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 August 3, 2015

Allegro Ophthalmics reported results of a phase II trial of Luminate (ALG-1001) in patients with vitreomacular traction (VMT) or vitreomacular adhesion (VMA). The prospective, randomized, double-masked, placebo-controlled trial evaluated Luminate in 106 study subjects. Sixty-five percent of eyes treated with the 3.2mg dose of Luminate achieved release of VMT or VMA by day 90 (end-of-study), compared to 9.7% of those in the placebo control group (p=0.0129). The study, which included three Luminate groups (2mg, 2.5mg or 3.2mg) and a balanced salt solution (BSS) placebo group, also found that Luminate was well tolerated, with no drug toxicity or intraocular inflammation noted with repeated intravitreal injections. Currently in phase II clinical trials for multiple indications, including diabetic macular edema and non-proliferative diabetic retinopathy, Luminate is an investigational drug not approved by the FDA for commercial sale in the U.S.

esoblast reported results of a phase II study of Mesenchymal Precursor Cells (MPCs) for the treatment of congestive heart failure (CHF). The dose-escalation, placebo-controlled study enrolled 60 subjects. Patients treated with the highest dose, MPC 150m, showed the greatest improvement in left ventricular remodeling compared to controls; this was evidenced by significant reductions in left ventricular end systolic volume (LVESV), p=0.015, and left ventricular end diastolic volume (LVEDV), p=0.02, at month six post treatment relative to controls. Patients treated with the highest dose, MPC 150m, showed the greatest improvement in functional exercise capacity compared to controls (6MTW: p=0.062) at month 12 post treatment. In a post-hoc analysis after all patients had completed 36 months of follow up, treatment with MPC 150m was shown to be associated with a significantly lower incidence of heart failure-related major adverse cardiovascular events (HF-MACE) events compared to the control group (0% v. 33% HF-MACE by Kaplan-Meier, p=0.026 by log-rank). A randomized, placebo-controlled, phase III trial using Mesoblast’s high-dose MPC 150m is being conducted by Mesoblast’s development and commercial partner, Teva Pharmaceutical Industries, and is actively enrolling patients across multiple clinical sites in North America.

Ohr Pharmaceutical released results of a phase II trial of OHR-102 (0.2% Squalamine lactate ophthalmic solution) plus Lucentis in patients with macular edema secondary to branch (BRVO) and central retinal vein occlusion (CRVO). The 38-week, investigator-sponsored trial enrolled 20 treatment naïve patients with macular edema due to retinal vein occlusion. All patients received OHR-102 topically for the first 10 weeks of treatment, with two injections of Lucentis given at week two and week six. The results demonstrated that, following an initial 10 week combination therapy treatment period, patients who continued to receive a combination of topical OHR-102 BID plus Lucentis achieved greater visual acuity gains than the control group who received Lucentis alone. At week 38, the mean gain in visual acuity from baseline for patients randomized (at week 10) to treatment with OHR-102 plus Lucentis PRN was +27.8 letters compared with +23.3 for patients randomized to treatment with Lucentis plus PRN alone (control group), a clinically meaningful difference of +4.5 letters. At week 38, 80% of patients in the OHR-102 plus Lucentis treated group had a gain in visual acuity, compared with 50% of patients treated with Lucentis alone. Additionally, at week 38, none of the patients in the OHR-102 plus Lucentis treated group lost any vision. Patients treated with OHR-102 plus Lucentis PRN required a mean of two Lucentis injections between weeks 10 and 38, compared with a mean of 3.3 Lucentis injections for the monotherapy group over the same time period.

Shire issued results of a phase III study of a 39-week, long-term maintenance of efficacy study of Vyvanse (lisdexamfetamine dimesylate) capsules (CII) in adults with moderate to severe binge-eating disorder (B.E.D.). The study consisted of a four-week screening period, a 12-week open-label treatment phase (four weeks of dose optimization and eight weeks of maintenance), and a 26- week, double-blind, randomized-withdrawal phase, as well as a follow-up visit one week after the last on-treatment visit. During the dose-optimization period, all Vyvanse-treated patients were initiated at the 30mg dose, and then titrated in 20mg increments to their optimal dose (either 50mg or 70mg). Patients who met response criteria—one or fewer binge days each week for four consecutive weeks prior to the last visit at the end of the 12-week open-label phase, and had a Clinical Global Impression-Severity (CGI-S) score of two or less at the same visit—were randomized to Vyvanse or placebo treatment groups. During this randomized-withdrawal phase, patients (N=275) received either ongoing treatment with the same optimized dose of Vyvanse from the open-label phase (N=137) or placebo (N=138). Vyvanse demonstrated superiority over placebo (p<0.001) on the primary efficacy endpoint of time to relapse. Additionally, at the conclusion of the study, the group continuing on Vyvanse had a significantly lower proportion of relapse of (5/136, 3.7%) as compared to the placebo group (42/131, 32.1%). Based on these results, as well as findings from a separate 12-month open-label safety extension study, the company plans to submit a supplemental NDA to the FDA by year-end. The FDA will evaluate adding these data to the current labeling for Vyvanse.