Utreloxastat

Utreloxastat
Clinical data
Other names	PTC857
Identifiers
	IUPAC name 2,3,5-trimethyl-6-nonylcyclohexa-2,5-diene-1,4-dione;
CAS Number	1213269-96-5 ;
PubChem CID	46174641;
IUPHAR/BPS	13260;
DrugBank	DB18154;
ChemSpider	115006754;
UNII	WW8KZK2PZT;
KEGG	D12510;
ChEMBL	ChEMBL5314565;
Chemical and physical data
Formula	C18H28O2
Molar mass	276.420 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CCCCCCCCCC1=C(C(=O)C(=C(C1=O)C)C)C;
	InChI 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]

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]

References

^ "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.

[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.

[Li_2024-5] 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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Utreloxastat

Contents

Pharmacology

Clinical trials

See also

References