Melbourne, Australia-based Mesoblast Limited announced promising data from a phase 2 trial of the company’s novel stem cell therapy for patients with rheumatoid arthritis (RA), which shows its therapeutic benefits continued 9 months after administration of a single intravenous (IV) dose.
The stem cell infusion of Mesoblast’s allogeneic Mesenchymal Precursor Cells (MPC)-300-IV was administered to 48 RA patients resistant to anti-Tumor Necrosis Factor (TNF) agents. A single IV dose of 2 million MPC/kg of MPC-300-IV was well tolerated and demonstrated a durable improvement in clinical symptoms, physical function, and disease activity relative to placebo over the follow-up period.
Approximately a third of RA patients develop resistance to TNF agents; others patients do not respond to the drugs or experience adverse events when using them. Second-line drugs such as Rituxan are not as effective as the anti-TNF agents and are associated with serious side effects.
The phase 2 study patients had active RA, were on a stable regimen of methotrexate, and had an inadequate prior clinical response to at least 1 anti-TNF agent. Of the 48 patients, 30 (68%) had previously received 1 to 2 biologic agents. Patients were randomized to a single IV infusion of 1 million MPCs/kg, 2 million MPCs/kg, or placebo. Patients were evaluated using the American College of Rheumatology (ACR) 20/50/70 measurement to assess clinical response and improvement in signs and symptoms of RA in terms of 20%, 50%, or 70% improvement from baseline. The study also used the ACR-N scale to assess mean or median magnitude of benefit, assessed health/disability with the HAQ-DI measure for functional status, and applied the DAS28 scale to measure RA disease activity in order to judge improvement.
The safety profile over 39 weeks was comparable among placebo and both MPC treatment groups, with no cell-related serious adverse events. Both MPC doses tested outperformed placebo at week 39 in each of the ACR20/50/70 responses. The 2 million MPCs/kg dose was found to be the most effective over 39 weeks; that dose also achieved the maximal ACR-N score earlier (at 12 weeks) and exhibited a more robust durable effect.
Mesoblast CEO Silviu Itescu said the company’s MPC therapies have demonstrated virtually no toxicity, and had generated no negative immune response. The cells also appear to be targeted, intrinsically moving toward sites of inflammation and embedding in tissue, he said. Receptors on the cells’ surface are activated by every major cytokine that is important in progressive RA, including TNF, interleukin (IL)-1, IL-6, and IL-17. Itescu believes MPC-300-IV has an edge on the biologics inhibiting TNF-alpha or other key targets because it is getting to the heart of the inflammation and disease, not just knocking back the immune system.
Although larger phase 3 studies are needed to validate these results, the data are intriguing in that they suggest what optimized and targeted regenerative medicines could potentially accomplish to address the unmet medical need among anti-TNF—resistant RA patients.
Physician and Patient Perspectives After Starting or Switching to Amgevita in IBD
March 23rd 2024A real-world study surveying physicians and patients on adalimumab biosimilar ABP 501 (Amgevita) in inflammatory bowel disease (IBD) found both patients initiating ABP 501 and those who had switched from the reference product had higher satisfaction levels.
What AmerisourceBergen's Report Reveals About Payers, Biosimilar Pricing Trends
May 28th 2023On this episode of Not So Different, Tasmina Hydery and Brian Biehn from AmerisourceBergen discussed results from a recent survey, that were also presented at Asembia 2023, diving into the payer perspective on biosimilars and current pricing trends across the US biosimilar industry.
The Role of Biosimilars: Advancing Access, Financial Health, and System Sustainability
March 11th 2024Kashyap Patel, MD, CEO of Carolina Blood and Cancer Care, a member of the Community Oncology Alliance, and member of The Center for Biosimilars® Advisory Board, glances back at the development of the biosimilar industry and the last 5 years of progress.
Pipelines and Preparation: How the US Can Prepare for More RA Biosimilars
April 16th 2023What can practices do to prepare for all the biosimilars to treat rheumatoid arthritis (RA) coming down the pipeline? And how can they ensure that the lower-than-anticipated adoption rates for infliximab biosimilars are not repeated? Robert Zutaut, RPh, from McKesson Provider Solutions, tackles all this and more on this episode of Not So Different.
Cardinal Health Report Showcases Biosimilar Growth, Provider and Payer Evolution, and More
February 29th 2024In its annual biosimilars report, Cardinal Health provided updates on how provider acceptance growth, evolving payer dynamics, and the growing pipeline for biosimilars will shape the biosimilar landscape over the next 5 years.
Biosimilar Substitution Within OCM Could Result in Lower Total Cost of Care
February 16th 2024Researchers found that the total cost of care per oncology episode was significantly lowered when biosimilar substitution was implemented in Medicare’s Oncology Care Model (OCM), suggesting that biosimilar uptake can serve as a critical tool to mitigate risk and improve financial performance for providers.