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+ [1]

Active Indication

Primary MyelofibrosisThrombocytopeniaPost-essential thrombocythemia myelofibrosis+ [4]

Inactive Indication

Acute Myeloid LeukemiaAdult Acute Myeloblastic LeukemiaB-Cell Lymphoma+ [12]

Originator Organization

CTI BioPharma Corp.

Active Organization

Swedish Orphan Biovitrum ABSobi, Inc.

Swedish Orphan Biovitrum Ltd.

+ [1]

Inactive Organization

S*BIO Pte Ltd.

Baxalta, Inc.

Drug Highest PhaseApproved

First Approval Date

US (28 Feb 2022),

Primary MyelofibrosisThrombocytopenia

RegulationPriority Review (US), Fast Track (US), Accelerated Approval (US), Orphan Drug (US)

Structure/Sequence

Molecular FormulaC28H32N4O3

InChIKeyHWXVIOGONBBTBY-ONEGZZNKSA-N

CAS Registry937272-79-2

Clinical Trials associated with Pacritinib

NCT06675123

/ Not yet recruitingPhase 1IIT

A Pilot Study of Pacritinib Combined With a BTK Inhibitor in Patients With Relapsed/Refractory Mantle Cell Lymphoma

This phase I trial tests the safety and side effects of pacritinib in combination with a Bruton's tyrosine kinase (BTK) inhibitor and how well it works in treating patients with mantle cell lymphoma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Pacritinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. BTK inhibitors block a protein called BTK which is present on B-cell (a type of white blood cell) cancers such as mantle cell lymphoma at abnormal levels. This may help keep tumor cells from growing and spreading. Giving pacritinib in combination with a BTK inhibitor may be safe, tolerable and/or effective in treating patients with relapsed or refractory mantle cell lymphoma.

Start Date01 Nov 2025

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06782373

/ Not yet recruitingPhase 2

PAXIS: A Randomized, Double-blind, Placebo-controlled Dose-finding Phase 2 Study (Part 1) Followed by an Open-label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome

This study is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 patients will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.

Start Date01 May 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

NCT06538181

/ Not yet recruitingPhase 1IIT

A Phase 1 Study of Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin- activating enzyme gene, UBA1, that is acquired in hematopoietic progenitor cells. Patients are often debilitated by autoinflammatory symptoms and there is currently no standard of care available. There is a clinically unmet need for better therapies in VEXAS Syndrome. There have been no prospective clinical trials of JAK-I in VEXAS syndrome. The investigators hypothesize that pacritinib, as a JAK2/IRAK1 inhibitor with a manageable safety profile in myelofibrosis patients with thrombocytopenia, will improve the autoinflammatory and hematologic manifestations of VEXAS syndrome with a tolerable toxicity profile.
The investigators propose a single arm, pilot Phase 1 study evaluating the safety and tolerability of pacritinib in patients with VEXAS syndrome with an initial safety run-in phase of 6 patients treated with pacritinib 200mg twice daily (BID) on days 1-28 of a continuous 28 day cycle. If no more than 1 patient experiences a dose-limiting toxicity (DLT), the investigators will enroll an expansion cohort to gain additional toxicity and efficacy data, for a total enrollment of 15 patients. If more than 1 patient experiences a DLT during the safety run-in phase, the investigators will decrease the dose to 100 mg BID, and if no more than 1 of 6 patients experiences a DLT, the investigators will complete the expansion cohort as above for up to a total enrollment of 15 patients. If more than 1 patient experiences a DLT at 100 mg BID, the investigators will discontinue the study. Patients will be treated for up to 12 cycles.

Start Date31 Jan 2025

Sponsor / Collaborator

Washington University School of Medicine

[+1]

100 Clinical Results associated with Pacritinib

100 Translational Medicine associated with Pacritinib

100 Patents (Medical) associated with Pacritinib

168

Literatures (Medical) associated with Pacritinib

01 Feb 2025·EUROPEAN JOURNAL OF HAEMATOLOGY

Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST‐2 Landmark Analysis of Survival

Article

Author: Mascarenhas, John ; Derkach, Andriy ; Rampal, Raajit K. ; Ajufo, Helen ; Palandri, Francesca ; Buckley, Sarah ; Kiladjian, Jean‐Jacques ; Gerds, Aaron ; Palmer, Jeanne ; Bewersdorf, Jan Philipp ; Harrison, Claire ; Roman‐Torres, Karisse

