pelabresib (DAK539)
/ Novartis
- LARVOL DELTA
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December 05, 2025
Efficacy and safety of ruxolitinib-based combination therapy in the patients with myelofibrosis (MF): A systematic review and meta-analysis
(ASH 2025)
- "Among JAK inhibitor-naïve patients, the combination of ruxolitinib with selinexor demonstrated the highest efficacy (SVR35: 92%; TSS50: 78%), followed by ruxolitinib plus BMS-986158 (SVR35: 90%). In addition, the efficacy ofthe combination of ruxolitinib with IFNα(SVR35:70%) and pelabresib (SVR35:66%; TSS50:53%) are also acceptable. For patients with prior JAK inhibitor exposure, ruxolitinib plus siremadlin (SVR35: 45%) and ruxolitinib plus selinexor (SVR35: 38%; TSS50: 33%) showed notable activity... For JAK inhibitor-naïve patients, ruxolitinib-based combination regimens demonstrated advantages over ruxolitinib monotherapy. For patients with prior JAK inhibitor exposure, the addition of combination therapy drugs may further enhance the efficacy. Personalized treatment selection remains essential, as therapeutic efficacy is significantly influenced by prior JAK inhibitor exposure."
Combination therapy • Retrospective data • Review • Chronic Eosinophilic Leukemia • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Neutropenia • Thrombocytopenia • IFNA1
November 04, 2025
Durable efficacy and long-term safety with pelabresib plus ruxolitinib in JAK Inhibitor–Naive myelofibrosis: 96-week Results from the Phase III MANIFEST-2 study
(ASH 2025)
- P3 | "Longer-term follow-up showed numerically fewer deathsand fewer progression events in the PELA+RUX arm vs the PBO+RUX arm. Overall findings suggest thatPELA+RUX provides clinically meaningful benefits vs RUX monotherapy, with potential for diseasemodification and improving survival in pts with MF."
Clinical • P3 data • Hematological Disorders • Myelofibrosis
November 04, 2025
The combination of ruxolitinib and the CDK4/6 inhibitor abemaciclib demonstrates safety and efficacy in previously treated myelofibrosis patients: Results of A phase I study
(ASH 2025)
- P1 | "Prior treatments included RUX plus pelabresib/placebo (n=3),hypomethylating agents (n=2), hydroxyurea (n=2), stem cell transplant (n=1). Combination therapy with RUX and the CDK 4/6 inhibitor ABE is safe and has encouragingobjective evidence of efficacy among pts with advanced, previously treated MF, confirming preclinicalhypotheses. These data nominate combined JAK and CDK4/6 inhibitor strategies for future investigationin JAK-STAT driven diseases. The combination of RUX and ABE will now advance to a phase II study inpreviously treated MF patients."
Clinical • P1 data • Breast Cancer • Fibrosis • Immunology • Myelofibrosis • Neutropenia • Solid Tumor • Thrombocytopenia • CALR • CDC25A • GNRP • JAK2
November 04, 2025
Identifying novel 'druggable' targets via Npm1A-turboid fusion and mass spectrometry to overcome genetic or adaptive resistance to menin inhibitors in mtNPM1 AML
(ASH 2025)
- "Treatment with revumenib may also cause emergenceof hot spot mutations in menin (e.g., S160T, M327V, M327I, G331D, G331R, and T349M) exhibitingreduced affinity to MI-binding...OCI-AML3 MEN1-M327I cells were resistant toSNDX-50469, ziftomenib, and DS1594b, but sensitive to the second-generation MI, bleximenib, aspreviously reported.To identify novel 'druggable' targets in mtNpm1 AML cells either sensitive or resistant to MI, we knockedin TurboID by CRISPR/Cas9 into the C-terminus of the mtNpm1 gene in OCI-AML3 cells...When combined with a BET inhibitor (pelabresib) ora novel dual BET/HAT inhibitor (NEO2734/EP31670), both RocA and talazoparib induced synergisticlethality in the sensitive OCI-AML3 and OCI-AML2-Npm1A KI, as well as the MI-resistant (OCI-AML3-Menin-M327I or the OCI-AML3 MITR) cells...Exvivo treatment with EP31670 and RocA or talazoparib induced synergistic loss of viability in MI (SNDX-50469)-resistant, patient-derived (N = 3) mtNpm1 AML cells. In the..."
