Paradigm Biopharmaceuticals Ltd (ASX: PAR) has achieved a number of critical developments required to progress investigation into the use of its core drug Pentosan Polysulfate Sodium (PPS) in the treatment of the rare disease mucopolysaccharidosis (MPS).
The company has gained ethics approval for its proposed pilot study for the ultra-rare Orphan disease Mucopolysaccharidosis Type 1 (MPS-1). The ethics approval is validation from the medical / scientific community that PPS may provide benefit to patients who continue to experience this unmet need.
Chairman and CEO, Paul Rennie, said the company has also received approval from the European Medical Agency (EMA) for its Orphan designation application for MPS-VI. Such approvals from the EMA can take up to 90 days; Paradigm received a decision on Day 60 of the process.
Mr Rennie said the company has also received positive feedback from a Parallel Scientific Advice (PSA) meeting with the Federal Drug Administration (FDA) and EMA.
Paradigm received clarification on the design of the clinical program and feedback on the regulatory pathway for its submission for a Phase 2/3 Clinical trial in MPS-VI.
Paradigm recently reported very positive pain reduction and functional improvement data in 10 ex- NFL players with osteoarthritis treated under the US FDA Expanded Access Programme (EAP),” Mr Rennie said.
“The mean reduction in WOMAC pain from baseline was 65% and this compares very favourably with the common oral treatments for patients with Osteoarthritis of roughly 30% reduction in WOMAC pain from baseline (NSAID’s and opioids).
“We had a number of important take-aways from the EAP notably (i) Paradigm was able to execute on this programme, in the USA, during the height of the COVID- 19 pandemic (on time and on budget), (ii) the EAP used the same batch of Zilosul (iPPS) that will be used in Paradigm’s Phase 3 clinical trial as well as using the same pain scoring system (WOMAC), and (iii) all subjects who commenced the program completed it with no drop outs and no serious adverse events reported. So good safety and outstanding efficacy are very pleasing outcomes as Paradigm prepares for its Phase 3 clinical trial.
“I am pleased to report that the second clinical program in our pipeline has also made considerable progress. MPS has gained orphan disease status from both the US FDA and EMA.
“Additionally, positive feedback on Paradigm’s clinical trial protocol and regulatory pathway was received from both the US FDA reviewers and EMA assessors at the Parallel Scientific Advice meeting. Paradigm is pleased to receive very clear directions on the regulatory pathway for the MPS programme.
“It is also important validation from the Investigators and Ethics Committee at the internationally recognised MPS centre, the Women’s and Children’s Hospital in Adelaide, with the Ethics Committee approval to commence a Phase 2 clinical trial in MPS-1. Current treatment options for MPS-1 (such as bone marrow transplant or Enzyme Replacement Therapy (ERT)) have limited effects on some organs, especially the skeletal system, with MPS-1 patients experiencing joint pain and dysfunction.
“The goals of this study are to investigate the safety and clinical effects, concerning mobility and pain, of PPS treatment in MPS I patients. Helping to relieve joint pain and stiffness is Paradigm’s mission and we are making rapid progress to achieving our goals.”
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