Therapy Detail
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| Therapy Name | Perifosine |
| Synonyms | |
| Therapy Description |
Perifosine is a synthetic alkylphospholipid that inhibits AKT resulting in apoptotic induction and antitumor activity (PMID: 16418332). |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Perifosine | KRX-0401|NSC-639966 | Akt Inhibitor (Pan) 19 | Perifosine (KRX-0401) is a synthetic alkylphospholipid that inhibits AKT resulting in apoptotic induction and antitumor activity (PMID: 16418332, PMID: 28864978, PMID: 31325145). |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| PIK3CA mutant | ovarian cancer | not predictive | Perifosine | Phase II | Actionable | In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent ovarian cancer, no significant difference in disease control rate (12.5%, 2/16 vs 40%, 2/5, p=0.22), median progression-free survival (HR=0.57, p=0.32), or median overall survival (HR=1.37, p=0.58) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). | 28864978 |
| PIK3CA mutant | cervical cancer | not predictive | Perifosine | Phase II | Actionable | In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent cervical cancer, no significant difference in disease control rate (11.1%, 2/18 vs 25%, 2/8, p=0.56), median progression-free survival (HR=1.06, p=0.81), or median overall survival (HR=1.66, p=0.26) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). | 28864978 |
| PIK3CA mutant | endometrial cancer | not predictive | Perifosine | Phase II | Actionable | In a Phase II trial , Perifosine (KRX-0401) treatment was well-tolerated, resulted in stable disease as best response in patients with recurrent endometrial cancer, no significant difference in disease control rate (47.1%, 8/17 vs 14.3%, 1/7, p=0.19), median progression-free survival (HR=1.71, p=0.30), or median overall survival (HR=0.92, p=0.88) was identified between PIK3CA wild-type and PIK3CA mutant patients (PMID: 28864978). | 28864978 |
| Unknown unknown | astrocytoma | not applicable | Perifosine | Phase II | Actionable | In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but demonstrated limited activity in patients with anaplastic astrocytoma (n=14), with only 1 patient achieved a partial response (PMID: 31325145). | 31325145 |
| Unknown unknown | Advanced Solid Tumor | not applicable | Perifosine | Phase I | Actionable | In a Phase I trial, Perifosine demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 25183650). | 25183650 |
| Unknown unknown | glioblastoma | no benefit | Perifosine | Phase II | Actionable | In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but did not demonstrate activity in patients with recurrent glioblastoma multiforme, with a 6-month progression-free rate of 0% (0/16), a median progression-free survival of 1.58 months, and a median overall survival of 3.68 months (PMID: 31325145). | 31325145 |
| AKT1 positive | ovarian clear cell carcinoma | predicted - sensitive | Perifosine | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Perifosine (KRX-0401) inhibited Akt1 signaling, inhibited proliferation, and induced apoptosis in human ovarian clear cell cancer cells with activated Akt1 in culture and inhibited tumor growth in cell line xenografts (PMID: 25519148). | 25519148 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| NCT00776867 | Phase I | Perifosine | Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors | Completed | USA | 0 |
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