A 62-year-old man was diagnosed with JAK2 mutation positive essential thrombocythemia (ET) after developing cerebral infarction. He was treated with anagrelide and aspirin, with anagrelide increased to 5.5 mg/day, but platelet count remained around 700,000/µl. In July 2024, he presented to the emergency department with abdominal pain and vomiting. CT angiography showed a large gastric intramural hematoma as well as pseudoaneurysm from the right gastroepiploic artery. Following transcatheter arterial embolization, his symptoms improved, the hematoma decreased in size, and his condition stabilized. Gastric intramural hematoma is an extremely rare condition, often secondary to factors like anticoagulant therapy. Reports of cases diagnosed after resection of a suspected tumor highlight the importance of accurate diagnosis. Although thrombosis prevention is crucial in ET, anagrelide is associated with a higher risk of bleeding events than other therapies. To our knowledge, this is the first reported case of gastric intramural hematoma associated with ET and anagrelide.

01 Jan 2026·EXPERIMENTAL HEMATOLOGY

Cytoreductive treatment differentially affects platelet size and cytoskeletal megakaryocyte organization during thrombopoiesis in myeloproliferative neoplasms

Article

Author: Kamiya, Laureano J ; Goette, Nora P ; Marta, Rosana F ; Cellucci, Adela S ; Lev, Paola R ; Yañuk, Danila B ; Lira, María C ; Glembotsky, Ana C ; Heller, Paula G ; De Luca, Geraldine

Cytoreductive treatment is a main strategy to reduce thrombotic complications and ameliorate symptoms in Phi-negative myeloproliferative neoplasms (MPNs) comprising essential thrombocythemia, polycythemia vera, and primary myelofibrosis. Based on the observation of differences in platelet size during microscopic analysis of blood smears from MPN patients, in this work we studied whether these differences could be dependent on the type of cytoreductive drug used for patients' treatment and whether changes in platelet size could be induced by the effect of these drugs on thrombopoiesis. Maximum platelet diameter (MPD) was measured in 120 patients with MPN. The effect of drugs on thrombopoiesis was evaluated in normal megakaryocytes (MKs) obtained from cord blood-derived CD34+ hematopoietic progenitors. Anagrelide (ANA), α-interferon (IFN), and ruxolitinib (Ruxo) increased, whereas hydroxyurea (HU) decreased platelet size. MK incubation with these drugs revealed that ANA and IFN induced abnormal proplatelet (PP) architecture and affected microtubular structure, but only ANA altered actin organization, whereas neither Ruxo nor HU modified MK cytoskeleton. By bioinformatic analysis, RANTES downregulation was identified as a candidate responsible for ANA-induced abnormalities. RANTES downregulation was confirmed in MK incubated with ANA but not with IFN. Addition of recombinant RANTES reverted ANA-induced cytoskeletal abnormalities. Evaluation of RANTES plasmatic levels and platelet RNA expression in patients with MPN showed RANTES decrease in both samples during ANA treatment, suggesting that in vitro findings could reflect ANA action in vivo. In conclusion, this study demonstrates the influence of cytoreductive drugs on platelet size and reveals their differential mechanisms of action during platelet production.

31 Dec 2025·DRUG DELIVERY

Pharmacokinetic profile and in vivo anticancer efficacy of anagrelide administered subcutaneously in rodents

Article

Author: Salmikangas, Sami ; Schöffski, Patrick ; De Sutter, Luna ; Merikoski, Nanna ; Wozniak, Agnieszka ; Sihto, Harri ; Duan, Jianmin ; Maksimow, Mikael ; Wyns, Karo ; Böhling, Tom ; Lahtinen, Maria ; Toivanen, Kirsi