IO biomarker • Acute Myelogenous Leukemia • AURKA • CDK9 • CDKN1A • EIF4A1 • EIF4A2 • FLT3 • HOXA9 • IL7R • IRAK4 • KMT2A • MEIS1 • MEN1 • MYC • NPM1 • PLK1 • S100A8 • SF3B1 • SMARCA2 • TP53
December 09, 2025
MANIFEST-2: 96-Week Data of Pelabresib Plus Ruxolitinib in JAK Inhibitor–Naive Myelofibrosis
(OncoDaily)
- "SVR35 at week 96: PELA+RUX: 91.5%; PBO+RUX: 57.6%. Kaplan–Meier curves for duration of spleen response showed an early separation between arms that remained throughout the 96-week follow-up. In the intent-to-treat population: Median duration of spleen response was not reached with PELA+RUX versus 138.3 weeks with PBO+RUX."
P3 data • Myelofibrosis
October 06, 2024
MODULE 4: Future Directions in the Management of MF
(ASH 2024)
- "This program is supported by educational grants from CTI BioPharma, a Sobi Company, Geron Corporation, GSK, Incyte Corporation and Karyopharm Therapeutics.Mechanism of antitumor activity of navitoclax and biological rationale for its evaluation for MF Available efficacy and safety findings from the Phase III TRANSFORM-1 study of navitoclax in combination with ruxolitinib versus ruxolitinib alone for patients with previously untreated MF Potential role of navitoclax in the up-front setting and ongoing evaluation for relapsed/refractory (R/R) disease in the Phase III TRANSFORM-2 study Rationale for the evaluation of BET inhibitors for MF; updated findings from the Phase III MANIFEST-2 study combining pelabresib to ruxolitinib for JAK inhibitor-naïve disease Scientific justification for the evaluation of selinexor for MF; early efficacy and safety findings with selinexor as monotherapy and in combination with ruxolitinib Ongoing evaluation of the combination of..."
Myelofibrosis • Oncology
December 03, 2023
Activity of Selinexor As a Single Agent and Synergistic Activity with Approved/Investigational Myelofibrosis Therapies in Vitro
(ASH 2023)
- "Despite the current standard of care, the JAK1/2 inhibitor (JAKi) ruxolitinib (RUX), a significant unmet need remains for patients (pts) with MF, due to lack of response or JAKi resistance... JAK2V617F mutant cell lines HEL ( TP53M113K), UKE-1 ( TP53WT), MUTZ-8 ( TP53WT), and JAK2WT ELF-153 ( TP53I251N) cells were treated with SEL alone or in combination with RUX, pacritinib (PAC), momelotinib (MMB), pelabresib (PELA), or navitoclax (NAV)... In a panel of MPN-derived cells with or without JAK2V617F, SEL showed single-agent antiproliferative activity, and synergism with other MF therapeutics at clinically achievable concentrations through regulation of XPO1, JAK/STAT signaling, and apoptosis/senescence regulators. In addition to positive Phase 1 clinical data in pts with JAKi-naïve MF who received SEL with RUX, these preclinical data support synergistic activity between SEL and other MF therapies, suggesting the potential for SEL as a backbone for novel treatment..."
IO biomarker • Preclinical • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Targeted Protein Degradation • CALR • CCND1 • CDKN1A • MCL1
November 06, 2024
ASXL1 Mutations in AML Are Associated with a Distinct Epigenetic State Which Highlights Vulnerabilities to Specific Epigenetic-Targeted Agents
(ASH 2024)
- "Notably, compared to parental cells, OCIAML3 ASXL1 Y591* cells exhibited reduced sensitivity to standard anti-AML chemotherapeutic agents, including cytarabine, etoposide and daunorubicin...Our findings also demonstrate and confirm that mtASXL1-expressing AML cells exhibited increased sensitivity to BETi (pelabresib or birabresib)...Importantly, in the NSG mice engrafted with luciferized OCIAML3 ASXL1 Y591*, monotherapy with NEO2734 (5 mg/kg QD), pelabresib (30 mg/kg QD) or SEL120-34A (40 mg/kg QD), compared to vehicle control, significantly reduced AML burden. Collectively these findings highlight previously uncharacterized biologic effects of the presence of mtASXL1 and support the rationale for further evaluating AML therapies incorporating BETi, HAT-BETi or inhibitor of mediator kinase."
