Pacira Announces 104-Week Safety and Efficacy Data Following Local Administration of PCRX-201 for Moderate to Severe Osteoarthritis of the Knee
Pacira BioSciences (NASDAQ: PCRX) announced promising 104-week safety and efficacy data for its gene therapy candidate PCRX-201 in treating knee osteoarthritis. The Phase 1 trial involving 72 patients demonstrated sustained improvements in knee pain, stiffness, and function. The pretreated cohort showed 48-65% pain improvement and 53-72% stiffness reduction from baseline. Over 70% of participants achieved >50% pain reduction by week 16. The therapy showed a well-tolerated safety profile with no serious treatment-related adverse events. PCRX-201, which received FDA's RMAT designation in March 2024, is designed to produce IL-1Ra protein to reduce inflammation. A Phase 2 study is planned for 2025.
Pacira BioSciences (NASDAQ: PCRX) ha annunciato dati promettenti sulla sicurezza e l'efficacia a 104 settimane per il suo candidato alla terapia genica PCRX-201 nel trattamento dell'osteoartrite del ginocchio. Lo studio di fase 1, che ha coinvolto 72 pazienti, ha dimostrato miglioramenti sostenuti nel dolore, nella rigidità e nella funzionalità del ginocchio. Il gruppo pretrattato ha mostrato un miglioramento del dolore del 48-65% e una riduzione della rigidità del 53-72% rispetto al baseline. Oltre il 70% dei partecipanti ha raggiunto una riduzione del dolore superiore al 50% entro la settimana 16. La terapia ha mostrato un profilo di sicurezza ben tollerato, senza gravi eventi avversi correlati al trattamento. PCRX-201, che ha ricevuto la designazione RMAT dalla FDA a marzo 2024, è progettato per produrre la proteina IL-1Ra per ridurre l'infiammazione. Uno studio di fase 2 è programmato per il 2025.
Pacira BioSciences (NASDAQ: PCRX) anunció datos prometedores de seguridad y eficacia a 104 semanas para su candidato de terapia génica PCRX-201 en el tratamiento de la osteoartritis de rodilla. El ensayo de fase 1, que involucró a 72 pacientes, demostró mejoras sostenidas en el dolor, la rigidez y la función de la rodilla. El grupo pretratado mostró una mejora del 48-65% en el dolor y una reducción del 53-72% en la rigidez desde el inicio. Más del 70% de los participantes alcanzó una reducción del dolor superior al 50% para la semana 16. La terapia presentó un perfil de seguridad bien tolerado, sin eventos adversos graves relacionados con el tratamiento. PCRX-201, que recibió la designación RMAT de la FDA en marzo de 2024, está diseñado para producir la proteína IL-1Ra para reducir la inflamación. Un estudio de fase 2 está planeado para 2025.
Pacira BioSciences (NASDAQ: PCRX)는 무릎 골관절염 치료를 위한 유전자 치료 후보물질 PCRX-201의 104주 안전성 및 효능에 대한 유망한 데이터를 발표했습니다. 72명의 환자를 포함한 1상 시험에서는 무릎의 통증, 뻣뻣함 및 기능에 지속적인 개선을 보였습니다. 치료 전 집단은 기준치에서 통증이 48-65% 개선되었고 뻣뻣함이 53-72% 감소했습니다. 참가자의 70% 이상이 16주 차에 50% 이상의 통증 감소를 달성했습니다. 이 요법은 심각한 치료 관련 부작용이 없는 잘 내성을 보이는 안전성 프로필을 보여주었습니다. PCRX-201은 2024년 3월 FDA의 RMAT 지정을 받았으며, 염증을 줄이기 위해 IL-1Ra 단백질을 생성하도록 설계되었습니다. 2025년에는 2상 연구가 계획되어 있습니다.
