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

General Information of Drug (ID: DM4XMZW)

Drug Name

BH4 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Discovery agent	N.A.	Investigative	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Approved Drug(s)

Clinical Trial Drug(s)

Investigative Drug(s)

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Drug(s) Targeting Phenylalanine hydroxylase (PAH)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Sapropterin hydrochloride	DM90TJP	Hyperphenylalaninemia	5C50	Approved	[2]
Pegvaliase	DM6NZ2P	Phenylketonuria	5C50.0	Approved	[3]
Phenylalanine hydroxylase	DMRP3CV	Autoimmune diabetes	5A10	Phase 2	[4]
HepaStem	DM8QGPE	Liver failure	DB99.7	Phase 2	[5]
HMI-102	DM8B85Y	Phenylketonuria	5C50.0	Phase 1/2	[6]
BMN-307	DMY0SKM	Phenylketonuria	5C50.0	Phase 1/2	[7]
fenclonine	DMUBO3E	Discovery agent	N.A.	Investigative	[5]
BMN-168	DM0VMZ4	Phenylketonuria	5C50.0	Investigative	[5]
ALTU-236	DMDLN9S	Genetic disease	8E02	Investigative	[5]
alpha-methylphenylalanine	DMAUH96	Discovery agent	N.A.	Investigative	[8]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Phenylalanine hydroxylase (PAH)	TTGSVH2	PH4H_HUMAN	Modulator	[1]

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References

1	Company report of BioMarin
2	2007 FDA drug approvals: a year of flux. Nat Rev Drug Discov. 2008 Feb;7(2):107-9.
3	Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria.Mol Genet Metab. 2018 Nov;125(3):217-227.
4	Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial. Lancet. 2014 Jul 5;384(9937):37-44.
5	URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 1240).
6	Clinical pipeline report, company report or official report of Homology Medicines
7	ClinicalTrials.gov (NCT04480567) A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With Phenylketonuria. U.S.National Institutes of Health.
8	Alpha-methylphenylalanine, a new inducer of chronic hyperphenylalaninemia in sucling rats. Science. 1976 Jun 4;192(4243):1007-8.