Acute Myelogenous Leukemia • Hematological Malignancies • Immunology • Oncology • Targeted Protein Degradation • ASXL1 • AURKA • BAP1 • BRD4 • CD14 • CDK9 • E2F1 • FZD5 • HOXA9 • MEIS1 • MYC • NDUFA2 • PLK1 • SPI1 • TCF7L2
November 25, 2025
Novartis will present data from over 70 abstracts, including investigator-initiated trials at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition and 2025 San Antonio Breast Cancer Symposium (SABCS).
(Novartis Press Release)
- "Scemblix data across clinical and real-world settings offer new evidence informing CML care amid evolving patient needs; 96-week pelabresib Phase III data represent longest follow-up of first-line myelofibrosis patients in randomized combination trial; Kisqali NATALEE and MONALEESA data add to evidence of long-term benefits for early and metastatic breast cancer patients."
Clinical data • Chronic Myeloid Leukemia • HER2 Negative Breast Cancer • Hormone Receptor Positive Breast Cancer • Myelofibrosis
December 07, 2024
A Phase 1/2 Study of Nuvisertib (TP-3654), an Investigational Selective PIM1 Kinase Inhibitor, in Combination with JAK Inhibitors Ruxolitinib or Momelotinib in Patients with Myelofibrosis
(ASH 2024)
- P1/2 | "Recent Phase 3 combination studies of RUX with Pelabresib (Rampal R, 2023) or Navitoclax (Pemmaraju N, 2023) in treatment naïve MF pts with platelet (PLT) count ≥100 x 109/L showed significant increase only in spleen responses; however, did not improve symptoms response, also were limited by overlapping toxicities of thrombocytopenia. For TP-3654 in combination with MMB (Arm 3), pts that have been previously treated with an approved JAK inhibitor (except MMB) for ≥12 weeks, or ≥4 weeks if JAK inhibitor therapy was complicated by a transfusion requirement of ≥4 units of RBC in 8 weeks, or Grade 3/4 AEs of thrombocytopenia, anemia, or hematoma. Combination arms are currently recruiting pts."
Clinical • Combination therapy • P1/2 data • Anemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Immunology • Myelofibrosis • Oncology • Thrombocytopenia • PIM1
November 03, 2023
Preclinical Studies Demonstrating Efficacy of Tasquinimod in Models of Advanced Myeloproliferative Neoplasm (MPN) in Blastic Phase
(ASH 2023)
- "Additionally, our findings showed that co-treatment with TM (5 to 30 µM) and ruxolitinib (250 to 1000 nM), BET inhibitor OTX015 (50 to 250 nM) or pelabresib (CPI-0610) (100 to 500 nM), or BCL2/Bcl-xL inhibitor navitoclax induced synergistic lethality in advanced MPN-BP cells exhibiting delta synergy scores of >1.0 (by the ZIP method). In a separate experiment on the same PDX model, treatment with TM (30 mg/kg/day) also induced significantly greater survival advantage than treatment with ruxolitinib (30 mg/kg/day) or OTX015 (30 mg/kg/day) by oral gavage. These findings clearly demonstrate preclinical efficacy of TM in advanced MPN-BP cellular models and create the rationale to further interrogate the efficacy of TM alone and in combinations with current, front-line therapies for advanced MPN with excess blasts."
