Spectrum Pharmaceuticals Announces Agreement with FDA on the Special Protocol Assessment (SPA) for the Upcoming Apaziquone Phase 3 Trial in Patients with Non-Muscle Invasive Bladder Cancer (NMIBC)

Spectrum Pharmaceuticals, Inc.

, a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology, announced today that the company has reached agreement with the U.S. Food and Drug Administration (FDA) on the Special Protocol Assessment (SPA) of the planned Phase 3 clinical trial of its novel, potent pro-drug, apaziquone. This trial will further evaluate the intravesical use of apaziquone for the treatment of patients with non-muscle invasive bladder cancer (NMIBC) as one or two instillations, immediately following transurethral resection of bladder tumor (TURBT). "Spectrum's agreement with the FDA on the SPA represents a significant milestone for bladder cancer patients," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "The learnings from previous Phase 3 studies and comments from the FDA have been incorporated in the new protocol to improve the chances of success. We look forward to initiating this trial and filing the apaziquone NDA by year-end. This NDA is based on data from the previously completed program that included two Phase 3 studies with a total of 1,615 patients. We believe there continues to be a significant unmet need for these patients, as no drugs have been approved in the U.S. for more than 40 years for the treatment of low-grade NMIBC. Due to the high rate of recurrence, the overall cost of the treatment of bladder cancer in the U.S. is a staggering $3.4 billion annually, most of which is related to direct treatment of the disease. We endeavor to bring this much needed therapy for patients and help reduce overall medical costs at the same time." In accordance with the SPA, the Phase 3 trial will be a randomized, double-blind, placebo-controlled, multicenter trial that will enroll patients with Ta G1 or G2 NMIBC. The patients will be randomized to receive either one instillation of apaziquone, two instillations of apaziquone, or placebo. The primary endpoint is Time to Recurrence. Since apaziquone is known to be inactivated in presence of blood, the new protocol includes a 30-60 minute waiting period post-TURBT, before apaziquone instillation. Patients that receive two instillations of apaziquone, will receive the second dose approximately two weeks after surgery minimizing the potential for drug inactivation due to bleeding. Further, it is recommended that patients with significant post-operative bleeding not receive apaziquone. Apaziquone is an anticancer pro-drug that is activated by bio-reductive enzymes that are over-expressed in bladder cancer cells, rendering it into a highly cytotoxic alkylating agent. Spectrum has conducted two multi-center, international Phase 3 trials of a single intravesical instillation of apaziquone (4 mg) into the bladder in the immediate post-operative period after surgical resection of low-grade NMIBC. Pooled data from the two studies (n=1,615) showed a statistically significant treatment effect for the primary study endpoint, 2-Year Recurrence Rates, in favor of apaziquone (p-value = 0.0218) and in a key secondary endpoint, Time to Recurrence (p-value = 0.0096).