Osteoarthritis, Knee

-- PCRX-201 is the first gene therapy product candidate to receive RMAT designation for osteoarthritis -- -- Designation supported by encouraging preliminary data from 72-patient Phase 1 study -- -- 52-Week Data Accepted for Presentation at OARSI 2024 and 104-week data to be submitted for presentation later this year -- TAMPA, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PCRX-201 (enekinragene inzadenovec), the company’s novel, intra-articular helper-dependent adenovirus (HDAd) gene therapy product candidate that codes for interleukin-1 receptor antagonist (IL-1Ra), for the treatment of osteoarthritis of the knee. “We are honored to receive FDA’s first-ever RMAT designation for a gene therapy product candidate in osteoarthritis," said Frank D. Lee, chief executive officer of Pacira BioSciences, Inc. “We continue to be encouraged by the preliminary clinical findings supporting PCRX-201 as a potential disease-modifying therapy for osteoarthritis, and we look forward to presenting additional follow-up data later this year.” 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 (WOMAC) pain and stiffness scores, as well as a meaningful improvement in (Knee Injury and Osteoarthritis Outcomes Score) KOOS functional assessment. Preliminary 36-week data were presented at the Osteoarthritis Research Society International (OARSI) 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. Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising therapies, including genetic therapies, that are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and for which preliminary clinical evidence indicates that the drug or therapy has the potential to address an unmet medical need. RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with 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 PCRX-201 PCRX-201 was acquired from GQ Bio Therapeutics GmbH, a privately held biopharmaceutical company headquartered in Hamburg, Germany. GQ Bio’s product candidates are next-generation gene transfer vehicles. These gene therapy vectors are highly efficient in entering joint cells to confer multi-year clinical benefit. In PCRX-201, the high-capacity adenoviral gene therapy vector codes for the expression of IL-1Ra, a cytokine inhibitor that plays a central role in blocking inflammation and catabolic processes that are associated with pain and disease progression in osteoarthritis. Its unique design includes an inducible promoter so that, only in the presence of inflammation signaling, the vector turns joint cells into factories to produce sustained therapeutic levels of IL-1Ra to manage pain and mitigate osteoarthritis-related joint damage while remaining localized to the joint space. About Pacira Pacira BioSciences, Inc. (Nasdaq: PCRX) is committed to providing a non-opioid option to as many patients as possible to redefine the role of opioids as rescue therapy only. 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. To learn more about Pacira, including the corporate mission to reduce overreliance on opioids, visit www.pacira.com. Forward-Looking Statements Any statements in this press release about Pacira’s future expectations, plans, trends, outlook, projections and prospects, and other statements containing the words “believes,” “anticipates,” “plans,” “estimates,” “expects,” “intends,” “may,” “will,” “would,” “could,” “can” and similar expressions, constitute forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and the Private Securities Litigation Reform Act of 1995, including, without limitation, statements related to our growth and future operating results and trends, our strategy, plans, objectives, expectations (financial or otherwise) and intentions, future financial results and growth potential, including our plans with respect to the repayment of our indebtedness, anticipated product portfolio, development programs, patent terms, development of products, strategic alliances and intellectual property and other statements that are not historical facts. For this purpose, any statement that is not a statement of historical fact should be considered a forward-looking statement. We cannot assure you that our estimates, assumptions and expectations will prove to have been correct. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks relating to, among others: the integration of our new chief executive officer; risks associated with acquisitions, such as the risk that the acquired businesses will not be integrated successfully, that such integration may be more difficult, time-consuming or costly than expected or that the expected benefits of the transaction will not occur; our manufacturing and supply chain, global and U.S. economic conditions (including inflation and rising interest rates), and our business, including our revenues, financial condition, cash flow and results of operations; the success of our sales and manufacturing efforts in support of the commercialization of EXPAREL, ZILRETTA and iovera°; the rate and degree of market