IO biomarker • Metastases • Preclinical • Fibrosis • Immunology • Inflammation • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • BCL2 • BCL2L1 • CALR • CCND1 • CD123 • CD33 • CD34 • CD99 • CDK6 • CDKN1A • CLEC12A • CXCR4 • IL3RA • IL6 • ITGAM • JAK2 • MPO • MYC • NLRP3 • S100A8 • S100A9 • TERT • TLR4 • TNFA
November 03, 2023
Assessment of Minimal Clinically Important Difference in Patient-Reported Myelofibrosis-Associated Symptoms Using an Anchor-Based Analysis Based on MANIFEST Arm 3 Data
(ASH 2023)
- "Pelabresib (CPI-0610) is an investigational oral small-molecule BET inhibitor, while the Janus kinase inhibitors (JAKis) ruxolitinib or fedratinib are the current standard of care for intermediate- or high-risk pts with MF. These findings provide valuable guidance for assessing treatment outcomes and evaluating symptom improvements' clinical significance in this patient population. Further exploration of these findings requires larger data sets, which will be addressed in the forthcoming Phase 3 MANIFEST-2 study evaluating pelabresib and ruxolitinib versus placebo and ruxolitinib in JAKi treatment-naïve pts with MF."
Clinical • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis
November 03, 2023
Pre-Clinical Efficacy of CDK7 Inhibitor-Based Combinations in Cellular Models of Advanced Myeloproliferative Neoplasms (MPN) Transformed to AML
(ASH 2023)
- "Treatment with JAK inhibitor (JAKi), e.g., ruxolitinib, venetoclax or hypomethylating agents alone or in combination are ineffective in improving the poor survival in MPN-sAML...Present studies demonstrate that treatment with ATP-competitive, covalent CDK7 inhibitors (CDK7i) SY-1365, and clinical grade SY-5609, dose-dependently (20 to 250 nM) increased % G1 while reducing the % of cell-cycle S phase SET2 and HEL cells...SY-5609 treatment also exerted synergistic lethality with the BETi pelabresib or BD2-selective BETi ABBV-744 or the CBP/p300 inhibitor GNE-049 in MPN-sAML cells...Additionally, compared to each drug or vehicle control, co-treatment with SY-5609 and OTX015 (30 mg/kg/day by oral gavage) reduced more MPN-sAML burden and significantly improved survival in a HEL-Luc/GFP xenograft model without inducing toxicity. These findings demonstrate promising preclinical activity of CDK7 inhibition against the cellular models of MPN-sAML, supporting the rationale to..."
Metastases • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • ASXL1 • AURKA • BCL2L1 • BRD4 • CALR • CASP9 • CCND1 • CD123 • CD34 • CD99 • CDK1 • CDK4 • CDK6 • CDK9 • CDKN1A • CLEC12A • HEXIM1 • IL3RA • ITGAM • JAK2 • MCL1 • MYC • PIM1 • PLK1 • RUNX1 • SRSF2 • STAT5 • TET2 • TGFB1
November 06, 2024
Updated Results from the Phase 3 Manifest-2 Study of Pelabresib in Combination with Ruxolitinib for Janus Kinase Inhibitor–Naïve Patients with Myelofibrosis
(ASH 2024)
- P3 | "Conclusion At Wk 48, PELA+RUX continued to show improvements in spleen volume, TSS, multiple measures of anemia, and the BM microenvironment vs PBO+RUX, impacting the four hallmarks of MF. These data suggest that PELA+RUX could lead to more profound and sustained responses in pts with MF vs PBO+RUX."
Clinical • Combination therapy • P3 data • Anemia • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis
November 06, 2024
Evaluation of the Lethal Activity and Its Mechanism of Tasquinimod in Advanced Myeloproliferative Neoplasm (MPN) in Blastic Phase
(ASH 2024)
- "Importantly, cotreatment with TQ (5 to 30 µM) and ruxolitinib (250 to 1000 nM), BET inhibitor OTX015 (50 to 250 nM) or pelabresib (CPI-0610) (100 to 500 nM), or BCL2/Bcl-xL inhibitor navitoclax, induced synergistic lethality in advanced MPN-BP cells, represented by delta synergy scores of >1.0 (by the ZIP method)...Co-treatment with TQ and RGFP966 (HDAC3i) induced synergistic lethality in post-MPN sAML cells...These findings demonstrate the pre-clinical efficacy of TQ and/or JAKi or BETi or with novel agents identified here in advanced MPN and MPN-AML cells. They also create the rationale to further interrogate the pre-clinical efficacy of the TQ-based combinations against cellular models of advanced MPN."