ABSTRACT:

Spleen volume reduction (SVR) is a key endpoint in inhibitors of Janus kinase (JAK) inhibitor studies. Retrospective analyses have demonstrated an association between SVR and improved overall survival (OS) among patients treated with ruxolitinib with a platelet count > 100 × 109/L. Whether this association occurs in patients with thrombocytopenia is unclear. Pacritinib, a JAK2/IRAK1/ACVR1 inhibitor, demonstrated improved SVR versus best available therapy (BAT [best available therapy]; including ruxolitinib) in patients with myelofibrosis and platelet counts ≤ 100 × 109/L in the PERSIST‐2 study. Patients on study at the start of the 12‐week SVR window on pacritinib 200 mg twice daily or BAT were included. OS was evaluated among SVR responders versus non‐responders using different SVR thresholds (≥ 35%, ≥ 20%, ≥ 10%, and > 0%). Among patients on pacritinib (n = 89), SVR ≥ 10% demonstrated the greatest separation in OS curves between responders and non‐responders (HR, 0.00; 95% CI, 0.00–0.14; p < 0.01), though SVR ≥ 0% and SVR ≥ 20% were also associated with improved OS. No SVR threshold conferred OS benefit on BAT (n = 84), including ruxolitinib (n = 39). In patients with myelofibrosis and platelets ≤ 100 × 109/L, achieving SVR on pacritinib, but not BAT (including ruxolitinib), was associated with significant OS benefit, suggesting that pacritinib may offer a unique survival advantage in patients with myelofibrosis and thrombocytopenia who achieve any SVR.Trial Registration:ClinicalTrials.gov number: NCT02055781

01 Dec 2024·BIOORGANIC CHEMISTRY

Muti-target rationale design of novel substituted N-phenyl-2-((6-phenylpyridazin-3-yl)thio)acetamide candidates as telomerase/JAK1/STAT3/TLR4 inhibitors: In vitro and in vivo investigations

Article

Author: Alzahrani, Abdullah Yahya Abdullah ; Al-Karmalawy, Ahmed A. ; El-Dessouki, Ahmed M ; Al-Karmalawy, Ahmed A ; Moussa, Sana Ben ; El-Dessouki, Ahmed M. ; Tawfik, Haytham O. ; Tawfik, Haytham O ; Shaldam, Moataz A. ; Sharaky, Marwa ; Saleh, Mohamed M ; Mousa, Mai H.A. ; Saleh, Mohamed M. ; Mousa, Mai H A ; Shaldam, Moataz A

In this work, additional effort was applied to design new BIBR1532-based analogues with potential inhibitory activity against telomerase and acting as multitarget antitumor candidates to overcome the resistance problem. Therefore, novel substituted N-phenyl-2-((6-phenylpyridazin-3-yl)thio)acetamide candidates (4a-n) were synthesized. Applying the lead optimization strategy of the previously designed compound 8e; compound 4l showed an improved telomerase inhibition of 64.95 % and a superior growth inhibition of 79 % suggesting its potential use as a successful "multitarget-directed drug" for cancer therapy. Accordingly, compound 4l was further selected to evaluate its additional JAK1/STAT3/TLR4 inhibitory potentials. Compound 4l represented a very promising JAK1 inhibitory potential with a 0.46-fold change, compared to that of pacritinib reference standard (0.33-fold change). Besides, it showed a superior STAT3-inhibitory potential with a 0.22-fold change compared to sorafenib (0.33-fold change). Additionally, compound 4l downregulated TLR4 protein expression by 0.81-fold change compared to that of resatorvid (0.29-fold change). Also, molecular docking was performed to investigate the binding mode and affinity of the superior candidate 4l towards the four target receptors (telomerase, JAK1, STAT3, and TLR4). Furthermore, the therapeutic potential of compound 4l as an antitumor agent was additionally explored through in vivo studies involving female mice implanted with Solid Ehrlich Carcinoma (SEC). Remarkably, compound 4l led to prominent reductions in tumor size and mass. Concurrent enhancements in biochemical, hematologic, histopathologic, and immunohistochemical parameters further confirmed the suppression of angiogenesis and inflammation, elucidating additional mechanisms by which compound 4l exerts its anticancer effects.

