Core Insights - Pacira BioSciences has completed patient enrollment in Part A of its Phase 2 ASCEND study for PCRX-201, a novel gene therapy for knee osteoarthritis, with topline results expected by the end of 2026 [1][6][10] Study Overview - The ASCEND study is a two-part, multicenter trial involving approximately 135 patients aged 45 to 80 with painful knee osteoarthritis at Kellgren-Lawrence Grades 2, 3, or 4 [4] - Part A randomizes about 45 patients to evaluate two doses of PCRX-201, with a 1:1:1 randomization to Dose A, Dose B, or saline, all receiving corticosteroid pretreatment [5][6] - The primary endpoint includes treatment-emergent adverse events and secondary endpoints assess pain and physical function changes at Weeks 38 and 52 [7] Product Details - PCRX-201 is designed to enhance IL-1Ra production locally in the knee joint, targeting chronic inflammation and pain associated with osteoarthritis [2][9] - The therapy utilizes a high-capacity adenovirus vector platform, allowing for efficient gene delivery and the potential for redosing [12][17] - The product has received RMAT designation from the FDA and ATMP designation from the EMA, highlighting its clinical promise [11] Company Background - Pacira BioSciences focuses on innovative, non-opioid pain therapies, with existing products including EXPAREL, ZILRETTA, and iovera° [15] - The company aims to address prevalent diseases like osteoarthritis through advanced gene therapy solutions [15]

Core Insights - Pacira BioSciences has announced an exclusive worldwide license and collaboration agreement with AmacaThera for the development of AMT-143, a novel long-acting formulation of the non-opioid analgesic ropivacaine aimed at postsurgical pain control [1][2] Company Strategy - The addition of AMT-143 to the pipeline aligns with Pacira's 5x30 growth strategy, focusing on clinical stage, derisked opportunities that complement existing pain management offerings [2] - The company aims to enhance its leadership in opioid-sparing pain management through this new asset, which is expected to improve patient outcomes and expand treatment options [2] Financial Terms of the Agreement - Under the agreement, Pacira will fund the clinical development up to commercial launch, with AmacaThera receiving an upfront payment of $5 million, along with potential future milestone payments and a tiered royalty on net sales [3] Product Details - AMT-143 is designed for postsurgical pain control and demonstrated sustained release of ropivacaine for up to 14 days in a Phase 1 study [4] - The product utilizes AmacaThera's hydrogel-based drug delivery platform, which allows for slow release while minimizing systemic side effects [5] Current Product Portfolio - Pacira currently offers three commercial-stage non-opioid treatments: EXPAREL, ZILRETTA, and iovera, which target various pain management needs [6]

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]

Core Insights - Pacira BioSciences, Inc. is set to present new data from its Phase 1 clinical trial of PCRX-201, a gene therapy for knee osteoarthritis, at the ACR Convergence 2025 meeting [1] - A Phase 2 study, known as the ASCEND study, is currently ongoing for PCRX-201 [2] Group 1: Clinical Data and Efficacy - PCRX-201 has shown sustained improvements in knee pain, stiffness, and function over two years in a Phase 1 study involving 72 patients, with a well-tolerated safety profile [4] - The therapy has received RMAT designation from the FDA and ATMP designation from the EMA, marking it as the first gene therapy to achieve such results in knee osteoarthritis [4] Group 2: Technology and Platform - The high-capacity adenovirus vector platform acquired from GQ Bio Therapeutics enables efficient gene delivery, addressing challenges in gene therapy for common diseases like osteoarthritis [5] - The HCAd vector can carry up to 30,000 base pairs of DNA, allowing for the potential administration of multiple or larger genes compared to traditional AAV vectors [6] Group 3: Company Overview - Pacira specializes in innovative, non-opioid pain therapies, with three commercial-stage products including EXPAREL, ZILRETTA, and iovera [6][7] - The company is advancing PCRX-201 as a locally administered gene therapy aimed at treating prevalent diseases such as osteoarthritis [7]

Core Insights - Preliminary data indicates that pre-existing and treatment-induced anti-Ad5 neutralizing antibodies do not affect the safety and effectiveness of PCRX-201 for knee osteoarthritis [1][2][4] - The findings will be presented at the ASGCT Annual Meeting, highlighting the potential of PCRX-201 as a durable treatment option for knee osteoarthritis [1][2] Company Overview - Pacira BioSciences, Inc. is focused on innovative, non-opioid pain therapies, with PCRX-201 being a key candidate for treating knee osteoarthritis [1][11] - The company has received RMAT designation from the FDA for PCRX-201, which facilitates efficient drug development and potential accelerated approval [6][7][10] Study Details - An open-label, phase 1 trial involved 72 participants aged 30 to 80, assessing the impact of anti-Ad5 neutralizing antibodies on PCRX-201 [3] - The study measured NAb titers and IL-1Ra levels over 52 weeks, with results showing no adverse effects on efficacy or safety across different dosing levels [4][5] Treatment Efficacy - All doses of PCRX-201 led to improvements in knee pain, stiffness, and function, as measured by WOMAC-A and WOMAC-B indices [4] - Corticosteroid pretreatment was found to reduce treatment-induced NAb titers and related knee swelling [5] Future Developments - A Phase 2 study (ASCEND study) is currently underway for PCRX-201, building on the promising Phase 1 results [8] - The innovative design of PCRX-201 utilizes a high-capacity adenovirus vector platform to enhance treatment efficacy for chronic inflammation in knee osteoarthritis [2][9]

