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news.Aeterna Zentaris has reported that the perifosine, an oral PI3K/Akt inhibitor, demonstrated significant activity against chemo-resistant and radio-resistant neuroblastoma, while allowing good quality of life and sparing vital organs in Phase 1 trial.
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The open-label dose-escalating trial evaluated toxicity and efficacy of perifosine, given in monotherapy to patients with neuroblastoma.
Aeterna Zentaris president and CEO Juergen Engel said the data emphasise perifosine’s anticancer activity, as was the case with the article published in Cancer, outlining perifosine’s anticancer activity in renal cell carcinoma.
"On both occasions, the authors alluded to perifosine’s potential as a combination therapy which further supports our current Phase 3 trial in multiple myeloma in which perifosine is used in combination with bortezomib and dexamethasone," Engel added.
The patients were dosed with 50mg tablets and a loading dose (100-200mg/m2) of perifosine on the first day, followed by daily maintenance doses (50-75mg/m2) until progressive disease or dose limiting toxicity.
Perifosine demonstrated anti-neuroblastoma activity with a 50% progression-free survival rate at 12 months and included one complete remission depending on a normalized MIBG scan and three patients with improved MIBG scan and normalized bone marrow histology over prolonged follow-up.
The study reported that perifosine, which was well tolerated without major toxicity, might have a possible role with chemotherapy, radiation therapy, and/or other agents active in PI3K/Akt pathway.