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 October 17, 2016

Alexion Pharmaceuticals released results of an ongoing, open-label extension of the pivotal phase III study for Kanuma (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D), also called Wolman disease. At 52 weeks, nearly all patients (97%) who had received Kanuma from the start of the double-blind period had a rapid and sustained reduction in alanine aminotransferase (ALT), with a mean percent reduction of 53%, and an increase from 31% (11/36) to 45% of patients (15/33) achieving ALT normalization. Similarly, after 52 weeks of Kanuma treatment, nearly all patients (97%) who had initially received placebo during the double-blind period had a reduction in ALT, with a mean percent reduction of 52%, and 48% of patients (14/29) achieving ALT normalization. Sustained improvements were also observed in both groups in markers of lipid abnormalities (including LDL cholesterol, non-HDL cholesterol, triglycerides and HDL cholesterol) through 52 weeks of Kanuma treatment. The safety profile of Kanuma during the extended open-label period was consistent with that observed in the double-blind period. Most treatment-emergent adverse events (TEAEs) were mild to moderate in severity, and no patient discontinued the open-label study because of adverse events. The most common TEAEs in the open-label period were headache (40%), nasopharyngitis (35%), and cough (28%). Twelve patients (18%) experienced an infusion-associated reaction during the open-label period; all but one reaction were mild or moderate in intensity.

Bionomics issued results of a single-center, double-blinded, placebo and lorazepam-controlled, four-way, cross-over, phase II clinical trial of BNC210 conducted in 24 patients with untreated generalized anxiety disorder (GAD). The objective of the study was to evaluate the capacity of BNC210 to engage brain systems relevant to anxiety while resting and in response to anxiety-related tasks. The co-primary endpoints were a change in cerebral perfusion measured by arterial spin labelling and a change in task-related brain activity, specifically in the amygdala as measured by functional Magnetic Resonance Imaging (fMRI) during the Emotional Faces Task (EFT). The results of the study show that BNC210 induced statistically significant changes in cerebral perfusion (300mg BNC210, p<0.05) and also significantly reduced amygdala activation in response to fearful faces during the EFT (300mg BNC210, p<0.05). In comparison, lorazepam exerted a modest suppressive effect on amygdala activation during performance of the EFT (1.5mg lorazepam, p=0.069). A secondary endpoint of the trial was to determine the effect of BNC210 on defensive behaviour using the Joystick Operated Runway Task (JORT) which uses a force-sensing interface to obtain an objective measure of the intensity of threat avoidance motivation. BNC210 administration was associated with a significant decrease in the intensity of threat avoidance behaviour (300mg BNC210, p=0.007; 2,000mg BNC210, p=0.033). BNC210 outperformed lorazepam in this regard (1.5mg lorazepam, p=0.165).

Janssen Research & Development reported results of a phase III study of guselkumab for the treatment of adults with moderate to severe plaque psoriasis. The randomized, double-blind, placebo and active comparator-controlled trial was designed to evaluate the efficacy and safety of guselkumab compared with adalimumab and placebo in adult patients with moderate to severe plaque psoriasis. Patients (n=837) were randomized to receive placebo at weeks zero, four and 12, followed by crossover to guselkumab at weeks 16 and 20 followed by every eight-week dosing; guselkumab 100mg at weeks zero, four and 12, followed by every eight-week dosing; or adalimumab 80mg at week zero and 40mg at week one, followed by every two-week dosing. In the VOYAGE 1 study, the co-primary endpoints were met at week 16, with 85.1% of patients receiving guselkumab 100mg at weeks zero and four and then every eight weeks achieving cleared (Investigator’s Global Assessment [IGA] 0) or minimal disease (IGA 1) compared with 6.9% of patients receiving placebo (p<0.001). Nearly three-quarters of patients receiving guselkumab (73.3%) achieved a PASI 90 response, or near complete skin clearance, compared with 2.9% percent of patients receiving placebo (p<0.001).

Merrimack Pharmaceuticals reported results of a phase III study of Onivyde (irinotecan liposome injection) in combination with fluorouracil (5-FU) and leucovorin for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) following treatment with gemcitabine-based therapy. The overall survival advantage of Onivyde in combination with 5-FU and leucovorin versus 5-FU and leucovorin alone was maintained in this extended analysis: 6.2 months versus 4.2 months (p=0.039, hazard ratio (HR) =0.75, 95% CI: [0.057- 0.99]). The probability of survival at one year was greater in the Onivyde combination arm of the study when compared to the 5-FU and leucovorin arm: 12-month overall survival estimates of 26% (95% CI, 18-35%) were observed in the Onivyde combination treatment arm compared to 16% (95% CI, 10-24%) for 5-FU and leucovorin arm. The overall response rate (ORR) for the Onivyde plus 5-FU and leucovorin arm was 16% versus 1% for the 5-FU and leucovorin arm (p<0.0001). Treatment with Onivyde in combination with 5-FU and leucovorin provided a two-fold improvement in disease control rate compared with 5-FU and leucovorin alone (52% v. 24%, respectively). Final results suggest that patients treated with Onivyde in combination with 5-FU and leucovorin had no notable deterioration in quality of life at 12 weeks despite the addition of a second chemotherapeutic agent to 5-FU and leucovorin. No new safety concerns were noted and the overall safety profile was manageable with the most common grade three adverse events of neutropenia, diarrhea, fatigue, vomiting and asthenia.