Pacira announces 104-week safety, efficay data for PCRX-201

Pacira (PCRX) BioSciences announced new data demonstrating its gene therapy for osteoarthritis candidate, PCRX-201, 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 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, 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. 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; 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 Activities of Daily Living scale, that were similar to improvements in WOMAC-A and WOMAC-B; By 16 weeks more than 70% of participants achieved greater than 50% 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 were the most common AE, occurring in 36% of patients who received steroid pretreatment vs 61% of patients who were not pretreated. The majority of effusions were mild to moderate in severity and resolved in a median of 33 days among patients in the pretreated group. PCRX-201 is a locally administered gene therapy, designed to produce interleukin-1 receptor antagonist, a naturally occurring, anti-inflammatory protein with a proven mechanism of action that reduces interleukin-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.