Delving into the Latest Updates on Pentamidine Isethionate with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

1,5-bis(4-amidinophenoxy)pentane, 4,4'-(1,5-pentanediylbis(oxy))bis-benzenecarboximidamide, 4,4'-(pentamethylenedioxy)dibenzamidine

+ [19]

Target-

Action-

Mechanism-

Therapeutic Areas

Infectious DiseasesRespiratory DiseasesOther Diseases+ [1]

Active Indication

Pneumonia, PneumocystisMyocardial Infarction

Inactive Indication

Advanced Pancreatic AdenocarcinomaHepatocellular CarcinomaMetastatic colon cancer+ [3]

Originator Organization

Fresenius Kabi AG

Active Organization

Fresenius Kabi USA LLC

Medical College of WisconsinSanofi KK

Inactive Organization

Verlyx Pharma, Inc.

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (16 Oct 1984),

Pneumonia, Pneumocystis

RegulationOrphan Drug (United States)

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Structure/Sequence

Molecular FormulaC23H36N4O10S2

InChIKeyYBVNFKZSMZGRAD-UHFFFAOYSA-N

CAS Registry140-64-7

The usual first treatment for Pneumocystis jirovecii pneumonia (PCP) is an antibiotic called trimethoprim-sulfamethoxazole (TMP-SMX). However, some patients cannot take this medication because of allergies, side effects, or lack of response.
This study asks the question:
When TMP-SMX cannot be used, which alternative treatment for PCP provides the best balance of effectiveness and safety?

Start Date01 Mar 2026

Sponsor / Collaborator-

NCT03730363

/ CompletedPhase 1IIT

A Phase I Study of Pentamidine in Combination With Salvage Chemotherapy for Relapsed/Refractory Classical Hodgkin Lymphoma

Primary Objective:
To evaluate dose limiting toxicity and to determine the recommended phase 2 dose (RP2D) of pentamidine in combination with salvage chemotherapy with ifosfamide, carboplatin and etoposide (ICE) on a 3-weeks schedule in relapsed/refractory classical Hodgkin lymphoma (cHL).
Secondary Objective:
* To estimate the overall best treatment response at 5- and 16-weeks from study enrollment. Although the clinical benefit of these drugs in combination has not been established, offering this treatment may provide a therapeutic benefit. The patients will be carefully monitored for tumor response and symptom relief, in addition to safety and tolerability.
* To estimate the duration of response to the proposed combined therapy.
* To measure the protein of regenerating liver-3 (PRL-3) level of expression in patients at time of relapse.
* To measure circulating biomarkers of response (soluble CD30 (sCD30), and thymus and activation-related chemokine (TARC)) in serum samples collected throughout treatment and inhibition of (pSTAT, pAKT) in peripheral blood mononucleated cells (PBMC).
Exploratory Objective:
* To measure cell-free messenger RNA (cfmRNA) in peripheral blood.
* To measure cell-free DNA in peripheral blood

Start Date19 Dec 2018

Sponsor / Collaborator

University of Kentucky

NCT03403621

/ TerminatedPhase 1/2IIT

Double Blind, Single-Center, Randomized, Within Subject Placebo, Phase I Study Evaluating the Effects of Novel Topical Gel in Prevention of Hypertrophic Scar Formation

Researchers are trying to find out more about the side effects of topical (applied to the skin) Pentamidine, to determine if it is safe for use in people. They also want to find out if topical use of Pentamidine can help treat hypertrophic scars.
Pentamidine is a medicine that is currently used to treat certain kinds of infection. It is most often given by intravenous (into a vein) or inhalation (through a breathing device). This medication is approved by the U.S. Food and Drug Administration (FDA) for use in these forms.
Everyone in this study will receive topical Pentamidine (TP) in a silicone based gel (PCCA Pracasil Plus). Topical treatment of Pentamidine is still experimental and has not been formally tested for safety or effectiveness in a randomized control trial within the United States. The FDA has allowed the use of topical Pentamidine in this research study.

