Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTHCF4J)

• DTT Name: Alpha-glucosidase (GLA)
• Synonyms: Maltase-glucoamylase; Maltase; Glucosidosucrase; Glucosidoinvertase; Glucoinvertase; Alpha-glucoside hydrolase; Alpha-glucopyranosidase; Alpha-D-glucoside glucohydrolase; Alpha-D-glucosidase; Alpha-1;4-glucosidase
• Gene Name: GAA
• DTT Type: Clinical trial target; [1]
• UniProt ID: LYAG_HUMAN ; GANAB_HUMAN ; GANC_HUMAN
• TTD ID: T61339
• Sequence:
  MGVRHPPCSHRLLAVCALVSLATAALLGHILLHDFLLVPRELSGSSPVLEETHPAHQQGA
  SRPGPRDAQAHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGA
  QMGQPWCFFPPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLH
  FTIKDPANRRYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLF
  FADQFLQLSTSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLA
  LEDGGSAHGVFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGY
  PFMPPYWGLGFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDG
  FRDFPAMVQELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKV
  WPGSTAFPDFTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELEN
  PPYVPGVVGGTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISR
  STFAGHGRYAGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVR
  WTQLGAFYPFMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAG
  ETVARPLFLEFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPV
  EALGSLPPPPAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQP
  MALAVALTKGGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQ
  LQKVTVLGVATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC
• Function: Breaks down starch and disaccharides to glucose.
• KEGG Pathway:
  Galactose metabolism (hsa00052)
  Starch and sucrose metabolism (hsa00500)
  Metabolic pathways (hsa01100)
  Lysosome (hsa04142)
• Reactome Pathway:
  Neutrophil degranulation (R-HSA-6798695)
  Glycogen breakdown (glycogenolysis) (R-HSA-70221)
  Glycogen storage disease type II (GAA) (R-HSA-5357609)

Molecular Interaction Atlas (MIA) of This DTT

1 Approved Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Cipaglucosidase alfa	DMAPDH2	Glycogen storage disease type II	5C51.3	Approved in EU	[2]

4 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Anamorelin	DM6HXTS	Carbohydrate metabolism disorder	5C51.Z	Phase 3	[3]
Deoxynojirimycin	DM2ATZB	Pompe disease	5C51.3	Phase 3	[1]
Maltose	DMH0ROP	N. A.	N. A.	Phase 1/2	[4]
AAV2/8-LSPhGAA	DMIX5HF	Pompe disease	5C51.3	Phase 1	[5]

8 Investigative Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
4,6-Dideoxyglucose	DMP5M9E	Discovery agent	N.A.	Investigative	[6]
6-Deoxy-Alpha-D-Glucose	DM9HBY5	Discovery agent	N.A.	Investigative	[6]
Beta-D-Glucose	DM5IHYP	Discovery agent	N.A.	Investigative	[6]
Double Oxidized Cysteine	DM6TU84	Discovery agent	N.A.	Investigative	[6]
JBP-1	DM0KMPN	Solid tumour/cancer	2A00-2F9Z	Investigative	[7]
Nicotinamide-Adenine-Dinucleotide	DM9LRKB	N. A.	N. A.	Investigative	[6]
POP-1	DMVTKCX	Solid tumour/cancer	2A00-2F9Z	Investigative	[7]
Tendamistat	DML53GP	Discovery agent	N.A.	Investigative	[8]

References

• [1] Nitrogen-in-the-ring pyranoses and furanoses: structural basis of inhibition of mammalian glycosidases. J Med Chem. 1994 Oct 28;37(22):3701-6.
• [2] Cipaglucosidase Alfa: First Approval. Drugs. 2023 Jun;83(8):739-745.
• [3] Absorption, elimination, and metabolism of CS-1036, a novel -amylase inhibitor in rats and monkeys, and the relationship between gastrointestinal distribution and suppression of glucose absorption.Drug Metab Dispos.2013 Apr;41(4):878-87.
• [4] DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Nucleic Acids Res. 2011 Jan;39(Database issue):D1035-41.
• [5] ClinicalTrials.gov (NCT03533673) AAV2/8-LSPhGAA in Late-Onset Pompe Disease. U.S. National Institutes of Health.
• [6] How many drug targets are there Nat Rev Drug Discov. 2006 Dec;5(12):993-6.
• [7] US patent application no. 2012,0251,516, PHARMACEUTICAL COMPOSITION FOR TREATING CANCER COMPRISING TRYPSINOGEN AND/OR CHYMOTRYPSINOGEN AND AN ACTIVE AGENT SELECTED FROM A SELENIUM COMPOUND, A VANILLOID COMPOUND AND A CYTOPLASMIC GLYCOLYSIS REDUCTION AGENT.
• [8] Influence of Specific Signal Peptide Mutations on the Expression and Secretion of the alpha -Amylase Inhibitor Tendamistat in Streptomyces lividans. J Biol Chem. 1996 Jun 21;271(25):15244-52.