Spectrum Pharmaceuticals’ Captisol-Enabled Melphalan Meets Endpoint in Pivotal Trial

Spectrum Pharmaceuticals said Wednesday that its 200 mg/m2 intravenous captisol-enabled melphalan met its primary endpoint of safety and toxicity as a myeloablative therapy prior to autologous stem cell transplant in multiple myeloma patients.

The pivotal, multicenter, Phase II trial also tracked the drug’s response rate and rates of myeloablation and engraftment. A previous study proved the drug bioequivalent to currently marketed intravenous melphalan, Spectrum said.

Aside from the stability and solubility granted by captisol, Spectrum’s formulation of melphalan eliminates propylene glycol, which allows physicians to administer higher doses of the drug over longer periods with less risk of cardiac and renal failure, the company said.

Spectrum’s melphalan was granted orphan drug status for use as a high-dose conditioning regimen prior to hematopoietic progenitor cell transplantation and, if approved, would be the first product approved for this indication, Spectrum claims.

The current intravenous melphalan market is about $130 million annually. Spectrum obtained worldwide rights to develop and commercialize the formulation from Ligand Pharmaceuticals in March 2013, and plans to file an NDA for the drug in the third quarter of this year. — Lena Freund

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