Start Date05 Mar 2018

Sponsor / Collaborator

Mayo Clinic

100 Clinical Results associated with Pentamidine Isethionate

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100 Translational Medicine associated with Pentamidine Isethionate

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100 Patents (Medical) associated with Pentamidine Isethionate

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2,919

Literatures (Medical) associated with Pentamidine Isethionate

01 Feb 2026·CARDIOVASCULAR DRUGS AND THERAPY

Mitigation of Injury from Myocardial Infarction by Pentamidine, an Inhibitor of the Acetyltransferase Tip60

Article

Author: Lough, John W ; Wan, Tina C ; Wang, Xinrui ; Auchampach, John A ; Qu, Zhuocheng ; Harty, Katherine R ; Smith, Brian C

PURPOSE:

There is an urgent unmet need for new pharmacologic approaches that promote re-muscularization and repair following myocardial infarction (MI). We previously reported that genetic depletion of the acetyltransferase Tip60 after MI in a mouse model activates the CM cell-cycle, reduces scarring, and restores cardiac function, and that these beneficial effects are mimicked by the Tip60-selective inhibitor TH1834. Here, we investigated whether the FDA-approved anti-microbial agent pentamidine, a Tip60 inhibitor from which TH1834 is derived, also protects from the damaging effects of MI.

METHODS:

Adult (10-14 weeks old) C57Bl/6 mice were subjected to permanent left coronary artery ligation to induce MI. Subsequently, echocardiography, electrocardiography, cardiac staining, and molecular analyses were performed to monitor the effects of treatment with pentamidine on cardiac injury and function.

RESULTS:

We report that transient systemic administration of pentamidine on days 3-16 post-MI at a daily dose of 3 mg/kg efficiently improved cardiac function for up to ten months. This was accompanied by improved survival, diminished scarring, and increased activation of cell-cycle markers in CMs located in the infarct border zone in the absence of hypertrophy. Histological assessments suggested that post-MI treatment with pentamidine reduced site-specific acetylation of the minor histone variant H2A.Z at lysines K4 and K7 in CMs, indicative of the dedifferentiation process which must occur prior to CM proliferation. Treating mice with pentamidine post-MI produced no prominent electrophysiological changes.

CONCLUSIONS:

These findings support the translational potential of pentamidine for treatment of MI, and provide evidence that functional improvement is mediated, in part, by CM renewal due to inhibition of the acetyltransferase activity of Tip60.

07 Jan 2026·ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Drug susceptibility of a clinical isolate of Balamuthia mandrillaris , a pathogenic free-living amoeba

Article

Author: Kobpornchai, Porntida ; Rice, Christopher A. ; Kulkeaw, Kasem ; Tongkrajang, Nongnat ; Dubey, Pratima ; Lu, Chenyang ; Chaiyaloom, Suwipa

ABSTRACT:

Balamuthia amoebic encephalitis (BAE) is a highly fatal infection caused by Balamuthia mandrillaris , an amoeba that lives in soil and water. In Thailand, three fatal cases of BAE have been documented, but no survivors have been reported, raising questions about current treatment regimens. Previous drug repurposing studies reveal some potent pharmacological compounds, but the drug susceptibility of the clinical isolate of pathogenic amoeba remains variable. Given the success in isolating B. mandrillaris from the human biopsied brain, this study aims to assess the amoebicidal effect of several previously repurposed drugs and suggested therapies for BAE. The trophozoites of a new clinical isolate, the KM-20 strain, were exposed to 12 compounds, including pentamidine, the most widely used antiprotozoal drug, and nitroxoline, the recent radical cure for BAE. The amoebicidal effect was assessed using the ATP level as a cell survival biomarker. The circularity and surface area of the cells were used as recrudescence indicators. Among all drugs tested, nitroxoline is the most potent amoebicidal drug without recrudescence. Topical antiseptic agents caused amoeba lysis at all doses tested, suggesting potential use for cutaneous balamuthiasis. Compared with two laboratory-adapted V039 and PRA-291 strains, the KM-20 isolate had reduced drug susceptibility to all of the tested compounds, suggesting strain dependency of amoebicidal activity. This study provides drug susceptibility data against a novel and geographically diverse clinical isolate of B. mandrillaris to assist in prioritizing anti- Balamuthia agents for further drug development testing, followed by in vivo efficacy testing animal models before clinical trials and drug repurposing.

