Chemical compound
Pharmaceutical compound
Utreloxastat (PTC857 ) is aninvestigational new drug developed byPTC Therapeutics for the treatment ofamyotrophic lateral sclerosis (ALS).[ 1]
Utreloxastat is a15-lipoxygenase (15-LO) inhibitor that has been used to studyα-synucleinopathies ,tauopathies ,traumatic brain injury , andischemic-reperfusion related injuries. Utreloxastat is a redox‐active inhibitor offerroptosis .[ 2]
Utreloxastat has reached Phase 2 clinical trials for the treatment ofamyotrophic lateral sclerosis (ALS).[ 3] [ 4] [ 5] [ 6]
^ "Utreloxastat - PTC Therapeutics" .AdisInsight . Springer Nature Switzerland AG.^ "Utreloxastat" .MedChemExpress .^ Gao L, Giannousis P, Thoolen M, Kaushik D, Latham J, Tansy A, et al. (February 2023)."First-in-Human Studies of Pharmacokinetics and Safety of Utreloxastat (PTC857), a Novel 15-Lipooxygenase Inhibitor for the Treatment of Amyotrophic Lateral Sclerosis" .Clinical Pharmacology in Drug Development .12 (2):141– 151.doi :10.1002/cpdd.1203 .PMC 10107758 PMID 36516010 . ^ Katz JS, Vu T, Chio A, McDermott C, Devos D, Johnston M, et al. (2024-04-14)."CARDINALS: A Phase 2, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Utreloxastat (PTC857) in Patients with Amyotrophic Lateral Sclerosis (P5-11.010)" .Neurology .102 (17_supplement_1): 3593.doi :10.1212/WNL.0000000000205241 . ^ Li X, Bedlack R (June 2024)."Evaluating emerging drugs in phase II & III for the treatment of amyotrophic lateral sclerosis" .Expert Opinion on Emerging Drugs .29 (2):93– 102.doi :10.1080/14728214.2024.2333420 PMID 38516735 . ^ "PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients" .PTC Therapeutics, Inc . 26 November 2024.
