Myelodysplastic Syndromes Drug Pipeline: 141 Assets by Phase

How many myelodysplastic syndromes drugs are in each phase of development?

Douglas Insights counts 11 myelodysplastic syndromes candidates in Phase III, 7.8% of the 141 assets in development; Phase II holds 31 and Phase I holds 99.

Phase I/II candidates sit in the Phase I row and Phase II/III candidates in the Phase II row, so each myelodysplastic syndromes asset appears in exactly one row. The 371 trials still recruiting or running carry 44% of the 839 registered trials in scope.

Which approved myelodysplastic syndromes medicines are still in trials?

1 medicines already on the market are still being tested in myelodysplastic syndromes Phase IV trials; Luspatercept has the most, with 24 registered trials.

Post-approval myelodysplastic syndromes trials test new doses, new patient groups and head-to-head comparisons. They set the standard of care that every candidate in development will be measured against, so the paid review profiles each one.

How many myelodysplastic syndromes trials are recruiting patients now?

202 myelodysplastic syndromes trials are recruiting patients and 42 more have not started recruiting yet, out of 839 interventional trials in scope; together they make up 29% of the trial base.

Terminated, suspended and withdrawn myelodysplastic syndromes trials stay in the trial base so that a stalled programme is visible, but a candidate whose only trials stopped is no longer counted as in development.

Which companies lead the myelodysplastic syndromes pipeline?

Janssen Research & Development, LLC leads the myelodysplastic syndromes pipeline with 9 candidates; the top 5 sponsors hold 26% of the 141 assets, and 89 sponsors run at least one.

Industry sponsors lead 292 of the 839 myelodysplastic syndromes trials (35%); universities, hospitals and public funders lead the rest. A sponsor is credited with an asset when it is the lead sponsor on most of that asset's industry trials.

Which drug types and mechanisms dominate the myelodysplastic syndromes pipeline?

Mechanism and drug-type classification for the 141 myelodysplastic syndromes candidates is done by the research desk asset by asset, each with a cited source, and is published in the paid review together with the white-space map.

What myelodysplastic syndromes trial readouts are coming next?

13 myelodysplastic syndromes candidates in Phase II or later have a registered primary completion date in the next 12 months; the next three are below, each linked to its registry record.

Primary completion is the date a sponsor expects the last patient to finish the main measurement, so data usually follow within months. Dates marked estimated are the sponsor's own forecast on the registry and often move.

How many myelodysplastic syndromes readouts fall in each quarter?

23 myelodysplastic syndromes candidates in Phase II or later reach registered primary completion over the next eight quarters, with the most, 7, due in Q4 2027.

Each candidate is counted once, at its next readout. The paid catalyst calendar names every readout with its trial, sponsor and design, adds filings and regulatory decisions, and runs 24 months ahead.

Which countries host the most myelodysplastic syndromes trials?

United States hosts the most myelodysplastic syndromes trials, with sites in 581 of the 839 studies (69%); trials in scope run in 60 countries in all.

A multinational myelodysplastic syndromes trial is counted once in every country where it has a site, so the shares add up to more than 100%. Country spread shows where sponsors are building the evidence that regulators in each market will ask for.

Where are myelodysplastic syndromes drugs being tested?

581 of the 839 myelodysplastic syndromes trials (69%) have a site in the United States, 197 (23%) in Europe and 95 (11%) in China; 101 of the 141 candidates in development are tested in the US.

Each region's trials are the evidence its regulator will review: the FDA in the United States, the EMA (with the MHRA and Swissmedic) in Europe, the NMPA in China and the PMDA in Japan. A multinational myelodysplastic syndromes trial counts once in every region where it has a site, so the shares add up to more than 100%. A candidate tested only outside the US and Europe is often a licensing opportunity for sponsors in those markets.

What has changed in the myelodysplastic syndromes pipeline since the last update?

This is the first edition of the myelodysplastic syndromes pipeline count, dated 4 Oct 2026. The next refresh is due by 2 Jan 2027, and each refresh lists the assets that entered, advanced or dropped out.

How does Douglas Insights count the myelodysplastic syndromes pipeline?

Douglas Insights read 839 interventional trials registered on ClinicalTrials.gov for the conditions "myelodysplastic syndromes" (excluding "paroxysmal nocturnal hemoglobinuria", "aplastic anemia", "graft vs host disease") and updated since 3 Oct 2021, then grouped every drug or biologic named in them into 141 unique candidates.

1. Code names, generic names and brand names of the same molecule are merged into one candidate, and every merge is reviewed by the research desk.
2. A candidate counts when it has a trial that is recruiting, running, or completed in the last 24 months, and at least one industry-sponsored trial.
3. Its phase is the highest phase among those trials; placebo, standard-of-care and comparator arms are not candidates.
4. Each figure on this page is drawn from one frozen dataset that passes ten checks before release, and the owner approves every edition.

Read the full approach on the research methodology page, or browse other Pharmaceuticals research.

Frequently asked questions about the myelodysplastic syndromes pipeline

Research & citation

This review was researched, written and reviewed by the Douglas Insights Research Desk under the Douglas Insights editorial standards. Material errors are logged in the corrections log. No section is sponsored.

Adjacent coverage