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 August 21, 2017

Bristol-Myers Squibb announced topline results from the CheckMate -214 trial investigating Opdivo (nivolumab) in combination with Yervoy (ipilimumab) versus sunitinib in intermediate and poor-risk patients previously untreated advanced or metastatic renal cell carcinoma. CheckMate -214 is a randomized, open-label study evaluating the combination of Opdivo plus Yervoy versus sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma. Patients in the combination group received Opdivo 3mg/kg plus Yervoy 1mg/kg every three weeks for four doses followed by Opdivo 3mg/kg every two weeks. Patients in the comparator group received sunitinib 50mg once daily for four weeks, followed by two weeks off before continuation of treatment. Patients were treated until progression or unacceptable toxic effects. The primary endpoints of the trial are progression-free survival, overall survival and objective response rate in an intermediate to poor-risk patient population (approximately 75% of patients). The combination met the co-primary endpoint of objective response rate (ORR) and achieved a 41.6% ORR versus 26.5% for sunitinib. Median duration of response was not reached for the combination of Opdivo and Yervoy and was 18.17 months for sunitinib. While there was an improvement in progression-free survival (PFS) (HR=0.82, [95% CI 0.64 – 1.05]; stratified 2-sided p=0.03), it did not reach statistical significance. The median PFS was 11.56 months (95% CI 8.71 – 15.51) for the Opdivo and Yervoy combination versus 8.38 months (95% CI 7.03-10.81) for sunitinib. The study will continue as planned to allow the third co-primary endpoint of overall survival to mature. The tolerability profile observed in CheckMate-214 was consistent with that observed in previously reported studies of this dosing schedule.

Lexicon Pharmaceuticals released results of a phase III study of sotagliflozin for type 1 diabetes. The study, known as inTandem2, was a double-blind, placebo-controlled, multicenter study of 782 patients in Europe and Israel with type 1 diabetes on insulin pump or multiple daily injection therapy who had an A1C level entering the study between 7.0% and 11.0%. The three-arm study evaluated two doses of sotagliflozin, 200mg and 400mg, each taken once daily before the first meal of the day, against placebo. Prior to randomization, insulin was optimized for all patients over a six-week period, with the objective of improving glycemic control using insulin alone. After completion of this optimization period, patients were maintained on optimized insulin and randomized to one of two doses of sotagliflozin or placebo, and their baseline, post-optimization A1C was measured. The mean baseline A1C levels after the six-week optimization period were 7.79%, 7.74% and 7.71% for patients randomized to the placebo, 200mg and 400mg arms, respectively. The primary endpoint of the study was a change in A1C from baseline after a 24-week period of treatment. The trial had a double-blind long-term extension of 28 weeks, with a total treatment duration of 52 weeks. There were 258 patients in the placebo arm, 261 patients in the 200mg dose arm and 263 patients in the 400mg dose arm. The overall mean placebo-adjusted A1C reduction at week 24 was 0.37% in the 200mg dose arm (p<0.001) and 0.35% in the 400mg dose arm (p<0.001). The A1C benefit achieved with sotagliflozin was sustained with statistically-significant results over the full 52-week duration of the study for both the 200mg and 400mg doses. Sotagliflozin was generally well-tolerated during the study. Across all three dose arms (placebo, 200mg, 400mg), over the full 52 weeks of treatment, the incidences of AEs were 61.2%, 68.2% and 68.8%, respectively; the incidences of SAEs were 6.6%, 10.0% and 8.0%, respectively; and discontinuations due to AEs were 3.5%, 3.8% and 6.8%, respectively. There were two deaths in the study in the placebo arm and no deaths in either sotagliflozin arm.

Ophthotech announced that the pre-specified primary endpoint of mean change in visual acuity at 12 months was not achieved in its phase III clinical trial investigating Fovista (pegpleranib) anti-PDGF therapy in combination with Eylea (aflibercept) or Avastin (bevacizumab) anti-VEGF therapy compared to Eylea or Avastin monotherapy for the treatment of wet age-related macular degeneration (AMD). The addition of 1.5mg of Fovista to an Eylea or Avastin regimen did not result in benefit as measured by the mean change in visual acuity at the 12-month time point. This clinical trial (also known as OPH1004) was an international, multicenter, randomized, double-masked, controlled study. In the OPH1004 trial, subjects receiving Fovista in combination with Eylea or Avastin therapy gained a mean of 9.42 letters of vision on the ETDRS standardized chart at 12 months, compared to a mean gain of 9.04 ETDRS letters in patients receiving Eylea or Avastin monotherapy, a resulting difference of 0.38 ETDRS letters (p=0.74). The results for the pre-specified primary efficacy analysis were not statistically significant. In addition, the company did not observe any clinically meaningful visual benefit in the pre-specified secondary endpoints when Fovista was added to Eylea or Avastin regimen. Based on these data, the company has decided to stop treating patients who are in the second year of the OPH1004 study. The phase III trial enrolled approximately 640 patients with wet AMD.