Giugliani, a leading geneticist and principal investigator.
The WORLD Symposium is the annual research conference focused on new therapies for lysosomal storage diseases such as MPS VI.
MPS-VI, or Maroteaux-Lamy Syndrome, is an inherited orphan disease classified as a rare autosomal recessive lysosomal storage disorder that affects between 0.36 and 1.30 of every 100,000 live births.
An unmet need to be addressed
Dr Giugliani, Principal Investigator, told the conference “Most MPS VI patients treated with enzyme replacement therapy still continue to experience pain and symptoms that reduce their quality of life.
“This is an unmet need to be addressed.”
Pentosan Polysulfate Sodium (PPS) is a semi-synthetic drug manufactured from European beech xylans that mimics glycosaminoglycans (GAGs).
These complex carbohydrates have a regulatory role in the body, interacting with proteins involved in inflammation. PPS also has a mild antithrombotic (anti-clotting) activity.
Brazil has one of the highest rates of MPS VI and researchers are evaluating the use of Paradigm’s PPS compared to a placebo in 12 patients in a 2:1 randomised trial.
Participants are dosed weekly for 24 weeks with the primary endpoint being safety. The secondary endpoints are improvements in pain and function.
The study is the largest of its kind in the world and has attracted the interest of medical researchers and MPS patient advocacy groups globally.
Encouraging levels of interest
Paradigm Biopharma global head of safety and head of MPS Dr Michael Imperiale is also attending the conference, where the company has both a physical and virtual booth to conduct meetings with parties interested in exploring the opportunity to license PPS for the treatment of pain and function in MPS patients.
Dr Imperiale said, “The level of interest from potential licensing partners in Paradigm’s MPS clinical program is encouraging, with several meetings scheduled during the conference.”
Paradigm also published insights from a landmark focus group among patients with MPS VI and their care givers in the Orphanet Journal of Rare Diseases.
Nine patients and care givers outlined the daily challenges of living with MPS VI and the painful and debilitating symptoms endured despite receiving enzyme replacement therapy.
The findings of the focus group highlight the unmet needs in this patient group.
Paradigm says they are important since patient-related outcomes in rare diseases are under-reported, and yet are a crucial part of assessing treatment response and measuring outcomes of current and novel treatment approaches.
Insights from the focus group have informed the clinical outcome assessments and endpoints for Paradigm’s MPS clinical trial program.
About Paradigm Biopharmaceuticals
Paradigm Biopharmaceuticals is a drug repurposing company driven by core competencies and experience at board and executive level in clinical and commercial pharmaceutical development.
The company’s approach is to take an existing approved drug, which has demonstrated safety in its approved indications, and repurpose the drug in a new patented therapeutic application.
Paradigm’s pipeline indications cover the use of pharmaceutical compound Pentosan Polysulfate Sodium (PPS), supported by full patent and IP protection, and its immediate commercial focus is the repurposing of PPS for the treatment of Osteoarthritis (OA).