Spectrum Pharmaceuticals Announces Publication of Pivotal EVOMELA™ (melphalan hydrochloride) for Injection Data in the Biology of Blood and Marrow Transplantation Journal

Spectrum Pharmaceuticals, Inc. SPPI, a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, today announced publication of results from the pivotal clinical study for EVOMELA, used for myeloablative conditioning in multiple myeloma (MM) patients undergoing autologous transplantation. (ASCT). The study, led by Dr. Parameswaran Hari from Froedtert Hospital and Medical College of Wisconsin, was published in the Biology of Blood and Marrow Transplantation (BBMT) journal. "We are pleased to have these clinical data selected for publication in the BBMT journal," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "These study data confirm the efficacy and acceptable safety profile of EVOMELA as a high-dose conditioning regimen for ASCT in patients with MM. Our novel EVOMELA formulation uses Captisol to improve the solubility and stability of Melphalan, and has eliminated the need for a propylene glycol-containing cosolvent. Importantly, this allows for longer use and infusion times with EVOMELA, which potentially simplifies its clinical use and administration logistics. Instead of propylene glycol, which is associated with toxicities including renal dysfunction and arrhythmias, this new formulation uses a standard aqueous diluent for reconstitution. We look forward to FDA's NDA decision on EVOMELA in October. Spectrum continues to deliver on its commitment to develop improved cancer therapies that benefit patients and health care providers." The BBMT journal publication includes data on 61 patients who were enrolled in this open-label Phase 2b pivotal study at five US study sites; 56 patients had newly diagnosed disease and five had relapsed MM following prior ASCT. Patients enrolled in this study received 200 mg/m2 of EVOMELA as two doses on Day -3 and Day -2 prior to ASCT (Day 0). Efficacy was assessed by clinical response at Day +100 with an ORR of 95% and CR rate of 31% (16% stringent CRs) based on investigators' assessments, and rates of 100% and 21%, respectively based on independent pathology review; the lower rate of confirmed CRs in the independent review was due to missing data. Importantly, the five patients who had previously relapsed from a prior ASCT were all shown to achieve a response to EVOMELA. All patients in the study achieved myeloablation with a median of 5 days post-ASCT, and all patients had successful neutrophil and platelet engraftment (median of 12 days and 13 days post-ASCT, respectively). Treatment-related mortality was 0%, and non-hematologic adverse events were mostly Grade 1 and Grade 2 in severity. The incidence of Grade 3 mucositis and Grade 3 stomatitis were 10% and 5%, respectively with no Grade 4 mucositis or stomatitis reported. Twenty percent of patients experienced treatment-emergent serious adverse events, most of which were Grade 3, and consisted of events commonly reported in patients undergoing myeloablative chemotherapy; no new safety signals were identified. In December 2014, Spectrum submitted an NDA to the FDA for the approval of EVOMELA for use as a high-dose conditioning treatment prior to ASCT in patients with MM. Spectrum is also seeking approval for the palliative treatment of patients with MM for whom oral therapy is not appropriate, which is the indication for the currently approved IV melphalan products. The NDA was accepted and a PDUFA decision is expected on October 23, 2015.