ALK2 inhibitors(Activin receptor type-1 inhibitors), CSF-1R antagonists(Colony stimulating factor 1 receptor antagonists), FLT3 inhibitors(Tyrosine-protein kinase receptor FLT3 inhibitors)

Therapeutic Areas

Hemic and Lymphatic DiseasesOther DiseasesNeoplasms+ [3]

Active Indication

Primary MyelofibrosisThrombocytopeniaThrombocythemia, Essential+ [9]

Inactive Indication

Acute Myeloid LeukemiaAdult Acute Myeloblastic LeukemiaAggressive-Phase Chronic Myelocytic Leukemia+ [10]

Originator Organization

CTI BioPharma Corp.

Active Organization

Swedish Orphan Biovitrum ABSobi, Inc.

CTI BioPharma Corp.

+ [1]

Inactive Organization

S*BIO Pte Ltd.

Baxalta, Inc.

License Organization

Baxalta Bioscience India Pvt Ltd.

CTI BioPharma Corp.

Onyx Pharmaceuticals, Inc.

+ [3]

Drug Highest PhaseApproved

First Approval Date

United States (28 Feb 2022),

Primary MyelofibrosisThrombocytopenia

RegulationFast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Priority Review (United States)

Structure/Sequence

Molecular FormulaC28H32N4O3

InChIKeyHWXVIOGONBBTBY-ONEGZZNKSA-N

CAS Registry937272-79-2

Clinical Trials associated with Pacritinib

NCT07387354

/ Not yet recruitingPhase 1/2IIT

A Phase 1/2 Study of Pacritinib in Combination With Azacitidine for the Treatment of IPSS-M Moderate Low to Very High Risk Myelodysplastic Syndrome

This study will be conducted as a phase 1/2 study of safety and preliminary efficacy of pacritinib in combination with azacitidine for IPSS-M moderate low to very high risk MDS. Phase one will be a 3 + 3 design to assess the dose for the phase two portion. The phase two portion will employ a simon min-max two-stage design whereby fifteen patients will be enrolled in the first stage then ten more if at least two patients in stage one have a response. The dosing of pacritinib for the phase two study will be based on the phase one findings. Standard dosing of azacitidine will be used. A correlative study will be conducted in conjunction with the trial where the investigators will measure whole blood collected pre-treatment and at four days post-treatment to measure intracellular flow and phosflow to detect JAK/STAT, NF-κβ, and AKT/mTOR signaling in patient samples and how treatment affects these pathways.

Start Date01 Apr 2026

Sponsor / Collaborator

Thomas Jefferson University

[+1]

NCT07148947

/ RecruitingPhase 2IIT

A Phase 2 Trial Investigating the Addition of Pacritinib to a Hypomethylating Agent as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant in Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

This phase II trial tests if adding pacritinib to standard of care azacitidine or decitabine increases the number of patients able to proceed to hematopoietic stem cell transplantation (bridging) for patients with accelerated and blast phase myeloproliferative neoplasms. Pacritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Azacitidine and decitabine are in a class of medications called hypomethylation agents. They work by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Adding pacritinib to standard of care azacitidine or decitabine may increase the number of patients able to proceed to hematopoietic stem cell transplantation for patients with accelerated and blast phase myeloproliferative neoplasms.

Start Date15 Mar 2026

Sponsor / Collaborator

University of Washington

[+1]

NCT06303193

/ Not yet recruitingPhase 1/2IIT

Phase I/II Trial of Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Background:
Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed.
Objective:
To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN.
Eligibility:
Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN.
Design:
Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone.
Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants.
Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years.
Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.

Start Date04 Mar 2026

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Pacritinib

100 Translational Medicine associated with Pacritinib

100 Patents (Medical) associated with Pacritinib

226

Literatures (Medical) associated with Pacritinib

31 Dec 2025·Hospital practice (1995)

Real-world experience with pacritinib for patients with myelofibrosis refractory to ruxolitinib: a report of three cases

Article

Author: Otsmane, Sonia ; Al-Rasheed, Mona ; Al Essa, Taghreed ; Zureiqi, Mohammad

OBJECTIVES:

Ruxolitinib, a Janus kinase (JAK) inhibitor, can lead to severe ruxolitinib discontinuation syndrome (RDS) upon abrupt cessation in myelofibrosis (MF). Pacritinib, a selective JAK2/IRAK1 inhibitor with minimal JAK1 inhibition, offers an alternative, particularly for patients with thrombocytopenia. This case report presents our experience of successfully switching from ruxolitinib to pacritinib in patients with MF and severe RDS.

