Delving into the Latest Updates on Forodesine Hydrochloride with Synapse

Phase I portion:
To confirm safety and tolerability in recurrent/refractory peripheral T-cell lymphoma patients during repeated oral administration of forodesine 300 mg twice daily (600 mg/day) for 28 days, and determine the recommended dose. Also, to evaluate pharmacokinetics.
Phase II portion:
To evaluate the efficacy, safety, and pharmacokinetics of the recommended dosage regimen determined in the phase I portion. The primary efficacy endpoint shall be objective response rate (ORR).

Start Date01 Jan 2013

Sponsor / Collaborator

Mundipharma KK

JPRN-JapicCTI-121985 / Unknown statusPhase 1/2

Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma

Start Date01 Nov 2012

Sponsor / Collaborator

Mundipharma KK

JPRN-jRCT2080221940 / Unknown statusPhase 1/2

Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma

Start Date12 Oct 2012

Sponsor / Collaborator

Mundipharma KK

100 Clinical Results associated with Forodesine Hydrochloride

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100 Translational Medicine associated with Forodesine Hydrochloride

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100 Patents (Medical) associated with Forodesine Hydrochloride

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215

Literatures (Medical) associated with Forodesine Hydrochloride

01 Dec 2024·Annals of Neurology

Guanylate Kinase 1 Deficiency: A Novel and Potentially Treatable Mitochondrial DNA Depletion/Deletions Disease

Article

Author: Saneto, Russell P. ; Tort, Frederic ; Dorschner, Michael O. ; Comi, Giacomo Pietro ; Shintaku, Jonathan ; Ronchi, Dario ; Hirano, Michio ; Marti, Ramon ; Sciacco, Monica ; Gort, Laura ; Hidalgo‐Gutierrez, Agustin ; Ramon, Javier ; Meneri, Megi ; Pesini, Alba ; Matas‐Garcia, Ana ; Gu, Yue ; Koganti, Lahari ; Barriocanal‐Casado, Eliana ; Milisenda, Jose Cesar ; Tadesse, Saba ; Garrabou, Gloria ; Horwitz, Marshall S. ; Ribes, Antonia ; Mansukhani, Mahesh ; Ugarteburu, Olatz ; Wang, Tian ; Tanji, Kurenai ; Wang, Shuang

ObjectiveMitochondrial DNA (mtDNA) depletion/deletions syndrome (MDDS) comprises a group of diseases caused by primary autosomal defects of mtDNA maintenance. Our objective was to study the etiology of MDDS in 4 patients who lack pathogenic variants in known genetic causes.MethodsWhole exome sequencing of the probands was performed to identify pathogenic variants. We validated the mitochondrial defect by analyzing mtDNA, mitochondrial dNTP pools, respiratory chain activities, and GUK1 activity. To confirm pathogenicity of GUK1 deficiency, we expressed 2 GUK1 isoforms in patient cells.ResultsWe identified biallelic GUK1 pathogenic variants in all 4 probands who presented with ptosis, ophthalmoparesis, and myopathic proximal limb weakness, as well as variable hepatopathy and altered T‐lymphocyte profiles. Muscle biopsies from all probands showed mtDNA depletion, deletions, or both, as well as reduced activities of mitochondrial respiratory chain enzymes. GUK1 encodes guanylate kinase, originally identified as a cytosolic enzyme. Long and short isoforms of GUK1 exist. We observed that the long isoform is intramitochondrial and the short is cytosolic. In probands’ fibroblasts, we noted decreased GUK1 activity causing unbalanced mitochondrial dNTP pools and mtDNA depletion in both replicating and quiescent fibroblasts indicating that GUK1 deficiency impairs de novo and salvage nucleotide pathways. Proband fibroblasts treated with deoxyguanosine and/or forodesine, a purine phosphatase inhibitor, ameliorated mtDNA depletion, indicating potential pharmacological therapies.InterpretationPrimary GUK1 deficiency is a new and potentially treatable cause of MDDS. The cytosolic isoform of GUK1 may contribute to the T‐lymphocyte abnormality, which has not been observed in other MDDS disorders. ANN NEUROL 2024;96:1209–1224

