TAMPA, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Pacira Biosciences Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PCRX-201 (enekinragene inzadenovec), the company’s novel, intra-articular helper-dependent adenovirus (HDAd) gene therapy product candidate that codes for interleukin-1 receptor antagonist (IL-1Ra), for the treatment of osteoarthritis of the knee..

佛罗里达州坦帕，2024年3月13日（环球通讯社）--Pacira Biosciences Inc.（纳斯达克：PCRX），致力于非阿片类药物疼痛管理和再生健康解决方案的行业领导者，今天宣布，美国食品和药物管理局（FDA）已将再生医学高级疗法（RMAT）指定为PCRX-201（enekinragene inzadenovec），该公司的小说，编码白细胞介素-1受体拮抗剂（IL-1Ra）的关节内辅助依赖性腺病毒（HDAd）基因治疗产品候选物，用于治疗膝关节骨关节炎。

“We are honored to receive FDA’s first-ever RMAT designation for a gene therapy product candidate in osteoarthritis,' said Frank D. Lee, chief executive officer of Pacira BioSciences, Inc. “We continue to be encouraged by the preliminary clinical findings supporting PCRX-201 as a potential disease-modifying therapy for osteoarthritis, and we look forward to presenting additional follow-up data later this year.”.

FrankD。Pacira BioSciences，Inc.首席执行官Lee说：“我们继续受到支持PCRX-201作为骨关节炎潜在疾病缓解疗法的初步临床发现的鼓舞，我们期待着在今年晚些时候提供更多的随访数据。”

The company’s RMAT application was supported by the preliminary safety and efficacy findings from a Phase 1 open-label, proof-of-concept, single ascending dose trial that enrolled 72 patients in two three-dose cohorts: a co-administered intra-articular steroid cohort and a cohort that did not receive a steroid.

该公司的RMAT应用得到了第一阶段开放标签，概念验证，单次递增剂量试验的初步安全性和有效性研究结果的支持，该试验在两个三剂量组中招募了72名患者：共同给药的关节内类固醇组群和未接受类固醇的队列。

PCRX-201 was well tolerated, with efficacy observed through at least 52 weeks at all doses and cohorts. The highest level of efficacy was achieved in the co-administered steroid group, which showed a greater percentage of patients with at least a 50% improvement in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain and stiffness scores, as well as a meaningful improvement in (Knee Injury and Osteoarthritis Outcomes Score) KOOS functional assessment.

PCRX-201耐受性良好，在所有剂量和队列中至少52周观察到疗效。联合使用类固醇组的疗效最高，西安大略省和麦克马斯特大学骨关节炎指数（WOMAC）疼痛和僵硬评分改善至少50%的患者比例更高，以及（膝关节损伤和骨关节炎结局评分）KOOS功能评估的有意义的改善。

Preliminary 36-week data were presented at the Osteoarthritis Research Society International (OARSI) 2023 World Congress, the premier annual international forum in osteoarthritis research and treatment. The 52-week data have been accepted for presentation at OARSI 2024 taking place in Vienna, Austria in April 2024 and the company expects to present 104-week efficacy and safety data later this year..

在国际骨关节炎研究学会（OARSI）2023年世界大会上提交了为期36周的初步数据，该大会是骨关节炎研究和治疗的主要年度国际论坛。52周的数据已被接受于2024年4月在奥地利维也纳举行的OARSI 2024会议上提交，该公司预计今年晚些时候将提交104周的疗效和安全性数据。

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising therapies, including genetic therapies, that are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and for which preliminary clinical evidence indicates that the drug or therapy has the potential to address an unmet medical need..

根据《21世纪治愈法案》建立的“RMAT指定”是一个专门的计划，旨在加速开发和审查有前途的疗法，包括基因疗法，这些疗法旨在治疗、修改、逆转或治愈严重或危及生命的疾病或状况，初步临床证据表明，该药物或疗法有可能解决未满足的医疗需求。

RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with 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.

RMAT指定提供了FDA关于有效药物开发的深入指导的好处，包括与FDA早期互动讨论替代或中间终点的能力，支持加速批准和满足批准后要求的潜在方法，生物制剂许可证申请（BLA）的潜在优先审查，以及其他加快开发和审查的机会。

PCRX-201 was also granted Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency in May 2023..

2023年5月，PCRX-201还被欧洲药品管理局授予高级治疗药品（ATMP）称号。

About PCRX-201

关于PCRX-201

PCRX-201 was acquired from GQ Bio Therapeutics GmbH, a privately held biopharmaceutical company headquartered in Hamburg, Germany. GQ Bio’s product candidates are next-generation gene transfer vehicles. These gene therapy vectors are highly efficient in entering joint cells to confer multi-year clinical benefit.

PCRX-201是从GQ Bio Therapeutics GmbH收购的，GQ Bio Therapeutics GmbH是一家总部位于德国汉堡的私营生物制药公司。GQ Bio的候选产品是下一代基因转移载体。这些基因治疗载体在进入关节细胞方面非常有效，可以带来多年的临床益处。

In PCRX-201, the high-capacity adenoviral gene therapy vector codes for the expression of IL-1Ra, a cytokine inhibitor that plays a central role in blocking inflammation and catabolic processes that are associated with pain and disease progression in osteoarthritis. Its unique design includes an inducible promoter so that, only in the presence of inflammation signaling, the vector turns joint cells into factories to produce sustained therapeutic levels of IL-1Ra to manage pain and mitigate osteoarthritis-related joint damage while remaining localized to the joint space..

在PCRX-201中，高容量腺病毒基因治疗载体编码IL-1Ra的表达，IL-1Ra是一种细胞因子抑制剂，在阻断与骨关节炎疼痛和疾病进展相关的炎症和分解代谢过程中起着核心作用。其独特的设计包括一个可诱导的启动子，因此，只有在存在炎症信号的情况下，载体才能将关节细胞转化为工厂，产生持续的治疗水平的IL-1Ra，以控制疼痛并减轻骨关节炎相关的关节损伤，同时保持局部关节间隙。

About Pacira

关于Pacira

Pacira BioSciences, Inc. (Nasdaq: PCRX) is committed to providing a non-opioid option to as many patients as possible to redefine the role of opioids as rescue therapy only. 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 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 ioveraº®, a novel, handheld device for delivering immediate, long-acting, drug-free pain control using precise, controlled doses of cold temperature to a targeted nerve.

Pacira BioSciences，Inc.（纳斯达克：PCRX）致力于为尽可能多的患者提供非阿片类药物选择，以重新定义阿片类药物仅作为救援治疗的作用。Pacira有三种商业阶段的非阿片类药物治疗：EXPAREL®（布比卡因脂质体注射混悬液），一种长效局部镇痛药，目前被批准用于浸润，筋膜平面阻滞，以及作为用于术后疼痛管理的肌间沟臂丛神经阻滞；Zilletta®（曲安奈德缓释注射混悬液），一种缓释关节内注射，用于治疗骨关节炎膝关节疼痛；和ioveraº®，一种新颖的手持设备，可通过精确、可控的低温剂量向目标神经提供即时、长效、无药物的疼痛控制。