Pacira Announces the Presentation of 104-Week Safety and Efficacy Data Following Local Administration of PCRX-201 for Moderate to Severe Osteoarthritis of the Knee

Rhea-AI Summary

Pacira BioSciences (NASDAQ: PCRX) announced the upcoming presentation of new 104-week safety and efficacy data for PCRX-201, its gene therapy candidate for moderate to severe osteoarthritis of the knee. The presentation will take place at the ACR Convergence annual meeting in Washington, D.C. on November 17.

PCRX-201 is a locally administered gene therapy designed to produce interleukin-1 receptor antagonist (IL-1Ra), which reduces IL-1 signaling, a factor in osteoarthritis progression. It uses an inducible promoter to express IL-1Ra when inflammation is present in the joint. In March 2024, PCRX-201 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, becoming the first gene therapy for osteoarthritis to do so. This designation provides benefits for expedited development and review. The therapy also received Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency in May 2023.

Positive

• PCRX-201 received RMAT designation from the FDA, potentially expediting development and review
• PCRX-201 is the first gene therapy for osteoarthritis to receive RMAT designation
• The therapy also received ATMP designation from the European Medicines Agency
• New 104-week safety and efficacy data to be presented at a major industry conference

Negative

• None.

Insights

Medical Research Analyst

The presentation of 104-week safety and efficacy data for PCRX-201 in treating moderate to severe osteoarthritis of the knee is a significant development for Pacira BioSciences. This gene therapy candidate represents a novel approach in the field of osteoarthritis treatment, potentially offering long-term relief from a single injection.

Key points to consider:

• PCRX-201 is designed to produce IL-1Ra, targeting a known inflammatory pathway in osteoarthritis
• The therapy uses an inducible promoter, potentially allowing for a more natural and responsive treatment
• PCRX-201 has received both RMAT designation from the FDA and ATMP designation from the EMA, indicating its potential to address unmet medical needs
• The 104-week data suggests sustained clinical effects, which could be a game-changer in osteoarthritis management

While promising, investors should note that this is still a candidate therapy. The upcoming presentation will be important in assessing its long-term efficacy and safety profile, which will influence its potential market impact and regulatory pathway.

Biotechnology Expert

The development of PCRX-201 represents a significant advancement in the field of osteoarthritis treatment. As a locally administered gene therapy, it addresses several key challenges in current treatments:

• Targeted delivery: By administering the therapy directly to the affected joint, it potentially reduces systemic side effects
• Long-lasting effects: The 104-week data suggests durability, which could greatly improve patient compliance and quality of life
• Responsive mechanism: The inducible promoter mimics natural inflammatory responses, potentially offering a more physiological treatment approach

The RMAT and ATMP designations are particularly noteworthy, as they may accelerate the regulatory process and market entry. However, investors should be aware that gene therapies often face unique challenges in manufacturing, pricing and reimbursement. The success of PCRX-201 could position Pacira as a leader in non-opioid pain management and regenerative medicine for osteoarthritis, potentially opening up a substantial market opportunity.

-- Poster to be presented at ACR Convergence annual meeting --

TAMPA, Fla., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies, today announced the upcoming presentation of new data in support of its gene therapy candidate, PCRX-201 (enekinragene inzadenovec). The data will be presented at the American College of Rheumatology’s annual ACR Convergence meeting, being held in Washington, D.C. November 14–19.

Presentation Title: Sustained Clinical Effects After a Single Intra-articular Injection of PCRX-201 for Moderate-to-Severe Osteoarthritis of the Knee

Presented By: Stanley Cohen, MD, a board-certified rheumatologist and Co-Medical Director of the Metroplex Clinical Research Center in Dallas, TX

Date & Time: Sunday, November 17 from 10:30 am – 12:30 pm EST

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 and 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 to reduce pain and disability and potentially slow structural progression.

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, and as an interscalene brachial plexus nerve block 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.

FAQ

What is PCRX-201 and what is it designed to treat?

PCRX-201 is a locally administered gene therapy designed to treat moderate to severe osteoarthritis of the knee. It produces interleukin-1 receptor antagonist (IL-1Ra), which reduces inflammation in the joint.

When and where will the new PCRX-201 data be presented?

The new 104-week safety and efficacy data for PCRX-201 will be presented on November 17 at the ACR Convergence annual meeting in Washington, D.C.

What designations has PCRX-201 received from regulatory agencies?

PCRX-201 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA in March 2024 and Advanced Therapy Medicinal Products (ATMP) designation from the European Medicines Agency in May 2023.

How does PCRX-201 differ from systemically administered gene therapies?

Unlike systemic gene therapies, PCRX-201 is locally administered and uses an inducible promoter to express IL-1Ra only when inflammation is present in the joint, mimicking the body's natural response.