Core Insights - Pacira BioSciences, Inc. is presenting new data on its gene therapy candidate PCRX-201 at the 2025 EULAR Annual Congress, highlighting its commitment to innovative, non-opioid pain therapies [1] Group 1: Clinical Data and Study Results - The Phase 1 study of PCRX-201 demonstrated that a single intra-articular injection was safe and provided sustained clinical benefits for patients with moderate-to-severe knee osteoarthritis over three years [2] - In a previous Phase 1 study, PCRX-201 showed sustained improvements in knee pain, stiffness, and function for two years post-administration, with a well-tolerated safety profile [3] - PCRX-201 has received RMAT designation from the FDA and ATMP designation from the EMA, marking it as the first gene therapy to achieve these clinical results in knee osteoarthritis [3] Group 2: Ongoing Research and Development - Following promising Phase 1 results, dosing is currently underway in a Phase 2 study (ASCEND study) for the treatment of knee osteoarthritis [4] - The company is advancing the development of PCRX-201 as a novel, locally administered gene therapy aimed at treating prevalent diseases like osteoarthritis [5] Group 3: Company Overview - Pacira BioSciences focuses on delivering innovative, non-opioid pain therapies, with three commercial-stage products: EXPAREL, ZILRETTA, and iovera® [5] - EXPAREL is a long-acting local analgesic approved for various postsurgical pain management applications, while ZILRETTA is an extended-release injection for osteoarthritis knee pain [5]

Core Insights - Pacira BioSciences is set to present findings from its Phase 1 study of PCRX-201, a gene therapy for knee osteoarthritis, at the ASGCT Annual Meeting on May 15, focusing on the impact of neutralizing antibodies on therapy efficacy and safety [1][2] - The company has reported promising Phase 1 results showing sustained improvements in knee pain and function over two years, leading to regulatory designations from the FDA and EMA [4][5] Company Overview - Pacira BioSciences specializes in innovative, non-opioid pain therapies, with three commercial-stage products: EXPAREL, ZILRETTA, and iovera, alongside the development of PCRX-201 [6] - PCRX-201 utilizes a proprietary high-capacity adenovirus vector platform and targets chronic inflammatory processes in knee osteoarthritis, a condition affecting over 14 million individuals in the U.S. [3][4] Upcoming Events - The presentation titled "Understanding the Clinical Immunogenicity of Locally Injected HCAd Vector Provides Insight into Optimizing Dosing Strategy" will be delivered by Dr. MiJeong Kim on May 15 [2] - A symposium on "High-Capacity Adenoviral Vectors: Advancing Gene Therapy Beyond AAV to Deliver Cost-Effective Therapies for Common Diseases" is scheduled for May 16 [3]

Core Insights - Pacira BioSciences, Inc. announced new data on its gene therapy candidate PCRX-201, showing sustained improvements in knee pain, stiffness, and function for up to two years after a single local administration in patients with varying severity of osteoarthritis [2][3][9] Study Findings - The study presented at the 2025 OARSI World Congress demonstrated that PCRX-201 provided clinically meaningful improvements across all structural severity subgroups, including advanced disease [1][2] - The open-label, phase 1 trial involved 72 patients aged 30 to 80, stratified by the severity of knee osteoarthritis, graded on the Kellgren-Lawrence scale [4] - Improvements in pain, stiffness, and function were assessed using the WOMAC and KOOS scores, showing significant reductions across all cohorts and severity levels [6][7] Treatment Efficacy - Patients receiving corticosteroid pretreatment experienced greater benefits, with pain reductions of 48%-65% and stiffness reductions of 53%-72%, compared to 41%-58% and 33%-53% in the non-pretreated cohort [7] - The greatest improvement was observed in patients with K/L grade 2 osteoarthritis [7] Safety Profile - No serious treatment-emergent adverse events were reported, with treatment-related joint effusions being the most common adverse event, occurring in 36% of the corticosteroid-pretreated group and 61% of the non-pretreated group [8] Regulatory Designations - PCRX-201 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA in March 2024, facilitating efficient drug development and potential accelerated approval [9][10] - The therapy also received Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency in May 2023 [10][13] Future Developments - Following promising Phase 1 results, a Phase 2 study (ASCEND study) is underway for PCRX-201, focusing on the treatment of knee osteoarthritis [11][13] - The company aims to advance its position as an innovative biopharmaceutical entity through this development [9]