Blood will tell: Hematology in review

Combing through my inbox, it seems news of therapeutics for (and research into) blood-related diseases, from hemophilia to leukemia, has picked up a little we thought we'd share some of that news with you

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I had planned, with last issue being the October one and all, to write this month’s editorial, as the “spooky” theme of Halloween held an amusing resonance with me on the topic I’m about to address, but there were other things to cover and only so much room to work with. And that topic: blood.
Well, most of you readers being scientific types or in closely allied professions, I suppose I should be more technical: hematology.
But while the October issue may have passed, it’s almost Halloween as I write this, so I’m not going to let the opportunity pass again. Besides, I like to do quick roundups periodically in this column, and issues of blood have been popping up with more than the usual frequency in my email inbox. Much of that, I’m sure, has to do with the upcoming annual meeting of the American Society of Hematology coming up in December.
And perhaps it also has a little to do with real life and market forecasts aligning, as GBI Research predicts that the hematological disorders therapy area, which covers indications such as sickle cell disease, paroxysmal nocturnal hemoglobinuria, anemia, thalassemia and hereditary hemochromatosis, is experiencing a significant level of first-in-class innovation, with 92 of its 327 pipeline programs with a disclosed molecular target identified as first in class. The business intelligence company’s latest report states that the hematological disorders market is relatively small, but with substantial unmet need, thus representing a significant investment opportunity.
And, as research and consulting firm GlobalData noted this summer, the hemophilia A and B market, which is set to approach $8 billion by 2026, will see a substantial number of newcomers capture market share from established companies.
“Before the approval of long-acting factor concentrates, the movement of patients from established treatment options to new short-acting recombinant factors had been minimal due to the lack of any clinical incentive,” wrote Dr. Chiara Marchetti, a healthcare analyst for GlobalData. “However, GlobalData expects that the improvements in convenience offered by long-acting factors and alternative coagulation promoters will help new entrants, such as Bioverativ and Roche, to establish themselves as key players in the hemophilia market. This process had already begun for hemophilia B, where the improved prophylactic dosing schedule of long-acting clotting factor IX has undermined Pfizer’s position in the U.S.”
Baxalta was the dominant player in the recombinant hemophilia market with its recombinant clotting factor VIII products Recombinante and Advate, but was acquired by Shire in 2016, noted GlobalData—and sales of Shire’s products in 2016 dwarfed those of its competitors in the hemophilia market, including Novo Nordisk, Pfizer, Bioverativ/Sobi and Bayer.
Marchetti concluded: “GlobalData forecasts that Roche will overtake Shire and become the hemophilia market leader in 2026, with global sales of around $2.5 billion, followed by a recent entrant, Bioverativ, which will have global sales of $1.4 billion in 2025. Overall, it is expected that the ability of companies to endure competition from emicizumab will be linked to their dependence on short-acting clotting factor VIII in their portfolios.”
However, that said, you also cannot count out established players. Shire announced recently a collaboration with MicroHealth to support a free and secure care monitoring tool for hemophilia A and B patients with inhibitors and their care teams. While prophylactic treatment is now recommended for managing many patients with hemophilia, the difficulties associated with maintaining this type of treatment regimen do make adherence challenging, leaving patients at risk for bleeds and subsequent joint damage.
“At Shire, we want to do everything we can to help people manage their hemophilia and to limit the burden of the disease on their everyday lives,” said Patty Torr, head of hematology in the United States for Shire. “Enhancing the existing MicroHealth app with new functionality and resources to address the needs of hemophilia patients with inhibitors is an important innovation for this group of patients.”
Now, how about one of those “new entrants” GlobalData mentioned—Bioverativ Inc. announced in the fall a research collaboration with Bicycle Therapeutics Ltd., a biotech with a proprietary bicyclic peptide (Bicycle) product platform, to discover, develop and commercialize innovative therapies for hemophilia and sickle cell disease. Bicycles are a new therapeutic modality that combine attributes of antibodies, small molecules and peptides within one molecule, reportedly enabling high selectivity and affinity while simultaneously being able to penetrate and bind to the targets of interest within the body.
“We are constantly exploring new ways to do innovative science to find new molecules that can advance the care of people living with rare blood disorders,” said Dr. Tim Harris, executive vice president of research and development at Bioverativ. “This collaboration offers a unique opportunity to identify an entirely new therapeutic modality that may lead to meaningful new treatments and outcomes for people living with hemophilia and sickle cell disease.”
Looking a bit farther down the R&D pipeline, Sangamo Therapeutics Inc. and Pfizer Inc. recently began a Phase 1/2 clinical trial, dubbed the Alta trial, which is designed to evaluate SB-525, an investigational gene therapy for patients with hemophilia A.
SB-525 consists of a recombinant adeno-associated virus vector carrying a factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter—a potential therapeutic for which the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Fast Track designations and for which the European Medicines Agency granted the Orphan Medicinal Product designation.
And finally, speaking of regulatory agencies and blood-focused therapeutics, the FDA recently gave Orphan Drug designation to Catalyst Biosciences Inc.’s CB 2679d/ISU304, a subcutaneous next-generation recombinant human factor IX variant for the treatment of hemophilia B; to Shire’s gene therapy candidate SHP654 (also designated as BAX 888), an investigational factor VIII gene therapy for the treatment of hemophilia A; and to H3 Biomedicine’s orally administered lead candidate, H3B-8800, for patients with acute myeloid leukemia and chronic myelomonocytic leukemia. FDA also granted Breakthrough Therapy designation to BioMarin Pharmaceutical’s valoctocogene roxaparvovec, or BMN 270, for treatment of patients with hemophilia A.
Hematology may not get as much play in these pages as many other therapeutic areas, but it does show up regularly and, as some of this news suggests, that may only increase going forward.

And for an outside opinion on a wholly different matter, since we lacked an official guest commentary this month...
Biosimilars may reduce U.S. health spending
Introducing biosimilar versions of complex biologic drugs used to treat illnesses such as cancer and rheumatoid arthritis could cut healthcare spending in the United States by $54 billion over the next decade, according to new analysis from the nonprofit research organization RAND Corporation. The savings estimate is about 20 percent larger than a similar analysis done by RAND researchers three years ago, representing both improved analysis methods and rapid growth in spending for biologics overall.
While expected to produce less-dramatic savings than an earlier generation of less-complex generic drugs, the introduction of biosimilars into the U.S. marketplace is expected to increase competition and drive down prices.
 “Biologics account for the fastest-growing segment of prescription drug spending, but biosimilars have the potential to help slow some of the increase,” said Andrew Mulcahy, lead author of the study and a policy researcher at RAND. “However, there remain many important industry, regulatory and policy decisions to be made that will influence whether such savings are realized.”
RAND researchers developed their estimate of savings from biosimilars by examining other studies that have examined the issue, reviewing the sales history of more than 100 biologic drugs and examining the brief experience of the one biosimilar drug that has been marketed in the United States. They estimate that biosimilars will cut spending on biologics by about 3 percent over the next decade. The range of the new savings estimate given reasonable ranges of key assumptions, like the price of biosimilars vs. reference biologics and biosimilar market share—varied from $24 billion to $150 billion from 2018 through 2027.

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