Pacira BioSciences Concludes Patient Enrollment in Part A of Phase 2 Study Evaluating Safety and Efficacy of PCRX-201 for the Treatment of Osteoarthritis of the Knee

-- Milestone advances two-part study evaluating novel, locally administered gene therapy designed to increase anti-inflammatory IL-1Ra production in the knee joint --

BRISBANE, Calif., Nov. 05, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to deliver innovative, non-opioid pain therapies to transform the lives of patients, today announced that it has concluded patient enrollment in Part A of its Phase 2 ASCEND study evaluating PCRX-201 (enekinragene inzadenovec) for the treatment of osteoarthritis (OA) of the knee. This milestone marks the first stage of a two-part, multicenter trial designed to evaluate the safety and efficacy of PCRX-201, the company’s novel, locally administered gene therapy candidate for osteoarthritis of the knee. The company expects to report topline results from Part A of the study near the end of 2026.

PCRX-201 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.

“Concluding enrollment in Part A of the ASCEND study for PCRX-201 represents a significant milestone in our Phase 2 development program,” said Jonathan Slonin, M.D., MBA, Chief Medical Officer at Pacira BioSciences. “Building on encouraging results from our Phase 1 study, this accomplishment underscores the strategic execution of our clinical team and the growing recognition of the potential of PCRX-201 to provide sustained relief, addressing the underlying inflammatory drivers of OA through local administration.”

Study Design
The two-part, multicenter ASCEND study will involve approximately 135 patients, 45 to 80 years old with painful OA of the knee at a Kellgren-Lawrence (K-L) Grade of 2, 3 or 4. Subjects are randomly assigned to a treatment dose group and stratified by K-L Grade, a semiquantitative method for evaluating the severity of OA on a scale of 0-4.

Part A of the study randomizes approximately 45 patients and will evaluate two doses of PCRX-201: Dose A (1.4 × 10¹⁰ genome copies [GC]) and Dose B (1.4 × 10¹¹ GC). Patients are randomized 1:1:1 to Dose A, Dose B, or saline. All cohorts receive pretreatment with an intra-articular corticosteroid (methylprednisolone 40 mg) to improve tolerability and gene transfer, a common technique in gene therapy dosing.

Part A will inform dose selection and manufacturing readiness for Part B, which will randomize approximately 90 patients. The drug product used in Part B of the study will be manufactured using the company’s proprietary commercial scale manufacturing process intended for commercial scale-up. Pacira expects to report topline results from Part A of the study before the end of 2026.

For both Parts A and B, the primary endpoint is the number and percentage of treatment-emergent adverse events, adverse events of special interest, and serious adverse events for PCRX-201 plus steroid pretreatment versus saline plus steroid pretreatment from Week 1 through Week 52. Secondary and exploratory endpoints include efficacy assessments such as changes in pain and physical function from baseline at Weeks 38 and 52, measured by the Numerical Rating Scale (NRS), the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), and the Knee Injury and Osteoarthritis Outcome Score (KOOS). Biomarkers, immunogenicity, biodistribution, and structural endpoints will also be evaluated. All participants will be followed for five years.

To learn more about the ASCEND study, visit clinicaltrials.gov.

About PCRX-201 (enekinragene inzadenovec)
PCRX-201 (enekinragene inzadenovec) features an innovative design based on the company’s proprietary high-capacity adenovirus vector platform. It is currently being studied in the fundamental, underlying chronic inflammatory processes that contribute to “wear and tear” over time in osteoarthritis of the knee, a condition that affects more than 15 million individuals in the U.S. today.

In June 2025, Pacira reported data from its ongoing clinical development program showing that PCRX-201 continues to demonstrate durable and clinically meaningful improvements in knee pain, stiffness, and function through three years following local administration, with a well-tolerated safety profile. PCRX-201 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration and Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency. PCRX-201 is the first gene therapy to achieve these clinical results and earn these regulatory designations in osteoarthritis of the knee – a testament to its promise and potential.

About the High-capacity Adenovirus Vector Platform
Pacira’s proprietary novel high-capacity adenovirus (HCAd) gene therapy vector platform solves many of the challenges in the field of gene therapy that have prevented its utilization in treating common diseases, such as osteoarthritis.

Key features include:

• The HCAd vector is much more efficient at delivering genes into cells compared to many other gene therapies that rely on adenovirus associated virus, or AAV, vectors. As a result, the desired effect can be achieved with much smaller doses.
• The vector used in the HCAd platform can carry up to 30,000 base pairs of DNA, which enables gene therapy with multiple or larger genes compared to AAV vectors.
• Genetic medicines based on the HCAd platform can be administered locally and have the potential for redosing at therapeutically appropriate intervals.
• Lower dose levels and efficient delivery of genes into cells means that thousands of doses can be produced in a single batch. As a result, therapies built on the HCAd platform are expected to have a commercially attractive and viable cost of goods profile.

Beyond PCRX-201 and other product candidates in preclinical development, the company has identified numerous well-validated cytokines that could also be the basis for locally administered genetic therapies using the HCAd platform.

About Pacira
Pacira delivers innovative, non-opioid pain therapies to transform the lives of patients. Pacira has three commercial-stage non-opioid treatments: EXPAREL® (bupivacaine liposome injectable suspension), a long-acting local analgesic currently approved for infiltration, fascial plane block, and as an interscalene brachial plexus nerve block, an adductor canal nerve block, and a sciatic nerve block in the popliteal fossa for postsurgical pain management; ZILRETTA® (triamcinolone acetonide extended-release injectable suspension), an extended-release, intra-articular injection indicated for the management of osteoarthritis knee pain; and iovera®º, a novel, handheld device for delivering immediate, long-acting, drug-free pain control using precise, controlled doses of cold temperature to a targeted nerve. The Company is also advancing the development of PCRX-201 (enekinragene inzadenovec), a novel, locally administered gene therapy with the potential to treat large prevalent diseases like osteoarthritis. To learn more about Pacira, visit www.pacira.com.

Forward-Looking Statements
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CONTACT: Investor Contact:
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Media Contact:
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