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news.Keryx Biopharmaceuticals has released Phase 1 data of KRX-0401(Perifosine), a new oral anti-cancer agent that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway, in the pediatric solid tumor.
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Keryx Biopharma has presented the data in the pediatric solid tumor poster discussion session held at the 46th Annual Meeting of the American Society of Clinical Oncology (ASCO) taking place in Chicago, IL.
Keryx Biopharmaceuticals’ Phase I Study of Perifosine for recurrent pediatric solid tumors is a single center, open-label, dose-escalating study to assess safety, tolerability, pharmacokinetics (PK) and to identify any dose limiting toxicity (DLT) of single agent perifosine in pediatric patients with any solid tumor that has failed standard therapy.
Most patients were heavily pretreated, with a median of three prior lines of therapy. Cohorts of three patients were treated at three dose levels: 25mg/m2/day, 50mg/m2/day and 75mg/m2/day using 50mg tablets of Perifosine after a loading dose on day 1, and taking into account the drug’s long half-life (>100hrs).
In the study, of particular interest are the early signs of clinical activity observed in two of the three patients with Stage 4 refractory neuroblastoma. Both patients were refractory to prior treatments upon entering the study and achieved stable disease for 48 weeks and 55+ weeks (ongoing).
The investigators concluded that Perifosine is well-tolerated in children with recurrent solid tumors and that these early signals of activity warrant further investigation in patients with advanced neuroblastoma and select brain tumors.
Keryx Biopharmaceuticals said that recently, NCI investigators published in-vitro and in-vivo data demonstrating that perifosine targets the activation of Akt in neuroblastoma cells and xenografts, inhibits tumor growth in-vivo and improves the survival of mice bearing neuroblastoma tumors.
Ron Bentsur, CEO of Keryx Biopharmaceuticals, said: “We are pleased at the safety and tolerability of single agent perifosine in pediatric patients and encouraged by the early signals of clinical benefit in neuroblastoma, as noted by the investigators.
“We are grateful to the researchers and we look forward to exploring Perifosine’s potential in future pediatric settings, including neuroblastoma.”
Perifosine is currently in Phase 3 clinical development for refractory advanced colorectal cancer and multiple myeloma, both of these Phase 3 programs being conducted under Special Protocol Assessment (SPA) agreements with the FDA with Fast Track designations obtained for both indications. Perifosine is also in Phase 1 and 2 clinical development for several other tumor types.