Dividing mesenchymal stem cells
It’s the kind of the data the field has worked towards for years.
Rheumatoid arthritis (RA) patients given a single intravenous dose of Mesoblast’s novel stem cell therapy were still demonstrating therapeutic benefits nine months later, according to new Phase 2 data from the Melbourne, Australia-based company.
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While much larger Phase 3 studies are needed to validate the results, the data offer a tantalizing look at what optimized and targeted regenerative medicines could do as the field moves closer to an approval.
The study involved 48 patients that were resistant to frontline TNF-alpha therapies, such as Enbrel, Remicade, and Humira.
While these drugs have revolutionized the field — and generated billions in revenue —around 20-40 percent of patients treated with a TNF inhibitor don’t achieve a significant reduction in symptoms. They’re non-responders. Others become resistant over time or experience adverse events.
When TNF inhibitors are off the table, patients are typically prescribed second-line drugs such as Rituxan. These, however, are not as effective and come with a range of serious side effects.
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CEO Silviu Itescu said Mesoblast’s mesenchymal precursor cells (MPCs) have demonstrated virtually no toxicity. The immune system doesn’t register them as foreign so there’s no negative immune response. The cells also appear targeted, intrinsically moving towards sites of inflammation and embedding themselves in the tissue.
“The way the cells work is, they have receptors on their surface that are activated by every major cytokine that is important in progressive rheumatoid arthritis, including TNF, IL-1, IL-6, IL-17,” Itescu explained. “Those cytokines drive the disease and also bind to receptors on our cells. And when they bind to our cells they activate the cells to release other factors that switch off the very cells that made those cytokines.”
In other words, MPCs interfere with the feed forward production of inflammatory molecules. Because the cells are addressing multiple pathways, he believes the therapy has an edge on the biologics inhibiting TNF-alpha or others key targets. It is also getting to the heart of the inflammation and disease, not simply knocking the immune system back.
Listed on both the NASDAQ and the Australian Stock Exchange (ASX), Mesoblast is applying its platform to a wide range of diseases. For each indication, the cells are delineated and optimized. Mesoblasts portfolio, Itescu said, is the most advanced in the stem cell field.
At the front of the pack is MSC-100-IV, a Phase 3 therapy for pediatric graft-versus-host disease (GvHD). MSC-100-IV secured orphan drug designation in the U.S., paving the way for an accelerated approval. The company is expecting a data readout in the third-quarter of this year.
Two other product candidates, MPC-150-IM and MPC-06-ID, are in late-stage development for advanced chronic heart failure and chronic low back pain due to degenerative disc disease.
In late December, U.S.-based Mallinckrodt Pharmaceuticals took an option on the GvHD and lower back pain programs.
When it comes to manufacturing, Itescu said the cells are designed to scale. The foundation for the supply chain is the allogeneic nature of the cells — they can be administered to any patient.
“It’s not a problem for us, it’s an advantage,” Itsecu said about the production logistics. “We’ve been at this for ten years and we’ve focused the entire thing on a scalable manufacturing platform using a unique subtype that can be used off-the-shelf and that can be industrially manufactured.”
There are plenty of potential customers if the therapy is approved.
Rheumatoid arthritis is particularly lucrative. As Global Business Intelligence notes, in 2013, three TNF-a-targeting biologics – Humira, Remicade, and Enbrel – were ranked among the top-10 best-selling drugs in the world, with global revenues of $11.1 billion, $9.9 billion, and $8.9 billion respectively.
Photo: Mesoblast
