Pacira BioSciences Announces Two-Year Efficacy Data Following a Single Local Administration of PCRX-201 in Patients with Mild to Severe Osteoarthritis of the Knee
Pacira BioSciences (Nasdaq: PCRX) has announced promising two-year efficacy data for its gene therapy candidate PCRX-201 in treating knee osteoarthritis. The study, presented at the 2025 OARSI World Congress, demonstrated sustained improvements in knee pain, stiffness, and function across all severity levels following a single injection.
The Phase 1 trial involved 72 patients aged 30-80, with two cohorts receiving different treatment protocols. The corticosteroid-pretreated cohort showed superior results with 48-65% pain reduction and 53-72% stiffness reduction, compared to 41-58% pain and 33-53% stiffness reductions in the non-pretreated group.
No serious treatment-related adverse events were reported. The most common side effect was joint swelling, occurring in 36% of pretreated patients versus 61% of non-pretreated patients. PCRX-201 received FDA's RMAT designation in March 2024 and EMA's ATMP designation in May 2023. A Phase 2 study (ASCEND) is currently underway.
Pacira BioSciences (Nasdaq: PCRX) ha annunciato dati promettenti sull'efficacia a due anni del suo candidato terapia genica PCRX-201 nel trattamento dell'osteoartrite del ginocchio. Lo studio, presentato al Congresso Mondiale OARSI 2025, ha mostrato miglioramenti duraturi nel dolore, nella rigidità e nella funzionalità del ginocchio in tutti i livelli di gravità dopo una singola iniezione.
La fase 1 ha coinvolto 72 pazienti di età compresa tra 30 e 80 anni, suddivisi in due gruppi con protocolli di trattamento differenti. Il gruppo pretrattato con corticosteroidi ha ottenuto risultati migliori, con una riduzione del dolore tra il 48% e il 65% e della rigidità tra il 53% e il 72%, rispetto al gruppo non pretrattato che ha mostrato riduzioni del dolore dal 41% al 58% e della rigidità dal 33% al 53%.
Non sono stati segnalati eventi avversi gravi correlati al trattamento. L'effetto collaterale più comune è stato il gonfiore articolare, presente nel 36% dei pazienti pretrattati contro il 61% di quelli non pretrattati. PCRX-201 ha ricevuto la designazione RMAT dalla FDA a marzo 2024 e la designazione ATMP dall'EMA a maggio 2023. Attualmente è in corso uno studio di fase 2 (ASCEND).
Pacira BioSciences (Nasdaq: PCRX) ha anunciado datos prometedores de eficacia a dos años para su candidato de terapia génica PCRX-201 en el tratamiento de la osteoartritis de rodilla. El estudio, presentado en el Congreso Mundial OARSI 2025, demostró mejoras sostenidas en el dolor, la rigidez y la función de la rodilla en todos los niveles de gravedad tras una única inyección.
El ensayo de fase 1 incluyó a 72 pacientes de entre 30 y 80 años, con dos cohortes que recibieron diferentes protocolos de tratamiento. La cohorte pretratada con corticosteroides mostró mejores resultados con una reducción del dolor del 48-65% y de la rigidez del 53-72%, en comparación con la reducción del dolor del 41-58% y de la rigidez del 33-53% en el grupo no pretratado.
No se reportaron eventos adversos graves relacionados con el tratamiento. El efecto secundario más común fue la inflamación articular, que ocurrió en el 36% de los pacientes pretratados frente al 61% de los no pretratados. PCRX-201 recibió la designación RMAT de la FDA en marzo de 2024 y la designación ATMP de la EMA en mayo de 2023. Actualmente se está llevando a cabo un estudio de fase 2 (ASCEND).
Pacira BioSciences (나스닥: PCRX)는 무릎 골관절염 치료를 위한 유전자 치료 후보물질 PCRX-201의 2년간 유효성 데이터를 발표했습니다. 2025년 OARSI 세계 학회에서 발표된 이 연구는 단일 주사 후 모든 중증도 단계에서 무릎 통증, 경직 및 기능의 지속적인 개선을 보여주었습니다.
1상 임상시험에는 30세에서 80세 사이의 72명의 환자가 참여했으며, 두 그룹이 서로 다른 치료 프로토콜을 받았습니다. 코르티코스테로이드 사전치료 그룹은 통증이 48-65%, 경직이 53-72% 감소하는 우수한 결과를 보였으며, 사전치료를 받지 않은 그룹은 통증 41-58%, 경직 33-53% 감소를 보였습니다.
치료 관련 심각한 부작용은 보고되지 않았습니다. 가장 흔한 부작용은 관절 부종으로, 사전치료 그룹에서 36%, 비사전치료 그룹에서 61% 발생했습니다. PCRX-201은 2024년 3월 FDA로부터 RMAT 지정을, 2023년 5월 EMA로부터 ATMP 지정을 받았습니다. 현재 2상 연구(ASCEND)가 진행 중입니다.
