Spectrum Pharma Reports Publication of Beleodaq Data Selected as Rapid Communication in JCO

Spectrum Pharmaceuticals SPPI, a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, announced today the publication of results from the pivotal BELIEF (CLN-19) Study, which was selected as a Rapid Communication in the Journal of Clinical Oncology (JCO), the journal of the American Society of Clinical Oncology. The study, led by Dr. Owen O'Connor from the Center for Lymphoid Malignancies, Department of Medicine, Columbia University Medical Center, New York, NY, showed that monotherapy with Beleodaq produced complete and durable responses with manageable toxicity in patients with R/R PTCL across the major subtypes, irrespective of the number or type of prior therapies. Beleodaq, previously known as belinostat, is a histone deacetylase (HDAC) inhibitor indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This indication is approved under accelerated approval based on tumor Response Rate and Duration of Response. An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial. Peripheral T-cell lymphomas are a diverse group of non-Hodgkin lymphomas with a poor prognosis and no accepted standard of care for relapsed or refractory patients. Unfortunately, current treatment options for most of these patients induce responses in only a minority of cases (<30%), and thus long-term survival is relatively poor. The BELIEF study evaluated the efficacy and tolerability of Beleodaq as a single agent in R/R PTCL. This study was an open-label, single-arm, non-randomized, international trial conducted at 62 centers that enrolled 129 patients with R/R PTCL, who had progressed following ≥1 prior therapy with a median number of prior therapies of two (1-8). These patients received Beleodaq (1,000 mg/m2) as daily 30-minute infusions on Days 1-5 every 21 days until disease progression or unacceptable toxicity. The primary endpoint of the BELIEF study was ORR as assessed centrally by an Independent Review Committee using the International Working Group (IWG) criteria. The ORR in the 120 evaluable patients was 25.8% (31 patients) (95% CI 18.3 - 34.6), including 13 Complete Responses (10.8%) (95% CI 5.9 - 17.8) and 18 Partial Responses (15%) (95% CI 9.1 - 22.7). Secondary endpoints included a median DoR of 13.6 months by IWG criteria and 8.4 months to disease progression or death, with the longest ongoing patient at ≥36 months. The most common Grade 3/4 adverse events were anemia (10.8%), thrombocytopenia (7%), dyspnea (6.2%), and neutropenia. No clinically relevant ECG changes were identified, and cardiovascular monitoring of ECGs is not required at baseline or during treatment. In this pivotal study, monotherapy with Beleodaq produced complete and durable responses with manageable toxicity in patients with R/R PTCL across the major disease subtypes, irrespective of the number or type of prior therapies and with a low incidence of Grade 3/4 thrombocytopenia. "We are pleased to have the results of the pivotal Beleodaq study selected for publication as a Rapid Communication in such a prominent journal," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "This is a highly distinguished category that JCO reserves for papers judged to have special impact to their broad clinical readership. Spectrum has a unique PTCL franchise, marketing two FDA approved drugs for this indication, Folotyn® (pralatrexate injection) and Beleodaq. We are very proud to be able to offer patients and clinicians more treatment options with two approved treatments for R/R PTCL." "This is a very exciting time in the treatment of patients with PTCL," said Dr. Owen A. O'Connor, MD, PhD, Director of the Center for Lymphoid Malignancies, Professor of Medicine and Experimental Therapeutics at Columbia Medical Center, New York Presbyterian Medical Center, one of the lead investigators in the BELIEF study. "At long last we finally have tools in the therapeutic armamentarium to help our patients. Belinostat represents the latest drug approved for patients with R/R PTCL that has relatively few side effects and produces long durations of benefit, even in patients who have received multiple conventional treatments in the past. Now that we have several new options to treat the disease when it comes back, we need to use these drugs to make better up-front treatment platforms; Belinostat will be an important part of that future."