IO biomarker • Metastases • Fibrosis • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • BCL2 • BCL2L1 • CALR • CCL2 • CCND1 • CD123 • CD33 • CD34 • CD99 • CDK6 • CDKN1A • CLEC12A • CXCL12 • CXCL8 • CXCR4 • HDAC3 • IL1A • IL3RA • IL6 • ITGAM • JAK2 • MPO • MYC • NLRP3 • NSD2 • S100A8 • S100A9 • TERT • TLR4 • TNFA • TP53
November 03, 2023
Pelabresib in Combination with Ruxolitinib for Janus Kinase Inhibitor Treatment-Naïve Patients with Myelofibrosis: Results of the MANIFEST-2 Randomized, Double-Blind, Phase 3 Study
(ASH 2023)
- P1/2, P3 | "As DIPSS Int-1 pts have been excluded or underrepresented in prior randomized trials in MF, MANIFEST-2 will also provide important insights in assessing the benefits of starting treatment at an earlier stage of the disease. Primary results from the pivotal Phase 3 MANIFEST-2 trial of pela and rux versus placebo and rux in JAKi treatment-naïve pts may have the potential to influence the MF treatment paradigm and will be presented at the ASH Annual Meeting 2023."
Clinical • Combination therapy • P3 data • Anemia • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytosis
October 27, 2025
PELABRESIB IN COMBINATION WITH RUXOLITINIB FOR JANUS KINASE INHIBITOR-NAIVE PATIENTS WITH MYELOFIBROSIS: 72-WEEK FOLLOW-UP WITH LONG-TERM EFFICACY OUTCOMES OF THE PHASE III MANIFEST-2
(SIE 2025)
- P3 | "Grade ≥3 TEAEs were similar and consistent between arms, with decreased imbalance in leukemic transformation over time and favorable trend in survival for PELA+RUX. Overall results suggest that PELA+RUX provides meaningful clinical benefits over RUX alone and support evidence of ongoing disease modification."
Clinical • Combination therapy • P3 data • Anemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Thrombocytopenia
September 19, 2025
MODULE 4: Novel Agents Under Investigation for MF
(ASH 2025)
- "This program is supported by educational grants from Blueprint Medicines, Bristol Myers Squibb, GSK, and Incyte Corporation. Rationale for the inhibition of BET proteins in MF; mechanism of action of pelabresib Key efficacy and safety findings from the Phase III MANIFEST-2 study combining pelabresib with ruxolitinib for patients with JAK inhibitor-naïve disease; potential role of BET inhibition as monotherapy and/or in combination with JAK inhibition Similarities and differences between pelabresib and investigational BET inhibitors (INCB57643); available data and ongoing evaluation of these agents Mechanism of antitumor activity of navtemadlin and biological rationale for its evaluation for patients with MF Design, eligibility criteria and key efficacy and safety endpoints of the Phase III BOREAS trial evaluating navtemadlin monotherapy for patients with MF whose disease was resistant or refractory to JAK inhibitors Ongoing Phase III POIESIS study of adding..."
Anemia • Hematological Disorders
August 26, 2025
Individualizing Treatment Selection in MF
(SOHO 2025)
- P3 | "These include the activin receptor ligand trap luspatercept, the telomerase inhibitor imetelstat, the selective inhibitor of nuclear export selinexor, the human double minute 2 inhibitor navtemadlin, and the bromodomain and extra-terminal protein inhibitor pelabresib...Elritercept — another activin receptor ligand trap — and the anti-hemojuvelin antibody DISC-0974 are being developed for anemia of MF...Currently, these patients are observed or managed for thrombotic risk similarly to those with essential thrombocythemia (ET) — receiving hydroxyurea for control of cytoses, ruxolitinib for symptoms, or interferon for long-term disease modification. Encouraging data on ruxolitinib in patients with intermediate-1-risk MF, along with analyses from the COMFORT trials demonstrating the benefits of early initiation of ruxolitinib in patients with intermediate-2/high-risk MF and intriguing evidence of event-free survival (EFS) improvement with both ruxolitinib and..."
