Phase 2 Trial of Captisol-Enabled, Propylene Glycol-Free Melphalan for Multiple Myeloma Patients Undergoing Autologous Transplantation

Ligand Pharmaceuticals announced results from a Phase 2 study of Captisol-enabled, propylene glycol-free (PG-free) melphalan in multiple myeloma patients undergoing autologous transplantation. The study compared the safety, tolerability and pharmacokinetic profiles of the PG-free melphalan intravenous (IV) formulation and the current clinically-used IV formulation of melphalan (Alkeran for Injection; GSK).

There were 24 patients treated in the study. The study successfully met all endpoints. The PG-free melphalan was bioequivalent to the current clinically-used IV formulation of melphalan, while also demonstrating a marginally higher systemic drug exposure (112%). PG-free melphalan, administered as half of a high-dose conditioning regimen, resulted in successful myeloablation (100% of patients) and subsequent engraftment (100% of patients) with no additional toxicity.

Captisol-enabled, PG-free melphalan, which has been granted Orphan designation by the FDA as a conditioning treatment for use in autologous transplant for patients with multiple myeloma, is a new IV formulation of melphalan (Alkeran for Injection).

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