This Market Spotlight report covers the Myelodysplastic Syndrome (MDS) market, comprising key marketed and pipeline drugs, recent events and analyst opinion, clinical trials, probability of success, upcoming and regulatory events, a 10-year disease incidence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• Datamonitor Healthcare estimates that in 2021, there were 253,000 incident cases of myelodysplastic syndrome (MDS) worldwide, and forecasts that number to increase to 313,500 incident cases by 2028.
• Approved drugs in the MDS space target DNA synthesis, erythropoietin receptor, BCR-ABL fusion protein, KIT/c-KIT, plateletderived growth factor receptor, PPAR gamma, cytidine deaminase, DNA methyltransferase, and fibroblast growth factor receptor. These therapies are administered via the intravenous, oral, and subcutaneous routes.
• The greatest proportion of industry-sponsored drugs in active clinical development for MDS are in Phase II. Therapies in development for MDS focus on a wide variety of targets. The majority of pipeline drugs in the MDS space are administered via the intravenous or oral routes, with the remainder being subcutaneous or intradermal formulations.
• High-impact upcoming events for drugs in the MDS space comprise topline Phase III trial results for magrolimab and SY-1425, updated Phase II/III trial results for imetelstat, topline Phase II trial results for sabatolimab, and topline Phase I/II trial results for eltanexor.
• The overall likelihood of approval of a Phase I hematologic asset is 7.6%, and the average probability a drug advances from Phase III is 55.4%. Drugs, on average, take 9.7 years from Phase I to approval, compared to 9.9 years in the overall oncology space.
• The distribution of clinical trials across Phase I–IV indicates that the vast majority of trials for MDS have been in the early and mid-phases of development, with 89% of trials in Phase I–II, and only 11% in Phase III–IV.
• The US has a substantial lead in the number of MDS clinical trials globally. France leads the major European markets, while Japan has the top spot in Asia.
• Clinical trial activity in the MDS space is dominated by completed trials. Bristol Myers Squibb has the highest number of completed clinical trials for MDS, with 110 trials.
• Bristol Myers Squibb leads the industry sponsors with by far the highest overall number of clinical trials for MDS