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 September 28, 2015

Intra-Cellular Therapies has issued results of a phase III trial of ITI-007 for schizophrenia. The randomized, double-blind, fixed-dose, placebo-controlled phase III clinical trial was conducted at 12 sites in the U.S. with 450 patients randomized (1:1:1) to receive either ITI-007 60mg or 40mg or placebo once daily in the morning for four weeks. Patients were required to have an acute exacerbation of psychotic symptoms. The pre-specified primary efficacy measure was changed from baseline v. placebo at study endpoint (four weeks) on the centrally rated Positive and Negative Syndrome Scale (PANSS) total score. ITI-007 60mg met the primary efficacy endpoint by demonstrating a statistically significant improvement v. placebo on the PANSS total score, in the intent-to-treat (ITT) study population (least squares [LS] mean change from baseline -14.5 points, effect size [ES]=0.30, p=0.022 v. -10.3 points change from baseline for placebo). ITI-007 showed a dose-related improvement in symptoms of schizophrenia with the 40mg dose approximating the trajectory of improvement seen with the 60mg dose, but the effect with 40mg did not reach statistical significance on the primary endpoint (-12.9 points, ES=0.18, p=0.164). In the trial, once-daily ITI-007 60mg met the primary endpoint and demonstrated antipsychotic efficacy with statistically significant superiority over placebo at week four (study endpoint) as measured by the change from baseline on the PANSS total score (p=0.022). Moreover, ITI-007 60mg showed significant antipsychotic efficacy as early as week one, which was maintained at every time point throughout the study. ITI-007 60mg also met the key secondary endpoint of statistically significant improvement on the Clinical Global Impression Scale for Severity of Illness (CGI-S; p=0.003). Consistent with previous studies, ITI-007 had a favorable safety and tolerability profile as evidenced by motoric, metabolic and cardiovascular characteristics similar to placebo, and no clinically significant changes in akathisia, extrapyramidal symptoms, prolactin, body weight, glucose, insulin or lipids.

Regeneron Pharmaceuticals and Sanofi have released results of a phase III study of Praluent (alirocumab) for heterozygous familial hypercholesterolemia (HeFH) patients to reduce low-density lipoprotein cholesterol (LDL-C). The analysis compared Praluent to placebo in 1,257 patients with HeFH. Data from four phase III ODYSSEY trials were included in the analysis. In those trials, patients either received Praluent or placebo, in addition to standard-of-care, which included maximally tolerated statins with or without other lipid-lowering therapies such as ezetimibe. In ODYSSEY LONG TERM and HIGH FH, patients were treated with Praluent 150mg (n=348) every two weeks administered as a single 1-milliliter (mL) injection or placebo (n=174). In those patients, the average LDL-C at baseline was 168mg/dL and 162mg/dL in the Praluent and placebo groups respectively. In ODYSSEY FH I and FH II, patients were treated with Praluent 75mg (n=490) every two weeks administered as a single 1mL injection or placebo (n=245). In ODYSSEY FH I and FH II, patients had their dose adjusted to 150mg at week 12 if they did not achieve their pre-specified LDL-C goal at week eight. In those patients, the average LDL-C level at baseline was 141mg/dL in both the Praluent and placebo groups. Across all primary and secondary endpoints assessed, there were statistical differences in favor of Praluent compared to placebo. Patients treated with Praluent achieved average LDL-C levels of less than 85mg/dL at week 12 and maintained reductions through 78 weeks of therapy. The effect of Praluent on CV morbidity and mortality has not been determined. In July, the EMA recommended the approval of Praluent in certain adult patients with hypercholesterolemia; a final decision from the European Commission is anticipated in September.

Trevi Therapeutics has reported results of a phase II/III trial of Nalbuphine ER for the treatment of moderate to severe uremic pruritus. The multicenter, randomized, double-blind, placebo-controlled, parallel, three-arm study evaluated Nalbuphine ER tablets dosed twice-daily at 60mg and 120mg in about 370 patients on hemodialysis in the U.S. and Europe. Patients with a wide range of mean moderate-to-severe itch intensity, ranging from 4.5 to 10 on the 10-point Numerical Rating Score (NRS) scale, were evaluated. Patients receiving 120mg of Nalbuphine ER (n=120) experienced a 3.5-point reduction in itch intensity from baseline, resulting in a highly statistically significant mean reduction in itch intensity as compared to placebo (p= 0.017). A statistically significant mean reduction for Nalbuphine ER compared to placebo was observed as early as one week following titration to the Nalbuphine ER fixed dose, and there was a statistically significant separation from placebo throughout the remaining blinded period. Sustained duration of drug effect continued to trend away from placebo through the eighth week of the study. On average, patients entered the study with a baseline mean NRS itch score of 6.9 (NRS considers a score of 7 to be severe), and at the end of the eight-week dosing period, average itch scores had been reduced to an NRS score of 3.4, which is considered mild on the NRS scale. Severe itch patients (those with NRS scores greater than or equal to 7) experienced on average an NRS score reduction of 4.5 points from baseline (p=0.007). The 60mg dose showed a numerically favorable reduction over placebo but did not achieve statistical significance. The company expects the open-label extension study to be completed by year-end.