Pacira granted RMAT designation for PCRX-201

Pacira BioSciences announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation to PCRX-201, the company’s novel, intra-articular helper-dependent adenovirus gene therapy product candidate that codes for interleukin-1 receptor antagonist, for the treatment of osteoarthritis of the knee. 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. 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 pain and stiffness scores, as well as a meaningful improvement in KOOS functional assessment. Preliminary 36-week data were presented at the Osteoarthritis Research Society International 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.