CASE PRESENTATION:

Three males in their early 20s, 60s, and 70s of Arab ethnicity presented with diverse clinical presentations, including post-polycythemia vera MF, primary MF, and primary triple-negative MF with multiple comorbidities. Ruxolitinib discontinuation was carefully managed through gradual tapering, concurrent corticosteroid administration, and pacritinib initiation, effectively preventing withdrawal syndrome. All patients demonstrated significant clinical improvements with pacritinib. Notable outcomes included reductions in spleen size (ranging from 7 to 8 cm within 1-6 months), stabilization or improvement in hematologic parameters, and resolution of transfusion dependency in previously transfusion-dependent cases. One patient achieved transfusion independence within six months of treatment, while another exhibited marked symptom relief and improved quality of life within one month. Adverse events, including gastrointestinal symptoms, weight loss, and transient voice changes, were manageable through dose adjustments and supportive care, enabling continued therapy.

CONCLUSION:

Our cases contribute to the growing body of evidence supporting pacritinib's role in the evolving treatment landscape of MF.

01 Dec 2025·Current Hematologic Malignancy Reports

Emerging Therapeutic Approaches for Anemia in Myelofibrosis

Review

Author: Harrington, Patrick ; Palumbo, Giuseppe A ; Chifotides, Helen T ; Duminuco, Andrea ; Torre, Elena ; Bose, Prithviraj ; Vetro, Calogero

PURPOSE OF REVIEW:

In this review, we highlight conventional agents and novel emerging therapeutic strategies to treat anemia in MF.

RECENT FINDINGS:

Anemia is a common and challenging feature of myelofibrosis (MF). The pathobiology of anemia is multifactorial, including progressive bone marrow fibrosis, decreased erythropoiesis due to high hepcidin levels leading to iron sequestration in the reticuloendothelial system, hypersplenism, erythropoiesis inhibition by myelosuppressive JAK inhibitors (ruxolitinib, fedratinib), and others. MF-associated anemia has a negative impact on survival. Conventional agents to manage anemia include erythropoiesis-stimulating agents, danazol, corticosteroids, and immunomodulatory agents, but responses are infrequent and lack durability. Notable advancements have emerged in developing novel treatments for anemia in MF, including the regulatory approval of momelotinib (ACVR1/JAK1/2 inhibitor) in 2023 and development of novel promising agents targeting hemojuvelin and activins. Momelotinib and pacritinib (ACVR1/JAK2 inhibitor) are the preferred JAK inhibitors for patients with cytopenias (anemia, thrombocytopenia). Luspatercept and elritercept are activin receptor ligand traps, promoting erythroid maturation and late-stage erythropoiesis. Currently, luspatercept is being evaluated in a phase 3 trial (INDEPENDENCE™) for anemia in MF patients who are on a JAK2 inhibitor and require transfusions, and in a phase 2 trial (ODYSSEY) in combination with momelotinib in MF patients who are transfusion dependent, whether or not on a JAK inhibitor. Interim results of the RESTORE trial demonstrated that elritercept significantly decreased transfusions in MF patients. DISC-0974 is a first-in-class anti-hemojuvelin (positive hepcidin regulator) monoclonal antibody that decreased hepcidin expression, increased serum iron, and enhanced erythropoiesis in anemic patients with MF in a phase 1b/2 study. Burgeoning studies of novel anemia-targeted agents and combinations are significantly improving the quality of life and outcomes of patients with MF. The recent approval of momelotinib to treat MF with anemia and the emerging novel anemia-directed strategies in early and advanced clinical development have ushered in a new era in the treatment of MF-related anemia.

