Pacira BioSciences has announced that its gene therapy candidate, PCRX-201 (enekinragene inzadenovec), has demonstrated sustained improvements in knee pain, function, and stiffness for up to two years in individuals with varying severities of knee osteoarthritis, as shown in a Phase I trial.
As part of the trial, the subjects received a single local administration of the therapy.
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The Phase I trial involved 72 subjects aged between 30 and 80 years, stratified by the structural severity of their knee osteoarthritis, as measured by the Kellgren-Lawrence (K/L) scale.
They were assigned to two groups. The first received a single dose of PCRX-201, while the second was pre-treated with an intraarticular corticosteroid to enhance gene transfer and tolerability.
Over the 104-week duration of the study, both cohorts were found to have exhibited significant improvements as evaluated by the Western Ontario and McMaster Universities Arthritis Index (WOMAC)-A, WOMAC-B, and Knee Injury and Osteoarthritis Outcome scores.
The corticosteroid-pretreated group experienced greater benefits, with a decrease in pain and stiffness.
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By GlobalDataNotably, improvements were observed in subjects with a K/L grade 2 classification of knee osteoarthritis without any serious treatment-emergent adverse events reported in relation to the treatment or procedure.
PCRX-201’s design is leveraged from the company’s high-capacity adenovirus gene therapy vector platform.
The therapy is administered directly into the knee joint to inhibit the activation of the interleukin-1 pathway and enhance interleukin-1 receptor antagonist (IL-1Ra) cellular production, consequently reducing chronic inflammation and improving joint function.
Its design also includes an inducible promoter, which is said to resemble the natural response of the body towards inflammation and turns on IL-1Ra expression when there is any presence of inflammation levels within the joint and turns off the expression when the inflammation is suppressed.
Pacira noted that following the promising Phase I outcomes, dosing in the Phase II ASCEND study is currently ongoing.
Pacira BioSciences CEO Frank Lee said: “This study reinforces the transformative potential of locally administered gene therapy to deliver long-lasting relief for the millions of people living with knee osteoarthritis, including the most severe subgroup—a condition that can severely limit mobility, quality of life, and overall well-being.”
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