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+ [2]

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)

Login to view timeline

Structure/Sequence

Molecular FormulaC23H36N4O10S2

InChIKeyYBVNFKZSMZGRAD-UHFFFAOYSA-N

CAS Registry140-64-7

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

NCT03201159

/ WithdrawnPhase 1IIT

A Pilot, Exploratory Dose Escalating Study on the Safety, Pharmacodynamics and Preliminary Efficacy of VLX103 in the Treatment of Moderate Alcoholic Steatohepatitis

The study drug (VLX103) is being developed for the treatment of Alcoholic Steatohepatitis and other liver diseases. Alcoholic Steatohepatitis is an inflammatory (associated with irritation, swelling and cell damage) disease that affects the liver. It is associated with heavy and chronic intake of alcohol and presence of fat in the liver. Signs and symptoms often include fever, yellowing of the skin, nausea and impairment of liver function.
The main objective of this study is to evaluate the safety, pharmacodynamics (what the drug does to the body) and pharmacokinetics (how the drug is handled by the human body, like absorption and elimination) of increasing doses of VLX103 in subjects with moderate Alcoholic Steatohepatitis. In other words, we will evaluate how your body tolerates VLX103 at a specific dose and the effects that this VLX103 dose has on your liver and your body in general. The secondary objectives of this study are to evaluate if VLX103 has the potential to treat Alcoholic Steatohepatitis patients, to determine the maximum dose that can be tolerated, and to measure the levels of VLX103 in your blood at different time points during the study.
VLX103 is an experimental drug. Experimental means that the drug has not been approved by the Food and Drug Administration (FDA) for the treatment of Alcoholic Steatohepatitis. The active ingredient in VLX103, pentamidine, is approved for treating parasitic (microorganisms) infections. Pentamidine is currently approved and marketed in about 20 countries, including the United States, for use by injection (administered by a syringe) and by inhalation (administered by a nebulizer) for other health conditions. However, VLX103 is the first oral form of pentamidine being developed, and is administered by mouth as an oral tablet.

Start Date25 Jun 2017

Sponsor / Collaborator

National Institute on Alcohol Abuse & Alcoholism

[+3]

100 Clinical Results associated with Pentamidine Isethionate

Login to view more data

100 Translational Medicine associated with Pentamidine Isethionate

Login to view more data

100 Patents (Medical) associated with Pentamidine Isethionate

Login to view more data

3,346

Literatures (Medical) associated with Pentamidine Isethionate

05 Aug 2025·Microbiology Spectrum

Pentamidine inhibition of streptopain attenuates Streptococcus pyogenes virulence

Article

Author: Trivedi, Keya ; LaRock, Christopher N.

ABSTRACT:

The obligate human pathogen Streptococcus pyogenes (also known as GAS; Group A Streptococcus ) carries high morbidity and mortality, primarily in impoverished or resource-poor regions. The failure rate of monotherapy with conventional antibiotics is high, and invasive infections by this bacterium frequently require extensive supportive care and surgical intervention. Thus, it is important to find new compounds with adjunctive therapeutic benefits. The conserved secreted protease streptopain (Streptococcal pyogenic exotoxin B; SpeB) directly contributes to disease pathogenesis by inducing pathological inflammation, degrading tissue, and promoting the evasion of antimicrobial host defense proteins. This study screened 400 diverse off-patent drugs and drug-like compounds for inhibitors of streptopain proteolysis. Lead compounds were tested for activity at lower concentrations and anti-virulence activities during in vitro infection. Significant inhibition of streptopain was seen for pentamidine, an anti-protozoal drug approved for the treatment of Pneumocystis pneumonia, leishmaniasis, and trypanosomiasis. Streptopain inhibition rendered GAS susceptible to killing by human innate immune cells. These studies identify unexploited molecules as new starting points for drug discovery and a potential for repurposing existing drugs for the treatment of infections by GAS.

IMPORTANCE:

Streptococcus pyogenes is a common cause of severe invasive infections. Repeated infections can trigger autoimmune diseases such as acute rheumatic fever and rheumatic heart disease. This study examines how targeting a specific, highly conserved virulence factor of the secreted cysteine protease streptopain can sensitize a serious pathogen to killing by the immune system. Manipulating the host-pathogen interaction, rather than attempting to directly kill a microbe, is a promising therapeutic strategy. Notably, its benefits include limiting off-target effects on the microbiota. Streptopain inhibitors, including the antifungal and antiparasitic drug pentamidine as identified in this work, may therefore be useful in the treatment of S. pyogenes infection.

01 Aug 2025·INTERNAL MEDICINE

Comparison between the Outcomes of Pentamidine and Trimethoprim-Sulfamethoxazole for <i>Pneumocystis</i> Pneumonia in Non-HIV Patients: A Nationwide Japanese Retrospective Cohort Study

Article

Author: Fushimi, Kiyohide ; Yasunaga, Hideo ; Aso, Shotaro ; Matsui, Hiroki ; Taniguchi, Jumpei

Objective Data on the first-line treatment options for patients with Pneumocystis pneumonia (PCP) without human immunodeficiency virus (HIV) infection are limited. Therefore, we evaluated the outcome of pentamidine compared to trimethoprim-sulfamethoxazole (TMP-SMX) in non-HIV patients with PCP. Methods We used data from the Japanese Diagnosis Procedure Combination Inpatient Database. We included non-HIV PCP patients who initially received TMP-SMX or pentamidine between July 2010 and March 2022. We categorized eligible patients into TMP-SMX and pentamidine groups and performed a propensity score overlap weighting analysis to compare in-hospital mortality between the groups. Results Among 5,870 eligible patients, 5,456 and 414 received TMP-SMX and pentamidine, respectively. Pentamidine treatment was associated with a higher in-hospital mortality than TMP-SMX treatment in the propensity score overlap weighting analysis (23.6% vs. 40.1%; risk difference, 16.5%; 95% confidence interval, 10.8-22.2%; p<0.001). Conclusions Based on these findings, pentamidine may not be as effective as TMP-SMX for treating PCP in non-HIV patients.