Clinical • Essential Thrombocythemia • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Non-melanoma Skin Cancer • Oncology • Polycythemia Vera • Skin Cancer • Solid Tumor • ACVR1 • IRAK1
August 26, 2025
Pelabresib in Combination With Ruxolitinib for Janus Kinase Inhibitor-Naive Patients With Myelofibrosis: 72-Week Follow-Up With Long-Term Efficacy Outcomes of the Phase 3 Manifest-2 Study
(SOHO 2025)
- P3 | "PELA+RUX showed sustained improvements over 72 weeks in splenic response, symptoms, SVR35/TSS50 dual response, BMF, and anemia vs PBO+RUX. The overall safety profile was similar across treatment arms, with imbalance in leukemic transformation decreasing over time. Survival outcomes show a trend in favor of PELA+RUX."
Clinical • Combination therapy • P3 data • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology
August 26, 2025
Is Combination Therapy Here for MPN?
(SOHO 2025)
- "2 Outside of the potential benefit of type 2 (AJ1-11095) and JAK2 V617F-selective (INCB160058) inhibitors under phase 1 evaluation, the research community has focused efforts on developing rational JAK inhibitor-based combination therapy regimens...3 Data from prospective and retrospective studies suggest a correlation between spleen reduction and OS with ruxolitinib and pacritinib...Nevertheless, the combination of pelabresib and ruxolitinib was well tolerated, and biomarkers of disease modification — including reduction in bone marrow fibrosis, driver mutation allele frequency, and inflammatory cytokine expression levels — all favored the combination, supporting the proposed mechanism of action and published preclinical synergy...11 The phase 3 trial will aim to confirm this activity in patients who have received at least 18 weeks of ruxolitinib therapy and meet rigorously defined criteria for suboptimal response, after which these patients will be randomized to..."
Combination therapy • IO biomarker • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • CD34 • XPO1
August 26, 2025
ASXL1 Mutations in AML Are Associated With a Distinct Epigenetic State That Results in Vulnerabilities to Epigenetic-Targeted Agents
(SOHO 2025)
- " Notably, compared with parental cells, OCIAML3 ASXL1 Y591* cells exhibited reduced sensitivity to chemotherapeutic agents, including cytarabine, etoposide, and daunorubicin. In contrast, mtASXL1-expressing AML cells exhibited increased sensitivity to bromodomain and extraterminal inhibitors (BETi; pelabresib or birabresib)... These findings highlight that rationally targeted agents including NEO2734, pelabresib, and SEL120 or their combinations can potentially exert significant anti-AML efficacy against AML cells with mtASXL1."
Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • ASXL1 • BAP1 • BCL9L • DVL1 • HOXA9 • MEIS1 • MYC • SPI1 • TCF7L2 • WNT5B • WNT7B
August 14, 2025
Inhibition of BRD4 prevents peribronchial fibrosis in mice with cutaneous lewisite exposure.
(PubMed, Front Mol Biosci)
- "Given CPI-0610's favorable clinical safety profile, it holds promise as a novel therapeutic strategy for treating arsenical-induced pulmonary complications, potentially improving outcomes where current countermeasures like BAL fall short. Further studies are warranted to explore its mechanism of action and therapeutic efficacy in broader exposure models."
Journal • Preclinical • Fibrosis • Hematological Disorders • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases • BRD4 • IL6
July 12, 2025
Highlights from MPN Asia 2025: Advances in Molecular Pathogenesis and Therapeutic Strategies in Myeloproliferative Neoplasms.
(PubMed, Curr Hematol Malig Rep)
- "Ropeginterferon alfa-2b, a novel interferon-based therapy, demonstrated durable clinical efficacy in polycythemia vera...Combination regimens involving ruxolitinib and agents such as pelabresib, selinexor, and interferon showed potential for enhanced efficacy...Its early use and personalized strategies are increasingly recognized. Real-world data and regional insights are shaping a more nuanced, globally informed approach to MPN care."
Journal • Review • Essential Thrombocythemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • ASXL1 • IL17A
June 27, 2025
An Extension Study for Patients Previously Enrolled in Studies With Pelabresib
(clinicaltrials.gov)
- P3 | N=50 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Jun 2029 ➔ Jun 2027 | Trial primary completion date: Jun 2029 ➔ Jun 2027
Trial completion date • Trial primary completion date • Hematological Malignancies • Oncology • Solid Tumor
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