Pacira launches Phase 2 osteoarthritis gene therapy trial

BRISBANE, Calif. - Pacira BioSciences, Inc. (NASDAQ:PCRX), a $1.15 billion market cap biopharmaceutical company with a robust 64% gross margin, announced the initiation of a Phase 2 study for its gene therapy candidate PCRX-201, marking a significant step in the development of potential new treatments for osteoarthritis (OA) of the knee. The first patient has been dosed in the ASCEND study, which is evaluating the safety and efficacy of the therapy designed to enhance the production of the anti-inflammatory protein IL-1Ra within the knee joint. The company’s stock has shown strong momentum, gaining over 56% in the past six months, and according to InvestingPro analysis, the stock appears undervalued based on its Fair Value assessment.

The ASCEND trial will enroll approximately 135 patients aged 45 to 80 with painful OA of the knee, across various severity grades. The study will test two doses of PCRX-201, comparing them to a saline placebo. All participants will receive a concurrent corticosteroid pretreatment to improve gene transfer and tolerability.

PCRX-201 is based on Pacira’s proprietary high-capacity adenovirus (HCAd) vector platform. This novel design allows the therapy to be injected locally into the knee, with an inducible promoter that regulates the expression of IL-1Ra in response to inflammation levels. The therapy aims to address chronic inflammation, which contributes to OA joint degeneration, by providing sustained pain relief and improved joint function.

The company expects to report the initial topline results from Part A of the study before the end of 2026, with primary endpoints focusing on the safety profile of PCRX-201 compared to the placebo. Secondary and exploratory endpoints will assess changes in pain and physical function, using established metrics such as the Numerical Rating Scale and the Western Ontario and McMaster Universities Index.

Pacira’s previous Phase 1 study of PCRX-201 demonstrated promising results, showing extended pain relief and a well-tolerated safety profile for up to two years post-administration. These findings were presented at the American College of Rheumatology’s annual meeting.

PCRX-201 has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration and Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency, reflecting its potential as a significant advancement over existing treatments.

Pacira, known for its commitment to non-opioid pain therapies, has three commercial-stage products and is exploring further uses of its HCAd vector platform for other cytokine-based genetic therapies. This study is part of the company’s broader ’5x30’ growth strategy, aiming to expand its portfolio and establish itself as a leading biopharmaceutical organization. With annual revenues of $701 million and an overall "GREAT" financial health score from InvestingPro, which offers comprehensive analysis and 10+ additional ProTips for this stock, the company appears well-positioned to execute its growth strategy. Analyst price targets for PCRX range from $21 to $48, reflecting varied expectations about the company’s potential.

The information in this article is based on a press release statement from Pacira BioSciences, Inc.

In other recent news, Pacira BioSciences has provided updates on its financial and strategic initiatives. The company has set its 2025 revenue guidance between $725 million and $765 million, representing a year-over-year growth of 3% to 9%, aligning with analysts’ expectations of $744 million. Needham has raised its price target for Pacira to $32, maintaining a buy rating, while H.C. Wainwright increased its target to $48, citing conservative revenue forecasts and positive developments for Exparel. Pacira’s recent acquisition of GQ Bio for $32 million, including $18 million in upfront cash, supports its "5x30" strategy by enhancing its pipeline with PCRX-201. Additionally, DOMA Perpetual Capital Management has nominated three candidates for Pacira’s board, aiming to address perceived deficiencies in financial and legal expertise. The nominations come amid Pacira’s focus on expanding its product use and developing new treatments. Pacira has also adopted a majority voting standard for director elections in uncontested scenarios, emphasizing enhanced corporate governance. These developments reflect Pacira’s ongoing efforts to optimize shareholder returns and strategic growth.

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