Market Spotlight: Myelodysplastic Syndrome (MDS)
Market Spotlight: Myelodysplastic Syndrome (MDS)
This Market Spotlight report covers the myelodysplastic syndrome market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
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.
- 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, an expected CHMP opinion, and an estimated PDUFA date.
- The overall likelihood of approval of a Phase I hematologic asset is 10.1%, and the average probability a drug advances from Phase III is 57.7%. 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
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
10 Supportive therapy
10 Growth factors
11 Immune treatments
11 Stem cell transplant
14 MARKETED DRUGS
16 PIPELINE DRUGS
30 RECENT EVENTS AND ANALYST OPINION
30 5F9 for MDS (July 11, 2019)
31 ASTX727 for MDS (June 6, 2019)
32 5F9 for MDS (May 15, 2019)
33 Estybon (Intravenous) for MDS (May 13, 2019)
34 Estybon (Oral) for MDS (May 13, 2019)
35 Revilimid for MDS (February 27, 2019)
36 Luspatercept for MDS (December 2, 2018)
38 KEY UPCOMING EVENTS
39 KEY REGULATORY EVENTS
39 MAA Positive Opinions
40 PROBABILITY OF SUCCESS
41 LICENSING AND ASSET ACQUISITION DEALS
41 HanX To Develop, Sell Onconova’s Rigosertib In China
41 Impressive Clinical Results For Argenx’s Oncology Candidate Seal The Deal for Janssen
43 PARENT PATENTS
45 REVENUE OPPORTUNITY
46 CLINICAL TRIAL LANDSCAPE
47 Sponsors by status
48 Sponsors by phase
49 Recent events
50 Prescription information
LIST OF FIGURES
13 Figure 1: Trends in incident cases of MDS, 2017–26
16 Figure 2: Overview of pipeline drugs for MDS in the US
16 Figure 3: Pipeline drugs for MDS, by company
17 Figure 4: Pipeline drugs for MDS, by drug type
17 Figure 5: Pipeline drugs for MDS, by classification
32 Figure 6: ASTX727 for MDS (June 6, 2019): Phase III – ASCERTAIN (vs. Decitabine)
37 Figure 7: Luspatercept for MDS (December 2, 2018): Phase III – MEDALIST
38 Figure 8: Key upcoming events in MDS
40 Figure 9: Probability of success in the MDS pipeline
41 Figure 10: Licensing and asset acquisition deals in MDS, 2014–19
43 Figure 11: Parent patents in MDS, 2019–28
44 Figure 12: Parent patents in MDS, 2029–34
46 Figure 13: Clinical trials in MDS
46 Figure 14: Top 10 drugs for clinical trials in MDS
47 Figure 15: Top 10 companies for clinical trials in MDS
47 Figure 16: Trial locations in MDS
48 Figure 17: MDS trials status
49 Figure 18: MDS trials sponsors, by phase
LIST OF TABLES
12 Table 1: Incident cases of MDS, 2017–26
15 Table 2: Marketed drugs for MDS
18 Table 3: Pipeline drugs for MDS in the US
30 Table 4: 5F9 for MDS (July 11, 2019)
31 Table 5: ASTX727 for MDS (June 6, 2019)
32 Table 6: 5F9 for MDS (May 15, 2019)
33 Table 7: Estybon (Intravenous) for MDS (May 13, 2019)
34 Table 8: Estybon (Oral) for MDS (May 13, 2019)
35 Table 9: Revilimid for MDS (February 27, 2019)
36 Table 10: Luspatercept for MDS (December 2, 2018)
45 Table 11: Historical global sales, by drug ($m), 2014–18
45 Table 12: Forecasted global sales, by drug ($m), 2019–23
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