Pacira BioSciences (NASDAQ: PCRX) a annoncé des données prometteuses de sécurité et d'efficacité sur 104 semaines pour son candidat à la thérapie génique PCRX-201 dans le traitement de l'arthrose du genou. L'essai de phase 1, impliquant 72 patients, a montré des améliorations durables de la douleur, de la raideur et de la fonction du genou. Le groupe prétraité a montré une amélioration de la douleur de 48 à 65 % et une réduction de la raideur de 53 à 72 % par rapport à la valeur de référence. Plus de 70 % des participants ont atteint une réduction de la douleur supérieure à 50 % d'ici la 16e semaine. La thérapie a montré un profil de sécurité bien toléré, sans événements indésirables graves liés au traitement. PCRX-201, qui a reçu la désignation RMAT de la FDA en mars 2024, est conçu pour produire la protéine IL-1Ra afin de réduire l'inflammation. Une étude de phase 2 est prévue pour 2025.
Pacira BioSciences (NASDAQ: PCRX) hat vielversprechende Daten zur Sicherheit und Wirksamkeit über 104 Wochen für seinen Gentherapiekandidaten PCRX-201 zur Behandlung von Kniearthrose veröffentlicht. Die Phase 1-Studie umfasste 72 Patienten und zeigte nachhaltige Verbesserungen bei Knieschmerzen, Steifheit und Funktion. Die vorbehandelte Gruppe zeigte eine Schmerzverbesserung von 48-65% und eine Steifigkeitsreduktion von 53-72% im Vergleich zur Ausgangssituation. Über 70% der Teilnehmer erreichten bis zur Woche 16 eine Schmerzlinderung von über 50%. Die Therapie wies ein gut verträgliches Sicherheitsprofil auf, ohne schwerwiegende behandlungsbedingte unerwünschte Ereignisse. PCRX-201, das im März 2024 von der FDA die RMAT-Übertragung erhielt, ist darauf ausgelegt, das IL-1Ra-Protein zu produzieren, um Entzündungen zu reduzieren. Eine Phase-2-Studie ist für 2025 geplant.
- Sustained efficacy demonstrated over 104 weeks with single injection
- 48-65% pain improvement and 53-72% stiffness reduction in pretreated cohort
- 70% of participants achieved >50% pain reduction by week 16
- No serious treatment-related adverse events reported
- Received FDA RMAT designation, potentially expediting development and review
- Phase 2 study advancement planned for 2025
- Treatment-related joint swelling occurred in 36% of pretreated patients and 61% of non-pretreated patients
Insights
The 104-week data for PCRX-201 represents a significant breakthrough in osteoarthritis treatment. The results show impressive durability with
The safety profile is particularly encouraging, with no serious treatment-related adverse events. The reduced incidence of joint effusions in pretreated patients (
The RMAT designation from the FDA and ATMP designation from EMA significantly enhance the product's regulatory pathway, potentially accelerating time to market for this first-in-class osteoarthritis gene therapy.
With 14 million U.S. patients suffering from knee osteoarthritis, PCRX-201 addresses a massive market opportunity. Current treatments typically last only 3-6 months, creating a significant competitive advantage for PCRX-201's demonstrated two-year durability. The local administration approach differentiates it from traditional systemic gene therapies, potentially offering a better risk-benefit profile and broader market acceptance.
The planned Phase 2 study in 2025 represents a critical value inflection point. The RMAT designation could accelerate the approval timeline and reduce development costs. Given Pacira's established presence in non-opioid pain management, successful commercialization could significantly expand their market position and revenue potential in the musculoskeletal therapeutics space.
-- Poster to be presented at ACR Convergence annual meeting --
PARSIPPANY, N.J., Nov. 14, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies to transform the lives of patients, today announced new data demonstrating its gene therapy for osteoarthritis candidate, PCRX-201 (enekinragene inzadenovec), provided sustained improvements in knee pain, stiffness, and function to 104 weeks following local administration, with a well-tolerated safety profile. The data, which indicate a potential for sustained clinical efficacy in patients with moderate to severe osteoarthritis of the knee (OAK), will be presented during a poster session at the American College of Rheumatology’s annual ACR Convergence meeting on Sunday, November 17 from 10:30 am – 12:30 pm EST.
