Delving into the Latest Updates on Vamifeport hydrochloride with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Vamifeport

Target

FPN1

Action

inhibitors

Mechanism

FPN1 inhibitors(Ferroportin-1 inhibitors)

Therapeutic Areas

Nervous System DiseasesCardiovascular DiseasesCongenital Disorders+ [3]

Active Indication

Beta-ThalassemiaSystemic Carnitine DeficiencyAnemia, Sickle Cell

Inactive Indication

Non-transfusion dependent thalassaemiaTransfusion-dependent Beta Thalassemia

Originator Organization

Vifor Pharma AG

Active Organization

Vifor (International) AG

CSL Behring LLCVifor Fresenius Medical Care Renal Pharma France S.A.S.

Inactive Organization-

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

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Structure/Sequence

Molecular FormulaC21H21FN6O2

InChIKeyKNYVRFXIVWUGBZ-UHFFFAOYSA-N

CAS Registry2095668-10-1

A Phase 1, Randomized, Open-label Study to Characterize the Pharmacokinetics, Pharmacodynamics, and Safety of Vamifeport After Multiple Oral Administration of One Immediate-release Formulation and After Single and Multiple Oral Administration of Two Prolonged-release Formulations in Healthy Adult Subjects

This is a phase 1, single-center, randomized, open-label study to characterize the pharmacokinetics (PK), pharmacodynamics (PD), and safety of vamifeport after multiple oral administrations of one immediate-release (IR) formulation and after single and multiple oral administrations of two prolonged-release (PR) formulation in healthy adult participants.

Start Date22 Nov 2024

Sponsor / Collaborator

CSL Behring LLC

NCT04817670

/ CompletedPhase 2

A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease (ViSionSerenity)

The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD). The safety, tolerability and clinical beneficial effects of VIT-2763 for the treatment of SCD are also explored.

Start Date18 Nov 2021

Sponsor / Collaborator

Vifor (International) AG

[+1]

NCT05077436

/ CompletedPhase 1

A Randomised, Open-Label, Food Effect and Formulation Bioavailability Study of Two Vamifeport Oral Formulations in Healthy Male and Female Adults

Two different vamifeport oral formulations will be administered in fed and fasted state to assess the vamifeport food-drug interaction and to assess the relative bioavailability (the proportion of drug entering the circulation) of 2 different vamifeport oral formulations in healthy adult participants.
Participants will be randomly allocated to one of four treatment sequences, with four dosing periods each, where different combinations of both formulations will be administered following fasted and fed state.
The total study duration for each participant is up to 7 weeks and 4 days.

Start Date14 Oct 2021

Sponsor / Collaborator

Vifor (International) AG

100 Clinical Results associated with Vamifeport hydrochloride

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100 Translational Medicine associated with Vamifeport hydrochloride

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100 Patents (Medical) associated with Vamifeport hydrochloride

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14

Literatures (Medical) associated with Vamifeport hydrochloride

09 Jan 2025·Blood

Genetic iron overload aggravates, and pharmacological iron restriction improves, MDS pathophysiology in a preclinical study

Article

Author: Manolova, Vania ; Dürrenberger, Franz ; Asperti, Michela ; Antypiuk, Ada ; Sharma, Richa ; Navaneethabalakrishan, Shobana ; Vance, S. Zebulon ; Vinchi, Francesca ; Passos, Sara

