This Market Spotlight report covers the myelodysplastic syndrome market, comprising key marketed and pipeline drugs, recent events and analyst opinion, clinical trials, probability of success, upcoming events, patent information, 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 2017, there were 367,200 incident cases of myelodysplastic syndrome (MDS) worldwide, and forecasts that number to increase to 401,700 incident cases by 2026.
• Approved drugs in the MDS space target DNA methyltransferase, BCR-ABL fusion protein, KIT/c-KIT, platelet-derived growth factor receptor, erythropoietin receptor, angiogenesis, E3 ubiquitin ligase, and the immune system. 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, with only one drug in the NDA/BLA stage.
• 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 II and Phase III trial results, Phase II/III and Phase III updated trial results, an expected CHMP opinion, and an estimated PDUFA date.
• The overall likelihood of approval of a Phase I hematologic asset is 9.6%, and the average probability a drug advances from Phase III is 58.3%. Drugs, on average, take 8.8 years from Phase I to approval, compared to 9.3 years in the overall oncology space.
• There have been 20 licensing and asset acquisition deals involving MDS drugs during 2014–19. The $1.8bn exclusive global collaboration and license agreement made in December 2018 between Argenx and Cilag to evaluate cusatuzumab (ARGX-110), an anti-CD70 SIMPLE Antibody, in acute myeloid leukemia (AML), high-risk MDS, and other hematological malignancies was the largest deal.
• 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 EU markets, while Japan has the top spot in Asia.
• Clinical trial activity in the MDS space is dominated by completed trials. Celgene has the highest number of completed clinical trials for MDS, with 93 trials.
• Celgene leads the industry sponsors with by far the highest overall number of clinical trials for MDS