A real-world trial using Paradigm Biopharmaceuticals’ (ASX: PAR) injectable pentosan polysulfate (iPPS) treatment has found the drug can provide a reduction in pain of more than 50% in patients with knee osteoarthritis.
Since October 2017, iPPS has been used on 205 patients over a series of small groups, and has been administered in private practices by doctors under the Therapeutic Goods Administration Special Access Scheme (TGA SAS).
All patients had been clinically-diagnosed with osteoarthritis and subchondral bone marrow lesions prior to treatment.
They had demonstrated pain for at least six months and had failed current standards of care such as analgesics, non-steroidal anti-inflammatories and corticosteroids.
Patients were administered two doses of iPPS per week for three, four or six weeks for a total of 6, 8 or 12 injections, and were followed up at four to six weeks following the last treatment.
During the course of PPS treatment, patients did not receive NSAIDs or corticosteroid treatment.
In the 205 patients treated, 184 (or 89.7%) responded with a reduction in joint pain and 91.2% had an improvement in knee function.
Patients pain scores were reduced by a “clinically meaningful” 51.3%, and function was improved by an average 58.4% from baseline pain scores, demonstrating superiority over 15% pain reduction scores reported for opioid treatments.
Paradigm chief executive officer Paul Rennie said the consistent reduction in knee pain was a promising outcome for the new drug.
“We have now accumulated data on 205 patients being successfully treated with iPPS for osteoarthritis and associated bone marrow lesions,” he said.
“The number of patients seeking treatment via the TGA SAS is strong feedback that they are receiving a clinically-meaningful benefit from iPPS.”
Real world evidence
When the pharmaceutical agent such as iPPS is used in patients outside a randomised clinical trial, the results are referred to as real world data and can provide important clinical (or real world) evidence on the safety and efficacy of a drug.
The results from Paradigm’s 205 patient trial provides important real world evidence which has the potential to be combined with data from the company’s randomised controlled clinical trials to support regulatory filings with agencies such as the US Food and Drug Administration.
Mr Rennie said the company had also received real world evidence from doctors treating patients with osteoarthritis of other joints such as the hip, ankle and hands.
The data will be released later this year.
Injectable PPS is registered in four of the seven major global pharmaceutical markets as an anti-thrombotic agent.
It is not currently registered in Australia and is not available for sale.
Injectable PPS for human use is only available by inclusion in a Paradigm-sponsored clinical trial or via a doctor who applies to use it in patients under the TGA SAS.
At midday, shares in Paradigm Biopharmaceuticals were down 2.03% to $1.450.