For patients with certain blood cancers, a stem cell transplant can often be a curative treatment. However, it is also a complex process riddled with severe toxicities and high rates of relapse or failure. These problems explain at least part of why about 60% of cancer patients, a third of patients with genetic diseases, and 5% of autoimmune patients eligible for a stem cell transplant receive one. It’s not hard to imagine that if a safer and more efficient process exists, the market could potentially expand. Enter Therapeutic magenta (NASDAQ: MGTA).
The lead compound of clinical-stage biotechnology, MGTA-145, has the potential to revolutionize the current standard of care transplant process for patients and donors, making it faster and safer for all parties.
Focus on donors
When you donate stem cells, it takes an average of five or more days to collect them. This is an inefficient process for a donor because it requires daily injections of a drug called G-CSF, which stimulates the release of stem cells from the bone marrow into the bloodstream for collection. These daily injections cause significant bone pain and a handful of other side effects. To make matters worse, around 15% of donors will need multiple collection attempts, which means more daily injections of G-CSF. It is perhaps not surprising that about 50% of registered donors refuse to donate when called upon to do so.
Patients receiving a stem cell transplant, on the other hand, must first undergo treatment with G-CSF to increase the release of their stem cells from the bone marrow. so undergo chemotherapy and radiation therapy to try to get rid of any bad or cancerous cells from the bone marrow before they receive their transplant. It’s a lot of steps, a lot of time, and a lot of potential side effects.
Magenta is working to streamline this process and MGTA-145 has the potential to become the first drug approved for this stem cell mobilization in both donors and transplant recipients. (Although G-CSF is commonly used by off-label clinicians for donor stem cell mobilization, the drug is actually not explicitly approved for this use.) Phase 1 studies have shown a three-fold increase the number of stem cells collected from healthy donors. when treated with MGTA-145 versus G-CSF, and surprisingly 88% of donors infused with MGTA-145 were able to have their stem cells collected on the same day, compared to zero for the G-CSF group. Finally, there was only one 1% rate of grade 2-4 side effects with MGTA-145; this rate is 38% for the G-CSF.
Data from two phase 2 trials are expected in the second half of 2021. A trial examines the effect of MGTA-145 on stem cell mobilization and collection, as well as the still significant progression-free survival rate, in cases of multiple myeloma. The other examines the effect of MGTA-145 on the mobilization, collection, and disease outcome of stem cells for acute myeloid leukemia (AML), acute lymphoid leukemia (ALL), and myelodysplastic syndrome (MDS).
How green can magenta turn?
So far, the MGTA-145 appears to be faster, safer, and requires fewer donor collections than the current standard of care, with no competition on the horizon. So what does the addressable market look like?
A place to start measuring is with Sanofi‘s (NASDAQ: SNY) plerixafor, which is currently given to about half of the patients undergoing stem cell therapy for certain blood cancers. This drug had sales of $ 200 million in 2019.
However, since MGTA-145 makes donation less complicated, it is no exaggeration to imagine that the drug could work as a catalyst to increase the number of stem cell transplants performed each year. Management estimates that it can grow the market from around 90,000 worldwide today to over 175,000. Magenta has also started to explore the utility of MGTA-145 in the gene therapy market, having already established a partnership with Therapeutic bundle (NASDAQ: BEAM), blue bird bio (NASDAQ: BLUE), and AVROBIO (NASDAQ: AVRO) mobilize stem cells more effectively for their gene therapy. Phase 2 trials in partnership are already underway.
If future trials of the MGTA-145 continue to be positive, which I think is likely, $ 200 million would likely be just the start of the addressable market. This could easily be more than double if you take into account the potential increase in donors and procedures, as well as the gene therapy market. With a current market cap of around $ 500 million and other promising candidates in the pipeline, Magenta is well positioned to add some green to health investor portfolios over the next few years – not to mention making a difference for tens of thousands of patients and donors.
This article represents the opinion of the author, who may disagree with the “official” recommendation position of a premium Motley Fool consulting service. We are motley! Challenging an investment thesis – even one of our own – helps us all to think critically about investing and make decisions that help us become smarter, happier, and richer.