Paradigm Biopharmaceuticals Ltd (ASX:PAR) will present positive interim data on a phase 2 pilot study of pentosan polysulfate sodium (PPS) for the treatment of mucopolysaccharidosis type I (MPS-I) at an international medical congress in Sydney today.
The 14th International Congress of Inborn Errors of Metabolism (ICIEM 2021) will hear that PPS was well tolerated with no serious adverse events reported over a 24-week period.
There have been meaningful improvements in pain, function and activities of daily living while an overall improvement in quality of life was observed in all patients.
What is MPS-I?
MPS-I is a rare disease caused by reduced levels, or the complete lack of, an enzyme responsible for the catabolism (break down) of glycosaminoglycans (GAG) resulting in progressive accumulation of GAG in the tissues.
The disorder causes problems with neurological, skeletal and cardiovascular development. There is no cure and children born with the most severe form of MPS-I do not typically survive beyond 10 years of age, without treatment.
Current standard treatments include bone marrow transplant and enzyme replacement therapy to address the underlying cause of the disease.
Encouraged by study data
During the conference, a poster detailing the promising data will be presented along with a discussion with the head of the Metabolic Unit at Adelaide Women and Children’s Hospital, Dr Drago Bratkovic, and Paradigm global head of Safety and MPS, Dr Michael Imperiale.
Paradigm interim chief executive officer Dr Donna Skerrett said: “We are very encouraged by the overall preliminary study data which supports our clinical development strategy for PPS as a viable treatment for children with residual musculoskeletal symptoms despite standard of care therapy in this difficult to treat disease.”
The study at the Adelaide Women’s and Children’s Hospital, South Australia, has enrolled three patients who are more than halfway through the 48-week treatment regime and PAR’s encouraging data from these patients was presented at ICIEM 2021 by Dr Bratkovic.
Presentation highlights
The poster provides evidence PPS could help address the unmet medical needs of MPS-I patients and supported further studies.
Summary highlights of the poster presentation include:
- PPS may address the unmet medical needs for patients who continue to experience pain and symptoms that affect function following treatment with the best current standard of care;
- PPS was well tolerated with no serious adverse events reported over a 24-week period;
- Meaningful improvements in pain, function and activities of daily living and an overall improvement in quality of life was observed in all patients;
- Administration of PPS resulted in improvements in 2- and 6-minute walk tests, range of motion and other standard tests of activities important to daily function of the patients;
- Pharmacokinetic results demonstrated consistency in serum concentrations that were dose-dependent; and
- Changes in the profile of biomarkers suggest PPS has the potential to modulate the inflammatory and joint degenerating biomarkers associated with arthralgia in MPS-I patients.
A phase 2 open-label study in up to 10 patients with MPS-I was initiated in September 2020. The primary aim is to evaluate safety and tolerability of PPS over an initial 48-week period, with a 6-month treatment extension available, in patients treated with the current standard of care.
Secondary and exploratory objectives include examining the effects of PPS on pain, function, and quality of life, pharmacokinetics, biomarkers, and inflammatory processes.
Study ongoing
The open-label study remains ongoing with additional patient recruitment expected in CY22. Paradigm is exploring strategic partnerships to progress current and future clinical studies to further evaluate PPS as a treatment to address the critical unmet need of ongoing musculoskeletal symptoms in this very rare patient population.
After conclusion of the conference, the poster presentation will be available on the Paradigm website.
About injectable PPS
PPS is a medication that has been used in humans for more than 60 years. Injectable PPS has previously been approved in European markets, where it is registered as an antithrombotic agent.
In Australia, injectable PPS for human use is not currently available for sale. Injectable PPS is available via a Paradigm sponsored clinical trial or under the TGA Special Access Scheme to physicians for individual patients who satisfy strict criteria and is subject to approval from the TGA.
Elmiron (the oral formulation utilised for interstitial cystitis) is the only PPS product approved in the US. A subcutaneous injectable formulation of PPS is being evaluated by Paradigm for the treatment of osteoarthritis and other inflammatory diseases in the US and other major global markets.