• Expected NDA filing mid-2014.
• Granted Orphan designation by the FDA as a conditioning treatment for use in autologous transplant for patients with multiple myeloma. If approved, will be the only drug approved for this indication.
• In a previous clinical study, Captisol-enabled melphalan met the requirements for establishment of bioequivalence to the current commercial intravenous formulation of melphalan.

Henderson, NV (Press Release) - Spectrum Phar­ma­ceu­ti­cals (NasdaqGS: SPPI), a bio­technology com­pany with fully integrated commercial and drug devel­op­ment operations and a pri­mary focus in hematology and on­col­ogy, announced today the completion of enrollment for the pivotal trial of Captisol-enabled®, propylene glycol-free (PG-free) high-dose mel­phalan as a con­di­tioning treat­ment prior to au­tol­o­gous trans­plant for patients with multiple myeloma.

"We are very pleased to have the patient enrollment com­pleted ahead of schedule," said Rajesh C. Shro­triya, MD, Chairman, Chief Executive Officer, and Pres­i­dent of Spectrum Phar­ma­ceu­ti­cals. "Melphalan re­mains a high priority for Spectrum; if approved, Captisol-enabled mel­phalan would be the only drug ap­proved as a con­di­tioning agent for stem cell trans­plant in multiple myeloma. This prod­uct will also fit seam­less­ly into our existing commercial and research infrastructure."

The phase 2 pivotal trial is a multi-center trial eval­u­ating safety and efficacy in 60 patients. The pri­mary ob­jective of the study is to determine the over­all safety and toxicity profile in multiple myeloma patients receiv­ing 200 mg/m2 of Melphalan HCl for Injection (Propylene Glycol-Free) as myeloablative ther­apy prior to au­tol­o­gous stem cell trans­plan­ta­tion (ASCT). The sec­ond­ary objective is to eval­u­ate the efficacy of Melphalan HCl (Propylene Glycol-Free) in this patient pop­u­la­tion as measured by multiple myeloma response rate (ac­cord­ing to International Myeloma Work­ing Group [IMWG] criteria), myeloablation, and engraft­ment. Several end­points require 90-100 day follow-up of patients on the trial.

There are an esti­mated 20,000 new cases of multiple myeloma in the United States each year, with an incidence of new cases in­creas­ing by approx­i­mately 1.7% per year.¹ The current in­tra­venous mel­phalan market is approx­i­mately $130 million annually, with predominant use in stem cell trans­plants. The rate of au­tol­o­gous stem cell trans­plants for patients with multiple myeloma is growing by approx­i­mately 3.3% annually.²

About Captisol-Enabled Melphalan

Captisol-enabled, PG-free mel­phalan is a novel in­tra­venous formulation of mel­phalan being in­ves­ti­gated for the multiple myeloma trans­plant setting, for which it has been granted Orphan desig­na­tion by the FDA. This formulation avoids the use of propylene glycol, which has been reported to cause renal and cardiac side effects that limit the ability to deliver higher doses of thera­peutic com­­pounds. The use of the Captisol^® tech­nol­ogy to reformulate mel­phalan is antic­i­pated to allow for slower infusion rates and longer admin­istra­tion durations, poten­tially enabling clinicians to safely achieve a higher dose intensity of pre-transplant chemo­ther­a­py.

About Captisol^®

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella at the University of Kansas' Higuchi Biosciences Center for specific use in drug devel­op­ment and formulation. This unique tech­nology has enabled six FDA-approved prod­ucts, in­­clud­ing Onyx Phar­ma­ceu­ti­cals' Kyprolis^®, Baxter Inter­na­tional's Nexterone^® and Pfizer's Vfend^® IV. There are also more than 30 Captisol-enabled prod­ucts cur­rently in clin­i­cal devel­op­ment.

About Spectrum Phar­ma­ceu­ti­cals, Inc.

Spectrum Phar­ma­ceu­ti­cals is a leading bio­technology com­pany focused on acquiring, devel­op­ing, and com­mer­cial­iz­ing drug prod­ucts, with a pri­mary focus in on­col­ogy and hematology. Spectrum and its affiliates market four on­col­ogy drugs ─ FUSILEV® (levoleucovorin) for Injection in the U.S.; FOLOTYN® (pralatrexate injection), also marketed in the U.S.; ZEVALIN® (ibritumomab tiuxetan) Injection for in­tra­venous use, for which the Company has world­wide market­ing rights and MARQIBO® (vinCRIStine sulfate LIPOSOME in­jec­tion) for in­tra­venous infusion, for which the Company has world­wide market­ing rights. Spectrum's strong track record in in-licensing and acquiring dif­fer­en­ti­ated drugs, and expertise in clin­i­cal devel­op­ment have generated a robust, di­vers­i­fied, and growing pipe­line of prod­uct can­di­dates in ad­vanced-stage Phase 2 and Phase 3 studies. More in­­for­ma­tion on Spectrum is avail­able at www.sppirx.com.

Forward-looking state­ment — This press release may con­tain forward-looking state­ments regarding future events and the future per­for­mance of Spectrum Phar­ma­ceu­ti­cals that involve risks and un­cer­tain­ties that could cause actual results to differ materially. These state­ments are based on man­agement's current beliefs and ex­pec­ta­tions. These state­ments in­clude, but are not limited to, state­ments that relate to our business and its future, in­­clud­ing sales of Spectrum's drug prod­ucts, certain com­pany mile­stones, Spectrum's ability to identify, acquire, develop and com­mer­cial­ize a broad and diverse pipe­line of late-stage clin­i­cal and commercial prod­ucts, leveraging the expertise of partners and employees around the world to assist us in the execution of our strategy, and any state­ments that relate to the intent, belief, plans or ex­pec­ta­tions of Spectrum or its man­agement, or that are not a state­ment of historical fact. Risks that could cause actual results to differ in­clude the possibility that our existing and new drug can­di­dates may not prove safe or effective, the possibility that our existing and new appli­ca­tions to the FDA and other regu­la­tory agencies may not receive approval in a timely manner or at all, the possibility that our existing and new drug can­di­dates, if approved, may not be more effective, safer or more cost efficient than competing drugs, the possibility that our efforts to acquire or in-license and develop addi­tional drug can­di­dates may fail, our lack of sustained revenue history, our limited market­ing ex­peri­ence, our customer con­cen­tra­tion, the possibility for fluctuations in customer orders, evolving market dynamics, our dependence on third parties for clin­i­cal trials, manu­fac­tur­ing, distribution, in­­for­ma­tion and quality control and other risks that are described in further detail in the Company's reports filed with the Securities and Exchange Com­mis­sion. We do not plan to update any such forward-looking state­ments and expressly disclaim any duty to update the in­­for­ma­tion con­tained in this press release except as required by law.

SPECTRUM PHARMACEUTICALS, INC.®, FUSILEV®, FOLOTYN®, ZEVALIN® and MARQIBO® are registered trademarks of Spectrum Phar­ma­ceu­ti­cals, Inc and its affiliates. REDEFINING CANCER CARE™ and the Spectrum Phar­ma­ceu­ti­cals logos are trademarks owned by Spectrum Phar­ma­ceu­ti­cals, Inc. Any other trademarks are the property of their re­spec­tive­ owners.

¹American Cancer Society, Cancer Facts & Figures 2012
² Leerink Swann Market Research

Source: Spectrum Pharma­ceu­ticals.