Petadeferitrin

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Petadeferitrin

CAS911714-45-9

MFC16H21NO6S MW355.4 g/mol

(4S)-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-5H-1,3-thiazole-4-carboxylic acid

(4S)-2-{2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl}-4-methyl4,5-dihydro-1,3-thiazole-4-carboxylic acid
iron chelating agent, SP 420, WBX54NZ436

Petadeferitrin is an orally bioavailable iron-chelating agent and derivative of desferrithiocin, with iron chelating and protective activities in diseases of iron overload. Upon oral administration, petadeferitrin targets, binds to and chelates free iron. This induces the excretion of iron, prevents iron accumulation and prevents cellular and/or tissue damage associated with iron overload.

Petadeferitrin (formerly known as SP-420) is an investigational, orally bioavailable, small-molecule iron chelator being developed by Pharmacosmos (and its subsidiary Abfero Pharmaceuticals) to treat patients with transfusion-dependent iron overload. The drug works by binding to excess free iron in the body and forming complexes that are primarily excreted through bile and feces.

Key Characteristics & Mechanisms

Drug Class: It is a tridentate iron chelator and a derivative of desferrithiocin.
Enhanced Efficiency: In preclinical studies, it demonstrated a higher iron clearance efficiency (ICE value of 26.7) compared to desferrithiocin.
Brain-Penetrant: It is uniquely characterized as a brain-penetrant agent, which could expand its potential protective use in specific diseases associated with iron accumulation.

Clinical Development Status

Investigational Status: The drug remains investigational and has not yet been approved for commercial use anywhere in the world.
Target Diseases: Clinical evaluation focuses on individuals who suffer from iron overload due to frequent blood transfusions, such as patients with β-thalassemia and sickle cell disease.
Ongoing Studies: Pharmacosmos is actively evaluating the drug in Phase II clinical trials (such as ClinicalTrials.gov ID NCT05693909) to assess its safety, tolerability, and dosing advantages over existing options. Early human data suggests it may offer effective clearance with less frequent dosing compared to some currently approved alternatives.

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic SyndromesCTID:NCT05693909Phase:Phase 2Status:RecruitingDate:2025-09-24
Safety of SP-420 in the Treatment of Transfusional Iron OverloadCTID:NCT04741542Phase:Phase 1Status:TerminatedDate:2024-12-27
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare AnemiasCTID:NCT03801889Phase:Phase 2Status:WithdrawnDate:2020-10-05
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-ThalassemiaCTID:NCT02274233Phase:Phase 1Status:TerminatedDate:2015-09-29

An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent β-thalassemiaEudraCT:2022-002395-36

Phase:Phase 2, Status:Trial now transitioned, Date:2022-12-16

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=US42268401&_cid=P21-MSCM3W-69668-1

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2006107626&_cid=P21-MSCM3W-69668-1

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=US43268075&_cid=P21-MSCM3W-69668-1

PAT

Desferrithiocin polyether analoguesPublication Number:

US-2017217912-A1Priority Date:

2005-04-04