15 march 2013
Spectrum Pharmaceuticals has licensed an investigational multiple myeloma drug from Ligand Pharmaceuticals in a deal that could be worth over $50 million.
The treatment in question is an intravenous formulation of melphalan, which is in a Phase III trial for use as a conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma. Spectrum is assuming the responsibility for the trial and hopes to file Captisol-enabled melphalan in the first half of 2014.
The Captisol technology used to reformulate melphalan allows for longer administration durations and slower infusion rates. It has been used with six US Food and Frug Administration-approved products, including Onyx Pharmaceuticals’ multiple myeloma drug Kyprolis (carfilzomib )and Pfizer’s antifungal Vfend (voriconazole).
Melphalan hydrochloride (trade name Alkeran) is a chemotherapy drug belonging to the class of nitrogen mustard alkylating agents.
An alkylating agent adds an alkyl group (CnH2n+1) to DNA. It attaches the alkyl group to the guanine base of DNA, at the number 7 nitrogen atom of the imidazole ring.
Otherwise known as L-Phenylalanine Mustard, or L-PAM, melphalan is a phenylalanine derivative of mechlorethamine.
It is used to treat multiple myeloma and ovarian cancer, and occasionally malignant melanoma.
The agent was first investigated as a possible drug for use in melanoma. It was not found to be effective, but has been found to be effective in the treatment of myeloma.
Oral or intravenous; dosing varies by purpose and route of administration as well as patient weight.
- Facon T, Mary JY, Hulin C, et al. (October 2007). “Melphalan and prednisone plus thalidomide versus melphalan and prednisone alone or reduced-intensity autologous stem cell transplantation in elderly patients with multiple myeloma (IFM 99-06): a randomised trial”. Lancet 370 (9594): 1209–18. doi:10.1016/S0140-6736(07)61537-2. PMID 17920916.