Anagrelide (ANA) is a phosphodiesterase 3A (PDE3A) inhibitor, commonly prescribed for essential thrombocythemia. It also functions as a molecular glue, inducing complex formation between PDE3A and Schlafen 12. This association either triggers apoptosis or inhibits proliferation in tumor cells, supporting its use in cancer therapy. Conventionally administered orally, ANA undergoes rapid metabolism and elimination, resulting in a short drug exposure time at the site of action. Here, we explored the pharmacokinetic profile of a subcutaneously (SC) injected ANA formulation in Sprague-Dawley rats by quantifying plasma ANA and metabolite concentrations using liquid-chromatography-tandem mass spectrometry. We further evaluated the in vivo tumor regression efficacy of orally and SC administered ANA in a patient-derived gastrointestinal stromal xenograft mouse model - UZLX-GIST2B - characterized by a KIT exon 9 driver mutation. The SC ANA exhibited extended-release plasma concentration-time profiles compared to intravenous and oral administrations. After a single administration in rats, plasma concentrations of ANA were detected up to 56 days later, and ANA metabolites up to 30 days later. The SC formulation also significantly reduced tumor volumes and demonstrated dose-dependent histological responses, nearly eradicating tumor tissue in 11 days with the highest dose. These findings suggest that the SC slow-release formulation maintains stable drug concentrations during treatment, potentially improving therapeutic efficacy at the target site.

News (Medical) associated with Anagrelide Hydrochloride

25 May 2026

·www.businesswire.com

PharmaEssentia to Present New Clinical Data Supporting the Use of Ropeginterferon Alfa-2b in Essential Thrombocythemia at ASCO and EHA 2026

PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced five upcoming presentations at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting and European Hematology Association (EHA) 2026 Congress. The ASCO annual meeting will be held May 29-June 2 in Chicago, and the EHA2026 Congress will be held June 11-14 in Stockholm, Sweden. PharmaEssentia will present new analyses from the Phase 3 SURPASS-ET study (NCT04285086) of ropeginterferon alfa-2b in patients with essential thrombocythemia (ET), including two-year outcomes comparing early versus delayed initiation (continuous treatment from baseline vs. initiation following prior anagrelide therapy) and post-hoc analyses of patients who transitioned from anagrelide. Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). The use of ropeginterferon alfa-2b-njft for ET is currently still investigational, but it is currently under review by FDA with a PDUFA goal date of August 30, 2026. Findings from SURPASS-ET demonstrate sustained hematologic control and progressive molecular improvement with longer interferon exposure, with deeper molecular responses and improved long-term disease control observed with earlier initiation. Patients who transitioned from anagrelide showed improved hematologic parameters over 12 weeks, supporting the feasibility of treatment transition. Importantly, estimated progression-free survival (PFS) at 24 months was 76.9% in patients who received ropeginterferon alfa-2b from baseline compared to 43.1% in those with delayed initiation. Together, these data support consideration of earlier ropeginterferon alfa-2b use in high-risk ET following hydroxyurea intolerance or resistance. PharmaEssentia will also present an integrated analysis of the SURPASS-ET and Phase 2b EXCEED-ET (NCT05482971) trials. The analysis includes 182 patients across both studies and evaluates the consistency of clinical benefit across treatment lines, including treatment-naïve and previously treated populations, as well as across molecular subtypes and ethnicities. Results demonstrated clinically meaningful hematologic and molecular responses in both treatment-naïve and pretreated patients. Efficacy was consistent across driver mutation subtypes, and the presence of additional non-driver mutations did not adversely impact outcomes. “These findings represent an important step forward in understanding the potential use of interferon-based approaches in essential thrombocythemia,” said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Advocate Health Cancer National Service Line. “Together, these data continue to build on the growing body of evidence supporting the use of ropeginterferon alfa-2b in ET and reinforce its potential role as a meaningful treatment option for patients.” In addition, PharmaEssentia will present real-world data evaluating the association between neutrophil-to-lymphocyte ratio (NLR) and thrombotic risk in 11,809 U.S. veterans with PV. An online-only abstract will also report findings from a meta-analysis evaluating dosing strategies for ropeginterferon alfa-2b in PV, suggesting that a higher initial dose with accelerated titration (HIDAT) may lead to improved early hematologic and molecular response rates compared to lower-dose currently approved regimens. Poster Presentations Title: Integrated Analysis of the Ropeginterferon alfa-2b Clinical Program in Essential Thrombocythemia to Demonstrate Molecular and Hematologic Responses with Safety Profile Across Treatment Lines, Ethnicities, and Driver Mutation Types Abstract Number: 6576 Poster: 369 Presenter: Ruben Mesa, M.D. Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant Date: June 1st Time: 9-12 pm CDT Title: Association of neutrophil‑to‑lymphocyte ratio and thrombotic events in US Veterans with Polycythemia vera Abstract Number: 6578 Poster: 371 Presenter: Ying Wang, PhD Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant Date: June 1st Time: 9-12 pm CDT Published Abstract Title: Effect of Higher Initial Dose and Accelerated Titration of Ropeginterferon Alfa-2b on Early Hematologic and Molecular Responses in Polycythemia Vera: A Meta-Analysis Abstract Number: e18592 EHA Oral Presentation Title: Early versus Delayed Initiation of Ropeginterferon Alfa-2b in High-Risk Essential Thrombocythaemia: Two-Year Results from the Phase 3 SURPASS-ET Study Abstract Number: S219 Presenter: Harry Gill, M.D. Session: Myeloproliferative neoplasms - Clinical Date: June 13th Time: 5:15 – 6:30 pm CEST Poster Presentations Title: Ropeginterferon alfa-2b Demonstrates Molecular and Hematologic Responses with a Favorable Safety Profile in Essential Thrombocythemia Abstract Number: PS1986 Presenter: Harry Gill, M.D. Session: Myeloproliferative neoplasms - Clinical Date: June 13th Time: 6:45 – 7:45 pm CEST Title: Hematologic Control and Improved Safety Following Switch from Anagrelide to Ropeginterferon Alfa-2b in Patients with Essential Thrombocythemia: 12-Week Pre-/Post-Switch Analysis from the SURPASS-ET study Abstract Number: PF912 Presenter: Lucia Masarova, M.D. Session: Myeloproliferative neoplasms - Clinical Date: June 12th Time: 6:45 – 7:45 pm CEST PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation, headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator and the developer and owner of BESREMi® (ropeginterferon alfa-2b-njft). Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. Essential thrombocythemia is a rare blood disorder and type of myeloproliferative neoplasm (MPN). It is characterized by the bone marrow overproducing platelets. Patients with ET are at an increased risk of blood clots, abnormal bleeding and enlarged spleens. ET is often caused by genetic mutations such as a JAK2 genetic mutation. Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). The Company plans to seek a ropeginterferon alfa-2b-njft label expansion to include ET and has submitted a sBLA with the U.S. FDA. BESREMi® holds orphan drug designation in the United States for the treatment of polycythemia vera (PV) in adults. It has received regulatory approval in over 40 countries, including from the European Medicines Agency (2019), the U.S. Food and Drug Administration (2021), and the Pharmaceuticals and Medical Devices Agency in Japan (2023). The product was developed by PharmaEssentia. PharmaEssentia retains full global intellectual property rights across all indications. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug ApprovalOrphan Drug