01 Dec 2025·CTS-Clinical and Translational Science

Janus Kinase Inhibitors During Pregnancy and Adverse Drug Reactions: A Pharmacovigilance Disproportionality Analysis in V igi B ase

Article

Author: Panchaud, Alice ; Noseda, Roberta ; Ceschi, Alessandro ; Girardin, François R. ; Dao, Kim ; Abolhassani, Nazanin ; Winterfeld, Ursula ; Bedussi, Francesca

ABSTRACT:

Janus kinase inhibitors (JAKIs) are immunomodulatory drugs used for autoimmune and inflammatory conditions. Their potential impact on pregnancy and fetal development remains a concern due to placental transfer and potential disruption of cytokine and growth factor signaling, with limited human data. This study analyzed VigiBase, the World Health Organization global pharmacovigilance database of individual case safety reports (ICSRs), to assess signals of disproportionate reporting (SDRs) for pregnancy‐related adverse drug reactions (ADRs) reported with systemic JAKIs, including abrocitinib, baricitinib, deucravacitinib, fedratinib, filgotinib, itacitinib, momelotinib, pacritinib, peficitinib, ritlecitinib, ruxolitinib, tofacitinib, and upadacitinib. As of May 26, 2024, 163 ICSRs met inclusion criteria, mainly from North America (41.7%) and Europe (39.3%). The most frequently reported JAKIs were tofacitinib (44.8%) and upadacitinib (34.4%), primarily indicated for rheumatoid arthritis (29.4%). Among 213 pregnancy‐related ADRs, spontaneous abortion was the most frequently reported event (47.9%) without representing an SDR compared with other drugs in the database (reporting odds ratio [ROR] 0.37, 95% confidence interval [CI] 0.30–0.46). Congenital anomalies were reported in 16.0% of ICSRs (43 events), but no specific organ‐related patterns were identified. Prematurity occurred in 9.2% of ICSRs, without SDR compared to the full database (ROR 0.07, 95% CI 0.04–0.11). Current pharmacovigilance data from VigiBase do not indicate SDRs for spontaneous abortion or prematurity following JAKI exposure during pregnancy. Findings should be interpreted cautiously given the limitations of spontaneous reporting systems and the exploratory nature of the analysis. Further studies are needed to better characterize the JAKI safety in pregnancy.

News (Medical) associated with Pacritinib

05 Dec 2025

·www.prnewswire.com

Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025

STOCKHOLM, Dec. 5, 2025 /PRNewswire/ -- Sobi® (STO: SOBI), today announced its participation at the 65th American Society of Hematology (ASH) Annual Meeting, taking place on 6 – 9 December in Orlando, Florida. At this year's meeting, Sobi will showcase its commitment to advancing care in haematology with 19 scientific abstracts, including two oral presentations. These feature the most recent clinical data and insights from completed and ongoing studies across Sobi's innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib. These presentations underscore Sobi's mission to deliver life-changing therapies for patients with rare and severe blood disorders. "We will present evidence at ASH from a post hoc analysis demonstrating that pacritinib reduces or stabilises spleen size, improves haematologic parameters, and lessens myelofibrosis symptoms in patients with highrisk disease. These findings matter because they address key drivers of morbidity which affect daytoday functioning and quality of life. The ASH meeting also offers an opportunity to discuss the latest research on emapalumab, efanesoctocog alfa, pegcetacoplan, avatrombopag, as well as loncastuximab tesirine," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Summary of full Sobi data to be presented at ASH 2025: Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria. Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report. User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria. About ALTUVOCT ® (efanesoctocog alfa) ALTUVOCT ® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (HA). ALTUVOCT can be used for all age groups and any disease severity. About the Sobi and Sanofi Collaboration Sobi and Sanofi collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the US, European Union, and other countries globally, and for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States. It is under regulatory review for C3G and primary IC-MPGN in the European Union and other countries globally. About the Sobi and Apellis Collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialisation. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® (avatrombopag) Doptelet (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure. Doptelet is also approved for the treatment of chronic ITP in pediatric patients. About Gamifant® (emapalumab-lzsg) Gamifant is an anti-IFNγ antibody that binds free and receptor-bound IFNγ, which when secreted in an uncontrolled manner can cause hyperinflammation. Gamifant is indicated for intravenous infusion over one hour and is FDA approved for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) in Still's disease. About Vonjo® (pacritinib) Vonjo (pacritinib) is a kinase inhibitor indicated in the United States for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. Vonjo is also being investigated for other rare hematologic conditions, including VEXAS syndrome. Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: 21% more press release views with Request a Demo