acceptance of EXPAREL, ZILRETTA and iovera°; the size and growth of the potential markets for EXPAREL, ZILRETTA and iovera° and our ability to serve those markets; our plans to expand the use of EXPAREL, ZILRETTA and iovera° to additional indications and opportunities, and the timing and success of any related clinical trials for EXPAREL, ZILRETTA and iovera°; the commercial success of EXPAREL, ZILRETTA and iovera°; the related timing and success of U.S. Food and Drug Administration supplemental New Drug Applications and premarket notification 510(k)s; the related timing and success of European Medicines Agency Marketing Authorization Applications; our plans to evaluate, develop and pursue additional product candidates utilizing our proprietary multivesicular liposome (“pMVL”) drug delivery technology; the approval of the commercialization of our products in other jurisdictions; clinical trials in support of an existing or potential pMVL-based product; our commercialization and marketing capabilities; our ability to successfully complete capital projects; the outcome of any litigation; the ability to successfully integrate any future acquisitions into our existing business; the recoverability of our deferred tax assets; assumptions associated with contingent consideration payments; and factors discussed in the “Risk Factors” of our most recent Annual Report on Form 10-K and in other filings that we periodically make with the Securities and Exchange Commission (the “SEC”). In addition, the forward-looking statements included in this press release represent our views as of the date of this press release. Important factors could cause actual results to differ materially from those indicated or implied by forward-looking statements, and as such we anticipate that subsequent events and developments will cause our views to change. Except as required by applicable law, we undertake no intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, and readers should not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from those expressed or implied by these statements. 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TAMPA, Fla., March 06, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX) today announced that it will participate in an analyst-led fireside chat at the Barclays 26th Annual Global Healthcare Conference at 1:35 PM ET on Wednesday, March 13, 2024 in Miami. Live audio of the event can be accessed by visiting the “Events” page of the company’s website at investor.pacira.com. A replay of the webcast will also be available for two weeks following the event. About Pacira Pacira BioSciences, Inc. (Nasdaq: PCRX) is committed to providing a non-opioid option to as many patients as possible to redefine the role of opioids as rescue therapy only. 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. To learn more about Pacira, including the corporate mission to reduce overreliance on opioids, visit www.pacira.com.

CytoMed diversifies into regenerative medicine SINGAPORE, March 04, 2024 (GLOBE NEWSWIRE) -- CytoMed Therapeutics Limited (NASDAQ: GDTC) (“CytoMed” or “Company”), a Singapore-based biopharmaceutical company focused on harnessing its proprietary technologies to develop novel donor-derived cell-based allogeneic therapies for the treatment of various cancers and aging-related diseases, today announced that it has entered into a Research Collaboration Agreement (“RCA”) with Sengkang General Hospital Pte Ltd (“SKH”), a public hospital in Singapore and a member of the SingHealth group to establish proof-of-concept use of an injectable cartilage regeneration therapy that has been developed with donor-sourced, (allogeneic) umbilical cord-derived mesenchymal stem cells (“UC-MSCs”) to treat cartilage injury. This injectable therapy will be implemented in vivo to support the application and commencement of a proposed Phase 1 clinical trial in Singapore, and is anticipated to complete within two years. The use of MSC as a potential treatment for tissue regeneration, inflammatory and regenerative diseases has been the subject of increasing interest in the global scientific community. Osteoarthritis of the knee is a debilitating disease affecting Singapore’s aging population and the clinical trial will pioneer stem cell use for regenerative medicine in Singapore. UC-MSCs are a type of adult stem cells that can be isolated from the umbilical cord tissue after childbirth. These cells possess unique properties that have demonstrated to be promising for various therapeutic applications, especially when these cells are freshly harvested and isolated from the umbilical cord, as compared to MSCs derived from frozen tissues, of which the yield and quality may potentially suffer. UC-MSCs are considered multipotent and are able to differentiate into different cell types, including bone, cartilage, fat, and muscle cells. They also exhibit immunomodulatory and anti-inflammatory properties, making them attractive for regenerative medicine and immune-related disorders. "Our