Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DM94Y6O)

• Drug Name: BMN-701 Drug Info
• Synonyms: IGF2-GAA; XC-701; ZC-701; Enzyme replacement therapy (GILT, Pompe disease), BioMarin/ZyStor

Indication

Disease Entry	ICD 11	Status	REF
Pompe disease	5C51.3	Phase 3	[1]
Type 2 glycogen storage disease	5C51.3	Phase 3	[2]

• Cross-matching ID: TTD Drug ID: DM94Y6O

Molecule-Related Drug Atlas

Drug(s) Targeting Lysosomal alpha-glucosidase (GAA)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Avalglucosidase alfa	DMPW5UF	Pompe disease	5C51.3	Approved	[4]
Deoxynojirimycin	DM2ATZB	Pompe disease	5C51.3	Phase 3	[5]
AT845	DMZJIIB	Pompe disease	5C51.3	Phase 2	[6]
SALACINOL	DMVKQT8	N. A.	N. A.	Phase 1/2	[7]
SPK-3006	DMK9DG5	Pompe disease	5C51.3	Phase 1/2	[8]
GZ402666	DMGO6M1	Pompe disease	5C51.3	Phase 1	[9]
5-(4-(4-Acetylphenyl)piperazin-1-ylsulfonyl)-6-chloroindolin-2-one	DMAJWLD	Pompe disease	5C51.3	Preclinical	[10]
Alpha-Homonojirimycin	DMT17H0	Discovery agent	N.A.	Investigative	[5]
7-O-b-D-Glucopyranosyl-a-homonojirimycin	DM8IY3D	Discovery agent	N.A.	Investigative	[5]
VALIOLAMINE	DM2VQN5	Discovery agent	N.A.	Investigative	[5]

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Lysosomal alpha-glucosidase (GAA)	TTLPC70	LYAG_HUMAN	Modulator	[3]

References

• [1] ClinicalTrials.gov (NCT01924845) BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study). U.S. National Institutes of Health.
• [2] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [3] Clinical pipeline report, company report or official report of BioMarin Pharma.
• [4] ClinicalTrials.gov (NCT02782741) Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET). U.S. National Institutes of Health.
• [5] In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures. Bioorg Med Chem. 2008 Aug 1;16(15):7330-6.
• [6] Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity. EMBO Mol Med. 2022 Jan 11;14(1):e13968.
• [7] Alpha-glucosidase inhibitor from Kothala-himbutu (Salacia reticulata WIGHT). J Nat Prod. 2008 Jun;71(6):981-4.
• [8] Clinical pipeline report, company report or official report of Spark Therapeutics
• [9] ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health.
• [10] 5-(4-(4-Acetylphenyl)piperazin-1-ylsulfonyl)indolin-2-one Analogs as Inhibitors of Acid alpha-Glucosidase for Potential Chaperone Treatment of Pompe Disease or Intervention for Diabetes Mellitus Type 2. N.A.. N.A.