23 Dec 2025·ACS Omega

Quantification of Interactions between Small Molecules and RNA Probes Representative of Myotonic Dystrophy Type 1 Using Affinity Capillary Electrophoresis and UPLC-UV

Article

Author: Lefebvre, Victor ; Blankert, Bertrand ; Beukens, Delphine ; Hambye, Stéphanie ; Wells, Mathilde ; Leveque, Mathieu

Myotonic dystrophy type 1 (OMIM #160900) is a multisystemic, autosomal, and dominantly inherited pathology. It is characterized by an expansion (>50) of trinucleotides [CTG] _n on the 3' untranslated region of the dystrophia myotonica protein kinase gene, transcribed into RNA [CUG] _n , aggregating in the nucleus as foci. The most accepted pathological mechanism considers the sequestration and dysregulation of proteins, including splicing factors (muscle-blind-like splicing regulator 1 and CUG-binding protein 1) by pathological RNA. Different therapeutic strategies to overcome this defect exist, such as the use of small molecules targeting the pathological RNA to prevent sequestration. Among these small molecules, pentamidine (PTMD), an antiprotozoal drug, has previously been shown to interact with a [CUG] _n repeat. In this context, we developed a fine-tuned affinity capillary electrophoresis (ACE) method that provides highly repeatable migration times. This is a crucial point, as all subsequent calculations for apparent affinity constant (Ka_app ) determination rely on them. Afterward, we quantified the interactions between pentamidine and two RNA probes: [CUG]₉₅, representative of the disease, and [CUG]₁₄ as a negative control. The results indicated an excellent affinity between [CUG]₉₅ and PTMD. Selectivity was assessed by comparing the Ka_app values: (6.1 ± 0.4) × 10³ M^-1 and (3.8 ± 0.3) × 10³ M^-1 for the positive and negative controls, respectively. For the first time, an orthogonal UPLC-UV methodology was applied to corroborate the ACE results. No significant differences were observed between the two analytical methods. To overcome the documented toxicity related to pentamidine, different libraries of small molecules have been investigated by the ACE method. Among these, two compounds, neomycin and chloroquine, demonstrated interactions with the pathological RNA model. Therefore, their affinities toward the positive and negative controls were quantified to assess the selectivity of these potential therapeutic candidates.

1

News (Medical) associated with Pentamidine Isethionate

07 Mar 2024

·www.businesswire.com

Montdorex Announces Acceptance of Its Abstract for Presentation at the AACR Annual Meeting 2024

MONTREAL--( BUSINESS WIRE )--Montdorex Inc. (“Montdorex” or “the Company”), a privately-owned precision medicine company, today announced that Terry Chow, Ph.D., Founder and CEO will present insights on the relevance of inhibiting the endo-exonuclease (EE) enzyme in a poster presentation at the American Association for Cancer Research (AACR) Annual Meeting, to be held in San Diego, CA, April 5-10, 2024. The endo-exonuclease enzyme plays a key role in DNA damage repair mechanisms, but it is also overexpressed in cancer tumors. In a proof-of-concept study, Montdorex showed that tumors with increased EE expression showed the best response to pentamidine, an endo-exonuclease inhibitor. The Company has also identified mono- and di-amidine analogs of pentamidine that are more effective than the parent drug in both in vitro and in vivo studies. “Our lead candidate, MTDX203, showed an increased anti-cancer activity with a higher safety index than pentamidine in preclinical animal studies,” said Terry Chow of Montdorex. “I look forward to presenting our detailed findings to the AACR scientific and investor community at the upcoming annual meeting.” Details of the AACR poster presentation are as follows: Poster Presentation (#5604): Modulating DNA repair through endo-exonuclease inhibition: a new therapeutic paradigm in Oncology [ link to the abstract ] Session Category: Molecular/Cellular Biology and Genetics Session Title: DNA Damage and Repair 2 Session Date and Time: Tuesday, April 9, 2024 1:30 PM – 5:00 PM (PT) Location: Poster Section 14, Poster Board Number 2 About Montdorex Based in Montreal, Canada, Montdorex Inc. is a privately-owned, early-stage precision medicine company, targeting the endo-exonuclease (EE) enzyme involved in cancer tumor growth and inflammatory processes. The company is developing proprietary mono-amidine and di-amidine EE inhibitors to modulate DNA repair in oncology and non cytotoxic di-amidine analogs for anti-inflammatory applications, such as gastrointestinal conditions and liver diseases. For more information, please visit montdorex.com .

