Branaplam

Branaplam
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
Synonyms	LMI070; NVS-SM1
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 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 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. PMID 26030728. doi:10.1038/nchembio.1837.
↑ "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.

This article is issued from Wikipedia. The text is licensed under Creative Commons - Attribution - Sharealike. Additional terms may apply for the media files.