Drug ApprovalASHAccelerated Approval

05 Dec 2025

·www.sobi.com

Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025

Press releases News Image gallery YouTube Subscribe Sobi® (STO: SOBI), today announced its participation at the 65th American Society of Hematology (ASH) Annual Meeting, taking place on 6 – 9 December in Orlando, Florida. At this year’s meeting, Sobi will showcase its commitment to advancing care in haematology with 19 scientific abstracts, including two oral presentations. These feature the most recent clinical data and insights from completed and ongoing studies across Sobi’s innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib. These presentations underscore Sobi’s mission to deliver life-changing therapies for patients with rare and severe blood disorders. “We will present evidence at ASH from a post hoc analysis demonstrating that pacritinib reduces or stabilises spleen size, improves haematologic parameters, and lessens myelofibrosis symptoms in patients with highrisk disease. These findings matter because they address key drivers of morbidity which affect daytoday functioning and quality of life. The ASH meeting also offers an opportunity to discuss the latest research on emapalumab, efanesoctocog alfa, pegcetacoplan, avatrombopag, as well as loncastuximab tesirine,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Summary of full Sobi data to be presented at ASH 2025: Efanesoctocog alfa Clinical outcomes up to four years of once-weekly Efanesoctocog Alfa Prophylaxis in previously treated adults, adolescents, and children with severe Haemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study. Oral Presentation Session Name: 322. Haemophilia A and B: Clinical and epidemiological: Innovations shaping the future of Haemophilia care Date: 7 December 2025 Time: 12:00 PM - 1:30 PM ET Presentation Time: 1:00 PM - 1:15 PM ET Room: OCCC – W304EFGH Publication Number: 539 Understanding unmet needs for people with Haemophilia A receiving factor and non-factor treatments. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 2679 Real-world experience of Efanesoctocog Alfa in Haemophilia A patients in the US: A retrospective analysis. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 1286 Patient characteristics, treatment patterns, and bleeding in people with Haemophilia A without inhibitors initiating Efanesoctocog alfa in the US: An administrative claims analysis. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 1290 Quality of life and functional improvements with Efanesoctocog alfa in patients with moderate to severe Haemophilia A: A real-world survey. Poster Presentation Session name: Poster Session III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 4846 Pegcetacoplan Consistent benefits of Pegcetacoplan treatment in PNH patients with and without a history of Aplastic Anaemia in real world: Analysis of the ongoing COMPLETE Phase 4 observational study. Poster Presentation Session Name: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Early results from the ongoing Pegcetacoplan Silo of the International Paroxysmal Nocturnal Haemoglobinuria Interest Group Registry. Poster Presentation Session Name: 508. Bone Marrow Failure: Acquired: Poster II Date: 7 December 2025 Time: 6:00:00 PM – 6:00:00 PM ET Location: OCCC - West Halls B3-B4 Real-world clinical characteristics and treatment outcomes in PNH patients prescribed Pegcetacoplan: Insights of complement inhibitor-experienced and -naïve patients across Europe, the United States and Canada. Poster Presentation Session Name: 508. Bone Marrow Failure: Acquired: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Pegcetacoplan - Publication Only Abstracts Real-world effectiveness of Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria: A systematic review of clinical and patient-reported outcomes. Publication only - published online on 3 November 2025, at 9:00 AM ET Low risk for Meningococcal and encapsulated bacteria infections with systemically administered Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria and C3 Glomerulopathies. Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria. Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report. User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria. Avatrombopag Real-world treatment patterns and outcomes among patients with immune thrombocytopenia (ITP) who switched treatment from Eltrombopag or Romiplostim to Avatrombopag in the United States: Results from the real-AVA 3.5 study. Poster Presentation Session Name: 905. Outcomes research: Non-malignant conditions excluding Hemoglobinopathies: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Real-world safety and efficacy of Avatrombopag in adults with Immune Thrombocytopenia: A systematic review and meta-analysis. Global Abstract Session Name: 311. Disorders of platelet number or function: Clinical and epidemiological: Poster I Date: 6 December 2025 Time: 5:30 PM – 7:30 PM ET Room: OCCC – West Halls B3-B4 Patient-reported outcomes of Avatrombopag for Chronic Immune Thrombocytopenia: Interim analysis of the Phase 4 ADOPT Study. Poster Presentation Session Name: 905. Outcomes research: Non-malignant conditions excluding Hemoglobinopathies: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Emapalumab Use of Emapalumab is associated with rapid and sustained benefits in pHLH subgroups, including CNS involvement and previously untreated patients: Pooled analysis of prospective trials NI-0501-04, NI-050105 and NI-050109. Poster Presentation Session Name: 201. Granulocytes, Monocytes, and Macrophages: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Emapalumab induces rapid, durable responses and reliable bridging to curative HSCT in patients with primary HLH: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09. Poster Presentation Session Name: 201. Granulocytes, Monocytes, and Macrophages: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Pacritinib Real-world treatment patterns and outcomes in patients with myelofibrosis who presented with thrombocytopenia and anaemia at initiation of Pacritinib treatment. Oral Presentation Session Name: 908. Outcomes Research: Myeloid Malignancies: Real-World Experiences Session date: 7 December 2025 Session time: 4:30 PM - 6:00 PM ET Presentation time: 5:30 PM - 5:45 PM Room: OCCC - W414CD Pacritinib in patients with high-risk myelofibrosis: Outcomes from post-hoc analyses of two Phase 3 studies. Poster Presentation Session Name: 634. Myeloproliferative Syndromes: Clinical and epidemiological: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Real-world treatment patterns and clinical outcomes in patients with Myelofibrosis treated with Pacritinib (PAC): Results from the MY-PAC Study. Poster Presentation Session Name: 908. Outcomes research: Myeloid Malignancies: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 PROSPERA (ABNL-MARRO 002): A randomised Phase 2 study of Pacritinib vs. Hydroxyurea in patients with Advanced Proliferative Chronic Myelomonocytic Leukaemia (CMML) Poster Presentation Session Name: 637. Myelodysplastic Syndromes: Clinical and epidemiological: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Treatment patterns and outcomes in patients with myelofibrosis treated with Pacritinib following a switch from Ruxolitinib: The MY-PAC Study. Session Name: 634. Myeloproliferative Syndromes: Clinical and epidemiological: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Incidence, prevalence, and clinical outcomes of Myelofibrosis with and without Cytopenia in the United States. About ALTUVOCT ® (efanesoctocog alfa) ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (HA). ALTUVOCT can be used for all age groups and any disease severity. About the Sobi and Sanofi Collaboration Sobi and Sanofi collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the US, European Union, and other countries globally, and for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States. It is under regulatory review for C3G and primary IC-MPGN in the European Union and other countries globally. About the Sobi and Apellis Collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialisation. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® (avatrombopag) Doptelet (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure. Doptelet is also approved for the treatment of chronic ITP in pediatric patients. About Gamifant® (emapalumab-lzsg) Gamifant is an anti-IFNγ antibody that binds free and receptor-bound IFNγ, which when secreted in an uncontrolled manner can cause hyperinflammation. Gamifant is indicated for intravenous infusion over one hour and is FDA approved for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) in Still’s disease. About Vonjo® (pacritinib) Vonjo (pacritinib) is a kinase inhibitor indicated in the United States for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. Vonjo is also being investigated for other rare hematologic conditions, including VEXAS syndrome. Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. To stay up to date with our development, please subscribe to our press releases. Sign up We are a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. At a glance Focus on rare diseases Business strategy Partnership Partnership Our commitment Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services Innovate disease awareness campaigns Value added services History Board of directors Executive committee Corporate Governance General Meetings Nomination committee Committees Remuneration Auditors Articles of association General Meetings Nomination committee Committees Remuneration Auditors Articles of association Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Specialty Care Pipeline Congresses Research & development Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Medical Grant We contribute to societies by improving access to treatment of rare diseases. Sustainability performance Key data Key data Sustainability governance Code of Conduct Policies Governance Code of Conduct Policies Governance Commitment to patients Access to treatment Patient centricity Humanitarian aid Access to treatment Patient centricity Humanitarian aid Act responsibly Caring for employees Environmental footprint Responsible sourcing Compliance Caring for employees Environmental footprint Responsible sourcing Compliance Transparency Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Find out more about our business and financial performance. Financial reports Presentations Financial data Calendar Pipeline Share information All trading venues Historical share price look-up Ownership Analyst coverage All trading venues Historical share price look-up Ownership Analyst coverage IR contacts Subscribe MTN-programme Rights issue 2023 Here we present our most recent press releases, stories, news articles, and images. Press releases News Image gallery YouTube Subscribe Every day, we work actively to find better ways to understand and meet patient needs. Our values Meet our employees Open positions About At a glance Focus on rare diseases Business strategy Partnership Our commitment Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services History Board of directors Executive committee Corporate Governance General Meetings Nomination committee Committees Remuneration Auditors Articles of association Therapeutic areas Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Specialty Care Pipeline Congresses Research & development Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Medical Grant Medicines Stories Sustainability Sustainability performance Key data Sustainability governance Code of Conduct Policies Governance Commitment to patients Access to treatment Patient centricity Humanitarian aid Act responsibly Caring for employees Environmental footprint Responsible sourcing Compliance Transparency Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Investors Financial reports Presentations Financial data Calendar Pipeline Share information All trading venues Historical share price look-up Ownership Analyst coverage IR contacts Subscribe MTN-programme Rights issue 2023 News Press releases News Image gallery YouTube Subscribe Careers Our values Meet our employees Open positions At a glance Focus on rare diseases Business strategy Partnership Our commitment Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services History Board of directors Executive committee Corporate Governance General Meetings Nomination committee Committees Remuneration Auditors Articles of association Partnership Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate disease awareness campaigns Value added services General Meetings Nomination committee Committees Remuneration Auditors Articles of association Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Specialty Care Pipeline Congresses Research & development Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Medical Grant Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Sustainability performance Key data Sustainability governance Code of Conduct Policies Governance Commitment to patients Access to treatment Patient centricity Humanitarian aid Act responsibly Caring for employees Environmental footprint Responsible sourcing Compliance Transparency Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Key data Code of Conduct Policies Governance Access to treatment Patient centricity Humanitarian aid Caring for employees Environmental footprint Responsible sourcing Compliance Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Financial reports Presentations Financial data Calendar Pipeline Share information All trading venues Historical share price look-up Ownership Analyst coverage IR contacts Subscribe MTN-programme Rights issue 2023 All trading venues Historical share price look-up Ownership Analyst coverage Press releases News Image gallery YouTube Subscribe Our values Meet our employees Open positions