01 Dec 2024·JOURNAL OF ONCOLOGY PHARMACY PRACTICE

Pacritinib and concurrent azole antifungal therapy is deliverable in patients with hematologic malignancies

Article

Author: Ali, Haris ; Tinajero, Jose ; Ngo, Dat ; Trang, Dina

Objective:

Pacritinib is a novel kinase inhibitor approved for the treatment of adults with intermediate or high-risk primary or secondary myelofibrosis. Strong and moderate CYP3A4 inhibitors, such as some azole antifungals, are contraindicated or recommended to be avoided in combination with pacritinib, respectively. We aim to report our experience in patients who received pacritinib with concurrent azole antifungal therapy.

Data Sources:

We queried for patients with hematologic malignancies in the electronic medical record who received concurrent pacritinib and azole antifungal therapy.

Data Summary:

There were five cases of concurrent pacritinib and azole antifungal therapy in which none of the patients experienced grade 3 or higher non-hematologic toxicities. Some patients required dose modifications and/or interruptions in pacritinib therapy.

Conclusion:

This is the first clinical experience describing concurrent pacritinib and azole antifungals. Our experience shows that in the setting where this interaction cannot be avoided, concurrent administration is feasible with close monitoring and possible empiric dose reductions in select patients.

News (Medical) associated with Pacritinib

07 Jan 2025

·www.prnewswire.com

BerGenBio Announces First Patient Entered Into Advanced Adenocarcinoma Lung Cancer Trial

BERGEN, Norway, Jan. 7, 2025 /PRNewswire/ -- BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing the novel, selective AXL kinase inhibitor bemcentinib for lung cancer announced today that the first patient has been included in a clinical trial sponsored by the Mays Cancer Center at The University of Texas Health Science Center at San Antonio (UT Health San Antonio). The study is led by Josephine A. Taverna, MD, a thoracic oncologist at the Mays Cancer Center and Associate Professor in the Division of Hematology and Oncology at UT Health San Antonio. "Dr. Taverna's research has shown that AXL and JAK-STAT3 work in tandem to transmit signals promoting tumor growth and metastasis in advanced lung cancer. We are pleased to see this research advancing into clinical application and believe this study aligns closely with BerGenBio's strategic focus on addressing the significant unmet needs of lung cancer patients, including first-line Non-Small Cell Lung Cancer patients with mutations in the STK11 gene," said Olav Hellebø, Chief Executive Officer of BerGenBio. The trial is designed to study BerGenBio's bemcentinib in combination with pacritinib, which is a JAK2 inhibitor indicated for treatment of the bone marrow disorder myelofibrosis in patients with platelet counts below 50 x 109/L. It works by blocking certain growth factors and cytokines. Pacritinib is marketed in the United States as VONJO® and owned by Swedish Orphan Biovitrum AB (Sobi®) (STO: SOBI), a specialized international biopharmaceutical company. Bemcentinib is a first-in-class, selective, oral once-a-day inhibitor of AXL receptor tyrosine kinase, a promising therapeutic target for serious diseases. The study will include patients with lung adenocarcinoma, the most common type of lung cancer in the United States, accounting for approximately 40% of all lung cancers. The study is funded by a grant from the National Cancer Institute (NCI) of the National Institutes of Health (NIH). Contacts Olav Hellebø, CEO, BerGenBio ASA [email protected] Rune Skeie, CFO, BerGenBio ASA [email protected] About the Trial (NCT 06516887) The trial sponsored by the Mays Cancer Center at The University of Texas Health Science Center at San Antonio is entitled "Study of Bemcentinib plus Pacritinib in Patients with Advanced adenocarcinoma" is a Phase Ib/II, open-label, single institution dose-escalation, safety, pharmacokinetics, pharmacodynamic and efficacy study. It is anticipated that a total of 44 patients will be enrolled in the trial. In the PhIb portion varying doses of bemcentinib and pacritinib will be studied in combination to determine safety, tolerability and the maximum tolerated dose. The Phase II portion of the trial will study the dose(s) selected in Phase Ib and will assess the overall response rate and progression free survival of treated patients. About BerGenBio ASA BerGenBio is a clinical-stage biopharmaceutical company focused on developing transformative drugs targeting AXL as a potential cornerstone of therapy for aggressive diseases, including cancer. The Company is focused on its proprietary lead candidate, bemcentinib, a potentially first-in-class selective AXL inhibitor in development for STK11 mutated NSCLC. BerGenBio is based in Bergen, Norway with a subsidiary in Oxford, UK. The company is listed on the Oslo Stock Exchange (ticker: BGBIO). For more information, visit . Forward looking statements This announcement may contain forward-looking statements, which as such are not historical facts, but are based upon various assumptions, many of which are based, in turn, upon further assumptions. These assumptions are inherently subject to significant known and unknown risks, uncertainties, and other important factors. Such risks, uncertainties, contingencies and other important factors could cause actual events to differ materially from the expectations expressed or implied in this announcement by such forward-looking statements. This information is subject to the disclosure requirements pursuant to section 5-12 of the Norwegian Securities Trading Act. This information was brought to you by Cision SOURCE BerGenBio ASA WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Phase 1