10 Feb 2026

·www.fiercepharma.com

Taiwan’s PharmaEssentia to build $46M manufacturing plant in Puerto Rico

PharmaEssentia will spend $46 million to build a manufacturing facility in Puerto Rico. The move forms part of the company's global expansion strategy to support demand for the rare blood disease treatment Besremi. PharmaEssentia will spend $46 million to build a new manufacturing facility in Puerto Rico. The move forms part of the Taiwanese biotech’s global production expansion strategy, with the goal that the plant will eventually support the supply of PharmaEssentia's rare blood disease treatment, Besremi.The project is expected to provide PharmaEssentia with enhanced supply security, operational flexibility, cost efficiencies and scalable manufacturing capacity for the future, the company said in a Feb. 9 press release. In its first phase, the plant is expected to create nearly 100 jobs, with room for the site's workforce to eventually grow in line with further scale-up projects, a company spokesperson told Fierce.The 40,000-square-foot facility will incorporate robust redundancy and contingency measures—like backup generation and infrastructure safeguards—to ensure uninterrupted operations and product supply, the spokesperson explained. “Expanding manufacturing capacity outside Taiwan is a core strategic priority for PharmaEssentia as we continue to strengthen supply resilience, scalability, and geographic diversification,” Ko-Chung Lin, Ph.D., PharmaEssentia’s founder and CEO, said in a statement. “We are proud to take this important step toward establishing U.S.-based manufacturing, with Puerto Rico serving as a future center for the U.S. market.”In January, the biotech unveiled plans to potentially expand the reach of its FDA-approved Besremi to another rare blood disorder after reviewing encouraging results from a phase 3 study.The company last month said it would head to regulators soon with data from its late-stage SURPASS-ET trial. In the study, which pitted Besremi against platelet-reducing anagrelide as a second-line therapy for patients with essential thrombocythemia (ET), PharmaEssentia's treatment was linked to durable clinical responses and also met a secondary endpoint concerning reductions in JAK2 V617F allelic burden.ET is a rare blood cancer typically caused by genetic mutations that cause bone marrow to produce too many platelets.The FDA approved Besremi in 2021 as the first polycythemia vera treatment that patients can take regardless of their treatment history, as well as the first interferon drug approved for the disorder.

Drug ApprovalPhase 3Clinical Result

12 Jan 2026

·www.biospace.com

PharmaEssentia Announces Positive Topline Phase 2b Data from EXCEED-ET Study Evaluating Ropeginterferon Alfa-2b-njft for Essential Thrombocythemia

EXCEED-ET Study Results Demonstrate Significant Efficacy, with Consistent Treatment Response Across the Overall Essential Thrombocythemia Population Results Extend Previously Reported Positive Phase 3 SURPASS-ET Study Data, Demonstrating Superior Efficacy Compared with Anagrelide in Hydroxyurea-Resistant or Intolerant Essential Thrombocythemia with Leukocytosis FDA Review Ongoing of Biologics License Application (sBLA) to Support Ropeginterferon alfa-2b (BESREMi ® ) Label Expansion BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced positive topline results from its Phase 2b EXCEED-ET clinical trial of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia (ET). The EXCEED-ET study (NCT05482971) is a North America–based, single-arm, Phase 2b clinical trial that serves as supportive and confirmatory evidence further substantiating the scientific rationale for ropeginterferon alfa-2b-njft’s efficacy in ET. The trial enrolled 91 patients with ET, including 24 patients previously treated with hydroxyurea (HU) and 67 treatment-naïve patients, providing a broad and representative dataset across key ET patient subgroups. The trial results demonstrated a consistent efficacy and treatment response across the overall ET population, with stronger results observed in treatment-naïve patients. Based on the overall ET population group (intent-to-treat), the response rate at months 10 and 13 was approximately 60.2%. A modified European LeukemiaNet (ELN) response was defined as meeting all three of the following criteria, with individual response rates as follows: Reduction in peripheral blood counts (Platelets ≤400 × 10⁹/L and WBC 40% of patients were influenza-like illness, arthralgia, fatigue, pruritus, nasopharyngitis, and musculoskeletal pain. Drug Interactions Patients on BESREMi ® who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification. Avoid use with myelosuppressive agents, narcotics, hypnotics, or sedatives, and monitor patients receiving the combination for effects of excessive CNS toxicity. Please see full Prescribing Information , including Boxed Warning. About PharmaEssentia PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation, headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website , LinkedIn or X (formerly Twitter). Contacts Media Contact Muriel Huang Director, Investor Relations and Corporate Communication muriel_huang@pharmaessentia.com

Clinical ResultPhase 2

100 Deals associated with Anagrelide Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
-	Anagrelide Hydrochloride	L01XX35	DB00261

