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 April 27, 2015

Icon Bioscience issued results from a phase III trial of IBI-10090 for treatment of inflammation post cataract surgery. The study was a prospective, randomized, double-masked, placebo-controlled, multicenter clinical trial involving 394 patients consisting of both men and women aged 40 years and older, undergoing unilateral cataract surgery by phacoemulsification with posterior chamber intraocular lens implantation. Three treatment arms included placebo and IBI-10090 at 342μg or 517μg placed into the anterior chamber of the eye at the conclusion of cataract surgery. Patients were followed for 90 days postoperatively. The primary endpoint in the clinical trial was the percentage of patients achieving anterior chamber cell (ACC) clearing at day 8, i.e., ACC=0. Secondary outcome measures included anterior chamber flare (ACF) and anterior chamber cell + flare (ACCF) clearing in the study eye. Ocular and non-ocular adverse events were assessed. In both drug treatment arms, the percentage of patients with an ACC=0 grade at day eight was statistically significant compared to placebo. Specifically, the percentage of patients with ACC clearing at day eight was 63.1% and 66% in the 342μg and 517μg treatment groups, respectively, (P<0.001) compared to 25% in the placebo group. The percentage of patients with ACF clearing at day eight was 92.4%, and 89.1%, respectively, in the 342μg and 517μg treatment groups (P<0.001) compared to 63.8% in the placebo group. The percentage of patients with ACCF clearing at day eight was 63.1% and 67.3% in the 342μg and 517μg treatment groups, respectively, (P<0.001) compared to 33.8% in the placebo group. Additionally, no ocular serious adverse events were reported up to day 90 and adverse events among the three groups were similar.

Novartis reported results of a phase III study of Arzerra (ofatumumab) plus chlorambucil in patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy was considered inappropriate, mainly due to advanced age or the presence of comorbidities. This prospective, randomized, open-label, phase III study included 447 patients with previously untreated CLL. Patients were randomized 1:1 to treatment with up to 12 cycles of ofatumumab in combination with chlorambucil or up to 12 cycles of chlorambucil alone. In the study, median PFS was improved by 71% in the group receiving ofatumumab plus chlorambucil compared to the chlorambucil alone group (22.4 months v. 13.1 months, respectively; HR 0.57 [95% CI 0.45, 0.72]; p<0.0001). Improvement in PFS was observed in most subgroups irrespective of age, gender, disease stage and prognostic factors. Additionally, patients in the combination arm also experienced significantly longer TTNT when compared to chlorambucil alone (39.8 months v. 24.7 months, respectively; HR 0.49 [95% CI: 0.36, 0.67]; p<0.0001). Patients in the combination arm had a higher ORR (82% of patients v. 69% of patients, respectively; odds ratio 2.16 [95% CI: 1.36-3.42]; p=0.001), with a better CR rate (14% of patients v. 1% of patients, respectively). Compared to those on chlorambucil alone, patients in the combination arm had a duration of response of 22.1 months v. 13.2 months (HR 0.56 [95% CI: 0.43, 0.74]; p<0.001). More patients in the group receiving ofatumumab plus chlorambucil (50%) experienced adverse events (AEs) of grade three or greater compared to chlorambucil alone (43%), with neutropenia being the most common adverse event (26% v. 14%). Grade 3/4 infusion-related reactions (IRRs) were reported in 10% of patients receiving ofatumumab plus chlorambucil, leading to drug withdrawal in 3% of patients and hospitalization in 2% of patients. No fatal IRRs were reported. These phase III data formed the basis for regulatory approvals in the U.S. and E.U. in 2014.

Ultragenyx Pharmaceutical released results of an investigator-sponsored trial of triheptanoin (UX007) for the treatment of movement disorders associated with glucose transporter type-1 deficiency syndrome (Glut1 DS). The open-label, investigator-sponsored trial enrolled eight Glut1 DS patients between seven and 47 years old with non-epileptic paroxysmal manifestations, a neurological symptom characterized by sudden, transient, involuntary movements. Patients completed comprehensive diaries to record all motor and non-motor events as well as a clinical global impression scale during a baseline, treatment, and withdrawal phase, each lasting two months. Six patients completed the study. During the baseline phase without triheptanoin, patients experienced on average 31 paroxysmal manifestations, including 16 dystonic events. During the triheptanoin treatment phase, patients reported a statistically significant 90% reduction in these events to an average of three paroxysmal manifestations, including two dystonic events (p=0.028). In the withdrawal phase, when triheptanoin treatment was discontinued, the rate of paroxysmal manifestations increased substantially to an average of 24 events per patient, including 12 dystonic events (p=0.043). In addition, patients reported an improvement in the clinical global impression scale during the treatment phase with triheptanoin and a worsening after withdrawal. This improvement also was associated with a normalization of induction of brain energy metabolism during visual stimulation in patients receiving triheptanoin as measured by 31P-NMR spectroscopy (p=0.021). Triheptanoin was well-tolerated in all patients. Two patients were considered not compliant for reasons unrelated to safety or tolerability. Ultragenyx intends to initiate a confirmatory randomized double-blind placebo-controlled study of triheptanoin in the Glut1 DS movement disorder phenotype and anticipates discussions with the FDA on final study design details in the second half of 2015.