Delving into the Latest Updates on Rimeporide with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

NHE1

Action

inhibitors

Mechanism

NHE1 inhibitors(Sodium/hydrogen exchanger 1 inhibitors)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]

Active Indication

Muscular Dystrophy, DuchenneMyotonic Dystrophy

Inactive Indication

Heart Failure

Originator Organization

Merck Serono SA

Active Organization

EspeRare FoundationMerck Serono SA

Inactive Organization-

Drug Highest PhasePhase 1

First Approval Date-

RegulationRare Pediatric Disease (United States), Orphan Drug (European Union)

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Structure/Sequence

Molecular FormulaC11H15N3O5S2

InChIKeyGROMEQPXDKRRIE-UHFFFAOYSA-N

CAS Registry187870-78-6

In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime. Sodium/proton type 1 exchanger (NHE-1) inhibition is an innovative pathway that has proved to efficiently prevent the accumulation of muscle damage (inflammation and fibrosis) in animal models of muscular dystrophies and heart failure. Based on prior safety and efficacy results in animal and humans, NHE-1 inhibition with Rimeporide represents a new therapeutic approach with no restriction on age and on genetic subtypes which could be combined to other treatments that restore or augment dystrophin.This study examines the safety and tolerability and effects on the muscles of rimeporide, in patients aged 6 to 14 years with Duchenne Muscular Dystrophy (DMD).

Start Date01 Mar 2016

Sponsor / Collaborator

EspeRare Foundation

100 Clinical Results associated with Rimeporide

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100 Translational Medicine associated with Rimeporide

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100 Patents (Medical) associated with Rimeporide

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31

Literatures (Medical) associated with Rimeporide

04 Feb 2025·ACS Omega

Quantitative Structure–Property Relationship Modeling with the Prediction of Physicochemical Properties of Some Novel Duchenne Muscular Dystrophy Drugs

Article

Author: K, Jyothish ; Santiago, Roy

Duchenne muscular dystrophy is a critical, progressively worsening, and ultimately deadly illness characterized by the deterioration of skeletal muscles, respiratory failure, and heart disease. The pharmaceutical industries are persistently innovating drug design processes to address the rise of infections and effectively treat emerging syndromes or genetically based disorders with the help of quantitative structure-property relationship models. These models are mathematical tools that correlate molecular structures with their physicochemical properties through structural characteristics. Different models can be generated based on the various structural features of the compounds, and topological indices are one such significant structural feature generated from the molecular graph and are key tools used in these models. This study focuses on creating quantitative structure-property relationship models using degree-based topological indices, which are highly effective in quantitative structure-property relationship analysis to explore the diverse physicochemical properties of Duchenne muscular dystrophy drugs with the prediction of properties of a recently approved drug givinostat. Furthermore, the drug discovery and development activities can be accelerated using the developed models to forecast the possible productiveness of novel Duchenne muscular dystrophy treatment drugs.

01 Jul 2022·International Journal of Biological Macromolecules

Exploring the interaction of guanidine ligands Amiloride, Rimeporide and Cariporide with DNA for understanding their role as inhibitors of Na+/H+ exchangers (NHEs): A spectroscopic and molecular docking investigation

Article

Author: Kumar, Pankaj ; Kaushik, Mahima ; Chaudhary, Swati

The inhibition of Na⁺/H⁺ Exchangers (NHEs) has shown efficacy in the pathology of several diseases like tumors, cardiovascular, and neurological disorders. The role of guanidine ligands such as amiloride, cariporide, and rimeporide as NHE inhibitors is very well documented but their interaction studies with genomic DNA are still unexplored. In this study, a combination of various biophysical and molecular docking studies was employed to investigate their binding aspects.UV-Visible, fluorescence, and circular dichroism (CD) studies indicated that guanidine ligands bind to the grooves of Calf Thymus DNA (ctDNA). Fluorescence titration studies depict that amiloride binds to ctDNA with a binding constant in the order of 10² M^-1 and free energy change (ΔG⁰) of -14.05 KJ mol^-1. Competitive fluorescence studies indicated the minor groove binding property of amiloride, whereas major groove binding mode was deduced for rimeporide and cariporide. Molecular docking studies were also found to be in accordance with the experimental results, revealing the information about the binding energy of the guanidine ligand-ctDNA complex. The docked structures depicted binding energy of -6.4 kcal mol^-1 for amiloride and - 6.6 kcal mol^-1 for rimeporide and cariporide. Such physicochemical studies of DNA-ligand interactions may facilitate the understanding of the mechanisms of NHE inhibition.

