The U.S. Food and Drug Admin­istra­tion has approved a Phase 3 clin­i­cal trial eval­u­ating perifosine in re­lapsed and refractory multiple myeloma patients. Perifosine, also known as KRX-0401, is an oral anti-cancer agent being devel­oped by Aeterna Zentaris and licensed in the U.S., Canada, and Mexico by Keryx Bio­pharma­ceuticals.

The trial, entitled “A Phase 3 Randomized Study to Assess the Efficacy and Safety of Perifosine Added to the Combination of Bortezomib and Dexamethasone in Multiple Myeloma Patients Previously Treated with Bortezomib,” will eval­u­ate the safety and efficacy of perifosine, com­pared to placebo, when added to a Velcade (bor­tez­o­mib) and dexamethasone (Decadron) regi­men.

To par­tic­i­pate, patients must have already received treat­ment with Velcade and either Revlimid (lena­lido­mide) or thalidomide (Thalomid). Though the study is not yet enrolling, the trial will involve approx­i­mately 400 patients. As its pri­mary objective, the study will assess whether perifosine extends pro­gres­sion-free survival, and it will also eval­u­ate over­all response rate, survival, and safety.

In pre­vi­ous clin­i­cal trials, perifosine has dem­onstrated sig­nif­i­cant anti-cancer activity. Acting through a novel mech­a­nism, perifosine inhibits the (PI3K)/Akt cellular path­way - thereby preventing can­cer­ous cell growth and maturation. In addi­tion, it regulates a number of other key cellular processes, in­clud­ing the JNK and MAPK path­ways, which are asso­ci­ated with pro­grammed cell death, growth, dif­fer­en­tiation and survival. Through these multiple effects, perifosine helps inhibit tumor growth and en­cour­ages cancer cells to die.

"Perifosine has shown con­siderable activity when com­bined with [Velcade] and dexa­meth­a­sone in heavily pre-treated patients with re­lapsed and refractory dis­ease,” explained Dr. Paul Richardson, the clin­i­cal director of the Jerome Lipper Multiple Myeloma Center, Dana Farber Cancer Institue, and the study chairman for the upcoming trial. “We are hopeful that the upcoming Phase 3 trial will dem­onstrate clin­i­cal benefit for our patients with less ad­vanced myeloma."

For more in­for­ma­tion, see the Keryx Bio­pharma­ceuticals and Aeterna Zentaris press releases announcing the Phase 3 trial approval.