Paradigm Biopharmaceuticals Ltd (ASX:PAR) has achieved a number of key approvals required to progress investigation into the use of its core drug Pentosan Polysulfate Sodium (PPS) in the treatment of the rare disease mucopolysaccharidosis (MPS).
Firstly, 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.
EMA approval
Paradigm 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, however, Paradigm received a decision on Day 60 of the process.
Positive feedback has been received from a Parallel Scientific Advice (PSA) meeting with the Federal Drug Administration (FDA) and EMA, where the company 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.
Shares higher
Shares have been more than 5.5% higher this morning to $3.04.
Interim chairman and CEO Paul Rennie said: “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 Program (EAP).
"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)."
Rennie said the company had a number of important takeaways from the EAP:
(i) Paradigm was able to execute on this program, in the USA, during the height of the COVID19 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 dropouts 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."
Program making “considerable progress”
Rennie also said: “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 program.”
MPS-I pilot study
Paradigm’s proposed open-label, single-centre pilot study to evaluate the safety and tolerability of PPS in subjects with MPS-I has been approved by the Human Research Ethics Committee, a precursor to commencing this pilot study.
The study will be conducted at the Adelaide Women’s and Children’s Hospital (WCH) with Dr David Ketteridge, the Principal Investigator (PI) and Dr Drago Bratkovic (Head of the Metabolic Clinic) leading the clinical trial.
Data from this trial will be used to support future regulatory filings and applications.
Investigating safety and clinical effects
Rennie continued: “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.
“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.”