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 29, 2017

Anika Therapeutics reported results of a phase III trial of CINGAL for the treatment of symptoms associated with osteoarthritis (OA) of the knee. The multicenter, double-blind, saline-controlled clinical trial evaluated 368 patients with knee osteoarthritis who were randomized for treatment with a single injection of CINGAL (n=149), MONOVISC (n=150) or saline (n=69). Changes in pain, stiffness and physical function were assessed using a variety of validated measurement tools including the Western Ontario and McMaster Universities Arthritis Index (WOMAC). CINGAL reduced WOMAC Pain by 70% at 12 weeks and 72% at 26 weeks as compared to saline—more improvement than that reported in previous viscosupplement studies. CINGAL demonstrated rapid pain relief following administration (a 59% improvement in WOMAC Pain at one week and 68% at three weeks), unmatched by prior trials of triamcinolone hexacetonide alone. CINGAL was shown to be safe, and was associated with a low incidence of adverse events (n=6) that resolved over time. There were no serious adverse events considered to be related to CINGAL. These phase III study results were part of the comprehensive clinical data package that formed the basis for CINGAL’s CE Mark and Health Canada approval in 2016. CINGAL is commercially available in certain countries of the European Union, and Canada, and is under regulatory review in the U.S. Anika recently completed site initiation in the E.U. to begin enrolling patients in a supplemental phase III study requested by the FDA. Anika anticipates FDA approval of CINGAL after it completes the study in 2018.

Celgene released results of a phase III trial of ozanimod for patients with relapsing multiple sclerosis (RMS) compared to weekly interferon (IFN) ß-1a (Avonex). RADIANCE is a multicenter, randomized, double-blind, double-dummy, active-controlled trial evaluating two doses of oral ozanimod (0.5mg and 1mg) against weekly intramuscular interferon beta-1a (Avonex) over a 24 month treatment period. The study included 1,313 RMS patients across 147 sites in 21 countries. Both ozanimod 0.5mg and 1mg doses demonstrated statistically significant and clinically meaningful reductions in the primary endpoint of annualized relapse rate (ARR) and the key secondary endpoints of the number of new or enlarging T2 MRI lesions over 24 months of treatment compared to Avonex and the number of gadolinium-enhancing MRI lesions at 24 months of treatment compared to Avonex. An NDA submission to the FDA, based on the combined SUNBEAM and RADIANCE trials for RMS, is expected by the end of 2017.

Otsuka Pharmaceutical issued positive top-line results from an additional phase III clinical trial of tolvaptan in adult patients with autosomal dominant polycystic kidney disease (ADPKD). The multicenter, international, randomized-withdrawal, placebo-controlled, double-blind trial compared the efficacy and safety of tolvaptan (45 to 120mg/day) to placebo. Trial enrollees were adults 18 to 65 years of age with ADPKD-induced chronic kidney disease between late stage two to early stage four (eGFR ranging from 65 to 25 mL/min) and not previously treated with tolvaptan. A total of 1,370 patients were randomized to either tolvaptan or placebo and were treated for a period of 12 months. As in the prior study, tolvaptan resulted in more patients than placebo with increased (>3x upper limit of normal (ULN)) levels of liver enzymes alanine aminotransferase (ALT; 5.6% vs. 1.2%) and aspartate aminotransferase (AST; 3.5% vs. 0.9%); however, none of these patients exhibited total bilirubin greater than 2x ULN. The most common adverse events associated with tolvaptan (incidence >5% and at least 1% more frequent than placebo) included diarrhea (6.9% vs. 3.4%), fatigue (6.8% vs 3.5%) and polyuria (5.3% vs. 1.6%). The trial was completed to supply confirmatory data to the previous study to address the Complete Response Letter (CRL) issued by the FDA in 2013 for an NDA for tolvaptan in the treatment of adults with ADPKD. Tolvaptan is approved for the treatment of adult patients with ADPKD in Japan, the EU and other countries.

Sunovion Pharmaceuticals issued results of a phase III clinical study evaluating Latuda (lurasidone HCI) in children and adolescents (10 to 17 years of age) with major depressive episodes associated with bipolar I disorder (bipolar depression). In the six-week, randomized, double-blind, placebo-controlled study, 347 children and adolescents 10 to 17 years of age received LATUDA flexibly dosed (20 to 80mg/day) or placebo. LATUDA was associated with statistically significant and clinically meaningful improvement in bipolar depression symptoms compared to placebo, based on the primary efficacy endpoint of change from baseline to week six on the Children’s Depression Rating Scale, Revised (CDRS-R) total score (-21.0 vs. -15.3; effect size = 0.45, p<0.0001). Statistically significant and clinically relevant change from baseline to week six on the Clinical Global Impression-Bipolar Version, Severity of Illness (CGI-BP-S) score (depression) was also seen with LATUDA compared to placebo (-1.49 vs. -1.05; effect size = 0.44, p<0.0001). LATUDA was generally well-tolerated. The most common treatment-emergent adverse events (TEAEs) reported for LATUDA compared to placebo were nausea (16% vs. 5.8%), somnolence (9.1% vs. 4.7%), weight gain (6.9% vs. 1.7%), vomiting (6.3% vs. 3.5%), dizziness (5.7% vs. 4.7%) and insomnia (5.1% vs. 2.3%). LATUDA is currently indicated in the U.S. for the treatment of adults with bipolar depression as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults and adolescents (13 to 17 years of age). These data have been submitted to the FDA to support an sNDA.