Pacira BioSciences (Nasdaq : PCRX) a annoncé des données prometteuses sur l'efficacité à deux ans de son candidat thérapie génique PCRX-201 dans le traitement de l'arthrose du genou. L'étude, présentée au Congrès mondial OARSI 2025, a démontré des améliorations durables de la douleur, de la raideur et de la fonction du genou à tous les niveaux de gravité après une injection unique.
L'essai de phase 1 a impliqué 72 patients âgés de 30 à 80 ans, répartis en deux cohortes recevant des protocoles de traitement différents. La cohorte prétraitée aux corticostéroïdes a montré des résultats supérieurs avec une réduction de la douleur de 48 à 65 % et de la raideur de 53 à 72 %, contre 41 à 58 % de réduction de la douleur et 33 à 53 % de réduction de la raideur dans le groupe non prétraité.
Aucun événement indésirable grave lié au traitement n'a été signalé. L'effet secondaire le plus fréquent était un gonflement articulaire, survenant chez 36 % des patients prétraités contre 61 % des patients non prétraités. PCRX-201 a reçu la désignation RMAT de la FDA en mars 2024 et la désignation ATMP de l'EMA en mai 2023. Une étude de phase 2 (ASCEND) est actuellement en cours.
Pacira BioSciences (Nasdaq: PCRX) hat vielversprechende Wirksamkeitsdaten über zwei Jahre für seinen Gentherapie-Kandidaten PCRX-201 bei der Behandlung von Kniearthrose bekannt gegeben. Die Studie, präsentiert auf dem OARSI Weltkongress 2025, zeigte anhaltende Verbesserungen bei Knieschmerzen, Steifheit und Funktion in allen Schweregraden nach einer einzigen Injektion.
Die Phase-1-Studie umfasste 72 Patienten im Alter von 30 bis 80 Jahren, die in zwei Kohorten mit unterschiedlichen Behandlungsprotokollen eingeteilt wurden. Die mit Kortikosteroiden vorbehandelte Kohorte zeigte überlegene Ergebnisse mit einer Schmerzreduktion von 48-65 % und einer Steifigkeitsreduktion von 53-72 %, verglichen mit 41-58 % Schmerz- und 33-53 % Steifigkeitsreduktion in der nicht vorbehandelten Gruppe.
Schwerwiegende behandlungsbedingte Nebenwirkungen wurden nicht berichtet. Die häufigste Nebenwirkung war Gelenkschwellung, die bei 36 % der vorbehandelten Patienten und 61 % der nicht vorbehandelten Patienten auftrat. PCRX-201 erhielt im März 2024 die RMAT-Zulassung der FDA und im Mai 2023 die ATMP-Zulassung der EMA. Eine Phase-2-Studie (ASCEND) läuft derzeit.
- Strong efficacy data with up to 65% pain reduction and 72% stiffness reduction over two years
- First-ever gene therapy to receive FDA's RMAT designation for osteoarthritis
- No serious treatment-related adverse events reported
- Phase 2 study already underway following promising Phase 1 results
- High incidence of joint swelling (61%) in non-pretreated patients
- Treatment requires corticosteroid pretreatment for optimal results
Insights
PCRX-201 showed impressive two-year efficacy data for knee osteoarthritis, representing significant advancement in Pacira's pipeline with regulatory fast-track designation.
The two-year efficacy data for PCRX-201 represents a potential breakthrough in osteoarthritis treatment. What makes this particularly significant is the sustained therapeutic effect lasting two years from a single injection - dramatically outperforming current standards that typically provide only 3-6 months of relief. The 48-65% pain reduction and 53-72% stiffness improvement in pretreated patients across all disease severity levels demonstrates meaningful clinical benefit.
The mechanism differentiates PCRX-201 from conventional approaches. This gene therapy uses a proprietary high-capacity adenovirus vector to boost IL-1Ra production with an inflammation-responsive promoter - essentially creating an "on-demand" anti-inflammatory response within the joint that activates specifically when inflammation is present. This addresses the underlying inflammatory pathway rather than merely masking symptoms.
The FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and EMA's Advanced Therapy Medicinal Products designation provide regulatory validation and potential acceleration pathways. Being the first-ever gene therapy for osteoarthritis to receive RMAT designation positions Pacira favorably from both scientific and competitive perspectives.
Efficacy across all structural severity subgroups (Kellgren-Lawrence grades 2-4) is particularly notable, as advanced osteoarthritis patients often have options before joint replacement. The safety profile appears manageable, with treatment-related effusions reduced by corticosteroid pretreatment and no serious treatment-related adverse events reported.
This represents a significant milestone in Pacira's strategic evolution beyond its historical focus on surgical pain management toward novel biologic therapies addressing chronic conditions with significant unmet needs.
Positive Phase 1 data and regulatory designations for PCRX-201 strengthen Pacira's diversification strategy and position in the massive osteoarthritis market.
The positive two-year data for PCRX-201 significantly enhances Pacira's strategic position in the $11+ billion global osteoarthritis treatment market. This gene therapy candidate demonstrates remarkable durability with effects lasting 8x longer than current standard treatments, potentially creating substantial competitive advantage and market disruption.
