Nizubaglustat

Nizubaglustat
Clinical data
Other names	AZ-3102
Legal status
Legal status	investigational;
Identifiers
	IUPAC name (2S,3R,4R,5S)-1-[5-[(3-fluoro-4-phenylphenyl)methoxy]pentyl]-2-(hydroxymethyl)piperidine-3,4,5-triol;
CAS Number	1633666-49-5;
PubChem CID	101882900;
IUPHAR/BPS	13906;
DrugBank	DB21603;
ChemSpider	58132507;
UNII	2X6NHN9RRN;
ChEBI	CHEBI:755338;
ChEMBL	ChEMBL3354637;
Chemical and physical data
Formula	C24H32FNO5
Molar mass	433.520 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C1[C@@H]([C@H]([C@@H]([C@@H](N1CCCCCOCC2=CC(=C(C=C2)C3=CC=CC=C3)F)CO)O)O)O;
	InChI InChI=InChI=1S/C24H32FNO5/c25-20-13-17(9-10-19(20)18-7-3-1-4-8-18)16-31-12-6-2-5-11-26-14-22(28)24(30)23(29)21(26)15-27/h1,3-4,7-10,13,21-24,27-30H,2,5-6,11-12,14-16H2/t21-,22-,23+,24+/m0/s1; Key:IRIRASPSMMWZOM-CJRSTVEYSA-N;

Nizubaglustat is an investigational new drug that is being evaluated by Azafaros BV for the treatment of Niemann–Pick disease type C. It is a dual inhibitor of glucosylceramide synthase (GCS; UGCG) and the non-lysosomal glucocerebrosidase (GBA2).^[1]^[2]^[3] In Niemann-Pick Type C Disease, metabolites such as unesterified cholesterol and glycosphingolipids build up in endosomes and lysosomes. By inhibiting the GCS and GBA2 enzymes, the buildup of these metabolites is reduced. Hence nizubaglustat is classified as a type of substrate reduction therapy.^[4]

Clinical trials

Nizubaglustat is in phase 3 clinical trials for Niemann-Pick Type C Disease.^[5]

↑ "Nizubaglustat - Amsterdam UMC; Leiden University". AdisInsight. Springer Nature Switzerland AG.
↑ Landskroner K, Singh K, Mitchell M, Walia JS (January 2026). "Therapeutic Effects of Nizubaglustat in a Mouse Model of GM2 Gangliosidosis". Journal of Inherited Metabolic Disease. 49 (1) e70130. doi:10.1002/jimd.70130. PMC 12779495. PMID 41500827.
↑ Hiwot T, Porter FD, Bremova-Ertl T, Ramaswami U, Hastings C, Héron B, et al. (May 2026). "2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C". Journal of Inherited Metabolic Disease. 49 (3) e70185. doi:10.1002/jimd.70185. PMC 13110917. PMID 42036217.
↑ Platt FM (October 2023). "The expanding boundaries of sphingolipid lysosomal storage diseases; insights from Niemann-Pick disease type C". Biochemical Society Transactions. 51 (5): 1777–1787. doi:10.1042/BST20220711. PMC 10657176. PMID 37844193.
↑ Clinical trial number NCT07082725 for "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) " at ClinicalTrials.gov