20 Dec 2024

·www.prnewswire.com

Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VGT-1849A, a Novel and Selective Antisense Oligonucleotide Candidate for the Treatment of Polycythemia Vera

WASHINGTON, Dec. 20, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans.1 PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage with exuberant red cell production and increased release of pro-inflammatory cytokines. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation leading to aberrant JAK2 production.2 Inhibiting JAK2 acts to suppress hematopoiesis, consequently reducing red blood cell, neutrophil, platelet, and lymphocyte production. JAK2 inhibitors have been shown to be efficacious in treating various JAK-dependent hematologic malignancies, including the treatment of PV. By selective reduction of JAK2 levels, the ASO VGT-1849A has the potential to reduce JAK2V617F-driven pathogenic signaling, ultimately suppressing the malignant proliferation and survival of hematopoietic cells. Currently available small molecule inhibitors targeting the JAK2 protein kinase, such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, lack sole selectivity for the target protein, which can result in off target effects. The adverse side effects that may occur from JAK inhibition emphasize the importance of selectively targeting JAK2 while avoiding inhibition of other JAK family members. By specifically targeting JAK2, Vanda seeks to reduce the risk of infection and toxic effects that are seen with inhibitors also blocking JAK1, JAK3, TYK2, or other kinases outside of the JAK family. If approved, VGT-1849A could offer targeted efficacy with an improved safety profile and convenient dosing. "This orphan designation for VGT-1849A is an important milestone in precision medicine-based therapeutics in the space of hematological malignancies. This milestone marks the second precision medicine therapeutic for Vanda following the development of VCA-894A for Charcot-Marie-Tooth3 that is expected to begin clinical testing in the coming months," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. VGT-1849A is a novel ASO treatment candidate for PV and other JAK2-driven hematologic malignancies. By selectively targeting JAK2, VGT-1849A reduces downstream signaling and JAK2V617F-driven autonomous cell proliferation, without any off-target kinase effects. The ability of VGT-1849A to reduce JAK2 activity may alleviate the disease burden that patients with PV face with a favorable safety profile, resulting in a higher quality of life for patients. Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers. References: Grunwald, M. R.; Stein, B. L.; Boccia, R. V.; Oh, S. T.; Paranagama, D.; Parasuraman, S.; Colucci, P.; Mesa, R. Clinical and Disease Characteristics From REVEAL at Time of Enrollment (Baseline): Prospective Observational Study of Patients With Polycythemia Vera in the United States. Clin Lymphoma Myeloma Leuk 2018, 18 (12), 788-795.e2. . P. Gou, W. Zhang, and S. Giraudier, "Insights into the Potential Mechanisms of JAK2V617F Somatic Mutation Contributing Distinct Phenotypes in Myeloproliferative Neoplasms," Myeloproliferative Neoplasms. Int. J. Mol. Sci, vol. 2022, p. 1013, 2022, S. Smieszek, C. Tyner, A. Kaden, C. Johnson, C. Polymeropoulos, G. Birznieks, M. Polymeropoulos. Potential treatment for CMT2S caused by IGHMBP2 cryptic splice variant, with ASO based therapeutic [abstract]. Mov Disord. 2023; 38 (suppl 1). . About Vanda Pharmaceuticals Inc. Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit and follow us on X @vandapharma. About VGT-1849A VGT-1849A is an antisense oligonucleotide (ASO) that selectively targets JAK2, reducing increased activity of JAK2 that may cause hematologic malignancies. ASOs have broad applicability in addressing a number of disorders caused by genetic variants. CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS Various statements in this press release, including, but not limited to statements regarding the estimated prevalence of PV, the potential therapeutic effects of VGT-1849A, the timing of the initiation of clinical testing of VCA-894A and the potential benefits of VGT-1849A, are "forward-looking statements" under the securities laws. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, the accuracy of the reporting and diagnosis of PV cases, the ability of VGT-1849A to safely and effectively treat PV, Vanda's ability to begin clinical testing of VCA-894A during the specified timeframe and Vanda's ability to successfully complete the clinical development of, and obtain regulatory approval for, VGT-1849A in the treatment of PV. There can be no assurance that the actual results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the U.S. Securities and Exchange Commission, which are available at . All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Corporate Contact: Kevin Moran Senior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc. 202-734-3400 [email protected] Jim Golden / Jack Kelleher / Dan Moore Collected Strategies [email protected] SOURCE Vanda Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphan DrugOligonucleotide

