Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTSWPH8)

• DTT Name: Growth/differentiation factor 8 (GDF-8)
• Synonyms: GDF8
• Gene Name: MSTN
• DTT Type: Clinical trial target; [1]
• BioChemical Class: Growth factor
• UniProt ID: GDF8_HUMAN
• TTD ID: T87749
• Sequence:
  MQKLQLCVYIYLFMLIVAGPVDLNENSEQKENVEKEGLCNACTWRQNTKSSRIEAIKIQI
  LSKLRLETAPNISKDVIRQLLPKAPPLRELIDQYDVQRDDSSDGSLEDDDYHATTETIIT
  MPTESDFLMQVDGKPKCCFFKFSSKIQYNKVVKAQLWIYLRPVETPTTVFVQILRLIKPM
  KDGTRYTGIRSLKLDMNPGTGIWQSIDVKTVLQNWLKQPESNLGIEIKALDENGHDLAVT
  FPGPGEDGLNPFLEVKVTDTPKRSRRDFGLDCDEHSTESRCCRYPLTVDFEAFGWDWIIA
  PKRYKANYCSGECEFVFLQKYPHTHLVHQANPRGSAGPCCTPTKMSPINMLYFNGKEQII
  YGKIPAMVVDRCGCS
• Function: Acts specifically as a negative regulator of skeletal muscle growth.
• KEGG Pathway: Cytokine-cytokine receptor interaction (hsa04060)
• Reactome Pathway: FOXO-mediated transcription of cell cycle genes (R-HSA-9617828)

Molecular Interaction Atlas (MIA) of This DTT

7 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
ACE-031	DMZ8PY0	Duchenne dystrophy	8C70	Phase 2	[2]
AMG 745	DMFXN2T	Muscle atrophy	FB32.Y	Phase 2	[3]
Domagrozumab	DMCT08V	Duchenne dystrophy	8C70	Phase 2	[4]
LY2495655	DMM5JAN	Disuse muscle atrophy	8B61	Phase 2	[1]
PF-06252616	DMQLZOF	Duchenne dystrophy	8C70	Phase 2	[1]
SAR391786	DMS1YPZ	Muscle atrophy	FB32.Y	Phase 2	[5]
Stamulumab	DMBCIXL	Duchenne dystrophy	8C70	Phase 1/2	[1]

Molecular Expression Atlas (MEA) of This DTT

Disease Name	ICD 11	Studied Tissue	p-value	Fold-Change	Z-score
Myopathy	8A00.0	Muscle tissue	1.64E-01	-0.06	-0.06

References

• [1] Myostatin inhibitors as therapies for muscle wasting associated with cancer and other disorders. Curr Opin Support Palliat Care. 2013 November; 7(4): 352-360.
• [2] A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers. Muscle Nerve. 2013 Mar;47(3):416-23.
• [3] The role of myostatin in muscle wasting: an overview. J Cachexia Sarcopenia Muscle. 2011 September; 2(3): 143-151.
• [4] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [5] Myostatin (GDF-8) as a key factor linking muscle mass and bone structure. J Musculoskelet Neuronal Interact. 2010 Mar;10(1):56-63.