Volanesorsen

Volanesorsen is a triglyceride-reducing drug. It is a second-generation[2] 2’-O-methoxyethyl (2’-MOE) chimeric antisense therapeutic oligonucleotide (ASO)[3] that targets the messenger RNA for apolipoprotein C3 (apo-CIII).

Volanesorsen
Clinical data
Other namesISIS 304801, ISIS-APOCIIIRx
Routes of
administration
Subcutaneous injection
ATC code
  • None
Identifiers
CAS Number
PubChem CID
ChemSpider
UNII
KEGG
Chemical and physical data
FormulaC230H320N63O125P19S19
Molar mass7165.05 g·mol−1

It is in phase III clinical trials for the treatment of hypertriglycidemia, familial chylomicronemia syndrome and familial partial lipodystrophy.[4][5]

The drug was discovered and developed by Ionis Pharmaceuticals.

Chemistry

The complete sequence of volanesorsen is:[1]:165–166

3’—A*—G*—mC*—T*—T*—dmC—dT—dT—dG—dT—dmCdmC—dA—dG—dmC—T*—T*—T*—A*—T*—5’
* = 2’-O-(2-methoxyethyl)
m = 5-methyl
d = 2’-deoxy
Chemical structure[1]:165–166

Names

Volanesorsen is the INN.[1]

References

  1. "International Nonproprietary Names for Pharmaceutical Substances (INN). Recommended International Nonproprietary Names: List 75" (PDF). World Health Organization. Retrieved 18 March 2017.
  2. Pechlaner, R; Tsimikas, S; Yin, X; Willeit, P; Baig, F; Santer, P; Oberhollenzer, F; Egger, G; Witztum, JL; Alexander, VJ; Willeit, J; Kiechl, S; Mayr, M (21 February 2017). "Very-Low-Density Lipoprotein-Associated Apolipoproteins Predict Cardiovascular Events and Are Lowered by Inhibition of APOC-III". Journal of the American College of Cardiology. 69 (7): 789–800. doi:10.1016/j.jacc.2016.11.065. PMC 5314136. PMID 28209220.
  3. Graham, MJ; Lee, RG; Bell TA, 3rd; Fu, W; Mullick, AE; Alexander, VJ; Singleton, W; Viney, N; Geary, R; Su, J; Baker, BF; Burkey, J; Crooke, ST; Crooke, RM (24 May 2013). "Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans". Circulation Research. 112 (11): 1479–1490. doi:10.1161/CIRCRESAHA.111.300367. PMID 23542898.
  4. Taylor, Nick Paul (20 December 2016). "Ionis' volanesorsen hits efficacy endpoint in phase 3". www.fiercebiotech.com. Retrieved 22 December 2016.
  5. Digenio, Andres; Dunbar, Richard L.; Alexander, Veronica J.; Hompesch, Marcus; Morrow, Linda; Lee, Richard G.; Graham, Mark J.; Hughes, Steven G.; Yu, Rosie; Singleton, Walter; Baker, Brenda F.; Bhanot, Sanjay; Crooke, Rosanne M. (August 2016). "Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes". Diabetes Care. 39 (8): 1408–1415. doi:10.2337/dc16-0126. PMID 27271183.
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