03 Dec 2024

·www.prnewswire.com

Sobi's strength in haematology to be showcased at ASH 2024

STOCKHOLM, Dec. 3, 2024 /PRNewswire/ -- New data from Sobi® and partners will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA (USA) from the 7th - 10th of December 2024. During the meeting several analyses will be presented on haemophilia A, haemophagocytic lymphohistiocytosis (HLH), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and immune thrombocytopenia (ITP). "Haemophilia A has a lasting impact on joint health and quality of life. At ASH 2024, three oral presentations will reveal important new data about long-term outcomes with ALTUVOCT® prophylaxis," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We will also present new data spanning different severe and debilitating rare diseases, including an oral presentation on the real-world effectiveness of Doptelet® in treating people with ITP, demonstrating our broader commitment to advancing innovative treatments for people with rare haematological diseases. We look forward to sharing these findings in San Diego." Key data to be presented at ASH 2024 About ALTUVOCT® ALTUVOCT® (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. ALTUVOCT builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019. It is approved and marketed as ALTUVOCT by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also 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 Gamifant® Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Vonjo® Vonjo (pacritinib), a JAK-1 sparing JAK2/IRAK1/ACVR1 inhibitor, is approved 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 in the United States. This indication is approved under FDA accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Currently, a Phase 3 study (PACIFICA) is being conducted to assess pacritinib in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. About Aspaveli®/ Empaveli® 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. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the United States, European Union, and other countries globally. Currently, a Phase 3 trial (VALIANT) is being conduted to assess the efficacy and safety of pegcetacoplan in patients with C3G and primary IC-MPGN. Aspaveli/Empaveli is also under investigation for several other rare diseases across haematology and nephrology. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® Doptelet (avatrombopag) is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved in over 30 countries worldwide, including the EU and the US, for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100,000 adults per year with a prevalence of 9.5 per 100,000 adults. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 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: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalPhase 3ASHOrphan DrugLicense out/in

