Trial Results

PQ Bypass Reports Positive 12-Month Results for DETOUR I Trial

July 9, 2018

PQ Bypass announced results from the DETOUR I Trial evaluating the PQ Bypass DETOUR System for percutaneous bypass. They showed promising 12-month durability for patients with extremely long blockages in the superficial femoral artery (SFA). The DETOUR I trial, a prospective, single-arm, multicenter, core lab-adjudicated study, enrolled and treated 77 patients and 81 lesions. Primary and primary-assisted patency at 12 months in all lesions of the DETOUR I trial was 73 percent and 80 percent, respectively. Secondary patency was achieved in 94 percent of patients. Additional safety and effectiveness outcomes include 100 percent freedom from amputation, 99 percent freedom from acute limb ischemia, and Rutherford improvement of ≥ 2 classes in 90 percent of patients. The DETOUR II trial aims to expand the body of evidence evaluating the safety and effectiveness of the DETOUR System to create a percutaneous femoropopliteal bypass. The DETOUR II trial is the first pivotal investigational trial in the United States for percutaneous bypass and is currently enrolling. The trial will enroll up to 292 patients at up to 40 sites across the United States and Europe.

Poxel Announces Completion of Patient Enrollment for TIMES 1 Trial
POXEL SA announced that patient enrollment for the TIMES 1 trial in the Phase III program for Imeglimin, an investigational therapeutic agent for type 2 diabetes, in Japan has been completed. The Imeglimin Phase III registration program includes three pivotal trials to evaluate the efficacy and safety of Imeglimin in approximately 1,100 patients. Referred to as TIMES (Trials of IMeglimin for Efficacy and Safety), the TIMES program includes three trials that will be performed using the dose of 1,000 mg twice daily. TIMES 1 is a Phase III, 24-week, multicenter, double-blind, placebo-controlled, randomized, monotherapy study to assess the efficacy, safety and tolerability of Imeglimin in over 200 Japanese patients with type 2 diabetes, using the change in HbA1c as the primary endpoint. TIMES 2 is a Phase III, 52-week, open-label, parallel-group study to assess the long-term safety and efficacy of Imeglimin in Japanese patients with type 2 diabetes. In this study, Imeglimin will be administrated orally as a monotherapy or combination therapy with existing hypoglycemic agents. TIMES 3 is a Phase III, 16-week, double-blind, placebo-controlled, randomized study with a 36-week open-label extension period to evaluate the efficacy and safety of Imeglimin in combination with insulin in Japanese patients with type 2 diabetes and inadequate glycemic control on insulin therapy.

Alnylam Announces Publication of APOLLO Phase III Clinical Study Results
Alnylam Pharmaceuticals announced that the pivotal study results from the APOLLO Phase III trial of patisiran. The trial enrolled 225 hATTR amyloidosis patients in 19 countries with 39 genotypes who were randomized 2:1, patisiran:placebo, with patisiran administered at 0.3 mg/kg intravenously once every three weeks for 18 months. The APOLLO Phase III trial was a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy. The primary endpoint of the study was the change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) relative to placebo at 18 months. Secondary endpoints included: the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) score; NIS-weakness (NIS-W); Rasch-built Overall Disability Scale (R-ODS); timed 10-meter walk (10-MWT); modified BMI (mBMI); and the composite autonomic symptom score-31 (COMPASS-31). The study showed that patisiran improved measures of polyneuropathy, quality of life, activities of daily living, ambulation, nutritional status and autonomic symptoms relative to placebo in patients with hereditary transthyretin-mediated (hATTR) amyloidosis, an inevitably progressive and generally fatal disease. Patisiran treatment also led to favorable effects on exploratory endpoints related to cardiac structure and function in patients with cardiac involvement. Further, the frequency and severity of adverse events (AEs) were similar in patients receiving patisiran and placebo, with the exception of peripheral edema and infusion-related events which were higher in patisiran-treated patients and generally mild to moderate in severity.

Ipsen and Exelixis Jointly Announce CELESTIAL Phase III Pivotal Trial Results
Ipsen and Exelixis jointly announced the results from the CELESTIAL Phase III pivotal trial of cabozantinib in patients with previously treated advanced hepatocellular carcinoma (HCC). CELESTIAL is a randomized, double-blind, placebo-controlled global Phase III study of cabozantinib versus placebo in patients with advanced hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. The study was conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced hepatocellular carcinoma (HCC)Median OS in CELESTIAL was 10.2 months with cabozantinib versus 8.0 months with placebo (HR 0.76, 95 percent CI 0.63-0.92; p=0.0049). Median progression-free survival (PFS) was more than doubled, at 5.2 months with cabozantinib and 1.9 months with placebo (HR 0.44, 95 percent CI 0.36-0.52; p<0.0001). Objective response rates per RECIST 1.1 were four percent with cabozantinib and 0.4 percent with placebo (p=0.0086). Disease control (partial response or stable disease) was achieved by 64 percent of patients in the cabozantinib group compared with 33 percent of patients in the placebo group.