The Paradigm Biopharmaceuticals Ltd (ASX: PAR) share price is climbing higher today on news the company has started Phase 2 trials of pentosan polysulphate sodium (iPPS).
In early morning trade, the Paradigm share price is up 1.62% to $3.13.
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Phase 2 trial commences
Paradigm advised that it has started Phase 2 trials of iPPS to treat patients who suffer from orphan disease Mucopolysaccharidosis Type 1 (MPS-1).
Led by principal investigator, Dr David Ketteridge, the open label study will recruit up to 10 participants who meet specific criteria. Those enrolled in the trial will receive one of two dosing cohorts, either 0.75mg/kg or 1.5mg/kg. The drug will be administered weekly as an injectable dose for the first 12 weeks and then every second week for the 11-month duration.
Paradigm will seek to primarily assess the safety of iPPS in patients and evaluate if treatment can successfully alleviate pain. The biopharma company said its first patient has been enrolled and had received the first dose of iPPS.
The Phase 2 study will be undertaken at the Adelaide's Women & Children's hospital.
What is MPS-1?
Mucopolysaccharidosis type 1 is a rare metabolic disorder caused by a genetic defect when born. The disorder catabolises heparan sulphate and dermatan sulphates which cause a raft of problems for sufferers. They include abnormal bone development, irregular growth, cardiac and respiratory problems, and sometimes cognitive impairment.
Current treatments available are enzyme replacement therapy (ERT) and hematopoietic stem cell therapy (HSCT). Both therapies work to reduce the accumulation glycosaminoglycans (heparan sulphate and dermatan sulphate).
What did the CEO say?
Commenting on the trial, Paradigm CEO Paul Rennie said:
The data collected from the Phase 2 trial will be vital to support Paradigm's future regulatory filings and applications for the development of PPS as a potential adjunctive therapy to enzyme replacement therapy (ERT) treatments. Our MPS programs will treat subjects as adjunct to ERT as well as previously bone marrow transplanted patients who may or may not remain on ERT.
Paradigm is making significant progress in the clinical development of PPS in both of our two programs of osteoarthritis (Zilosul) and the rare disease of MPS.
It is important to note both the US FDA and EU EMA have confirmed MPS is an orphan indication and as such the commercial advantage of an orphan drug is the 7-year regulatory exclusivity awarded to orphan drugs.
About the Paradigm share price
The Paradigm share price has been up and down over the past 12 months, reaching as high as $4.50 and as low as $1.08.
The company has a market capitalisation of $702.3 million and trades an average volume of 1.1 million shares daily. This shows investors are active when it comes to buying and selling Paradigm shares.