Drug ApprovalASHPhase 3Clinical ResultPhase 2

07 Nov 2025

·www.prnewswire.com

Shinobi Therapeutics Announces the Appointment of Dr. Laurent Fischer as Chair of its Board of Directors

Dr Fischer brings 30+ years of experience leading and exiting innovative biotech companies focused on developing and commercializing breakthrough therapies, including Adverum Biotechnologies, recently acquired by Eli Lilly Shinobi prepares to advance two immune evasive iPSC therapies toward the clinic in 2026, and today announces its first data presentation at the upcoming Society for Immunotherapy of Cancer (SITC) Annual Meeting SAN FRANCISCO and KYOTO, Japan, Nov. 7, 2025 /PRNewswire/ -- Shinobi Therapeutics, the biotechnology company developing a new class of immune evasive iPS therapies, today announced the appointment of Laurent Fischer, MD, as its first independent Chair of the Board of Directors, alongside the company's first data presentation at the Society for Immunotherapy of Cancer (SITC) Annual Meeting taking place in National Harbor, MD from November 5-9, 2025. These milestones collectively reflect Shinobi's continued momentum as it advances its oncology and autoimmune pipeline toward clinical entry in 2026. Dr. Fischer brings more than 30 years of senior leadership experience in biotechnology, with a demonstrated track record of building, scaling and exiting companies focused on breakthrough therapies. His accomplishments include the sale of Jennerex Biotherapeutics to SillaJen (2013), leading Tobira Therapeutics through its IPO and subsequent $1.7 billion acquisition to Allergan (2016), chairing CTI BioPharma through its approval and launch of Vonjo™ and subsequent $1.7 billion sale to SOBI and most recently, the sale of Adverum Biotechnologies to Eli Lilly in October 2025, handing off an advanced and potentially curative gene therapy for wet AMD. Dr. Fischer brings extensive board experience, including current positions as first independent Director of Lycia Therapeutics, Director of Mirum Pharmaceuticals and Director of AtaiBeckley. He previously served as Chair of Tobira Therapeutics, Chairman of CTI BioPharma, Director of Senti Biosciences, and Senior Advisor of Frazier Healthcare Partners. "Following the recent acquisition of Adverum, I'm excited to turn my focus to Shinobi Therapeutics to help lead advancement of the company's promising immune evasive iPSC-derived cell therapy programs," said Dr. Fischer. "Combining the ability to overcome traditional cell therapy cost and access barriers with a re-dosable, predictable, and potent treatment could transform the way we care for cancer and autoimmune patients." The appointment comes as Shinobi publishes its first public data in a poster presentation at the 2025 SITC Conference, titled, "Systemic and regionally infused iPSC-Derived CD8 αβ T Cells Mediate Tumor Killing via TCR- and Innate Receptor-Dependent Mechanisms Enhanced by Cytokine Armoring." The poster will be presented on Saturday, Nov. 8th as Abstract 1010. Dr. Fischer joins the board following a strong Series A financing and a $71M non-dilutive grant from the Japanese Government's AMED program. These company milestones position Shinobi to continue development of NJA-001, an iPS-T cell therapy targeting GPC3 for solid tumors, and NJA-201, an iPS-NK cell therapy targeting CD19 for autoimmune diseases; and to advance Katana, the company's plug-and-play platform for rapid pipeline and cell type expansion. About Shinobi Therapeutics Shinobi Therapeutics is a biotechnology company developing a new class of patient and physician friendly off-the-shelf immune evasive iPSC-derived cell therapies to overcome conventional clinical and commercial barriers to cell therapies. The technology is based on the research of scientific co-founders Shin Kaneko, M.D., Ph.D., at Kyoto University and Tobias Deuse, M.D., at University of California, San Francisco. Shinobi closed a $51M Series A in 2023 and has a $71M non-dilutive grant from AMED to advance NJA-001 for solid tumor cancers, NJA-201 for autoimmune diseases, and the Katana platform for rapid pipeline expansion. SOURCE Shinobi Therapeutics 21% more press release views with Request a Demo