“The results of this large phase 1 study demonstrate durable pain relief across all levels of disease severity for at least 2 years following a single injection. This is promising, considering traditional pain management interventions provide an average of three to six months of effect,” said Stanley Cohen, MD, a board-certified rheumatologist and Co-Medical Director of the Metroplex Clinical Research Center in Dallas, TX, who was lead investigator in this trial and primary author on the poster presentation. “Unlike other treatments that temporarily alleviate symptoms, PCRX-201 addresses a root cause of osteoarthritis knee pain—inflammation—to help control patients’ pain for years rather than months.”
The new data is derived from an open-label, phase 1 trial investigating the safety and efficacy of PCRX-201 administered via ultrasound-guided intraarticular injection in 72 patients with OAK graded at 2, 3, or 4 on the Kellgren-Lawrence scale, a semiquantitative method for evaluating the severity of osteoarthritis on a scale of 0-4.
Participants were broken into two cohorts. The first cohort received one of three doses of PCRX-201. The second cohort received concurrent pretreatment with an intraarticular corticosteroid (methylprednisolone 40 mg), a technique common in gene therapy dosing to improve tolerability and gene transfer.
Pain and function benefits were observed at all doses and across both cohorts over the full 104 weeks studied, with patients in the second cohort achieving greater pain reduction and fewer adverse events (AEs). Additional results in the pretreated cohort, across all doses, include:
48% -65% improvement in pain from baseline, as measured by the Western Ontario and McMaster Universities Arthritis Index-A (WOMAC-A)53% -72% improvement in stiffness from baseline, as measured by WOMAC-B- Improvements in function from baseline, as measured by the Knee Injury and Osteoarthritis Outcome Score (KOOS) Activities of Daily Living (ADL) scale, that were similar to improvements in WOMAC-A and WOMAC-B
- By 16 weeks more than
70% of participants achieved greater than50% reductions from baseline pain.
No serious treatment-emergent AEs related to the treatment or procedure were reported regardless of steroid pretreatment or dose level administered. Treatment-related joint effusions (swelling) were the most common AE, occurring in
“We look forward to continuing to advance the clinical investigation of PCRX-201 following these promising results, with a Phase 2, double-blind, active-controlled study planned for 2025,” said Frank D. Lee, chief executive officer of Pacira BioSciences. “Unlike traditional gene therapies, which are administered systemically and have primarily been limited to the treatment of rare diseases, we believe PCRX-201 holds the broad potential to provide a long-term pain management solution for the 14 million U.S. patients suffering from the negative impacts of osteoarthritis of the knee. With a local administration that delivers relief directly to the source—the knee joint capsule—PCRX-201 is on the leading edge of what could be possible for gene therapies and offers patients the hope for a long-lasting pain management solution that improves their ability to comfortably engage in activities of daily living, like climbing stairs and exercising.”
PCRX-201 is a locally administered gene therapy, designed to produce interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring, anti-inflammatory protein with a proven mechanism of action that reduces interleukin-1 (IL-1) signaling, a known factor in the development and progression of osteoarthritis of the knee. Unlike systemically administered gene therapies, PCRX-201 delivers the medicine where it matters to reduce pain and disability and potentially slow structural progression at the site of the disease. PCRX-201 uses 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 IL-1Ra expression once inflammation is quelled.
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.
About Pacira BioSciences
Pacira BioSciences 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, interscalene brachial plexus nerve block in adults, sciatic nerve block in the popliteal fossa in adults, and adductor canal block in adults 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. The company is also advancing the development of PCRX-201, 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.
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FAQ
What were the efficacy results of PCRX-201 in the 104-week knee osteoarthritis trial?
What is the safety profile of PCRX-201 for knee osteoarthritis treatment?
When did PCRX-201 receive FDA RMAT designation?
What are Pacira's next steps for PCRX-201 development?
