SAGE-324

SAGE-324
Clinical data
Other names	BIIB124
Legal status
Legal status	Investigational;
Identifiers
	IUPAC name 1-[(3R,5S,8R,9R,10S,13S,14S,17S)-3-Hydroxy-3-(methoxymethyl)-13-methyl-2,4,5,6,7,8,9,10,11,12,14,15,16,17-tetradecahydro-1H-cyclopenta[a]phenanthren-17-yl]-2-(5-methyltetrazol-2-yl)ethanone;
CAS Number	1832596-09-4;
PubChem CID	118548467;
Chemical and physical data
Formula	C24H38N4O3
Molar mass	430.593 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CC1=NN(N=N1)CC(=O)[C@H]2CC[C@@H]3[C@@]2(CC[C@H]4[C@H]3CC[C@@H]5[C@@H]4CC[C@@](C5)(COC)O)C;
	InChI InChI=1S/C24H38N4O3/c1-15-25-27-28(26-15)13-22(29)21-7-6-20-19-5-4-16-12-24(30,14-31-3)11-9-17(16)18(19)8-10-23(20,21)2/h16-21,30H,4-14H2,1-3H3/t16-,17-,18+,19+,20-,21+,23-,24+/m0/s1; Key:QILSKKMYIGWGLH-YRTSKKSESA-N;

SAGE-324, also known as BIIB124, is an experimental drug. It is a neurosteroid that works as a GABA_A receptor positive allosteric modulator.^[1]^[2]^[3]

SAGE-324 was being developed by Biogen for the treatment of essential tremor.^[4] Its development was discontinued in 2024 due to lack of efficacy in Phase 2 clinical trials.^[5]

References

^ Cerne, Rok; Lippa, Arnold; Poe, Michael M.; Smith, Jodi L.; Jin, Xiaoming; Ping, Xingjie; Golani, Lalit K.; Cook, James M.; Witkin, Jeffrey M. (June 2022). "GABAkines – Advances in the discovery, development, and commercialization of positive allosteric modulators of GABAA receptors". Pharmacology & Therapeutics. 234: 108035. doi:10.1016/j.pharmthera.2021.108035. PMC 9787737.
^ Witkin, Jeffrey M.; Lippa, Arnold; Smith, Jodi L.; Jin, Xiaoming; Ping, Xingjie; Biggerstaff, Andrew; Kivell, Bronwyn M.; Knutson, Daniel E.; Sharmin, Dishary; Pandey, Kamal P.; Mian, Md Yeunus; Cook, James M.; Cerne, Rok (February 2022). "The imidazodiazepine, KRM-II-81: An example of a newly emerging generation of GABAkines for neurological and psychiatric disorders". Pharmacology Biochemistry and Behavior. 213: 173321. doi:10.1016/j.pbb.2021.173321. PMID 35041859. S2CID 245963990.
^ Quagliato, Laiana A.; Carta, Mauro G.; Nardi, Antonio E. (September 2022). "Panic Disorder Seeks More Specific Drugs for Treatment: Might the Amygdala Be the Best Target?". Journal of Clinical Psychopharmacology. 42 (5): 427–428. doi:10.1097/JCP.0000000000001591. PMID 36099401. S2CID 252219658.
^ Ondo, William (1 January 2022). "Chapter Eleven - Enhancing GABA inhibition is the next generation of medications for essential tremor". International Review of Neurobiology. Vol. 163. Academic Press. pp. 317–334. doi:10.1016/bs.irn.2022.02.007. ISBN 9780323899741. PMID 35750368. S2CID 250022192.
^ "Sage Therapeutics and Biogen Announce Topline Results from Phase 2 KINETIC 2 Study of SAGE-324 (BIIB124) for the Treatment of Essential Tremor". Biogen (Press release). 2024-07-24. Retrieved 2024-07-24.

[1] Cerne, Rok; Lippa, Arnold; Poe, Michael M.; Smith, Jodi L.; Jin, Xiaoming; Ping, Xingjie; Golani, Lalit K.; Cook, James M.; Witkin, Jeffrey M. (June 2022). "GABAkines – Advances in the discovery, development, and commercialization of positive allosteric modulators of GABAA receptors". Pharmacology & Therapeutics. 234: 108035. doi:10.1016/j.pharmthera.2021.108035. PMC 9787737.

[2] Witkin, Jeffrey M.; Lippa, Arnold; Smith, Jodi L.; Jin, Xiaoming; Ping, Xingjie; Biggerstaff, Andrew; Kivell, Bronwyn M.; Knutson, Daniel E.; Sharmin, Dishary; Pandey, Kamal P.; Mian, Md Yeunus; Cook, James M.; Cerne, Rok (February 2022). "The imidazodiazepine, KRM-II-81: An example of a newly emerging generation of GABAkines for neurological and psychiatric disorders". Pharmacology Biochemistry and Behavior. 213: 173321. doi:10.1016/j.pbb.2021.173321. PMID 35041859. S2CID 245963990.

[3] Quagliato, Laiana A.; Carta, Mauro G.; Nardi, Antonio E. (September 2022). "Panic Disorder Seeks More Specific Drugs for Treatment: Might the Amygdala Be the Best Target?". Journal of Clinical Psychopharmacology. 42 (5): 427–428. doi:10.1097/JCP.0000000000001591. PMID 36099401. S2CID 252219658.

[4] Ondo, William (1 January 2022). "Chapter Eleven - Enhancing GABA inhibition is the next generation of medications for essential tremor". International Review of Neurobiology. Vol. 163. Academic Press. pp. 317–334. doi:10.1016/bs.irn.2022.02.007. ISBN 9780323899741. PMID 35750368. S2CID 250022192.

[5] "Sage Therapeutics and Biogen Announce Topline Results from Phase 2 KINETIC 2 Study of SAGE-324 (BIIB124) for the Treatment of Essential Tremor". Biogen (Press release). 2024-07-24. Retrieved 2024-07-24.

[1]

[2]

[3]

[4]

[5]