AbstractAlthough iron overload is a common feature in myelodysplastic syndromes (MDS), it remains unclear how iron excess is detrimental for disease pathophysiology. Taking advantage of complementary approaches, we analyzed the impact of iron overload and restriction achieved through genetic activation of ferroportin (FPN) via the C326S mutation (FPNC326S) and pharmacologic inhibition (vamifeport) of the iron exporter FPN, respectively, in a MDS mouse model. Although FPNC326S-induced iron overload did not significantly improve the late stages of erythroid maturation, vamifeport-mediated iron restriction ameliorated anemia and red blood cell maturation in MDS mice, through the reduction of oxidative stress and apoptosis in erythroid progenitors. Iron overload aggravated, and restriction alleviated, reactive oxygen species formation, DNA damage, and cell death in hematopoietic stem and progenitor cells (HSPCs), resulting in altered cell survival and quality. Finally, myeloid bias, indicated by expanded bone marrow myeloid progenitors and circulating immature myeloid blasts, was exacerbated by iron excess and attenuated by iron restriction. Overall, vamifeport treatment resulted in improved anemia and significant survival increment in MDS mice. Interestingly, the combined therapy with vamifeport and the erythroid maturation agent luspatercept has superior effect in improving anemia and myeloid bias as compared with single treatments and offers additive beneficial effects in MDS. Our results prove, to our knowledge, for the first time in a preclinical model, that iron plays a pathologic role in transfusion-independent MDS. This is likely aggravated by transfusional iron overload, as suggested by observations in the FPNC326SMDS model. Ultimately, the beneficial effects of pharmacologic FPN inhibition uncovers the therapeutic potential of early prevention of iron toxicity in transfusion-independent MDS.

01 Sep 2024·HemaSphere

The oral ferroportin inhibitor vamifeport prevents liver iron overload in a mouse model of hemochromatosis

Article

Author: Altermatt, Patrick ; Nyffenegger, Naja ; Flace, Anna ; Sundstrom, Hanna ; Manolova, Vania ; Varol, Ahmet ; Doucerain, Cédric ; Dürrenberger, Franz

AbstractHemochromatosis is an inherited iron overload condition caused by mutations that reduce the levels of the iron‐regulatory hormone hepcidin or its binding to ferroportin. The hepcidin–ferroportin axis is pivotal to iron homeostasis, providing opportunities for therapeutic intervention in iron overload disorders like hemochromatosis. The aim of this study was to evaluate the efficacy of the oral ferroportin inhibitor vamifeport in the Hfe C282Y mouse model, which carries the most common mutation found in patients with hemochromatosis. A single oral dose of vamifeport lowered serum iron levels in Hfe C282Y mice, with delayed onset and shorter duration than observed in wild‐type mice. Vamifeport induced transient hypoferremia by inhibiting ferroportin and resulted in a feedback regulation of liver Hamp in wild‐type mice, which was absent in Hfe C282Y mice, reflecting the dysregulated systemic iron sensing in this hemochromatosis model. Chronic dosing with vamifeport led to sustained serum and liver iron reductions in Hfe C282Y mice, as well as markedly reducing liver Hamp expression in Hfe C282Y mice, suggesting distinct regulation of liver Hamp expression following acute or continuous iron restriction via vamifeport. At the tested dose, vamifeport retained its activity when combined with phlebotomy and did not significantly interfere with liver iron removal by phlebotomy in Hfe C282Y mice. These data demonstrate that chronic vamifeport treatment significantly reduces serum iron levels and prevents liver iron loading in the Hfe C282Y mouse model of hemochromatosis, thus providing preclinical proof of concept for the efficacy of vamifeport in hemochromatosis with or without phlebotomy.

01 Oct 2023·Haematologica

Ferroportin inhibitor vamifeport ameliorates ineffective erythropoiesis in a mouse model of β-thalassemia with blood transfusions.

Article

Author: Altermatt, Patrick ; Nyffenegger, Naja ; Flace, Anna ; Dürrenberger, Franz ; Kalleda, Natarajaswamy ; Doucerain, Cédric ; Ingoglia, Giada ; Manolova, Vania

