Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of November 20, 2017

Boehringer Ingelheim issued results from two new analyses of the phase III RE-VERSE AD study, which evaluated the safety and efficacy of idarucizumab, marketed in the U.S. as Praxbind, in reversing the anticoagulant effect of Pradaxa (dabigatran etexilate mesylate) in patients in diverse emergency situations, including those with acute bleeding or requiring an urgent surgery or procedure, such as an orthopedic, vascular or abdominal procedure. Mortality and thromboembolic event rates for patients with a GI bleed were consistent with the overall findings of RE-VERSE AD. One of the subanalyses focused on patients with GI bleeding, the most common type of bleeding event among patients who were enrolled in the study due to acute bleeding. Of the 137 patients enrolled with a GI bleed, complete reversal of the anticoagulant effect of Pradaxa was observed in more than 95% of patients. The median time to cessation of bleeding post-idarucizumab administration was less than three hours when the location of the GI bleed was known and 6.4 hours when the location was unknown. In the subanalysis, as presented in the abstract, at 90 days, there were low thromboembolic event rates of 5.1% in patients with a GI bleed, compared to 6.3% in non-GI bleed patients. Mortality in the first five days following administration was 6% in patients with a GI bleed, compared to 9% in non-GI bleed patients. At 90 days, mortality rates were 16% and 23%, respectively.

Puma Biotechnology reported results of a phase III trial of neratinib in patients with early stage HER2-positive breast cancer. The ExteNET trial was a double-blind, placebo-controlled, phase III trial of neratinib versus placebo after adjuvant treatment with trastuzumab (Herceptin). The ExteNET trial randomized 2,840 patients in 41 countries with early stage HER2-positive breast cancer who had undergone surgery and adjuvant treatment with trastuzumab. After completion of adjuvant treatment with trastuzumab, patients were randomized to receive extended adjuvant treatment with either neratinib or placebo for a period of one year. Patients were then followed for invasive recurrent disease, ductal carcinoma in situ (DCIS), or death for a period of five years after randomization in the trial. The primary endpoint of the trial was invasive disease free survival (iDFS). The results of the trial demonstrated that after a median follow up of 5.2 years, treatment with neratinib resulted in a 27% reduction of risk of invasive disease recurrence or death versus placebo (hazard ratio=0.73, p=0.008). The five-year iDFS rate for the neratinib arm was 90.2% and the five-year iDFS rate for the placebo arm was 87.7%. Neratinib was approved by the FDA in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy, and is marketed in the U.S. as NERLYNX (neratinib) tablets.

Sage Therapeutics announced positive top-line results from two phase III clinical trials with its proprietary i.v. formulation of brexanolone (USAN; formerly SAGE-547) in postpartum depression (PPD). The Hummingbird program included two phase III multicenter, randomized, double-blind, parallel-group, placebo-controlled trials (Study 202B and Study 202C). Trial participants in 202B were required to have a Hamilton Rating Scale for Depression (HAM-D) score of 26 or above prior to treatment. These patients were randomized to one of three treatment groups (brexanolone 90μg/kg/hour, brexanolone 60μg/kg/hour, or placebo) on a 1:1:1 basis. Trial participants in 202C were required to have a HAM-D score of between 20 and 25 prior to treatment. These patients were randomized to one of two treatment groups (brexanolone 90μg/kg/hour or placebo) on a 1:1 basis. Brexanolone achieved the primary endpoint in both trials, a mean reduction from baseline in the HAM-D total score compared to placebo at 60 hours (Study 202B: p=0.0242 for 90µg/kg/h dose and p=0.0011 for 60µg/kg/h dose; Study 202C: p=0.0160 for 90µg/kg/h dose). Patients treated with brexanolone demonstrated mean reductions from baseline in HAM-D total scores of 14 to 20 points at 60 hours maintained to 30 days in both trials. Brexanolone was generally well-tolerated and showed a similar safety profile as seen in earlier studies.