Delving into the Latest Updates on Egaptivon pegol with Synapse

Overview

Basic Info

Drug Type

DNA aptamer

Synonyms

Egaptivon pegol (USAN)

Target

vWF

Mechanism

vWF inhibitors(Von willebrand factor inhibitors)

Therapeutic Areas

Immune System DiseasesNervous System DiseasesCardiovascular Diseases+ [3]

Active Indication-

Inactive Indication

Brain InfarctionCarotid StenosisIntracranial Embolism+ [3]

Originator Organization

Archemix Corp.

Active Organization-

Inactive Organization

Archemix Corp.

Drug Highest PhaseDiscontinuedPhase 2

First Approval Date-

Regulation-

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Structure

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Gene Sequence

Sequence Code 271898487

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The purpose of this study is to determine, in patients undergoing carotid endarterectomy, the effect of ARC1779 Injection on the number of microembolic signals detected by transcranial Doppler immediately after surgery. This study will also evaluate the safety of ARC1779 Injection with respect to bleeding risk in patients in the peri-operative (during surgery) period.

Start Date01 Feb 2009

Sponsor / Collaborator

Archemix Corp.

[+1]

EUCTR2008-001872-54-IT / Not yet recruitingNot Applicable

A Randomized, Double-blind, Placebocontrolled, Clinical Outcome Study of ARC1779 Injection in Patients with Thrombotic Microangiopathy - ND

Start Date28 Jan 2009

Sponsor / Collaborator

Archemix Corp.

100 Clinical Results associated with Egaptivon pegol

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100 Translational Medicine associated with Egaptivon pegol

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100 Patents (Medical) associated with Egaptivon pegol

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23

Literatures (Medical) associated with Egaptivon pegol

01 Jul 2024·Seminars in Thrombosis and Hemostasis

Progress in von Willebrand Disease Treatment: Evolution towards Newer Therapies

Review

Author: Jilma, Bernd ; Moser, Miriam M. ; Schoergenhofer, Christian

Abstractvon Willebrand disease (VWD) is a very heterogenous disease, resulting in different phenotypes and different degrees of bleeding severity. Established therapies (i.e., desmopressin, antifibrinolytic agents, hormone therapy for heavy menstrual bleeding, and von Willebrand factor [VWF] concentrates) may work in some subtypes, but not in all patients. In recent years, progress has been made in improving the diagnosis of VWD subtypes, allowing for more specific therapy. The impact of VWD on women's daily lives has also come to the fore in recent years, with hormone therapy, tranexamic acid, or recombinant VWF as treatment options. New treatment approaches, including the replacement of lacking factor VIII (FVIII) function, may work in those subgroups affected by severe FVIII deficiency. Reducing the clearance of VWF is an alternative treatment pathway; for example, rondaptivon pegol is a VWFA1 domain-binding aptamer which not only improves plasma VWF/FVIII levels, but also corrects platelet counts in thrombocytopenic type 2B VWD patients. These approaches are currently in clinical development, which will be the focus of this review. In addition, half-life extension methods are also important for the improvement of patients' quality of life. Targeting specific mutations may further lead to personalized treatments in the future. Finally, a few randomized controlled trials, although relatively small, have been published in recent years, aiming to achieve a higher level of evidence in future guidelines.

01 Jan 2022·[Rinsho ketsueki] The Japanese journal of clinical hematology

[Development of a novel aptamer blocking the interaction between the VWF A1 domain and platelet GP Ib for the treatment of arterial thrombosis].

Article

Author: Harada, Kaori ; Matsumoto, Masanori

A DNA aptamer to the von Willebrand factor (VWF) A1 domain, TAGX-0004, inhibits the binding of VWF to platelets. Nucleic acid aptamers are single-stranded DNA or RNA molecule forming three-dimensional structures that are capable of specifically binding to proteins and are expected to contribute to alternative medicine. ARC1779, an aptamer targeting the VWF A1 domain, had been evaluated in a phase II clinical trial of patients with acquired thrombotic thrombocytopenic purpura (aTTP); however, its development was terminated. Caplacizumab, an anti-VWF A1 domain nanobody, is now increasingly employed as first-line therapy for the treatment of aTTP in Western countries. However, there have been reports regarding adverse bleeding events and the high cost of the treatment. In this study, the inhibitory effects of TAGX-0004 were compared with those of ARC1779 and caplacizumab on in vitro platelet aggregation and thrombus formation. TAGX-0004 had an excellent high affinity to the VWF A1 domain and superior efficacy, such as its potent inhibitory activity in in vitro platelet aggregation and thrombus formation. Therefore, it can potentially overcome the problems associated with caplacizumab and can be developed as a promising drug not only for aTTP treatment but also for the treatment of the various VWF-mediated thrombotic disorders.