Executive ChangeImmunotherapyCell TherapyIPOAcquisition

100 Deals associated with Pacritinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11768	Pacritinib	-	DB11697

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Primary Myelofibrosis	United States	Sobi, Inc.	28 Feb 2022
Thrombocytopenia	United States	Sobi, Inc.	28 Feb 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 3	Belgium	CTI BioPharma Corp.	03 Dec 2012
Post-essential thrombocythemia myelofibrosis	Phase 3	Belgium	CTI BioPharma Corp.	03 Dec 2012
Post-polycythemia vera myelofibrosis	Phase 3	Belgium	CTI BioPharma Corp.	03 Dec 2012
Thrombocythemia, Essential	Phase 3	Belgium	CTI BioPharma Corp.	03 Dec 2012
Waldenstrom's macroglobulinaemia refractory	Phase 2	United States	Sobi, Inc.	21 Nov 2025
VEXAS syndrome	Phase 2	United States	Swedish Orphan Biovitrum AB	28 May 2025
VEXAS syndrome	Phase 2	Japan	Swedish Orphan Biovitrum AB	28 May 2025
VEXAS syndrome	Phase 2	Canada	Swedish Orphan Biovitrum AB	28 May 2025
VEXAS syndrome	Phase 2	France	Swedish Orphan Biovitrum AB	28 May 2025
VEXAS syndrome	Phase 2	Germany	Swedish Orphan Biovitrum AB	28 May 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Myelofibrosis	169	Pacritinib	mgkukcnwit(ravvvxmakk) = rmcdqflcks wcfltfmytu (ndrjafoeuo, 0.0 - 1.0) View more	Positive	06 Dec 2025
NCT07033598 (PROSPERA, ASH2025)	Phase 2	Chronic Myelomonocytic Leukemia	66	Pacritinib 200 mg orally twice daily	qphxxhhceq(ofayxzanxc) = trpbmpjlli xqccsqmyyb (cjcmohczab ) View more	Positive	06 Dec 2025
NCT07033598 (PROSPERA, ASH2025)	Phase 2	Chronic Myelomonocytic Leukemia	66	Hydroxyurea	znsshuvkgz(pbiatlwtfz) = dxknyhkkak mjiwukdtxx (ffhmlvdxir ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Myelofibrosis	236	Pacritinib	dmljewxmhq(qsyuqniosc) = The proportion of PAC vs MOM-treated pts discontinuing therapy at 3, 6, and 12 mo was 26% vs 17%, 52% vs 29%, and 70% vs 42%, respectively. ibxsutcbcc (udgnaarfrt )	Positive	06 Dec 2025
ASH2025	Not Applicable	Myelofibrosis	236	Momelotinib		Positive	06 Dec 2025
NCT01773187 (PERSIST-1, ASH2025)	Phase 3	Primary Myelofibrosis	114	Pacritinib	eodwbrxjbv(zvvwgluzxy) = zpemmmdphe zbzhohftkc (pbywwcsrpb, 86.1 - NE) View more	Positive	06 Dec 2025
NCT01773187 (PERSIST-1, ASH2025)	Phase 3	Primary Myelofibrosis	114	Best Available Therapy (BAT)	eodwbrxjbv(zvvwgluzxy) = gymmxihkeu zbzhohftkc (pbywwcsrpb, 43.6 - NE) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Myelofibrosis	169	Pacritinib (PAC)	brmwnmdccp(whbgsquroq) = owslbwsgny mnhdlswiuh (wjqphhwfdk, 8.1 - 9.8) View more	Positive	06 Dec 2025
ASCO2025	Not Applicable	Myelofibrosis First line \| Second line	212	Pacritinib	kbkufxjybi(lpvruqpltw) = fpaicyrwzi fxrczrdldn (jcwhtzfoyi, 61.5 - 75.3) View more	Positive	30 May 2025
NCT02055781 (PERSIST-2, ASCO2025)	Phase 3	Myelofibrosis	-	Pacritinib	hjnmuwwngq(itzjoheurg) = evcpnbyxpi rpwdmrbjfc (tfxarejgjh ) View more	Positive	30 May 2025
NCT02055781 (PERSIST-2, ASCO2025)	Phase 3	Myelofibrosis	-	Best Available Therapy (BAT)	hjnmuwwngq(itzjoheurg) = uktvqinbvs rpwdmrbjfc (tfxarejgjh ) View more	Positive	30 May 2025
ASCO2025	Not Applicable	Myelofibrosis	148	Pacritinib	rotdjiudcw(fjdmqhkdmr) = yftxaqofrz tbtwlndzzl (qyiirzrzca, 27 - 84) View more	Positive	30 May 2025
PS1827 (EHA2025)	Not Applicable	Anemia \| Thrombocytopenia \| Myelofibrosis First line	179	Pacritinib	srgfrjpzsi(vlrwiqnkap) = pkcsktszgd fhsdbcvssk (bxnyvwoath, 6.9 - 7.6) View more	Positive	14 May 2025
MY-PAC STUDY (EHA2025)	Not Applicable	Myelofibrosis	35	Pacritinib (PAC)	miaizzdukw(tlhspjukjb) = dnyaxxvyfe iqeciqucqs (imdaawkntw, -1.1% - 12.5%) View more	-	14 May 2025

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis