• CE Melphalan is being developed as a novel version of the well-established conditioning treatment in autologous transplant for patients with multiple myeloma. CE Melphalan does not contain propylene glycol, an ingredient in currently available products which has been associated with renal and cardiac side effects.
• In its pivotal trial, CE Melphalan met its primary endpoint. The improved stability profile of CE Melphalan could lead to rapid adoption in the transplant conditioning market.
• The company expects to file an NDA in the 3rd quarter and plans to launch this drug with its existing hematology/oncology sales force next year pending approval.

Henderson, Nevada (Press Release) - Spectrum Pharma­ceu­ticals (NasdaqGS: SPPI), a bio­technology com­pany with fully integrated commercial and drug devel­op­ment operations with a pri­mary focus in Hematol­ogy and Oncology, today announced that its pivotal trial of Captisol-enabled™ (propylene glycol-free) Melphalan met its pri­mary end points.

"I am pleased with the focus and alacrity in which our clin­i­cal team has executed this trial," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharma­ceu­ticals. "With the positive results from this trial, we are one step closer to providing patients and health­care providers with a much needed new formulation of Melphalan. Melphalan is cur­rently a well-established con­di­tioning treat­ment in au­tol­o­gous trans­plant for patients with multiple myeloma. Our novel Captisol-enabled Melphalan is free of propylene glycol, which itself is asso­ci­ated with renal and cardiac side effects. Additionally, the im­proved stability profile of CE Melphalan could be valuable in its rapid adoption in the trans­plant con­di­tioning market. This prod­uct also fits seamlessly into our existing commercial and research infrastructure. With key upcoming catalysts related to CE Melphalan, Beleodaq and SPI-2012, we con­tinue to be committed to build­ing a world-class Hematology/Oncology com­pany."

The phase 2 pivotal trial eval­u­ating CE Melphalan was a multi-center trial eval­u­ating safety and efficacy. The pri­mary objective of the study was to determine the over­all safety and toxicity profile in multiple myeloma patients receiving 200 mg/m² of CE Melphalan as myeloablative ther­apy prior to au­tol­o­gous stem cell trans­plan­ta­tion (ASCT). The sec­ond­ary objectives eval­u­ated the efficacy of CE Melphalan in this patient pop­u­la­tion as measured by multiple myeloma response rate (according to International Myeloma Work­ing Group [IMWG] criteria), and the rates of myeloablation, and engraftment. The pri­mary end­point of this Phase 2 trial was met, and addi­tional analyses are cur­rently underway. The com­pany plans to file an NDA in the third quarter.

In a pre­vi­ous clin­i­cal study, Captisol-enabled Melphalan met the require­ments for estab­lish­ment of bioequivalence to the current commercial in­tra­venous formulation of mel­phalan.

Spectrum Pharma­ceu­ticals gained global devel­op­ment and com­mer­cial­iza­tion rights to CE Melphalan from Ligand Pharma­ceu­ticals Incorporated (NASDAQ: LGND) in March 2013. Spectrum assumed the responsibility for the pivotal clin­i­cal trial and is responsible for filing an NDA. Under the license agree­ment, Ligand received a license fee and is eli­gible to receive mile­stone payments, as well as royalties fol­low­ing poten­tial com­mer­cial­iza­tion.

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 an Orphan Drug Designation by the FDA. This formulation elim­i­nates 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 also im­proves its stability and is antic­i­pated to allow for slower infusion rates and longer admin­istra­tion durations, poten­tially enabling clinicians to safely achieve a higher dose intensity for 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 Merck's NOXAFIL 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 ^[1].

Forward-looking state­ment — This press release may con­tain forward-looking state­ments regarding future events and the future per­for­mance ofSpectrum 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 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 dependence on third parties for clin­i­cal trials, manu­fac­tur­ing, distribution 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. BELEODAQ™, 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.

Source: Spectrum Pharma­ceu­ticals.