Perifosine

Perifosine (also KRX-0401) is a former drug candidate that was under development for a variety of cancer indications. It is an alkyl-phospholipid^[1] structurally related to miltefosine. Perifosine interrupts the PI3K/AKT/mTOR pathway by acting as an allosteric AKT inhibitor targeting the pleckstrin homology domain of AKT.^[2] It was being developed by Keryx Biopharmaceuticals who had licensed it from Æterna Zentaris Inc.^[3]

Perifosine

Names
IUPAC name 1,1-Dimethylpiperidinium-4-yl octadecyl phosphate
Other names D 21266; KRX 0401
Identifiers
CAS Number	157716-52-4 Y
3D model (JSmol)	Interactive image
ChEMBL	ChEMBL372764
ChemSpider	130628
ECHA InfoCard	100.217.789
IUPHAR/BPS	7424
PubChem CID	148177
UNII	2GWV496552 Y
CompTox Dashboard (EPA)	DTXSID3048946
InChI InChI=1S/C25H52NO4P/c1-4-5-6-7-8-9-10-11-12-13-14-15-16-17-18-19-24-29-31(27,28)30-25-20-22-26(2,3)23-21-25/h25H,4-24H2,1-3H3
SMILES [O-]P(=O)(OCCCCCCCCCCCCCCCCCC)OC1CC[N+](C)(C)CC1
Properties
Chemical formula	C25H52NO4P
Molar mass	461.668 g·mol−1
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa). Infobox references

In 2010, perifosine received orphan drug status in the U.S. for the treatment of multiple myeloma and neuroblastoma, and for multiple myeloma in the EU.^[4] However, both were later withdrawn.^[5][6]

In 2011 it was in a phase III trial for colorectal cancer,^[7] and another for multiple myeloma.^[4][8] On April 2, 2012, it was announced that perifosine failed its phase III clinical trial for treatment of colon cancer.^[9] Detailed results were released in June 2012.^[10] On March 11, 2013 Aeterna Zentaris announced the discontinuing of Phase 3 clinical trial of perifosine for the treatment of relapsed and refractory multiple myeloma.^[11]