Pacira BioSciences announces two-year efficacy data following a single local administration of PCRX-201 in patients with osteoarthritis of the knee
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Pacira BioSciences, Inc., the industry leader in its commitment to deliver innovative, non-opioid pain therapies to transform the lives of patients, today announced new data demonstrating its locally administered gene therapy candidate, PCRX-201 (enekinragene inzadenovec), provided sustained improvements in knee pain, stiffness, and function for up to two years following a single local administration in patients with mild, moderate, as well as severe osteoarthritis of the knee. The data was presented during a poster session at the 2025 Osteoarthritis Research Society International (OARSI) World Congress in Incheon, South Korea, on Friday, April 25, and Saturday, April 26.
These results highlight the promise of PCRX-201 to reshape the treatment landscape for knee osteoarthritis by delivering long-lasting, inflammation-targeted relief with a single injection—regardless of the severity of the condition. The power of PCRX-201 is that it targets much more than the symptoms of osteoarthritis, it addresses the root cause by targeting the inflammatory endotype and chronic inflammation at the cellular level with a locally administered genetic medicine that that mimics the body’s natural inflammatory response. There is a clear need for innovative, disease-modifying treatments for osteoarthritis as current options are based on decades-old mechanisms that only provide three to six months of relief.
said Ali Mobasheri, Professor of Musculoskeletal Biology at the University of Oulu in Finland and Chief Researcher at the State Research Institute Centre for Innovative Medicine in Lithuania, who was lead investigator and primary author on the poster presentation.
PCRX-201 features an innovative design based on the company’s proprietary high-capacity adenovirus, or HCAd, gene therapy vector platform. It is injected locally into the knee joint to boost cellular production of interleukin-1 receptor antagonist (IL-1Ra), and block interleukin-1 pathway activation to improve chronic inflammation, pain, and function. PCRX-201’s unique design also features an inducible promoter to mimic the body’s natural response to inflammation by “turning on” the expression of IL-1Ra when inflammation is present in the joint and turning off expression once inflammation is quelled.
Study Details
The open-label, phase 1 trial investigated the efficacy of PCRX-201 administered by ultrasound-guided intraarticular injection in 72 patients aged 30 to 80, stratified by structural severity of osteoarthritis of the knee. The research included participants with osteoarthritis of the knee graded at 2, 3 and 4 on the Kellgren-Lawrence (K/L) scale, a method for evaluating the severity of osteoarthritis on a scale of 0-4.
Participants were divided into two cohorts. The first cohort was administered one injection of PCRX-201 at a low, middle or high dosage. The second cohort received concurrent pretreatment with an intraarticular corticosteroid (methylprednisolone 40 mg) to improve tolerability and gene transfer.
Improvements in pain, stiffness and function from baseline were shown across both cohorts at all three doses and severity levels over the full 104-week study period; they were assessed using the Western Ontario and McMaster Universities Osteoarthritis Index pain score (WOMAC-A), the stiffness score (WOMAC-B), and Knee Injury and Osteoarthritis Outcome Score (KOOS).
Patients in the corticosteroid -pretreated cohort achieved greater benefits (48%-65% reductions in pain and 53%-72% reductions in stiffness) than the first cohort (41%-58% reduction in pain and 33%-53% reduction in stiffness). Improvement was observed across all severity subgroups with the greatest improvement observed among individuals with K/L grade 2 osteoarthritis of the knee.
No serious treatment-emergent adverse events (TEAEs) related to the treatment or procedure were reported regardless of corticosteroid pretreatment or dose level administered. Treatment-related joint effusions (swelling) were the most common AE, occurring in 36% of patients who received corticosteroid pretreatment vs 61% of patients who were not pretreated. The majority of effusions were mild to moderate in severity and resolved in a median of 33 days among patients in the pretreated group. The percentage of participants with treatment related knee effusions was similar across subgroups.
“This study reinforces the transformative potential of locally administered gene therapy to deliver long-lasting relief for the millions of people living with knee osteoarthritis, including the most severe subgroup—a condition that can severely limit mobility, quality of life, and overall well-being,” said Frank D. Lee, chief executive officer of Pacira BioSciences. “As we advance our Phase 2 study, PCRX-201 represents a key milestone on our path to becoming an innovative biopharmaceutical company and delivering on our 5x30 strategy for growth and value creation.”
In March 2024, PCRX-201 became the first-ever gene therapy product candidate in osteoarthritis to receive Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA).
RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with the FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the Biologics License Application (BLA), and other opportunities to expedite development and review. PCRX-201 was also granted Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency in May 2023.
Given the promising Phase 1 results, dosing is underway in a Phase 2 study of PCRX-201 (the ASCEND study) for the treatment of osteoarthritis of the knee. To learn more about PCRX-201 and the company’s clinical development program, please visit the investor events page of the company’s investor website.
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