01 May 2022·Pflügers Archiv - European Journal of PhysiologyQ4 · MEDICINE

Hypotension in hereditary cardiomyopathy

Q4 · MEDICINE

Article

Author: Jacques, Danielle ; Al-Khoury, Johny ; Bkaily, Ghassan

It is well accepted that hypertension may lead to the development of heart failure (HF). However, little is known about the development of hypotension that may contribute to the onset of hereditary cardiomyopathy (HCM), thus promoting heart failure and early death. The purpose of this study is to verify whether a decrease in blood pressure takes place during different phases of HCM (asymptomatic, necrosis, hypertrophy, and heart failure). Using the well-known animal model, the UM-X7.1 hamster strain of HCM (HCMH), our results showed the absence of a change in mean arterial pressure (MAP) during the asymptomatic phase preceding the development of necrosis in HCMHs when compared to age-matched normal hamster (NH). However, there was a progressive decrease in MAP that reached its lowest level during the heart failure phase. The MAP during the development of the necrosis phase of HCM was accompanied by a significant increase in the level of the sodium-hydrogen exchanger, NHE1. Treatments with the potent NHE1 inhibitor, EMD 87580 (rimeporide), did not affect MAP of NH. However, treatments with EMD 87580 during the three phases of the development of HCM significantly reversed the hypotension associated with HCM.Our results showed that the development of HCM is associated with hypotension. These results suggest that a decrease in blood pressure could be a biomarker signal for HCM leading to HF and early death. Since the blockade of NHE1 significantly but partially prevented the reduction in MAP, this suggests that other mechanisms can contribute to the development of hypotension in HCM.

1

News (Medical) associated with Rimeporide

05 Aug 2023

·esperare.org

Florence Porte talks about EspeRare’s Mission and Innovations on ‘Objectif Croissance’

Florence Porte-Thomé, R&D Director and co-Founder of EspeRare Foundation, was recently interviewed on Objectif Croissance by Vincent Touraine. This insightful discussion, brought to you by MEDIAS FRANCE in partnership with BFM Business, delves into the mission and objectives of EspeRare. What Sets EspeRare Apart? Florence sheds light on EspeRare’s patient-centric approach and the 'venture philanthropy' model which drives the Foundation to innovate and push the boundaries of rare disease drug development. EspeRare's Pioneering Projects Rimeporide – A drug exhibiting a therapeutic potential in children affected by Duchenne Muscular Dystrophy (DMD). ER004 – A groundbreaking pre-natal therapy for X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), being developed in partnership with Pierre Fabre Group. Partnership and Progress The collaboration with Pierre Fabre Group has been instrumental since 2020, reflecting their unwavering trust and enthusiasm for EspeRare's ambitious goals. The Foundation is also proud to be collaborating with 8 medical institutions across Europe and the US, among which is l'Hôpital Necker Enfants Malades in Paris, France to provide an innovative solution for vulnerable patients. Watch the Video! To learn more about our journey and commitment to transforming lives through science and partnerships: https://vimeo.com/991451978?share=copy

Clinical Study

100 Deals associated with Rimeporide

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External Link

KEGG	Wiki	ATC	Drug Bank
-	Rimeporide	-	DB12861

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Heart Failure	Phase 2	Germany	Merck Serono SA	-
Muscular Dystrophy, Duchenne	Phase 1	-	Merck Serono SA	-
Myotonic Dystrophy	Preclinical	Switzerland	EspeRare Foundation	-

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02710591 (RIM4DMD, CTgov)	Phase 1	Muscular Dystrophy, Duchenne	20	Rimeporide (Cohort 1)	(phlzzahdqz) = qifrgmevrr snfjgrkgvc (yhyvjuqjyp, tgjhmkbfct - inopxolywx) View more	-	18 Jul 2019
				Rimeporide (Cohort 2)	(phlzzahdqz) = gvnizvtnmk snfjgrkgvc (yhyvjuqjyp, qjmjerqjhf - lqlxqrhmrz) View more