Pelabresib Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Pelabresib comprises 5 peer-reviewed publications across 5 journals, 1 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Primary Myelofibrosis. Most recent publication: Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial., Nat Med, 2025.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial.
    Rampal RK, Grosicki S, Chraniuk D, et al. · Nat Med · 2025
  2. Phase III MANIFEST-2: pelabresib + ruxolitinib vs placebo + ruxolitinib in JAK inhibitor treatment-naive myelofibrosis.
    Harrison CN, Gupta VK, Gerds AT, et al. · Future Oncol · 2022
  3. Results from phase 1 of the MANIFEST clinical trial to evaluate the safety and tolerability of pelabresib in patients with myeloid malignancies.
    Stein EM, Fathi AT, Harb WA, et al. · Leuk Lymphoma · 2024
    PubMed: PMID 38259250 · NCT02158858 · Primary Myelofibrosis
  4. MANIFEST: Pelabresib in Combination With Ruxolitinib for Janus Kinase Inhibitor Treatment-Naïve Myelofibrosis.
    Mascarenhas J, Kremyanskaya M, Patriarca A, et al. · J Clin Oncol · 2023
    PubMed: PMID 36881782 · NCT02158858 · Primary Myelofibrosis
  5. Matching-adjusted indirect comparison of the pelabresib-ruxolitinib combination vs JAKi monotherapy in myelofibrosis.
    Gupta V, Mascarenhas J, Kremyanskaya M, et al. · Blood Adv · 2023
    PubMed: PMID 37530627 · NCT02158858 · Primary Myelofibrosis

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT04603495
MANIFEST-2
Primary MyelofibrosisNumber of Participants With Splenic Response by Central Radiology Reads at Week 24
Week 24
Pelabresib + Ruxolitinib (Experimental Arm)141 Participants
Placebo + Ruxolitinib (Control Arm)76 Participants

Publications by year

20222025: 5 publications.

2022
1
2023
2
2024
1
2025
1

Publications by indication

Primary Myelofibrosis (5)

  • Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial.
    Nat Med · 2025 · PMID 40065169 · NCT04603495
  • Results from phase 1 of the MANIFEST clinical trial to evaluate the safety and tolerability of pelabresib in patients with myeloid malignancies.
    Leuk Lymphoma · 2024 · PMID 38259250 · NCT02158858
  • MANIFEST: Pelabresib in Combination With Ruxolitinib for Janus Kinase Inhibitor Treatment-Naïve Myelofibrosis.
    J Clin Oncol · 2023 · PMID 36881782 · NCT02158858
  • Matching-adjusted indirect comparison of the pelabresib-ruxolitinib combination vs JAKi monotherapy in myelofibrosis.
    Blood Adv · 2023 · PMID 37530627 · NCT02158858
  • Phase III MANIFEST-2: pelabresib + ruxolitinib vs placebo + ruxolitinib in JAK inhibitor treatment-naive myelofibrosis.
    Future Oncol · 2022 · PMID 35950489 · NCT04603495

Publications by journal

Trial-results highlights

Pelabresib has been studied in clinical trials for primary myelofibrosis. One pivotal study, NCT04603495 (MANIFEST-2), investigated Pelabresib in combination with ruxolitinib for this condition. The trial assessed the number of participants who achieved a splenic response, as determined by central radiology reads, at Week 24.

In the NCT04603495 trial, the experimental arm, which received Pelabresib + Ruxolitinib, included 141 participants who achieved a splenic response by central radiology reads at Week 24. In the control arm of the same study, which received Placebo + Ruxolitinib, 76 participants achieved a splenic response by central radiology reads at Week 24.

All values presented here are sourced from primary registry reporting; individual scientific papers should be consulted for clinical decisions.

All Pelabresib publications (5)

2025 (1 paper)

  1. Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial.
    Rampal RK, Grosicki S, Chraniuk D, et al. · Nat Med · 2025 · Trial result

2024 (1 paper)

  1. Results from phase 1 of the MANIFEST clinical trial to evaluate the safety and tolerability of pelabresib in patients with myeloid malignancies.
    Stein EM, Fathi AT, Harb WA, et al. · Leuk Lymphoma · 2024 · Derived
    PubMed: PMID 38259250 · NCT02158858 · Primary Myelofibrosis

2023 (2 papers)

  1. MANIFEST: Pelabresib in Combination With Ruxolitinib for Janus Kinase Inhibitor Treatment-Naïve Myelofibrosis.
    Mascarenhas J, Kremyanskaya M, Patriarca A, et al. · J Clin Oncol · 2023 · Derived
    PubMed: PMID 36881782 · NCT02158858 · Primary Myelofibrosis
  2. Matching-adjusted indirect comparison of the pelabresib-ruxolitinib combination vs JAKi monotherapy in myelofibrosis.
    Gupta V, Mascarenhas J, Kremyanskaya M, et al. · Blood Adv · 2023 · Derived
    PubMed: PMID 37530627 · NCT02158858 · Primary Myelofibrosis

2022 (1 paper)

  1. Phase III MANIFEST-2: pelabresib + ruxolitinib vs placebo + ruxolitinib in JAK inhibitor treatment-naive myelofibrosis.
    Harrison CN, Gupta VK, Gerds AT, et al. · Future Oncol · 2022 · Trial result

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).