Key Takeaways
- The publisher estimates that in 2019, there were 238,000 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, angiogenesis, E3 ubiquitin ligase, the immune system, transforming growth factor-beta receptor, erythropoietin receptor, BCR-ABL fusion protein, KIT/c-KIT, platelet-derived growth factor receptor, cytidine deaminase, and DNA methyltransferase. 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 pevonedistat, magrolimab, and Reblozyl; topline Phase II trial results for eprenetapopt, KER-050, and MBG453; and updated Phase II/III trial results for imetelstat.
- The overall likelihood of approval of a Phase I hematologic asset is 8.7%, and the average probability a drug advances from Phase III is 58.9%. Drugs, on average, take 9.4 years from Phase I to approval, compared to 9.6 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 106 trials.
- Bristol Myers Squibb leads the industry sponsors with by far the highest overall number of clinical trials for MDS.