Pacira BioSciences has initiated a Phase 2 clinical trial, ASCEND, for PCRX-201, a locally administered gene therapy designed to treat osteoarthritis (OA) of the knee. PCRX-201 utilizes a high-capacity adenovirus vector to deliver the gene for interleukin-1 receptor antagonist (IL-1Ra) directly into the knee joint, aiming to combat inflammation and alleviate pain. The study will assess two doses of PCRX-201 compared to a saline control, with topline results from the first part of the study anticipated by the end of 2026.
This development represents a significant advancement in the treatment of OA, a debilitating condition impacting millions. Current treatments offer limited, short-term pain relief. PCRX-201’s unique mechanism of action offers the potential for long-lasting pain reduction by addressing the underlying chronic inflammation driving OA progression, a significant improvement over existing therapies. This localized approach may also minimize systemic side effects often associated with other treatments.
The ASCEND study will involve approximately 135 patients with varying degrees of OA severity. Participants will be randomized into three groups, receiving either one of two doses of PCRX-201 or a saline injection, all with a concurrent corticosteroid pretreatment. The primary endpoint focuses on safety and tolerability over one year. Secondary endpoints include assessments of pain and physical function using standardized metrics like the Numerical Rating Scale (NRS), the Western Ontario and McMaster Universities Index (WOMAC), and the Knee Injury and Osteoarthritis Outcome Score (KOOS). Biomarkers, structural endpoints, immunogenicity, and biodistribution will also be evaluated over a five-year follow-up period. The second part of the study will utilize a manufacturing process designed for commercial scalability.
Positive results from the ASCEND trial could transform the OA treatment landscape. This novel gene therapy approach has the potential to offer a disease-modifying solution, providing long-term pain relief and improved joint function, ultimately enhancing the quality of life for OA patients. Success could also position Pacira as a leader in the emerging field of gene therapy for musculoskeletal conditions, opening up new avenues for growth and innovation.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