19 Jul 2025·Scientific Reports

Efficient adsorption of basic fuchsin dye using thermally engineered novel smart nanocomposites.

Article

Author: Abdelrahman, Ehab A

Basic fuchsin dye is known for its genotoxic, neurotoxic, and carcinogenic effects on humans and its long-term persistence and ecological toxicity in aquatic environments. In this study, novel MgO/BaCO₃/BaCrO₄/C nanocomposites were fabricated through a Pechini-type sol-gel strategy and thermally treated at 600 and 800 ^oC to yield MB600 and MB800, respectively, for the effective adsorption of basic fuchsin from water-based solutions. The crystalline phases were identified using X-ray diffraction patterns, indicating the formation of crystalline MgO, BaCO₃, and BaCrO₄ phases, with average crystallite sizes of 60.70 and 75.64 nm for MB600 and MB800, respectively. Energy dispersive X-ray spectroscopy revealed the atomic composition, with MB600 containing 21.3% C, 51.1% O, 10.2% Mg, 2.8% Cr, and 14.6% Ba, while MB800 showed enhanced metal content and reduced carbon residues. Field emission scanning electron microscope and high-resolution transmission electron microscope micrographs demonstrated morphological evolution with increasing calcination temperature and average particle sizes of 13.7 and 32.1 nm for MB600 and MB800, respectively. MB600 exhibited a maximum adsorption capacity of 442.48 mg/g, outperforming MB800 at 375.94 mg/g. The sorption mechanism was spontaneous, exothermic, physisorption, and best described by the pseudo-first-order model in addition to the Langmuir isotherm, demonstrating monolayer coverage and uniform surface binding.

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

Login to view more data

External Link

KEGG	Wiki	ATC	Drug Bank
D00834	Pentamidine Isethionate	P01CX01	DB00738

R&D Status

Approved

10 top approved records.

Login

to view more data

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

Developing

10 top R&D records.

Login

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
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

Login to view more data

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03403621 (CTgov)	Phase 1/2	Cicatrix, Hypertrophic	6	Pentamidine Isethionate (Topical Pentamidine Isethionate)	tiammlcdnt = tqyhyevwhd wzrffwumgo (alppliuycc, unxsrqjrvl - hpmtkfqdzo) View more	-	22 Nov 2023
NCT03403621 (CTgov)	Phase 1/2	Cicatrix, Hypertrophic	6	Placebo (Placebo Control)	tiammlcdnt = clzozjhypn wzrffwumgo (alppliuycc, zdskmzejag - roeyupeeok) View more	-	22 Nov 2023
NCT01360762 (CTgov)	Phase 3	Acquired Immunodeficiency Syndrome \| Leishmaniasis, Visceral	74	Pentamidine	cozbcsepjb = feqrdunbry diijugclpe (zyzbzsyktn, xlribyceut - kpogplwfdd) View more	-	15 Feb 2019
NCT02669706 (Pubmed \| Bone Marrow Transplant)	Not Applicable	Hematopoietic stem cell transplantation	50	Intravenous pentamidine	yycommgvpi(eumnwjjijq) = oqccbeicmn gmylqgzplk (fjwdefbbfb )	-	01 Mar 2018
NCT01360762 (Pubmed \| Clin Infect Dis)	Phase 3	HIV Infections \| Leishmaniasis, Visceral	74	Pentamidine secondary prophylaxis	anozmfonjg(raxixyvrpz) = lxbzvmyznz xsityxrgvg (ovpyhujdnt ) View more	-	18 Jan 2018
Tandem Meetings ASTCT and CIBMTR2017	Not Applicable	Hematologic Neoplasms \| Pneumonia, Pneumocystis	-	IV Pentamidine	mzobbkdbag(pstqcujzhm) = wlwcabdkpr lbmqwswgyq (ieluuobdja ) View more	-	01 Mar 2017
NCT00729807 (CTgov)	Phase 2	Refractory Melanoma \| Melanoma, Cutaneous Malignant	6	pentamidine	pynkuexfae = gjpwqrwhbp dzcmwmzwuo (pxkrvwtftf, daiszudbty - vtswnsdefr) View more	-	21 Oct 2016
Tandem Meetings ASTCT and CIBMTR2016	Not Applicable	Pneumonia, Pneumocystis \| Hematopoietic stem cell transplantation	58	Intravenous Pentamidine	pnwhoscusv(hbwkgmhjjb) = sbsgzhehld givtncwcfz (xhoihaiorg )	Positive	01 Mar 2016
ARVO2013	Not Applicable	Acanthamoeba Keratitis	24	Pentamidine isethionate	nydqzobqdt(vvocawidyp) = omuczscpdp bcnyadzubm (uayvpekxua, 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	hwsqojjfqd = fkxvnmnera lbeldqvnrx (sbxgejwcpf, dswqqfgeho - wtzwqllfds) View more	-	17 Oct 2012
NCT00078559 (CTgov)	Phase 1/2	Kidney Diseases \| Renal transplant rejection	10	Kidney transplant+Acyclovir+Acetaminophen+Bactrim+Nystatin+Sirolimus+Clotrimazole+Trimethoprim+Diphenhydramine+TMP+Alemtuzumab+Valgancyclovir+Sulfa+Methylprednisolone+Tacrolimus+Pentamidine	lzdnqqwial = wnarnxxzzg gtdskujpys (byytsvjrtz, nzgrsmwysi - ymwwhzktzz) View more	-	09 Jul 2012