From Earlier: Ligand Presents Full Data from Successful Phase II Trial of Captisol-Enabled, Propylene Glycol-Free Melphalan

Ligand Pharmaceuticals Incorporated

announced that results from the Phase II study of its Captisol-enabled, propylene glycol-free (PG-free) Melphalan program were featured this evening in a poster presentation at the combined annual meetings of the Center for International Blood & Marrow Transplant Research (CIBMTR) and the American Society for Blood and Marrow Transplantation (ASBMT) in San Diego. The Phase II study was completed in late 2011 and compared the safety, tolerability and pharmacokinetic profiles of the PG-free Melphalan intravenous formulation and the current clinically-used intravenous formulation of melphalan for multiple myeloma patients undergoing autologous transplantation. The Captisol-enabled product is expected to allow for longer administration durations and slower infusion rates, potentially enabling clinicians to safely achieve a higher dose intensity of pre-transplant chemotherapy, which may lead to better therapeutic outcomes. The key findings and new data presented include: The Phase II study successfully met all endpoints. The new PG-free Melphalan was bioequivalent to Alkeran per guidance requirements, while also demonstrating a marginally higher systemic drug exposure (112%). 37.5% (9 of 24) of the multiple myeloma patients treated in the study were at Stage 2 at study entry and 62.5% (15 of 24) were at Stage 3 at study entry. PG-free Melphalan, administered as half of a high-dose conditioning regimen, clearly resulted in successful myeloablation (100% of patients) and subsequent engraftment (100% of patients) with no additional toxicity. Based on the successful Phase II results, the follow-on Pivotal study will utilize a dosing regimen comparable to Alkeran. The Pivotal study is designed to expose patients exclusively to the PG-free Melphalan formulation and will further elaborate the safety and efficacy measures for the product.