Keryx Provides Updates On Phase 1/2 Data For Perifosine

Keryx Biopharmaceuticals (Keryx) has provided updated efficacy and safety data as well as new survival data on the clinical activity of KRX-0401 (perifosine) in combination with Bortezomib (Velcade) (+/- dexamethasone) in patients with relapsed/refractory multiple myeloma.

In the trials 84 patients with relapsed/refractory multiple myeloma were enrolled in a combined Phase 1/2 study (18 patients in the Phase 1 component and 66 patients in the Phase 2 component). The patients enrolled were pre-treated with a median of 5 prior lines of therapy (range 1 – 13).

100% of patients had been treated with Bortezomib (55% of the patients were previously treated with at least two Bortezomib-based therapies (range 1 – 4) and 81% were previously treated with Bortezomib plus Dexamethasone).

Reportedly, Overall Response Rate (ORR), partial or minor response (CR, PR or MR), was the primary endpoint, with Time to Progression (TTP), Progression-Free Survival (PFS), Overall Survival (OS) and safety as secondary endpoints.

The company said that approximately 60% of patients demonstrated progression at some point in their treatment and received 20mg Dexamethasone, four times per week, in addition to Perifosine plus Bortezomib. Responses occurred both with patients taking Perifosine in combination with Bortezomib and with patients receiving the combination plus Dexamethasone.

The study result demonstrated that 5 patients have achieved an initial response on Perifosine + Bortezomib alone, and subsequently responded again with the addition of Dexamethasone. 3 additional patients achieved stable disease on Perifosine + Bortezomib alone, and subsequently achieved stable disease again with the addition of Dexamethasone.

Ron Bentsur, CEO of Keryx, said: “We wish to thank the impressive team of multiple myeloma investigators in the Phase 1/2 trial, led by Richardson and Anderson. The updated overall response rate of 41%, an extended PFS, and median overall survival of 25 months particularly in a patient population where 73% were previously refractory to Bortezomib, is encouraging. We look forward to starting the Phase 3 trial by year-end.”

Keryx has been granted a Special Protocol Assessment (SPA) from the FDA for the Phase 3 study of perifosine in multiple myeloma. Additionally, the FDA has granted Perifosine Orphan Drug and Fast Track designations in this indication.