Pacira BioSciences Reports Positive Three-Year Data for PCRX-201 Gene Therapy in Knee Osteoarthritis

BRISBANE, Calif. – June 11, 2025 – Pacira BioSciences, Inc. (NASDAQ: PCRX) today announced new long-term follow-up data from its Phase 1 clinical trial of PCRX-201 (enekinragene inzadenovec), a novel gene therapy for moderate-to-severe osteoarthritis (OA) of the knee. Results presented at the EULAR Congress in Barcelona show that a single intra-articular injection of PCRX-201 was well tolerated and provided sustained improvements in pain, stiffness, and function for up to three years.

PCRX-201, based on Pacira’s proprietary high-capacity adenovirus (HCAd) gene therapy vector platform, is designed to boost local production of interleukin-1 receptor antagonist (IL-1Ra) in the knee joint. This aims to block inflammation, a root cause of OA, and features an inducible promoter to regulate IL-1Ra expression based on inflammation levels.

Lead author Professor Philip G. Conaghan noted that current OA treatments offer only short-term symptomatic relief. PCRX-201, by contrast, targets chronic inflammation at the cellular level, offering the potential for long-term pain and function improvements with a single administration.

Key Findings from the Phase 1 Trial (72 patients over 156 weeks):

• Safety: PCRX-201 showed an acceptable safety profile with no serious treatment-related adverse events. The most common side effect was joint swelling (effusion), mostly mild to moderate and resolving quickly, especially with corticosteroid pretreatment.
• Efficacy: Patients experienced sustained, clinically meaningful reductions in pain and stiffness, and improvements in function. In the corticosteroid-pretreated cohort, mean WOMAC-A pain scores decreased by 51–53%, WOMAC-B stiffness scores by 38–76%, and KOOS daily living function scores improved by 26–28 points.

Frank D. Lee, CEO of Pacira BioSciences, highlighted the “excellent results” and “three-year durability,” reinforcing PCRX-201’s potential as a unique disease-modifying gene therapy for OA.

PCRX-201 is the first gene therapy for OA to receive both Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA (March 2024) and Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency (May 2023), recognizing its promise and potential expedited development pathway.

Dosing is currently underway in a Phase 2 study (the ASCEND study) of PCRX-201 for knee OA.