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Indication	Country/Location	Organization	Date
Thrombocythemia, Essential	European Union	Takeda Pharmaceuticals International AG	15 Nov 2004
Thrombocythemia, Essential	Iceland	Takeda Pharmaceuticals International AG	15 Nov 2004
Thrombocythemia, Essential	Liechtenstein	Takeda Pharmaceuticals International AG	15 Nov 2004
Thrombocythemia, Essential	Norway	Takeda Pharmaceuticals International AG	15 Nov 2004
Thrombocytosis	United States	Takeda Pharmaceuticals U.S.A., Inc.	14 Mar 1997

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Indication	Highest Phase	Country/Location	Organization	Date
Neoplasms	NDA/BLA	Canada	Septa Pharmaceuticals, Inc.	01 Jan 2026

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


SURPASS-ET (EHA2025)	Phase 3	Thrombocythemia, Essential Second line	174	Ropeginterferon alfa-2b	deifblobzb(kbdhrueaht) = bveijqkrnp nznmakhitz (kdhqcnpsco ) View more	Positive	14 May 2025
SURPASS-ET (EHA2025)	Phase 3	Thrombocythemia, Essential Second line	174	Anagrelide	deifblobzb(kbdhrueaht) = spvmorxfmo nznmakhitz (kdhqcnpsco ) View more	Positive	14 May 2025
NCT03866434 (CTgov)	Phase 1	-	20	Omeprazole+SPD422	eywcfiduth = vwgxfiamsf rqofckacac (ahhxvulbcl, kalxapiuel - zbbpruxctj) View more	-	24 Apr 2020
EHA2019	Not Applicable	First line	53	Anagrelide	vbctrpysgq(waflkdzysk) = dsgmofyapv cpuxmhacpp (hsxcwzbjcc ) View more	-	16 May 2019
NCT01467661 (Pubmed \| Int J Hematol) Manual	Phase 3	Thrombocythemia, Essential	53	anagrelide	hjasykbnwa(xawhjfyzgi) = 1088.3 × 10^9/L to 473.5 × 10^9/L mixgmfalyv (uglvfuqkfr )	Positive	01 Nov 2018
NCT00202644 (CTgov)	Phase 4	Thrombocythemia, Essential	150	Anagrelide (Anagrelide)	xzgfutvjos(dkpumqopvz) = shcmsfkhiz hpfleqfrsl (fqfhyquhwu, 4.81) View more	-	24 Jan 2018
NCT00202644 (CTgov)	Phase 4	Thrombocythemia, Essential	150	Hydroxyurea (Hydroxyurea)	xzgfutvjos(dkpumqopvz) = icwtqvuvoi hpfleqfrsl (fqfhyquhwu, 4.59) View more	-	24 Jan 2018
NCT01467661 (CTgov)	Phase 3	Thrombocythemia, Essential	41	SPD422	ngxezccron(uadomreofe) = ydrmreqweo vnintgkylj (yorbeohvas, 433.14) View more	-	08 Jun 2016
EHA2015	Phase 2	Myeloproliferative Disorders \| Thrombocytosis	18	Anagrelide CR	zivkfyfafu(vtjnlxcjxl) = dyfkpodovd annbfllvkr (zojxmtajas )	-	21 May 2015
Pubmed \| Int J Hematol	Phase 3	Thrombocythemia, Essential Second line	53	Anagrelide	jjvsnxepik(maomlkathq) = ubbvantxgp rmwerzqnwn (lhrjgacesg ) View more	-	01 Oct 2014
NCT01552928 (CTgov)	Phase 1	-	60	Placebo	yjisxsntcz(uracabfmxv) = vrpdqemnih jpnoqtsdjy (lbqkhymgst, wkruvftsch - xgatnezozf) View more	-	10 Jan 2014
NCT01214915 (CTgov)	Phase 3	Thrombocythemia, Essential	53	Anagrelide Hydrochloride	xxksmmduby = alapzyylpq nzizmuiogd (nmtqqfsadw, sbsmugnxrt - aawdasobue) View more	-	25 Sep 2013

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