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 23, 2016

Envisia Therapeutics reported an interim three-month analysis of an ongoing safety and efficacy evaluation of the low dosage form of ENV515 XR (travoprost) for sustained reduction in intraocular pressure (IOP). The second cohort of the ongoing phase II trial was a 12-month safety and efficacy evaluation of the low dosage form of ENV515 that was designed as an open-label trial that enrolled five glaucoma patients at sites within the U.S. The low dosage form of ENV515, administered once on day one, achieved the interim efficacy endpoint in this three-month analysis, time-matched 8 a.m. IOP over the three-month post-dose period, with -7.1mmHg or -27% change from IOP baseline that was comparable to topical timolol 0.5% twice daily with -7.4mmHg or -28% change from IOP baseline, administered to the non-study eye. ENV515 was well-tolerated and there were no serious adverse events, no changes in corneal endothelial cell counts evaluated by an independent reading center and no changes in corneal thickness. The most common adverse event was early-onset transient hyperemia, or eye redness, related to the dosing procedure.

F Hoffmann-La Roche, Genentech issued results of a phase II trial of tocilizumab for systemic sclerosis. The randomized, double-blind, placebo-controlled trial enrolled 87 subjects: 43 assigned to tocilizumab and 44 assigned to placebo. Patients were randomly assigned (1:1) to weekly subcutaneous tocilizumab 162mg or placebo. The least squares mean change in modified Rodnan skin score at 24 weeks was −3.92 in the tocilizumab group and −1.22 in the placebo group (difference −2.70, 95% CI −5.85 to 0.45; p=0.0915). The least squares mean change at 48 weeks was −6.33 in the tocilizumab group and −2.77 in the placebo group (treatment difference −3.55, 95% CI −7.23 to 0.12; p=0.0579). In one of several exploratory analyses, fewer patients in the tocilizumab group than in the placebo group had a decline in percent-predicted forced vital capacity at 48 weeks (p=0.0373). Forty-two (98%) of 43 patients in the tocilizumab group versus 40 (91%) of 44 in the placebo group had adverse events. Fourteen (33%) versus 15 (34%) had serious adverse events. Serious infections were more common in the tocilizumab group (seven [16%] of 43 patients) than in the placebo group (two [5%] of 44). One patient died in relation to tocilizumab treatment.

Melinta Therapeutics has released results a phase III study of Baxdela (delafloxacin) for acute bacterial skin and skin structure infections (ABSSSI). The pivotal study, the second of a phase III program, was a randomized, double-blind study. Patients in the Baxdela arm received 300mg of intravenous (IV) Baxdela every 12 hours for six doses followed by 450mg oral Baxdela every 12 hours. The recommended vancomycin dose was 15mg/kg of IV vancomycin every 12 hours based on actual body weight plus 1-2g of IV aztreonam every 12 hours. Duration of treatment in either the Baxdela or active control arms was 5-14 days. In the intent-to-treat population (ITT), IV-to-oral Baxdela met the FDA’s primary endpoint of statistical non-inferiority (10% non-inferiority margin) at the early clinical response at 48-72 hours after initiation of therapy (83.7%) compared to IV vancomycin combination therapy with aztreonam (80.6%). The 95% confidence interval for the treatment difference had lower and upper bounds of -2% and 8.3%, respectively. Baxdela also met the EMA’s required endpoint of statistical non-inferiority (57.7%) compared to vancomycin plus aztreonam (59.7%) based on the investigator’s assessment of complete cure (resolution of all baseline signs and symptoms) at the follow up visit in the ITT population. Lower and upper bounds of the 95% confidence interval for the treatment difference were -8.6% and 4.6%, respectively. In addition, Baxdela was comparable to vancomycin plus aztreonam in achieving treatment success at follow up (cure or improved, with no further antibiotics needed) with success rate of 87.2% v. 84.8%, respectively. IV/oral Baxdela monotherapy successfully eradicated Gram-positive pathogens, including MRSA, and Gram-negative pathogens at rates comparable to IV vancomycin/aztreonam combination treatment. Both IV and oral Baxdela were well-tolerated in the study participants. The company anticipates submitting an NDA to the FDA in the second half of this year.

Sunovion Pharmaceuticals reported results of a post-hoc analysis of a long-term safety study evaluating Brovana (arformoterol tartrate) Inhalation Solution in people with moderate-to-severe chronic obstructive pulmonary disease (COPD). Data for this post hoc analysis came from a multicenter, randomized, double-blind, 52-week safety trial. The Brovana group reported a greater reduction in hospitalization risk relative to placebo, and these results were more pronounced in a subset of patients who were responders on the St. George’s Respiratory Questionnaire (SGRQ). For the intent-to-treat population, the adjusted annualized hospitalization rate was significantly reduced with Brovana compared with placebo (relative risk=0.60 [95% confidence interval (CI)=0.41, 0.87; p=0.007]). For those patients who were SGRQ responders, the adjusted relative risk was the lowest at 0.34 (95% CI=0.12, 0.99; p=0.048); SGRQ non-responders had an adjusted relative risk of 0.81 (95% CI=0.39, 1.67; p=0.573). Brovana is approved by the FDA for the long-term maintenance treatment of bronchoconstriction in people with COPD, including chronic bronchitis and emphysema.