{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Levin J"],"funding":["Parkinson's UK"],"pagination":["104021"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9065877"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["80"],"pubmed_abstract":["Background
Synucleinopathies such as Parkinson ́s disease (PD), Dementia with Lewy bodies (DLB) and Multiple System Atrophy (MSA) are characterized by deposition of misfolded and aggregated α-synuclein. Small aggregates (oligomers) of α-synuclein have been shown to be the most relevant neurotoxic species and are targeted by anle138b, an orally bioavailable small molecule compound which shows strong disease-modifying effects in animal models of synucleinopathies.Methods
Anle138b was studied in a single-centre, double-blind, randomised, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy subjects. Eligible participants were randomly assigned (1:1 for sentinel subjects and 1:5 for main group) to placebo or anle138b (dose range 50 m"],"journal":["EBioMedicine"],"pubmed_title":["Safety, tolerability and pharmacokinetics of the oligomer modulator anle138b with exposure levels sufficient for therapeutic efficacy in a murine Parkinson model: A randomised, double-blind, placebo-controlled phase 1a trial."],"pmcid":["PMC9065877"],"funding_grant_id":["G-1102","G-1703","G-0701"],"pubmed_authors":["Sing N","Melbourne S","Prager K","Griesinger C","Schmidt F","Morgan A","Mariner C","Langer S","Spillantini MG","Dalley JW","Levin J","Weckbecker D","Ryazanov S","Matthias T","Wegrzynowicz M","Leonov A","Giese A"],"additional_accession":[]},"is_claimable":false,"name":"Safety, tolerability and pharmacokinetics of the oligomer modulator anle138b with exposure levels sufficient for therapeutic efficacy in a murine Parkinson model: A randomised, double-blind, placebo-controlled phase 1a trial.","description":"Background
Synucleinopathies such as Parkinson ́s disease (PD), Dementia with Lewy bodies (DLB) and Multiple System Atrophy (MSA) are characterized by deposition of misfolded and aggregated α-synuclein. Small aggregates (oligomers) of α-synuclein have been shown to be the most relevant neurotoxic species and are targeted by anle138b, an orally bioavailable small molecule compound which shows strong disease-modifying effects in animal models of synucleinopathies.Methods
Anle138b was studied in a single-centre, double-blind, randomised, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy subjects. Eligible participants were randomly assigned (1:1 for sentinel subjects and 1:5 for main group) to placebo or anle138b (dose range 50 m","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Jun","modification":"2026-05-30T15:17:30.145Z","creation":"2025-04-04T10:01:57.106Z"},"accession":"S-EPMC9065877","cross_references":{"pubmed":["35500536"],"doi":["10.1016/j.ebiom.2022.104021"]}}