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 22, 2016

AcelRx Pharmaceuticals reported results of the phase III trial SAP302 of ARX- 04 (sufentanil sublingual tablet, 30mcg) in patients who presented to the emergency room with moderate to severe acute pain associated with trauma or injury. The ARX- 04 program is comprised of three studies in patients with moderate-to-severe acute pain: SAP301, a double-blind, placebo-controlled trial in ambulatory abdominal surgery patients; SAP302, an open-label trial in adult emergency room patients; and SAP303, an open-label trial in postoperative patients. SAP302 enrolled patients in two cohorts. The initial phase enrolled 40 adults who were administered a single dose of ARX-04 and an extension phase enrolled 36 adults who were eligible to receive up to four doses of ARX-04, given hourly as needed for pain. Overall, the 76 adults treated with ARX-04 in this study experienced a mean pain intensity difference to baseline of 2.9 from a baseline of 8.1, or 35%, on a zero to 10 numeric rating scale at 60 minutes. Of these 36 patients, seven received a second dose of ARX-04, and two received a third dose. For 75% of patients in the second cohort, a single dose of ARX04 was sufficient for pain relief and only 8% of patients received morphine in addition to ARX-04. Patients reported a mean pain intensity decrease of 1.1 compared to baseline 15 minutes following first administration of ARX-04, and a decrease of 1.9 after 30 minutes. Overall ARX-04 was well-tolerated in this study, with 79% of patients reporting no adverse events.

Aurinia Pharmaceuticals issued results of a phase IIb trial of voclosporin for active lupus nephritis (LN). The AURA–LV study enrolled 265 patients at centers in over 20 countries worldwide. Patients were randomized to one of two dosage groups of voclosporin (23.7mg BID and 39.5mg BID) or placebo, with all patients also receiving mycophenolate mofetil and oral corticosteroids as background therapy. All patients had an initial IV dose of steroids (500-1000mg) and then were started on 20-25mg/daily, which was tapered down to a low dose of 5mg daily by week eight and 2.5mg daily by week 16. The trial achieved its primary endpoint, demonstrating statistically significantly greater complete remission (CR) (as defined by confirmed urinary protein/creatinine ratio of =0.5mg/mg at 24 weeks and confirmed at 26 weeks) in patients treated with 23.7mg of voclosporin twice daily (p=0.045). Both treatment arms, 23.7mg and 35.9mg twice daily, also showed a statistically significant improvement in the rate of achieving partial remission at 24 weeks (p=0.007; p=0.024). No unexpected safety signals were observed and voclosporin was shown to be well-tolerated. Based on the results of the 24-week analysis, Aurinia plans to meet with the FDA in the fourth quarter of 2016 to discuss these data and the drug’s subsequent clinical development and path to registration in LN.

Indivior released results of a phase III trial of RBP-6000 buprenorphine monthly depot for the treatment of opioid use disorder as part of a complete treatment plan to include counseling and psychosocial support. The multicenter, randomized, double-blind, placebo-controlled study randomized 489 subjects. RBP-6000 achieved the primary endpoint of the cumulative distribution function of the percentage of urine samples negative for opioids combined with self-reports negative for illicit opioid use collected from week five through week 24 (p<0.0001 for both dosage regimens v. placebo). The key secondary endpoint in this study was treatment success defined as any subject with 80% of urine samples negative for opioids combined with self-reports negative for illicit opioid use from week five through week 24. The secondary endpoint was also achieved for both dosage regimens at p<0.0001 v. placebo. RBP-6000 was generally well-tolerated in this study. Available safety findings suggest that 2.8% of subjects on RBP-6000 experienced a serious treatment-emergent adverse event (TEAE) compared with 5.1% of subjects on placebo. There were no related serious TEAEs across groups. RBP-6000 has previously received Fast Track designation from the FDA. Subject to satisfactory completion of the analysis and, assuming the FDA review and approval is achieved within the assumed six month Priority Review timeline, it is possible that a marketing authorization could be granted in Q4 2017 per previous guidance.

NovaDigm Therapeutics reported results of a phase IIa study of NDV-3A for recurrent vulvovaginal candidiasis (RVVC). The multicenter, double-blind, randomized, placebo-controlled study enrolled 188 patients over 20 U.S. study sites. Patients were assigned one dose of either 300μg NDV-3A immunotherapy or a placebo. The study met its primary endpoint of safety and tolerability. There were no significant differences between NDV-3A and placebo for injection site reactions and systemic reactions of grade three or greater. A single dose of NDV- 3A generated very rapid and robust immune responses. Exploratory efficacy measures based on patient-reported symptom scores showed a trend toward significance at the 12-month follow-up period (p=0.10). Younger patients showed higher efficacy rates. In patients under 40 years of age (80% of the study population), NDV-3A recipients were about 50% more likely to be recurrence free at the end of the study compared to placebo recipients (p<0.05).