Branaplam

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Branaplam
Branaplam skeletal.svg
Clinical data
SynonymsLMI070; NVS-SM1
Identifiers
CAS Number
PubChem CID
ChemSpider
UNII
Chemical and physical data
FormulaC22H27N5O2
Molar mass393.491 g/mol
3D model (JSmol)

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[edit]

  1. ^ 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.
  2. ^ "LMI070". SMA News Today. Retrieved 2017-03-10.
  3. ^ "An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. Retrieved 2017-03-10.
  4. ^ "Novartis Releases Update on LMI070 (Branaplam) Clinical Trial". CureSMA. 2017-09-20. Retrieved 2017-10-07.