Pacira BioSciences Presents Updated Three-Year Data Demonstrating Sustained Efficacy and Safety of PCRX-201 Gene Therapy for Patients with Moderate-to-Severe Osteoarthritis of the Knee

Core Insights - Pacira BioSciences, Inc. announced updated three-year results for PCRX-201, a gene therapy for knee osteoarthritis, showing sustained clinical improvements in pain, stiffness, and function for up to 156 weeks following a single injection [1][12][8] Study Details - The open-label Phase 1 trial involved 72 patients aged 30 to 80 with moderate to severe knee osteoarthritis, assessing safety and efficacy of PCRX-201 at three different doses over 156 weeks [4][6] - Patients were evaluated using the WOMAC-A pain score, WOMAC-B stiffness score, and KOOS for function [4] Key Findings - PCRX-201 demonstrated sustained, clinically meaningful reductions in pain and stiffness, and improvements in function across all doses for three years [6][7] - The treatment had an acceptable safety profile with no serious treatment-related adverse events reported [6][7] - In the corticosteroid pretreated cohort, patients with K/L grade 2 osteoarthritis showed greater pain reduction compared to those with advanced grades [6] - Pre-existing neutralizing antibodies did not affect the treatment's effectiveness [6][7] Regulatory Designations - PCRX-201 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency, marking it as the first gene therapy candidate in osteoarthritis to achieve these designations [8][9][12] Future Developments - A Phase 2 study (ASCEND) of PCRX-201 is currently underway, focusing on a lower dose with corticosteroid pretreatment [10][12] - The innovative high-capacity adenovirus vector platform used in PCRX-201 allows for efficient gene delivery and potential for redosing [14]