Captisol-enabled® Propylene Glycol-Free Melphalan

Ligand's Captisol-enabled® melphalan program is a new IV formulation of melphalan that has the potential to offer multiple advantages for clinicians and patients in the multiple myeloma transplant setting. Ligand's formulation completely avoids the use of propylene glycol, which is used as a co-solvent in the current formulation of melphalan (sold as Alkeran® for Injection), and has been reported to cause renal and cardiac side-effects that limit the ability to deliver higher quantities of intended therapeutic compounds. The use of Captisol® technology to reformulate melphalan is anticipated to allow for longer administration durations and slower infusion rates, potentially enabling clinicians to safely achieve a higher dose intensity of per-transplant chemotherapy.

The Captisol-enabled Melphalan was granted Orphan Drug status by the FDA as a conditioning treatment for use in autologous transplant for patients with multiple myeloma. Ligand's Propylene Glycol-Free (PG-Free) formulation of melphalan will be the first product approved for use as a high-dose conditioning regimen prior to stem cell transplantation.

Projected Timing

Multiple Myeloma (MM)

 MM is an incurable blood cancer caused by overgrowth of abnormal plasma cells, manifesting as tumors in the bone marrow
 More than 50,000 patients in the U.S. suffer from MM with an estimated 20,000 new cases diagnosed each year
 Approximately 11,000 people died from MM in 2010

Autologous Stem Cell Transplantation

Autologous Stem Cells are stem cells that are harvested from a patient's own blood or bone marrow.  High-dose chemotherapy is used prior to transplant of healthy stem cells in multiple myeloma patients in order to destroy the diseased cells in the bone marrow.  After completion of the chemotherapy, the healthy stem cells are infused back into the patient to take over the job of making new blood cells.  High-dose chemotherapy combined with stem-cell transplantation improves the overall survival in patients with Multiple Myeloma.