β-thalassemia is an inherited anemia characterized by ineffective erythropoiesis. Blood transfusions are required for survival in transfusion-dependent β-thalassemia and are also occasionally needed in patients with non-transfusion-dependent β-thalassemia. Patients with transfusion-dependent b-thalassemia often have elevated transferrin saturation (TSAT) and non-transferrin-bound iron (NTBI) levels, which can lead to organ iron overload, oxidative stress, and vascular damage. Vamifeport is an oral ferroportin inhibitor that was previously shown to ameliorate anemia, ineffective erythropoiesis, and dysregulated iron homeostasis in the Hbbth3/+ mouse model of β-thalassemia, under non-transfused conditions. Our study aimed to assess the effects of oral vamifeport on iron-related parameters (including plasma NTBI levels) and ineffective erythropoiesis following blood transfusions in Hbbth3/+ mice. A single dose of vamifeport prevented the transient transfusion-mediated NTBI increase in Hbbth3/+ mice. Compared with vehicle treatment, vamifeport significantly increased hemoglobin levels and red blood cell counts in transfused mice. Vamifeport treatment also significantly improved ineffective erythropoiesis in the spleens of Hbbth3/+ mice, with additive effects observed when treatment was combined with repeated transfusions. Vamifeport corrected leukocyte counts and significantly improved iron-related parameters (serum transferrin, TSAT and erythropoietin levels) versus vehicle treatment in Hbbth3/+ mice, irrespective of transfusion status. In summary, vamifeport prevented transfusion-mediated NTBI formation in Hbbth3/+ mice. When given alone or combined with blood transfusions, vamifeport also ameliorated anemia, ineffective erythropoiesis, and dysregulated iron homeostasis. Administering vamifeport together with repeated blood transfusions additively ameliorated anemia and ineffective erythropoiesis in this mouse model, providing preclinical proof-of-concept for the efficacy of combining vamifeport with blood transfusions in β-thalassemia.

100 Deals associated with Vamifeport hydrochloride

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Beta-Thalassemia	Phase 2	Greece	Vifor (International) AG	11 Jun 2020
Beta-Thalassemia	Phase 2	Italy	Vifor (International) AG	11 Jun 2020
Beta-Thalassemia	Phase 2	Lebanon	Vifor (International) AG	11 Jun 2020
Beta-Thalassemia	Discovery	Greece	Vifor (International) AG	11 Jun 2020
Beta-Thalassemia	Discovery	Italy	Vifor (International) AG	11 Jun 2020
Non-transfusion dependent thalassaemia	Discovery	Israel	Vifor (International) AG	11 Jun 2020
Non-transfusion dependent thalassaemia	Discovery	Thailand	Vifor (International) AG	11 Jun 2020
Non-transfusion dependent thalassaemia	Discovery	Greece	Vifor (International) AG	11 Jun 2020
Non-transfusion dependent thalassaemia	Discovery	Lebanon	Vifor (International) AG	11 Jun 2020
Non-transfusion dependent thalassaemia	Discovery	Italy	Vifor (International) AG	11 Jun 2020

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04817670 (ViSionSerenity, CTgov)	Phase 2	Anemia, Sickle Cell	25	VIT-2763 (Cohort 1: VIT-2763 60 mg BID (120 mg/Day))	fyiqsjpycl(vhyukiruab) = wslccncuzv vlvtneubud (qildwalnqf, odkaqkkbrk - hbjvxerzrv) View more	-	18 Feb 2025
				VIT-2763 (Cohort 2: VIT-2763 120 mg BID (240 mg/Day))	fyiqsjpycl(vhyukiruab) = uqwlcvdgcf vlvtneubud (qildwalnqf, pmokyggrtb - asbmzlgnid) View more
NCT04364269 (VITHAL, CTgov)	Phase 2	Beta-Thalassemia \| Non-transfusion dependent thalassaemia	35	Placebo	vcucfuupuy(lsvorfxuov) = lnqzwnbmfx flahkrqsir (haiizlqggs, bagvssxpzt - bplbnkknws) View more	-	14 Dec 2023
NCT04364269 (EHA2022) Manual	Phase 2	Transfusion-dependent Beta Thalassemia	25	Vamifeport hydrochloride (QD)	(qbjulhmtzv) = rpsmvdduei drfczomvfs (dsbcuxosyp ) View more	Positive	12 May 2022
				Vamifeport hydrochloride (BID)	(dddxqampec) = ayupwrdvzi wkkzmixfzl (dqiykmbcea ) View more