Pacira BioSciences Announces Two-Year Efficacy Data Following a Single Local Administration of PCRX-201 in Patients with Mild to Severe Osteoarthritis of the Knee

Core Insights - Pacira BioSciences, Inc. announced new data on its gene therapy candidate PCRX-201, showing sustained improvements in knee pain, stiffness, and function for up to two years after a single local administration in patients with varying severity of osteoarthritis [2][3][9] Study Findings - The study presented at the 2025 OARSI World Congress demonstrated that PCRX-201 provided clinically meaningful improvements across all structural severity subgroups, including advanced disease [1][2] - The open-label, phase 1 trial involved 72 patients aged 30 to 80, stratified by the severity of knee osteoarthritis, graded on the Kellgren-Lawrence scale [4] - Improvements in pain, stiffness, and function were assessed using the WOMAC and KOOS scores, showing significant reductions across all cohorts and severity levels [6][7] Treatment Efficacy - Patients receiving corticosteroid pretreatment experienced greater benefits, with pain reductions of 48%-65% and stiffness reductions of 53%-72%, compared to 41%-58% and 33%-53% in the non-pretreated cohort [7] - The greatest improvement was observed in patients with K/L grade 2 osteoarthritis [7] Safety Profile - No serious treatment-emergent adverse events were reported, with treatment-related joint effusions being the most common adverse event, occurring in 36% of the corticosteroid-pretreated group and 61% of the non-pretreated group [8] Regulatory Designations - PCRX-201 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA in March 2024, facilitating efficient drug development and potential accelerated approval [9][10] - The therapy also received Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency in May 2023 [10][13] Future Developments - Following promising Phase 1 results, a Phase 2 study (ASCEND study) is underway for PCRX-201, focusing on the treatment of knee osteoarthritis [11][13] - The company aims to advance its position as an innovative biopharmaceutical entity through this development [9]