Trial of Melphalan in Multiple Myeloma Meets Primary Endpoint

A phase II trial of propylene glycol-free (Captisol-enabled) melphalan in patients with multiple myeloma has met its primary endpoint, according to a statement released by the drug manufacturer.

Propylene glycol-free melphalan is an intravenous formulation of melphalan being investigated for the multiple myeloma transplant setting. The drug currently has Orphan Drug Designation by the US Food and Drug Administration. This formulation of melphalan is designed to eliminate the use of propylene glycol, which has been reported to cause renal and cardiac side effects that limit the ability to deliver higher doses of therapeutic compounds.

The phase II, multicenter trial was designed to evaluate the safety and efficacy of propylene glycol-free melphalan. The primary objective of the study was the determine the safety and toxicity of a 200 mg/m² dose of the drug as a myeloablative therapy in patients with multiple myeloma prior to autologous stem cell transplantation.  Spectrum Pharmaceuticals recently announced that the drug formulation has met its phase II trial safety-related primary endpoints.

The secondary objectives evaluated the efficacy of propylene glycol-free melphalan in this patient population as measured by multiple myeloma response rate, according to International Myeloma Working Group (IMWG) criteria, and the rates of myeloablation, and engraftment.

According to a statement by the manufacturer, the use of the Captisol technology to reformulate melphalan also improves its stability and is anticipated to allow for slower infusion rates and longer administration durations, potentially enabling clinicians to safely achieve a higher dose intensity for pretransplant chemotherapy.

Additional analyses of the drug are underway and Spectrum plans to file a New Drug Application in the third quarter of 2014. If approved, it would be the only drug approved as a conditioning treatment for use in autologous transplants for patients with multiple myeloma.