01 Nov 2024·Journal of NeuroInterventional Surgery

Two year follow-up of distal unruptured intracranial aneurysms treated with a surface modified flow diverter under prasugrel monotherapy

Article

Author: Abud, Thiago Giansante ; de Oliveira, Ricardo Santos ; Machado, João Paulo ; de Castro-Afonso, Luis Henrique ; Abud, Daniel Giansante ; Nakiri, Guilherme Seizem ; Monsignore, Lucas Moretti ; Colli, Benedicto Oscar ; Freitas, Rafael Kiyuze

BackgroundA new generation of modified surface flow diverters (FDs) and monotherapy using new antiplatelets may reduce both ischemic and hemorrhagic complications during the treatment of intracranial aneurysms. Previous preliminary safety analysis of distal unruptured intracranial aneurysms treated with the FD p48 MW HPC (phenox-Wallaby, Bochum, Germany) under antiplatelet monotherapy with prasugrel showed promising results. However, the long term outcomes of distal intracranial aneurysms treated with FDs under antiplatelet monotherapy are not known.MethodsThis was a single center, prospective, pivotal, open single arm study. The primary (safety) endpoint was absence of any new neurological deficits after treatment until the 24 month follow-up. The primary (efficacy) endpoint was the incidence of complete aneurysm occlusion 24 months after treatment. The secondary (efficacy) endpoints were any incidence of aneurysm dome reduction 24 months after treatment.Results21 patients harboring 27 distal aneurysms of the anterior circulation were included. No patient had neurologic deficits in the time from treatment to the 24 month follow-up. Complete aneurysm occlusion occurred in 20 (74%) of 27 aneurysms at the 24 month follow-up. Four aneurysms (14.8%) had dome reduction, and three aneurysms (11.1%) remained unchanged.ConclusionsIn this pilot trial, treatment of distal unruptured intracranial aneurysms with an FD under monotherapy with prasugrel, followed by monotherapy with aspirin, appeared to be safe and effective. Randomized studies with long term follow-up are needed to confirm these results.

01 Sep 2024·World Neurosurgery

Telescoping Flow Diverters for the Treatment of Brain Aneurysms: Indications and Outcome

Article

Author: Jabbour, Pascal M ; Momin, Arbaz A. ; Lan, Matthews ; Winiker, Sarah ; Musmar, Basel ; Teichner, Eric M ; Momin, Arbaz A ; Roy, Joanna M. ; Ghanem, Marc ; El Naamani, Kareem ; Gooch, Michael R. ; Tjoumakaris, Stavropoula I ; Rosenwasser, Robert H. ; Ghosh, Ritam ; Ahmed, Meah T. ; Gooch, Michael R ; Zarzour, Hekmat ; Schmidt, Richard F ; Teichner, Eric M. ; Rosenwasser, Robert H ; Schmidt, Richard F. ; Roy, Joanna M ; Ahmed, Meah T ; Jabbour, Pascal M. ; Tjoumakaris, Stavropoula I.

