Ligand and Spectrum Pharmaceuticals Announce Specialty Formulation of Melphalan for Multiple Myeloma

Using Captisol technology, scientists have developed a more stable formulation of melphalan that may facilitate higher-intensity regimens in patients with multiple myeloma undergoing autologous stem cell transplant procedures.

On May 9, 2014, Ligand Pharmaceuticals and partner Spectrum Pharmaceuticals announced favorable results with its Captisol-enabled (propylene glycol-free) formulation of Melphalan in treatment of multiple myeloma (MM).

Melphalan, which was approved in 1964, has been used as a treatment for multiple myeloma for over 40 years, and is still widely used in conditioning regimens before autologous stem cell transplant in patients with MM.

In a phase II clinical trial, patients with MM received melphalan at a dose of 200 mg/m² before receiving an autologous stem cell transplant. Although the results remain unpublished, in a press release, representatives of Ligand and Spectrum stated that phase II trials met their primary end point goals, which included measures of adverse events, and treatment-related mortality (for more information, see ClinicalTrials.gov entry NCT01660633).

Captisol technology helps improve the stability of melphalan, allowing for slower rates of infusion, longer drug administration times, and higher-intensity regimens. The drug delivery technology is already used in 6 existing products in current clinical use, and 30 additional products incorporating the Captisol delivery technology are in development.

In a press release, Ligand’s President and Chief Executive Officer, John Higgins stated, “We congratulate the team at Spectrum Pharmaceuticals on their success in running a thoughtful pivotal trial with a favorable outcome.”

Submission of the new drug application is expected to occur in the third quarter of 2014.