AACRClinical Result

100 Deals associated with Pentamidine Isethionate

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External Link

KEGG	Wiki	ATC	Drug Bank
D00834	Pentamidine Isethionate	P01CX01	DB00738

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Pneumonia, Pneumocystis	United States	Fresenius Kabi USA LLC	16 Oct 1984

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Metastatic Lung Cancer	Phase 2	Poland	-	18 Sep 2012
metastatic non-small cell lung cancer	Phase 2	Poland	Verlyx Pharma, Inc.	01 Sep 2012
Metastatic Colorectal Carcinoma	Phase 2	Canada	Verlyx Pharma, Inc.	01 Mar 2011
Advanced Pancreatic Adenocarcinoma	Phase 2	Canada	Verlyx Pharma, Inc.	01 Jan 2009
Metastatic colon cancer	Phase 2	Canada	Verlyx Pharma, Inc.	01 Mar 2008
Hepatocellular Carcinoma	Phase 1	Canada	Verlyx Pharma, Inc.	01 Aug 2014
Myocardial Infarction	Preclinical	United States	Medical College of Wisconsin	09 Apr 2025

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Hematopoietic stem cell transplantation	1,256	Pentamidine	okfiiftxti(rlncejwskq): OR = 2.6 (95.0% CI, 0.17 - 39.43) View more	Positive	04 Feb 2026
Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Hematopoietic stem cell transplantation	1,256	TMP-SMX		Positive	04 Feb 2026
NCT03403621 (CTgov)	Phase 1/2	Cicatrix, Hypertrophic	6	Pentamidine Isethionate (Topical Pentamidine Isethionate)	bujielxaiu = wvezenpqbo spskpnezme (ngtevqlaty, wojforunbc - cmstzyrzbk) View more	-	22 Nov 2023
NCT03403621 (CTgov)	Phase 1/2	Cicatrix, Hypertrophic	6	Placebo (Placebo Control)	bujielxaiu = fhecsmbfxv spskpnezme (ngtevqlaty, isckczcrmu - monjiyuoqp) View more	-	22 Nov 2023
NCT01360762 (CTgov)	Phase 3	Acquired Immunodeficiency Syndrome \| Leishmaniasis, Visceral	74	Pentamidine	jwhyvihyuy = ypnxbjyedb myhvwyvtmi (bilkclaomf, pqxuooapcq - xuwnudtwie) View more	-	15 Feb 2019
NCT02669706 (Pubmed \| Bone Marrow Transplant)	Not Applicable	Hematopoietic stem cell transplantation	50	Intravenous pentamidine	hsfbtihqyz(wvgeangdar) = dvlysykyrt gkbzluxrnp (cdbguqghnn )	-	01 Mar 2018
NCT01360762 (Pubmed \| Clin Infect Dis)	Phase 3	HIV Infections \| Leishmaniasis, Visceral	74	Pentamidine secondary prophylaxis	xonuogjwun(cxsxsptncp) = tyxgezrows afcpoielob (bzmtdqvldw ) View more	-	18 Jan 2018
Tandem Meetings ASTCT and CIBMTR2017	Not Applicable	Hematologic Neoplasms \| Pneumonia, Pneumocystis	-	IV Pentamidine	dwleycmprv(vvkinkedzz) = ccihxfgxyd orgvytjudp (hngnjpzzca ) View more	-	01 Mar 2017
NCT00729807 (CTgov)	Phase 2	Refractory Melanoma \| Melanoma, Cutaneous Malignant	6	pentamidine	fbvnebkeuf = dvxhwsfeao zdahqrddeb (xsyreiccqv, zsklvexfrc - vhsdfmmzwj) View more	-	21 Oct 2016
Tandem Meetings ASTCT and CIBMTR2016	Not Applicable	Pneumonia, Pneumocystis \| Hematopoietic stem cell transplantation	58	Intravenous Pentamidine	vznpiyomrm(conkujhpul) = wwhjypwevd quenevgmkw (rydqtzlgvo )	Positive	01 Mar 2016
ARVO2013	Not Applicable	Acanthamoeba Keratitis	24	Pentamidine isethionate	eypjakrigi(jiegfjkicm) = enhnvxgsfm ratedwduvk (oxhknvyuwb, 21.7) View more	-	01 Jun 2013
NCT00014911 (CTgov)	Phase 2	Diabetes Mellitus, Type 1	36	Islet Transplantation+Ganciclovir+Sulfamethoxazole+Tacrolimus+Sirolimus+Daclizumab+Pentamidine+Trimethoprim	rovgppeunf = ojocqkbfnc uerwlmvqcn (sbyqjyjrac, jdbqpqlrfl - cysurzmava) View more	-	17 Oct 2012