Paradigm Biopharmaceuticals (ASX: PAR) has completed treatment of its Zilosul injectable pentosan polysulfate (iPPS) drug under the US Food and Drug Administration’s expanded access program, while collaring FDA orphan status for using it in treating MPS-I.
Also known as compassionate use, the expanded access program provides a pathway for doctors to use investigational drugs, biologics and medical devices.
These can be used to treat patients with serious diseases of conditions that currently don’t have a satisfactory option.
Under the program, Paradigm was able to treat 10 knee osteoarthritis sufferers with Zilosul, with all 10 patients now completing treatment.
A final evaluation will take place six weeks after the last injection was received for each patient.
Paradigm anticipates it will publish the data from the program during the September quarter.
“Paradigm is excited to have achieved this milestone, especially during the current health conditions created by COVID-19 and would like to thank all those involved in the expanded access program for their continued diligence to the treatment program,” Paradigm chief executive officer Paul Rennie said.
Orphan designation for MPS-1 and MPS-VI
In addition to advancing its Zilosul drug in osteoarthritis sufferers, Paradigm has now secured orphan designation for using the drug to treat MPS-I and MPS-VI, which will provide tax credits for qualified clinical testing, fee waivers and eligibility for seven-year marketing exclusivity once approved.
According to Paradigm, MPS-I is a rare metabolic disorder that is caused by a genetic defect. It results in abnormal bone development, growth retardation, cardiac and respiratory programs and sometimes cognitive impairment.
Meanwhile, MPS-VI is a rare autosomal recessive disorder that leads to accumulations of glycosaminoglycans in the lysosomes and physical manifestations.
Current treatments for MPS patients involve enzyme replacement therapy (ERT) which can reduce non-neurological symptoms and pain. However, these patients continue to report ongoing stiffness, pain, inflammation and heart and airway soft tissue manifestations.
Paradigm noted these treatments are not adequate in negating pain and claims its iPPS drug may be an effective adjunct or combination therapy with ERT.
In the US, there are more than 13,000 MPS patients, with Paradigm projecting a potential market for its iPPS in this space between US$605 million and US$1.3 billion per annum.
Paradigm is gearing up to begin a pilot clinical program evaluating iPPS in MPS-I sufferers that have lingering symptoms following ERT or a bone marrow transplant.