collaboration with CytoMed marks a significant step in advancing research on allogeneic in-vivo UC-MSC for cartilage repair, including osteoarthritis of the knee. Through our commitment to the Investigator Initiated Trial (IIT), we aim to gather crucial safety and efficacy data and evidence to make this intervention more accessible to patients," said Assistant Professor Francis Wong, Director of Research at SKH. “The unique advantages of UC-MSCs include their abundant supply, compared to cord blood-derived MSCs or adult stem cell sources like bone marrow-derived MSCs. Freshly harvested UC-MSCs are believed to possess higher proliferation capacity and greater differentiation potential. Additionally, UC-MSCs have been shown to exhibit lower immunogenicity and are less likely to be rejected by the recipient’s immune system, which opens the possibility for allogeneic (donor-to-recipient) transplantation. Therefore, UC-MSC would serve as an ideal source of regenerative cells for treating osteoarthritis of the knee. This hypothesis has also been supported by many international published clinical papers. We are excited to embark on this collaboration, which would potentially be the first stem cell trial in Singapore,” added Dr Tan Wee Kiat, co-CEO and Chief Operating Officer of CytoMed. Under the RCA, CytoMed will provide the allogeneic UC-MSC, processed and expanded in its own state-of-the-art GMP laboratory while SKH will provide the infrastructure, manpower and scientific data analysis. CytoMed is focused on cancer immunotherapies. The RCA has been established within a year of approval of CytoMed’s Phase 1 IIT with the National University Hospital of Singapore, which sought to evaluate the safety and efficacy of CytoMed’s novel allogeneic chimeric antigen receptor T-cell therapy (“CAR-T”), which is the first home-grown CAR-T therapy in Singapore, for the treatment of liquid and solid cancers. For more information about the Company's services, latest news, and ongoing initiatives, please visit About CytoMed Therapeutics Limited (CytoMed) Incorporated in 2018, CytoMed was spun off from the Agency for Science, Technology and Research (A*STAR), Singapore’s leading research and development agency in the public sector. It is a biopharmaceutical company focused on harnessing its licensed proprietary technologies, namely gamma delta T cell and iPSC-derived gamma delta Natural Killer T cell, to create novel cell-based allogeneic immunotherapies for the treatment of human cancers. The development of novel technologies has been inspired by the clinical success of existing CAR-T therapies in treating haematological malignancies, as well as the current clinical limitations and commercial challenges in extrapolating the CAR-T principle into the treatment of solid tumors. For more information, please visit and follow us on Twitter (“X”) @CytomedSG, on LinkedIn, and Facebook. Forward-Looking Statements This press release contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning plans, objectives, goals, strategies, future events or performance, and underlying assumptions and other statements that are other than statements of historical facts. When the Company uses words such as "may, "will, "intend," "should," "believe," "expect," "anticipate," "project," "estimate" or similar expressions that do not relate solely to historical matters, it is making forward-looking statements. Forward-looking statements are not guarantees of future performance and involve risks and uncertainties that may cause the actual results to differ materially from the Company's expectations discussed in the forward-looking statements. These statements are subject to uncertainties and risks including, but not limited to, the following: the Company's plans to develop and commercialize its product candidates; the initiation, timing, progress and results of the Company's current and future pre-clinical studies and clinical trials and the Company's R&D programs; the Company's expectations regarding the impact of the ongoing COVID-19 pandemic on its business, the Company's industry and the economy; the Company's estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company's ability to successfully acquire or obtain licenses for additional product candidates on reasonable terms; the Company's ability to establish and maintain collaborations and/or obtain additional funding and assumptions underlying or related to any of the foregoing and other risks contained in reports filed by the Company with the SEC. For these reasons, among others, investors are cautioned not to place undue reliance upon any forward-looking statements in this press release. Additional factors are discussed in the Company's filings with the SEC, which are available for review at The Company undertakes no obligation to publicly revise these forward-looking statements to reflect events or circumstances that arise after the date hereof. Contact: CytoMed Therapeutics enquiry@cytomed.sg Attention: Ms. Lynne Ng, Legal Consultant