This development aligns perfectly with Pacira's stated "5x30 strategy for growth and value creation" and represents a critical pillar in their transformation from an acute surgical pain company to a diversified biopharmaceutical organization. The single-administration model offers compelling pharmacoeconomic advantages over current treatments requiring multiple annual injections.
The dual regulatory accelerations are particularly significant market catalysts. The RMAT designation provides tangible benefits including intensive FDA guidance, potential accelerated approval pathways, and priority review. These designations typically reduce time-to-market by approximately 25-30% and signal regulatory recognition of substantial unmet need.
Perhaps most compelling is the demonstrated effectiveness across all severity subgroups, including advanced disease (K/L grade 4). This dramatically expands the potential patient population beyond what many competing development programs can address. The 72-patient Phase 1 trial showed consistent efficacy across dose levels with a manageable safety profile.
With Phase 2 dosing already underway in the ASCEND study, Pacira is maintaining development momentum. While substantial clinical development work remains before potential commercialization, these results represent a meaningful de-risking event for this program, which could become the cornerstone of Pacira's future growth strategy in addressing chronic inflammatory conditions.
-- PCRX-201 demonstrated two years of clinically meaningful improvements in pain, stiffness and function across all structural severity subgroups, including advanced disease --
-- Study Findings Presented at 2025 OARSI World Congress--
BRISBANE, Calif., April 28, 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 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,” 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. “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.”
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 (
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
“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.
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 14 million individuals in the U.S. today.
In November 2024, Pacira reported promising data from a large Phase 1 study in which PCRX-201 provided sustained improvements in knee pain, stiffness, and function through two 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 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
Any statements in this press release about Pacira’s future expectations, plans, trends, outlook, projections and prospects, and other statements containing the words “believes,” “anticipates,” “plans,” “estimates,” “expects,” “intends,” “may,” “will,” “would,” “could,” “can” and similar expressions, constitute forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and the Private Securities Litigation Reform Act of 1995, including, without limitation, statements related to: the settlement described herein, ‘5x30’, our growth and business strategy; our future outlook, our intellectual property and patent terms, our growth and future operating results and trends, our strategy, plans, objectives, expectations (financial or otherwise) and intentions, future financial results and growth potential, including our plans with respect to the repayment of our indebtedness, anticipated product portfolio, development programs, development of products, strategic alliances and other statements that are not historical facts. For this purpose, any statement that is not a statement of historical fact should be considered a forward-looking statement. We cannot assure you that our estimates, assumptions and expectations will prove to have been correct. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks relating to, among others: the failure to realize the anticipated benefits and synergies from the acquisition of GQ Bio Therapeutics GmbH; risks associated with acquisitions, such as the risk that the acquired businesses will not be integrated successfully, that such integration may be more difficult, time-consuming or costly than expected or that the expected benefits of the transaction will not occur; our manufacturing and supply chain, global and U.S. economic conditions (including inflation and rising interest rates), and our business, including our revenues, financial condition, cash flow and results of operations; the success of our sales and manufacturing efforts in support of the commercialization of EXPAREL, ZILRETTA and iovera°; the rate and degree of market acceptance of EXPAREL, ZILRETTA and iovera°; the size and growth of the potential markets for EXPAREL, ZILRETTA and iovera° and our ability to serve those markets; our plans to expand the use of EXPAREL, ZILRETTA and iovera° to additional indications and opportunities, and the timing and success of any related clinical trials for EXPAREL, ZILRETTA and iovera°; the commercial success of EXPAREL, ZILRETTA and iovera°; the related timing and success of U.S. Food and Drug Administration supplemental New Drug Applications and premarket notification 510(k)s; the related timing and success of European Medicines Agency Marketing Authorization Applications; our plans to evaluate, develop and pursue additional product candidates utilizing our proprietary multivesicular liposome (“pMVL”) drug delivery technology; the approval of the commercialization of our products in other jurisdictions; clinical trials in support of an existing or potential pMVL-based product; our commercialization and marketing capabilities; our ability to successfully complete capital projects; the outcome of any litigation; the recoverability of our deferred tax assets; assumptions associated with contingent consideration payments; assumptions used for estimated future cash flows associated with determining the fair value of the Company; the anticipated funding or benefits of our share repurchase program; and factors discussed in the “Risk Factors” of our most recent Annual Report on Form 10-K and in other filings that we periodically make with the Securities and Exchange Commission (the “SEC”). In addition, the forward-looking statements included in this press release represent our views as of the date of this press release. Important factors could cause actual results to differ materially from those indicated or implied by forward-looking statements, and as such we anticipate that subsequent events and developments will cause our views to change. Except as required by applicable law, we undertake no intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, and readers should not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.
These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from those expressed or implied by these statements. These factors include the matters discussed and referenced in the “Risk Factors” of our most recent Annual Report on Form 10-K and in other filings that we periodically make with the SEC.
Investor Contact: Susan Mesco, (973) 451-4030 susan.mesco@pacira.com Media Contact: Sara Marino, (973) 370-5430 sara.marino@pacira.com
FAQ
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