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Skeletal formula of perifosine
Space-filling model of the perifosine zwitterion
IUPAC name
1,1-Dimethylpiperidinium-4-yl octadecyl phosphate
Other names
D 21266; KRX 0401
3D model (JSmol)
ECHA InfoCard 100.217.789
Molar mass 461.67 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

Perifosine (also KRX-0401) is a drug candidate being developed for a variety of cancer indications. It is an alkyl-phospholipid[1] structurally related to miltefosine. It acts as an Akt inhibitor and a PI3K inhibitor. It was being developed by Keryx Biopharmaceuticals who have licensed it from Æterna Zentaris Inc.[2]

In 2010 perifosine has reached phase II.[3] In one phase II trial for metastatic colon cancer perifosine doubled time to progression.[2]

It has orphan drug status in the U.S. for the treatment of multiple myeloma and neuroblastoma, and for multiple myeloma in the EU.[4]

In 2011 it was in a phase III trial for colorectal cancer,[5] and another for multiple myeloma.[4][6] On April 2, 2012, it was announced that perifosine failed its phase III clinical trial for treatment of colon cancer.[7] Detailed results were released in June 2012.[8] 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