Utreloxastat

Last updated

Utreloxastat
Utreloxastat.png
Clinical data
Other namesPTC857
Identifiers
  • 2,3,5-trimethyl-6-nonylcyclohexa-2,5-diene-1,4-dione
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
Chemical and physical data
Formula C18H28O2
Molar mass 276.420 g·mol−1
3D model (JSmol)
  • CCCCCCCCCC1=C(C(=O)C(=C(C1=O)C)C)C
  • InChI=InChI=1S/C18H28O2/c1-5-6-7-8-9-10-11-12-16-15(4)17(19)13(2)14(3)18(16)20/h5-12H2,1-4H3
  • Key:IJWAQTHZBDBIID-UHFFFAOYSA-N

Utreloxastat (PTC857) is an investigational new drug developed by PTC Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS). [1]

Contents

Pharmacology

Utreloxastat is a 15-lipoxygenase (15-LO) inhibitor that has been used to study α-synucleinopathies, tauopathies, traumatic brain injury, and ischemic-reperfusion related injuries. Utreloxastat is a redox‐active inhibitor of ferroptosis. [2]

Clinical trials

Utreloxastat has reached Phase 2 clinical trials for the treatment of amyotrophic lateral sclerosis (ALS). [3] [4] [5] However this trial failed to show significant efficacy in slowing disease progression. [6]

See also

References

  1. ↑ "Utreloxastat - PTC Therapeutics". AdisInsight. Springer Nature Switzerland AG.
  2. ↑ "Utreloxastat". MedChemExpress.
  3. ↑ 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.
  4. ↑ 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.
  5. ↑ 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.
  6. ↑ "PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients". PTC Therapeutics, Inc. 26 November 2024.
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