CyDex Pharmaceuticals, Inc. has announced that the FDA has accepted the company’s Investigational New Drug (IND) application for a clinical study of Captisol-Enabled® melphalan HCL (CDX-353). Melphalan is an FDA-approved low dose chemotherapy for multiple myeloma marketed under the brand name Alkeran® by GlaxoSmithKline. In December 2008, CyDex received orphan-drug designation from the FDA for melphalan “as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation.” This designation provides an important economic incentive, granting CyDex seven years of exclusive marketing rights. “IND acceptance for Captisol-enabled melphalan is an important milestone for CyDex as we work to develop a portfolio of new drugs for the hospital acute care market,” said Theron E. Odlaug, CyDex’s president and chief executive officer. “We look forward to establishing a relationship with a strategic out-licensing partner to advance CDX-353 into the clinic and, longer-term, develop and commercialize this promising oncology product.” Upcoming clinical studies are expected to begin with a phase 2(a) trial that compares the pharmacokinetics of CyDex’s CDX-353: Propylene Glycol-Free Melphalan HCL with Alkeran, and evaluate safety parameters. Alkeran is packaged as two separate vials that must be combined prior to injection and, due to its limited stability, administered immediately thereafter. CyDex’s version of melphalan is enabled by Captisol®, the company’s proprietary and patented sulfobutylether -cyclodextrin. Captisol-Enabled® melphalan is a one-vial formulation that does not contain harsh co-solvents and remains stable at room temperature for an extended period of time. “CDX-353 is a novel form of melphalan that is more stable and could potentially allow for longer administration durations and slower infusion rates compared to current pre-transplant treatments for multiple myeloma,” said Parameswaran Hari, M.D., Clinical Director of the Adult Bone Marrow Transplant Program and Associate Professor of Medicine at the Medical College of Wisconsin. “These advantages have the potential to enable doctors to safely achieve a higher dose intensity of pre-transplant chemotherapy, which could lead to better therapeutic outcomes.”