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	US	Swedish Orphan Biovitrum Ltd.	28 Feb 2022
Thrombocytopenia	US	Swedish Orphan Biovitrum Ltd.	28 Feb 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Post-essential thrombocythemia myelofibrosis	Phase 3	BE	CTI BioPharma Corp.	01 Jan 2013
Post-essential thrombocythemia myelofibrosis	Phase 3	NL	CTI BioPharma Corp.	01 Jan 2013
Post-essential thrombocythemia myelofibrosis	Phase 3	NZ	CTI BioPharma Corp.	01 Jan 2013
Post-polycythemia vera myelofibrosis	Phase 3	BE	CTI BioPharma Corp.	01 Jan 2013
Post-polycythemia vera myelofibrosis	Phase 3	NL	CTI BioPharma Corp.	01 Jan 2013
Post-polycythemia vera myelofibrosis	Phase 3	NZ	CTI BioPharma Corp.	01 Jan 2013
VEXAS syndrome	Phase 2	US	Swedish Orphan Biovitrum AB	01 May 2025
VEXAS syndrome	Phase 2	CA	Swedish Orphan Biovitrum AB	01 May 2025
VEXAS syndrome	Phase 2	FR	Swedish Orphan Biovitrum AB	01 May 2025
VEXAS syndrome	Phase 2	DE	Swedish Orphan Biovitrum AB	01 May 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04635059 (BLAST, CTgov)	Phase 2	Prostatic Cancer	6	Pacritinib	vyvjmudckc(ilbbpawxne) = aqdpgmkucs alvywfwixs (indmkkairi, qcghfqjvby - myhgdtxvnd) View more	-	17 Dec 2024
NCT01773187 \| NCT02055781 \| NCT04884191 (PERSIST-1, PERSIST-2, PAC203, ASH2024)	Phase 2/3	serum albumin \| cholesterol	484	Pacritinib 400 mg QD	bmhhcohtem(rfmmmphkgr) = qntjmdauan qtbdgcvtqa (acjjqcrcwv, -1.3 to +3.14)	Positive	08 Dec 2024
ASH2024	Not Applicable	Myelofibrosis	-	Pacritinib	mwhxqlahyr(bpzadnfmst) = miipjyzcow qxloctyxcw (bniiifgjvw, 63.2 - 99.0) View more	-	08 Dec 2024
SOHO2024	Not Applicable	Myelofibrosis	-	Pacritinib 200 mg twice daily	ugafnfemfm(iuntoyrhkk) = rcuyyfsads zavrahwtuv (epruvnjxup ) View more	-	04 Sep 2024
				Best Available Therapy (BAT)	ugafnfemfm(iuntoyrhkk) = vmpekafyxw zavrahwtuv (epruvnjxup ) View more
NCT02807207 (MPN-302, SOHO2024)	Phase 1	-	42	Moxifloxacin 400 mg	yvyxmlyepw(nhdfczhxwq) = 29.3% dnplexcokt (ukgoemyqxo ) View more	Positive	04 Sep 2024
SOHO2024	Not Applicable	Myelofibrosis	-	Pacritinib	qbabblscaa(rqrgpnvlce) = jabeuaiflm mvebkhawcu (ucvsrvelya, 7.9 - 10.3) View more	-	04 Sep 2024
NCT02055781 (PERSIST-2, SOHO2024)	Phase 3	Anemia \| Thrombocytopenia \| Myelofibrosis	-	Pacritinib 200 mg twice daily	ndxntcxmtz(minsfimgos) = uouypipxhp pplgwlslip (tssdhyyvsh ) View more	Positive	04 Sep 2024
				Best Available Therapy (BAT)	ndxntcxmtz(minsfimgos) = mpxdzxaznc pplgwlslip (tssdhyyvsh ) View more
NCT04404361 (PRE-VENT, CTgov)	Phase 2	COVID-19 \| Neoplasms	200	Pacritinib (Pacritinib and SOC)	rfvaetgaqe(myxdotvaqu) = jqqcwgolen nuojbazaio (jhmngxhhce, rdzzmyovss - pkrjeisamn) View more	-	05 Jun 2024
				Placebo (Placebo and SOC)	rfvaetgaqe(myxdotvaqu) = tknrxywmak nuojbazaio (jhmngxhhce, brujcogbis - jxbwbnovhu) View more
NCT02055781 (PERSIST-2, ASCO2024) Manual	Phase 3	Myelofibrosis	93	Pacritinib 200 mg twice daily	vudpxxbckc(kqffqehwpb) = shkdfctlzy rapxarzzae (hpcyvksdvy ) View more	Positive	24 May 2024
				Best Available Therapy (BAT)	vudpxxbckc(kqffqehwpb) = nphfpudnlv rapxarzzae (hpcyvksdvy ) View more
NCT02055781 (PERSIST-2, ASCO2024) Manual	Phase 3	Thrombocytopenia \| Myelofibrosis	92	Pacritinib 200 mg twice daily	zdmbjbrpxu(ztxmnmauct) = duixijhlov mnlvozilqg (pozfmetezq ) View more	Positive	24 May 2024
				Best Available Therapy (including Ruxolitinib)	zdmbjbrpxu(ztxmnmauct) = vndbqmjgpf mnlvozilqg (pozfmetezq ) View more

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