PCRX-201 (enekinragene inzadenovec)
Pacira is developing gene therapy approaches intended to broaden access, with an initial focus on knee osteoarthritis (OA). PCRX-201 is currently being studied in the fundamental, underlying chronic inflammatory processes that contribute to “wear and tear” over time in knee OA.
These are investigational treatments that have not yet been evaluated or approved by the US Food and Drug Administration.
Osteoarthritis of the knee
A serious disease starved for innovation
Knee OA is a highly prevalent, degenerative, and painful condition affecting approximately 14 million people in the United States alone. Patients aren’t satisfied with current treatment options, which only provide 3-6 months of relief and are based on decades-old mechanisms that don’t address the drivers of disease.1-3
This lack of innovation has created apathy, leading patients to believe they need to accept their pain and the impact it has on their life. Millions of patients per year are receiving total knee replacements as a last resort, which are expensive, can have complications, and aren’t guaranteed solutions.
We have an innovative pipeline, including an investigational gene therapy product candidate (PCRX-201) that has the potential to transform chronic pain management, beginning with knee OA.
PCRX-201: Developing gene therapy approaches intended to support broader therapeutic use
PCRX-201’s innovative design, manufacturing process, and local administration aims to solve many of the challenges that have made gene therapy inaccessible for common diseases.
PCRX-201 is designed to be injected locally into the knee joint to boost cellular IL-1Ra production and block IL-1 pathway activation, intended to reduce chronic inflammation.
Designed to make genetic medicines more efficient and enable the use of large and multiple gene constructs. PCRX-201 is the lead program from this platform.
PCRX-201 is designed to use an inflammation-responsive promoter intended to only produce IL-1Ra when needed, mimicking the body’s natural response to inflammation.
The IL-1 pathway: A root cause of disease
It’s been known for years that chronic IL-1 activation is an underlying driver of knee inflammation, joint degeneration, and pain. Several FDA-approved drugs have successfully targeted the IL-1 pathway in other joint inflammatory conditions, proving that blocking its activation is a well-validated, de-risked strategy for reducing inflammation. However, these existing drugs aren’t practical for knee OA because they would require very high doses or daily injections into the knee.
A new direction in knee OA treatment
PCRX-201 is designed to supplement receptor antagonist (IL-1Ra) production, which blocks IL-1 pathway activation and dampens down chronic inflammation in the knee.
PCRX-201 is poised to transform how we approach osteoarthritis treatment with the goal of helping patients return to normal function.
PCRX-201 has shown promise in early-stage clinical trials
PCRX-201 is currently being evaluated in a Phase 1 trial with 72 knee OA patients (NCT04119687).
References
- Osteoarthritis Research Society International White Paper Osteoarthritis: A Serious Disease, Submitted to the U.S. Food and Drug Administration Dec. 1, 2016.
- A National Public Health Agenda for Osteoarthritis: 2020 Update; Osteoarthritis Action Alliance (Centers for Disease Control and Arthritis Foundation).
- Voice of the Patient; Osteoarthritis Foundation Summary Report from FDA’s Patient-Focused Drug Development Meeting Sep. 30, 2017.