Branaplam

Branaplam
Clinical data
Synonyms	LMI070; NVS-SM1
Identifiers
	IUPAC name (6E)-3-(1H-pyrazol-4-yl)-6-[3-(2,2,6,6-tetramethylpiperidin-4-yl)oxy-1H-pyridazin-6-ylidene]cyclohexa-2,4-dien-1-one;
CAS Number	1562338-42-4;
PubChem CID	89971189;
ChemSpider	34980709;
UNII	P12R69543A;
Chemical and physical data
Formula	C22H27N5O2
Molar mass	393.491 g/mol
3D model (JSmol)	Interactive image;
	SMILES CC1(CC(CC(N1)(C)C)Oc2ccc(nn2)c3ccc(cc3O)c4c[nH]nc4)C;
	InChI InChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24)/b18-17+; Key:YIFFDXMJVNKGBL-ISLYRVAYSA-N; ; InChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24); Key:STWTUEAWRAIWJG-UHFFFAOYSA-N;

Branaplam, also known as LMI070 and NVS-SM1, is a highly potent, selective and orally active small molecule experimental drug being developed by Novartis to treat spinal muscular atrophy (SMA). It is a pyridazine derivative that works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.^[1]^[2]

As of March 2017, branaplam is in a phase-II clinical trial in children with SMA type 1.^[3]^[4]

References

↑ Palacino, James; Swalley, Susanne E; Song, Cheng; Cheung, Atwood K; Shu, Lei; Zhang, Xiaolu; Van Hoosear, Mailin; Shin, Youngah; Chin, Donovan N; Keller, Caroline Gubser; Beibel, Martin; Renaud, Nicole A; Smith, Thomas M; Salcius, Michael; Shi, Xiaoying; Hild, Marc; Servais, Rebecca; Jain, Monish; Deng, Lin; Bullock, Caroline; McLellan, Michael; Schuierer, Sven; Murphy, Leo; Blommers, Marcel J J; Blaustein, Cecile; Berenshteyn, Frada; Lacoste, Arnaud; Thomas, Jason R; Roma, Guglielmo; et al. (2015). "SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice". Nature Chemical Biology. 11 (7): 511. doi:10.1038/nchembio.1837. PMID 26030728.
↑ "LMI070". SMA News Today. Retrieved 2017-03-10.
↑ "An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. Retrieved 2017-03-10.
↑ "Novartis Releases Update on LMI070 (Branaplam) Clinical Trial". CureSMA. 2017-09-20. Retrieved 2017-10-07.

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