Aug 06, 2026 —
Pacira BioSciences announced publication of positive two-year results from a Phase 1 clinical trial evaluating PCRX-201, also known as enekinragene inzadenovec, its investigational locally administered gene therapy for knee osteoarthritis.
The data were published in Annals of the Rheumatic Diseases. The study showed that a single intra-articular injection of PCRX-201 into the knee was well tolerated in patients with moderate-to-severe knee osteoarthritis, with exploratory clinical outcome measures showing improvements in pain, stiffness, and function through two years among patients who remained in the study.
PCRX-201 is based on Pacira’s proprietary high-capacity adenovirus, or HCAd, vector platform. The therapy is designed to address biomechanical and inflammatory processes involved in knee osteoarthritis through local gene delivery.
The Phase 1 trial was a two-part, open-label study enrolling 72 adults aged 30 to 80 years with symptomatic, radiographically confirmed moderate-to-severe knee osteoarthritis. In Part 1, 36 participants received a single injection of PCRX-201 at low, middle, or high dose levels. In Part 2, 36 participants received methylprednisolone immediately before PCRX-201 administration to improve tolerability and evaluate whether glucocorticoid pretreatment could enhance vector transduction.
The primary endpoint was safety. Secondary and exploratory endpoints included systemic biodistribution and clinical outcomes measuring pain, stiffness, and function, with follow-up through 104 weeks.
Pacira reported that glucocorticoid pretreatment was associated with a lower rate of joint effusion and greater improvement in clinical outcomes. This approach has been incorporated into the company’s ongoing Phase 2 ASCEND study of PCRX-201 in knee osteoarthritis.
The company recently initiated enrollment in Part B of ASCEND and said it has successfully transitioned PCRX-201 to a scalable manufacturing process. The first topline readout from the Phase 2 ASCEND trial remains on track for year-end 2026.
PCRX-201 has received Regenerative Medicine Advanced Therapy, or RMAT, designation from the U.S. Food and Drug Administration and Advanced Therapy Medicinal Product, or ATMP, designation from the European Medicines Agency.
Pacira said its HCAd vector platform offers several potential advantages for locally administered genetic medicines. Compared with many AAV-based gene therapies, the HCAd platform is designed to deliver genes efficiently at lower doses, carry larger genetic payloads of up to 30,000 base pairs, and potentially support redosing at appropriate therapeutic intervals.
The update highlights a differentiated approach to gene therapy for a common musculoskeletal disease. While most gene therapy programs have focused on rare genetic disorders, PCRX-201 is being developed for knee osteoarthritis, a condition affecting more than 15 million people in the United States.
