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 May 30, 2016
Allergan and Gedeon Richter issued results from one of two pivotal phase III clinical trials evaluating the efficacy and safety of ulipristal acetate in women with uterine fibroids. The study was a multicenter, randomized, double-blind, placebo-controlled clinical trial in premenopausal women between 18 and 50 years old with cyclic (22 to 35 days) abnormal uterine bleeding in four of the last six menstrual cycles. Eligible patients were randomized 1:1:1 to ulipristal acetate 5mg, 10mg or placebo for one 12-week treatment course followed by a 12- week treatment-free follow-up period. The study included 157 patients, with 101 patients randomized to ulipristal acetate 5 and 10mg and 56 to placebo. The study met all the co-primary and secondary endpoints with both ulipristal treatment arms achieving statistically significant results over placebo (p<0.0001). The co-primary efficacy endpoints were percentage of patients with absence of uterine bleeding and time to absence of uterine bleeding. Significantly more patients in the 10mg group (58.3%; p<0.0001) and the 5mg group (47.2%; p<0.0001) achieved absence of bleeding compared to placebo (1.8%). A new drug application for the treatment of uterine fibroids is planned to be submitted in 2017.
Encore Vision released results of a phase I-II study of EV06 ophthalmic solution 1.5% for the treatment of presbyopia. The randomized, double-masked, multicenter study examined the safety and efficacy of EV06 compared to placebo. A total of 75 subjects between the ages of 45 and 55 with distance corrected near visual acuity (DCNVA) worse than 20/40 and best corrected distance visual acuity (BCDVA) of 20/20 or better in each eye were randomized 2:1 to receive one drop of EV06 (n=50) or placebo (n=25) twice daily over 90 days. The mean change in DCNVA and BCDVA was evaluated throughout the study. The study met both primary safety and efficacy outcomes. A significant improvement of DCNVA from baseline was observed in the EV06 group compared to placebo, with onset of DCNVA improvement beginning at day 15 (p=0.017) and continuing throughout the 90-day study period (p=0.005). EV06 outperformed placebo in objective and subjective measures throughout the study duration. EV06 was well-tolerated. Encore intends future clinical studies of EV06.
Pfizer released results of two phase III studies demonstrating the immunogenicity of TRUMENBA (Meningococcal Group B Vaccine) against invasive meningococcal B (MnB) strains representative of prevalent strains in the U.S. and Europe. One phase III study was a randomized, active-controlled, observer-blinded study that included approximately 3,600 healthy individuals 10 through 18 years of age. Individuals were randomized to receive one of three different lots of TRUMENBA in a zero, two, six-month schedule or a control, licensed hepatitis A (HAV) vaccine, at zero and six months and saline at two months. The hSBA responses one month after doses two and three against the four primary MnB test strains as defined by hSBA responses (titers ≥LLOQ) were 64.0%-99.1% and 87.1%-99.5%, respectively. As these four primary strains are representative, they predict the ability of antibodies elicited by TRUMENBA to be active against diverse circulating strains. The hSBA responses to the 10 additional MnB test strains were 61.1%-100.0% and 75.1%-98.6% one month after dose two and three, respectively. The second phase III study was a randomized, placebo-controlled, observer-blinded study that included approximately 3,300 healthy individuals 18 through 25 years of age. Individuals were randomized to receive TRUMENBA in a zero, two, six-month schedule or a saline control. The hSBA responses one month after dose two and three among TRUMENBA recipients against the four primary MnB test strains as defined by hSBA responses (titers ≥LLOQ) were 68.3%-97.4% and 87.4%-99.4%, respectively. The hSBA responses to the 10 additional MnB test strains were 51.6%-97.9% and 71.3%-99.3% one month after dose two and three, respectively. TRUMENBA is currently approved in the U.S. These data support additional upcoming global regulatory submissions and the planned U.S. supplement to request the conversion of Accelerated Approval to Traditional Approval for TRUMENBA.
RegeneRx Biopharmaceuticals issued results of a phase II/III trial of RGN-259 for the treatment of dry eye. The 317-patient trial demonstrated statistically significant improvements in both signs and symptoms of dry eye with 0.05% and 0.1% RGN-259 compared to placebo in a dose dependent manner during a 28-day dosing period. While the primary outcome measures were not met, several key related pre-specified endpoints and subgroups of patients with more severe dry eye showed statistically significant treatment effects. On the final day of dosing (day 28), patients receiving 0.1% RGN-259 had a statistically significant reduction in ocular discomfort during Controlled Adverse Environment (CAE) Model exposure when compared to placebo (Intent-to-Treat Population (ITT), p=0.043). Importantly, this result was also observed in the previous phase II trial in patients treated with 0.1% RGN-259 (ITT, p=0.024), thereby demonstrating a symptom endpoint in two independent trials. A statistically significant ocular discomfort improvement after CAE exposure on day 28 was also observed in the 0.05% and 0.1% RGN-259 treatment arms when compared to placebo (ITT, p=0.0366 and p=0.0072, respectively) indicating a dose dependent response. Efficacy in an environmental setting was also demonstrated in more symptomatic patients at baseline, with statistically significant improvements in ocular discomfort observed at day 28 prior to CAE in patients receiving 0.05% and 0.1% RGN-259 compared to placebo (p=0.022 and p=0.006, respectively). The company plans to conduct a confirmatory phase III study to start by the fourth quarter of 2016.