• PDUFA decision expected October 23, 2015, 10 months from NDA filing.
• Approval is being sought for use as a high-dose con­di­tion­ing treatment prior to stem cell transplantation in multiple myeloma and for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.
• Spectrum’s formulation is propylene-glycol free and is more stable with a longer use time, which could simplify clinical administration logistics.
• The Company plans to launch this drug with its existing hematology/oncology sales force pending ap­proval.

Henderson, NV (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 New Drug Application (NDA) for Captisol-Enabled Melphalan (CE-Melphalan), has been accepted by the U.S. Food and Drug Admin­istra­tion (FDA). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of October 23, 2015 for the CE-Melphalan NDA, which is 10 months from the filing date. Spectrum is seeking FDA approval for its use as a high-dose con­di­tion­ing treat­ment prior to au­tol­o­gous hematopoietic (progenitor) stem cell trans­plan­ta­tion (AHCT) in patients with multiple myeloma (MM), an orphan drug desig­na­tion. Spectrum is also seeking approval for the palliative treat­ment of patients with MM for whom oral ther­apy is not appro­pri­ate.

“We are excited the CE-Melphalan filing has been accepted by the FDA, rep­re­senting another important com­pany mile­stone,” said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharma­ceu­ticals. “CE-Melphalan met all pivotal trial end­points, and we ex­pec­t to launch this drug if ap­proved, using our existing sales force to­wards the end of the year. The drug's im­proved solubility and stability should make it an attractive treat­ment option for both trans­plant con­di­tioning and the palliative treat­ment of patients with MM. Eliminating the need for propylene glycol in the preparation of CE-Melphalan elim­i­nates the risk of the toxicities asso­ci­ated with this excipient. In addi­tion, CE-Melphalan's in­­creased stability sim­pli­fies the logistics for pharmacies and nursing staff, and is antic­i­pated to allow for longer infusion times which may permit the admin­istra­tion of higher dose intensities. We antic­i­pate these char­ac­ter­istics of CE-Melphalan will facilitate rapid adoption. We look forward to bringing this drug to market and providing ad­di­tional treat­ment options to patients suffering with cancer.”

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 MM patients receiving 200 mg/m2 of CE-Melphalan as myeloablative ther­apy prior to AHCT. 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. Study results sup­port the safety and efficacy of high-dose CE-Melphalan as a high-dose con­di­tion­ing treat­ment prior to AHCT in patients with MM. CE-Melphalan led to successful myeloablation and sub­se­quent engraftment in all (100%) of the MM patients studied with no mortality or unexpected trans­plant-related toxicity. Overall, 95% of subjects (n=61) responded to high dose CE-Melphalan, and 67% VGPR or better responses were achieved in the subgroup of high risk patients (15%). There were no deaths by Day 100, and the most common Grade 3 and 4 toxicities were the ex­pec­ted hema­to­logic events (neutropenia, leu­ko­penia, lymphopenia, thrombo­cytopenia and anemia). The most frequent non-hematologic adverse events in­cluded diarrhea, nausea, and fatigue. Importantly, the incidence of severe mucositis was low (Grade 3/4; 10%).

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 was responsible for filing the 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 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 five 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; MARQIBO® (vinCRIStine sulfate LIPOSOME injection) for in­tra­venous infusion, for which the Company has world­wide market­ing rights and BELEODAQ® (belino­stat) for Injection in the U.S.. 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.

About Captisol-Enabled Melphalan

Captisol-Enabled, Propylene Glycol -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 need to use propylene glycol con­taining custom diluent, which has been reported to cause renal and cardiac side effects, which in turn 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.

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 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®, MARQIBO®, and BELEODAQ® 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.

Source: Spectrum Pharma­ceu­ticals.