01 Feb 2021·Biochemistry (Moscow)Q4 · BIOLOGY

The Use of Fluorescently Labeled ARC1779 Aptamer for Assessing the Effect of H2O2 on von Willebrand Factor Exocytosis

Q4 · BIOLOGY

Article

Author: Goncharov, Nikolay V ; Tsitrina, Aleksandra A ; Avdonin, Pavel V ; Avdonin, Piotr P ; Trufanov, Sergey K ; Rybakova, Elena Yu

AbstractHere, we propose a new approach for quantitative estimation of von Willebrand factor (vWF) exposed on the surface of endothelial cells (ECs) using the ARC1779 aptamer that interacts with the vWF A1 domain. To visualize complex formation between vWF and the aptamer, the latter was conjugated with the Cy5 fluorescent label. Cultured human umbilical vein endothelial cells (HUVEC) were stained with the ARC1779-Cy5 conjugate and imaged with a fluorescence microscope. The images were analyzed with the CellProfiler software. vWF released from the Weibel–Palade bodies was observed as bright dot-like structures of round and irregular shape, the number of which increased several times after HUVEC exposure to histamine or thrombin. Staining with ARC1779-Cy5 also revealed long filamentous vWF structures on the surface of activated HUVEC. vWF secretion by ECs is activated by the second messengers cAMP and Ca2+. There is evidence that hydrogen peroxide also acts as a second messenger in ECs. In addition, exogenous H2O2formed in leukocytes can enter ECs. The aim of our study was to determine the effect of H2O2on the vWF exposure at the surface of HUVEC using the proposed method. It was shown that hydrogen peroxide at concentration 100 µM, which is lower than the cytotoxicity threshold of H2O2for cultured HUVEC, increased several times the number of dot-like structures and total amount of vWF exposed on plasma membrane of HUVEC, which suggest that H2O2acts as a mediator that activates exocytosis of Weibel–Palade bodies and vWF secretion in the vascular endothelium during inflammation and upon elevated generation of endogenous reactive oxygen species in ECs.

100 Deals associated with Egaptivon pegol

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External Link

KEGG	Wiki	ATC	Drug Bank
D09892	-	-	DB05202

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Brain Infarction	Phase 2	US	Archemix Corp.	01 Feb 2009
Brain Infarction	Phase 2	GB	Archemix Corp.	01 Feb 2009
Carotid Stenosis	Phase 2	GB	Archemix Corp.	01 Feb 2009
Carotid Stenosis	Phase 2	US	Archemix Corp.	01 Feb 2009
Intracranial Embolism	Phase 2	US	Archemix Corp.	01 Feb 2009
Intracranial Embolism	Phase 2	GB	Archemix Corp.	01 Feb 2009
Purpura, Thrombotic Thrombocytopenic	Phase 2	AT	Archemix Corp.	01 Jan 2008
Von Willebrand Disease, Type 2	Phase 2	AT	Archemix Corp.	01 Jan 2008
Von Willebrand Disease, Type 2	Phase 2	AT	Archemix Corp.	01 Jan 2008
Thrombosis	Phase 2	US	Archemix Corp.	01 Dec 2006

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00742612 (Pubmed \| Cochrane Database Syst Rev)	Phase 1	-	-	ARC1779	kjhyayoiaa(prscjxalaq) = arkiirvyoj owqxaromxm (jkioaxstbj )	-	01 Aug 2011
NCT00742612 (Pubmed \| Cochrane Database Syst Rev)	Phase 1	-	-	Placebo	kjhyayoiaa(prscjxalaq) = gukhtcnxee owqxaromxm (jkioaxstbj )	-	01 Aug 2011
NCT00432770 (Pubmed \| J Am Coll Cardiol)	Phase 1	-	47	ARC1779	dxmhtigyhw(vaescqimzo) = ukffpmyews zlxsgfmqys (mllkcueesa ) View more	-	04 Dec 2007