OBJECTIVEFew studies have reported the impact of telescoping flow diverters (FDs) in intracranial aneurysm treatment. Our study compared aneurysms treated using telescoping FDs to those treated with a single FD and identified predictors of telescoping.METHODSThis was a single-center retrospective review of a prospectively maintained database of aneurysms treated with FDs between 2011 and 2023. All patients who were treated with FDs for intracranial aneurysms were included in the study.RESULTSThe study comprised 750 patients with 750 aneurysms treated using 871 FDs. The study cohort was divided into 85 patients requiring telescoping FDs and 655 who did not. Rates of hemorrhage (7.1% vs. 1.8%, P < 0.001), symptomatic stroke (5.9% vs. 2.6, P < 0.001), and asymptomatic stroke (1.2% vs. 0.8%, P < 0.001) were significantly higher in the telescoping cohorts. At final follow-up, the rate of nonocclusion (9.8% vs. 5.1%, P = 0.029) and the rate of complete occlusion (88.5% vs. 81.1%, P = 0.029) were significantly higher in the telescoping cohort. On multivariate analysis, fusiform morphology (odds ratio [OR]: 2.4, 95% confidence interval [CI] 1.0-5.0, P = 0.03), increasing aneurysm height (OR: 1.0, 95% CI 1.0-1.1, P= 0.034), and the use of the Pipeline Embolization Device FD (OR: 2.4, 95% CI 1.3-4.4, P = 0.005) were independent predictors of telescoping.CONCLUSIONSAneurysms with fusiform morphology, increasing aneurysm height and those that underwent flow diversion using Pipeline Embolization Device had higher odds for telescoping. Significantly higher rates of angiographic occlusion with the use of telescoping FD add to the literature on its efficacy in treating aneurysms of varying morphology.

100 Deals associated with Forodesine Hydrochloride

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External Link

KEGG	Wiki	ATC	Drug Bank
D04245	Forodesine Hydrochloride	L01XX	DB06185

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Peripheral T-Cell Lymphoma	JP	Mundipharma KK	31 Mar 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cutaneous T-Cell Lymphoma	Phase 2	FR	BioCryst Pharmaceuticals, Inc.	01 Jul 2007
Cutaneous T-Cell Lymphoma	Phase 2	AU	BioCryst Pharmaceuticals, Inc.	01 Jul 2007
Cutaneous T-Cell Lymphoma	Phase 2	DE	BioCryst Pharmaceuticals, Inc.	01 Jul 2007
Recurrent T Lymphoblastic Lymphoma	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Jul 2006
Chronic Lymphocytic Leukemia	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Jun 2005
CD19-positive B-cell acute lymphoblastic leukemia	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Sep 2004
Precursor B-Cell Lymphoblastic Leukemia-Lymphoma	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Sep 2004
T-Cell Leukemia	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Mar 2004
T-Cell Leukemia	Phase 2	GE	BioCryst Pharmaceuticals, Inc.	01 Mar 2004
CD30-Positive recurrent or refractory Cutaneous T-Cell Lymphoma	Phase 2	US	BioCryst Pharmaceuticals, Inc.	01 Feb 2003

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Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01776411 (Pubmed \| Ann Hematol) Manual	Phase 2	Peripheral T-Cell Lymphoma Second line	48	forodesine	(nxjbqjcaxh) = ejvtfczgak rsshfvdgpk (sqkxdxpcvv, 14 - 38) View more	Positive	01 Jan 2019
NCT01776411 (ASCO2016)	Phase 1/2	T-cell lymphoma recurrent	44	Forodesine 300 mg BID	(jijbeyfdho) = gerexfrouk npddzjgrme (tgmywzfopl, 12.0 - 35.3) View more	Positive	20 May 2016
NCT00501735 (Pubmed \| Ann Oncol)	Phase 2	Cutaneous T-Cell Lymphoma \| Mycosis Fungoides \| Sezary Syndrome	144	Forodesine	(dqtjmkgzkd) = oeybqqcuwz unkxgzdhfe (tputrzefvp ) View more	-	01 Sep 2014
NCT00289549 (Pubmed \| Circulation)	Phase 2	Chronic Lymphocytic Leukemia	8	Oral forodesine 200 mg/d	lvzrfxtsxv(vjcuzfkxvz) = cdfladyohr svnsfayauv (czpgtcdpxc ) View more	-	12 Aug 2010
ASCO2009	Not Applicable	Cutaneous T-Cell Lymphoma	64	Forodesine	(gcltjuforz) = tizxjzjmve tvyehhhokh (pilynafnzs ) View more	-	20 May 2009