Biogen Announces Five-Year Results from ENDORSE Phase 3 Study Show TECFIDERA Provides Strong, Sustained Efficacy for RRMS

Today Biogen Idec BIIB announced that five-year results from the ENDORSE Phase 3 extension study show TECFIDERA^® (dimethyl fumarate) provides strong and sustained efficacy in a broad range of people living with relapsing-remitting multiple sclerosis (RRMS). These data will be presented at the Sixth Triennial Joint Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis and the European Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS-ECTRIMS). Across all patients in the ENDORSE study who received TECFIDERA, including some patients who were treated for up to seven and a half years, the safety profile remained consistent with no new or worsening safety signals. Additional analyses in patients who were newly diagnosed with multiple sclerosis (MS) when the parent studies DEFINE and CONFIRM began indicate that TECFIDERA had a robust long-term effect on MS relapse rates, disability progression and MRI measures in these patients. “TECFIDERA continues to provide patients with effective oral treatment for MS that is supported by a growing body of data reinforcing its benefits and favorable safety profile,” said Alfred Sandrock, M.D., Ph.D., group senior vice president and chief medical officer at Biogen Idec. “These new ENDORSE results provide further insight into the positive impact of using TECFIDERA early in the course of MS and for long-term treatment of this chronic disease.” ENDORSE Clinical Efficacy and MRI Outcomes ENDORSE is a global, dose-blind extension study evaluating the long-term safety and efficacy of TECFIDERA (240 mg, dosed twice a day [BID] or three times a day [TID]). Patients who received up to two years of TECFIDERA in the pivotal Phase 3 DEFINE and CONFIRM studies continued on the same dose in ENDORSE. Patients who previously received placebo or glatiramer acetate (GA; 20 mg subcutaneous daily injection; CONFIRM only) in DEFINE and CONFIRM were randomized 1:1 to TECFIDERA BID or TID. At five years (two years in DEFINE or CONFIRM and three years in ENDORSE), interim results show that patients who continued on TECFIDERA BID treatment experienced sustained clinical efficacy on relapse and disability progression endpoints as measured by annualized relapse rate (ARR) and 24-week Expanded Disability Status Scale (EDSS), similar to what was observed after two years in DEFINE and CONFIRM. These patients also maintained a low frequency of brain lesions over five years as measured by new or enlarging T2-hyperintense lesions, new non-enhancing T1-hypointense lesions and gadolinium-enhanced [Gd+] lesions. These data will be presented in poster presentations on Thursday, Sept.11 at 3:30 p.m. ET: * Five-Year Follow-up of Delayed-Release Dimethyl Fumarate in RRMS: Integrated Clinical Efficacy Data from the DEFINE, CONFIRM, and ENDORSE Studies (P110) * Five-Year Follow-up of Delayed-Release Dimethyl Fumarate in Relapsing-Remitting Multiple Sclerosis: MRI Outcomes from DEFINE, CONFIRM, and ENDORSE (P059) Clinical Efficacy in Newly Diagnosed Patients An analysis of data from ENDORSE evaluated the long-term efficacy of TECFIDERA in newly diagnosed patients, defined as those diagnosed within one year prior to enrolling in DEFINE or CONFIRM and either disease modifying treatment-naïve or previously treated with corticosteroids alone. Over the five-year observation period, newly diagnosed patients taking TECFIDERA BID experienced sustained reductions in relapses (measured by ARR and proportion of patients who relapsed), disability progression (measured by 24-week EDSS) and number of brain lesions. These effects are similar to the results reported for the overall ENDORSE study population, supporting the consistent efficacy of TECFIDERA in this subpopulation. These data will be presented in a poster presentation on Thursday, Sept. 11 at 3:30 p.m. ET: * Long-Term Efficacy of Delayed-Release Dimethyl Fumarate in Newly Diagnosed Patients With RRMS: An Integrated Analysis of DEFINE, CONFIRM and ENDORSE (P064) “As shown in the ENDORSE study, TECFIDERA provides consistent outcomes across a broad range of relapsing-remitting MS patients, including those who are newly diagnosed, highlighting its utility in the range of patients we see in practice,” said Ralf Gold, M.D., professor and chair of the Department of Neurology at St. Josef-Hospital, Ruhr-University in Bochum, Germany. “The long-term efficacy of TECFIDERA in reducing key measures of disease activity and its favorable safety profile help support its role as an important therapeutic option for people living with MS.” Effect of TECFIDERA on No Evidence of Disease Activity Another analysis from ENDORSE evaluated the long-term effects of TECFIDERA on the emerging measure of No Evidence of Disease Activity (NEDA) over five years. Patients were assessed annually and were considered to have NEDA if they had: no relapses, no 24-week confirmed disability progression, no Gd+ lesions, and no new or enlarging T2-hyperintense lesions. Results over five years show that the proportion of patients achieving NEDA annually was maintained in patients who continued on TECFIDERA, and was improved in patients who switched from placebo to treatment with TECFIDERA BID in the ENDORSE study. These data will be presented in a platform presentation on Friday, Sept. 12 at 9:00 a.m. ET: * Long-term Follow-up of the Effect of Delayed-Release Dimethyl Fumarate on No Evident Disease Activity in Patients with Multiple Sclerosis (FC3.5) ENDORSE Safety The safety profile of TECFIDERA observed in ENDORSE was consistent with the favorable findings reported in the DEFINE and CONFIRM studies. There were no new or worsening safety findings observed in patients who continued treatment with TECFIDERA from DEFINE and CONFIRM or in patients who switched to TECFIDERA therapy after the conclusion of the pivotal studies. All patients in ENDORSE had received treatment with TECFIDERA for at least five years if previously on TECFIDERA in DEFINE or CONFIRM, including some patients who had received TECFIDERA for up to seven and a half years cumulatively. Patients previously on placebo in DEFINE or CONFIRM had received TECFIDERA in ENDORSE for at least three years. The most common adverse events in patients who switched to TECFIDERA from placebo or GA were flushing and gastrointestinal (GI) events, the incidences of which were generally similar to what was observed in DEFINE and CONFIRM. In patients continuing on TECFIDERA therapy, the most common adverse events were MS relapse and nasopharyngitis (common cold). In patients continuing on, or new to, TECFIDERA treatment there was no overall increased risk of serious infections (including opportunistic infections) or malignancies. There was no overall increased risk of renal dysfunction, urinary events or hepatic adverse events in any treatment group; and mean white blood cell and lymphocyte counts remained stable. These data will be presented in a poster presentation on Thursday, Sept. 11 at 3:30 p.m. ET: * Long-term Follow-up of the Safety of Delayed-Release Dimethyl Fumarate in RRMS: Interim Results From the ENDORSE Extension Study (P066)