In the phase 2 study, 37 patients were treated with Perifosine 150mg daily for six cycles. Over 41% of the patients had three or more lines of prior therapy and 78% had two or more prior lines of therapy. Such prior therapies include nucleoside analogues, bortezomib, alkylating agents and rituximab, which are not approved for, but are often used in the treatment of Waldenstrom’s.
The median percent involvement of the bone marrow with lymphoplasmacytic cells was 70%, indicating advanced disease. Stable or responding patients were allowed to continue therapy until progression.
Reportedly, of the 37 patients, 11% achieved a partial response, 24% achieved a minimal response, and 54% showed stable disease. Overall, 89% of patients treated with single agent Perifosine were reported to have stable disease or better, while 11% demonstrated progression. The median progression-free survival in the study was 12.6 months, with a median overall survival of 26 months. Perifosine was generally well-tolerated.
Ron Bentsur, chief executive officer of Keryx, said: “We are very excited to see this single agent activity of Perifosine, which further demonstrates its potential as a targeted agent. This Waldenstrom’s data is of particular interest because, similar to multiple myeloma, Waldenstrom’s is also a bone-marrow-based hematologic tumor.
“Moreover, Waldenstrom’s represents an unmet medical need for which there are no approved drugs and therefore could potentially provide us with an additional registration strategy for Perifosine. Finally, we wish to thank Ghobrial and her impressive team of investigators for their dedication to this Waldenstrom’s program.”