Branaplam

Branaplam
Clinical data
Other names	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 g·mol−1
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 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^[update], branaplam is in a phase I–II clinical trial in children with SMA type 1.^[3]

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.

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