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 June 20, 2016

Array BioPharma issued results of a phase III trial of binimetinib in patients with advanced NRAS-mutant melanoma. The trial enrolled 402 patients randomized 2:1 to receive continuous 45mg BID binimetinib or 1,000 mg/m2 dacarbazine dosed every three weeks. The study found binimetinib significantly extended median progression-free survival (PFS), the study’s primary endpoint, at 2.8 months, as compared with 1.5 months observed with dacarbazine [hazard ratio (HR)=0.62 (95% CI 0.47-0.80), p<0.001] the first trial to ever meet a PFS endpoint in patients with advanced NRAS-mutant melanoma. In the pre-specified subset of patients who received prior treatment with immunotherapy, patients who received binimetinib experienced 5.5 months of median PFS (95% CI, 2.87.6), compared with 1.6 months for those receiving treatment with dacarbazine (95% CI, 1.52.8). Based on the strength of these data, the company plans to submit a regulatory filing for binimetinib in NRAS-mutant melanoma later this month.

Avanir Pharmaceuticals released results of an open-label, multicenter study of NUEDEXTA (dextromethorphan hydrobromide/quinidine sulfate) for Alzheimer’s disease (AD) and other dementias, stroke and traumatic brain injury (TBI). PRISM II was designed to evaluate the safety, tolerability and effectiveness of NUEDEXTA capsules that contains 20mg dextromethorphan hydrobromide (DM) and 10mg quinidine sulfate (Q). The nationwide, open-label study enrolled 367 patients at 74 study centers. The Center for Neurologic Study Lability Scale (CNS-LS) score improved significantly from a mean of 20.5 at baseline to 12.8 (p<0.001) at the 90-day endpoint, which is consistent with results seen with NUEDEXTA in the phase III trial. PBA episodes were reduced by 72.6% (p<0.001) compared to baseline at the 90-day endpoint. PBA episode counts over the seven days prior to study visit decreased from a median of 12 at baseline to two at the 90-day endpoint; 35.5% of patients experienced no PBA episodes at the 90-day endpoint. The adverse event (AE) profile in PRISM II was consistent with the known safety profile of NUEDEXTA. The most frequently occurring AEs were diarrhea (5.4%), headache (4.1%), urinary tract infection (2.7%) and dizziness (2.5%); 9.8% of patients had AEs that led to discontinuation. Serious AEs were reported in 6.3% of patients; however, none were considered to be related to NUEDEXTA treatment.

Eli Lilly and Incyte released results of two phase III trials of baricitinib compared to methotrexate or adalimumab (Humira) in patients with rheumatoid arthritis (RA). In the RA-BEGIN trial, nearly 600 patients were randomized to one of the following treatment groups: once-weekly oral methotrexate monotherapy, 4mg once-daily oral baricitinib monotherapy and 4mg once-daily oral baricitinib in combination with once-weekly oral methotrexate. At 24 weeks, 81% of patients receiving baricitinib monotherapy and 79% of patients receiving baricitinib plus methotrexate had clinically meaningful improvement in physical function compared with 70% among those receiving methotrexate alone (p<0.05). Clinically meaningful improvement was defined as an improvement in the Health Assessment Questionnaire-Disability Index (HAQ-DI) score of 0.22. At 52 weeks, 68% of patients on baricitinib monotherapy and 72% of patients on baricitinib in combination with methotrexate saw clinically meaningful improvements in physical function compared to 57% of those treated with methotrexate alone (p<0.05). RA-BEAM was a 52-week trial of 1,305 patients who had active, moderate-to-severe RA, despite ongoing treatment with methotrexate. Patients were randomized to placebo once-daily (n=488), baricitinib 4mg once-daily (n=487) or adalimumab 40mg biweekly (n=330). All patients received background methotrexate. At week 24, patients taking placebo were crossed over to the baricitinib treatment group. At 12 weeks, 75% of patients treated with baricitinib reported clinically meaningful improvement in physical function compared with 71% of patients on adalimumab (p=0.302). Clinically meaningful improvement was defined as an improvement in HAQ-DI score of 0.22. At 24 weeks, 73% of patients treated with baricitinib reported clinically meaningful improvement in physical function compared with 64% of patients on adalimumab (p<0.05). At 52 weeks, 68% of patients treated with baricitinib reported clinically meaningful improvement in physical function compared with 58% of patients on adalimumab (p<0.01).