- Paradigm Biopharmaceuticals (PAR) lands key regulatory approvals for a second-phase clinical trial in Brazil
- The trial will test PAR’s pentosan polysulfate sodium (PPS) drug in patients with a rare disease known as mucopolysaccharidosis VI (MPS VI)
- The primary endpoint for the trial will be to test the safety and tolerability of the drug, with its ability to treat the condition secondary
- Paradigm says this will be the largest clinical trial conducted using PPS in subjects with MPS
- Shares in Paradigm Biopharmaceuticals are up 6.03 per cent and trading at $2.11 at 10:35 am AEST
Paradigm Biopharmaceuticals (PAR) has opened strong in Friday trade after landing some key regulatory approvals for a clinical trial in Brazil.
The drug developer has received regulatory approval from Brazil’s National Health Surveillance Agency (ANVISA) and ethics approval from the National Research Ethics Commission (CONEP) for a phase two trial of its injectable pentosan polysulfate sodium (PPS) drug.
The trial will test PPS in patients with a rare disease known as mucopolysaccharidosis VI, or MPS VI.
This disease is a progressive condition that causes tissues and organs to enlarge and become inflamed or scarred. It can often cause skeletal abnormalities and joint deformities.
Life expectancy for people with severe MPS VI is often very short, with many only surviving until late childhood.
The PAR trial
With regulatory approvals locked in, Paradigm is now set to design and begin the phase two trial, which will test PPS against a placebo in MPS VI patients.
Paradigm will recruit 12 patients for the trial, with eight to receive PPS and four to receive the placebo.
The main goal of the trial, however, is not to test the drug’s ability to treat MPS VI, but to test its safety in patients.
PAR said the primary endpoint for the second-phase study will be to evaluate the safety and tolerability of PPS in MPS VI patients at six, 12 and 24 weeks.
Secondary endpoints will measure PPS’ effect on pain, function, and glycosaminoglycan levels in patients.
Paradigm CEO Paul Rennie said the company is “delighted” to have received this Brazilian regulatory milestone as it progresses its Investigative New Drug (IND) application with the US Food and Drug Administration (FDA) for PPS treating osteoarthritis.
“Paradigm will be monitoring the current COVID situation in Brazil to plan the trial commencement and will update the market once a timeline is determined,” Mr Rennie said.
Under the Brazilian trial, patients over nine years old will receive one weekly 1.5-milligram-per-kilogram (mg/kg) dose of PPS for 24 weeks, while patients under nine will receive a 1.0-mg/kg dose.
According to Paradigm, this will be the largest clinical trial conducted using PPS in subjects with MPS.
Shares in Paradigm Biopharmaceuticals were up 6.03 per cent and trading at $2.11 at 10:35 am AEST.