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 2, 2016

ALK issued results of a phase III trial of Acarizax for house dust mite (HDM) allergic asthma. The randomized, double-blind, placebo-controlled trial enrolled 834 adult patients. The trial was conducted at 109 sites in 13 European countries and forms part of ALK’s ongoing clinical development program for Acarizax, which has recently been approved in 11 European countries and where it is currently being launched. Patients were treated daily with either a 12 SQ-HDM or a 6 SQ-HDM dose, or with placebo in addition to inhaled corticosteroids (ICS) and short-acting beta-agonists (SABA). After a period of treatment varying between seven and 12 months, daily ICS use was reduced by half for three months and subsequently withdrawn completely for another three months for patients who did not experience an asthma exacerbation. The trial showed that 12 SQ-HDM (the dose approved in the E.U.) significantly reduced the risk of a moderate or severe asthma exacerbation relative to placebo with a hazard ratio (HR) of 0.66, corresponding to a 34% risk reduction. This included a 36% reduction in risk of nocturnal awakening or increase in daily symptoms (HR: 0.64); a 48% reduction in the risk of increased use of SABA treatments (HR: 0.52); and a 42% reduction in the deterioration of lung function (HR: 0.58). 

Amgen issued results of a phase III, randomized, double-blind, placebo-controlled study of Nplate (romiplostim) in children with symptomatic immune thrombocytopenia (ITP). The study enrolled 62 children who had ITP for more than six months to weekly Nplate or placebo (2:1) for 24 weeks. By the final eight weeks of the study, noncutaneous bleeding had decreased with Nplate, and rates of durable platelet response were 52% compared to 10% with placebo (p=0.002, odds ratio 9.1, 95% CI: 1.9, 43.2). Rates of overall platelet response with Nplate were 71% (30/42) compared with 20% with placebo (p=0.0002, odds ratio 9, 95% CI: 2.5, 32.3), and rates of any platelet response were 81% (34/42) with Nplate compared to 55% (11/20) with placebo (p=0.0313). The overall safety profile on the pediatric subjects who received Nplate in this study was similar to the known safety profile of Nplate. The most frequently reported AEs included contusion, epistaxis, headache and upper respiratory tract infections. Oropharyngeal pain occurred more frequently with Nplate [26.2% (11/42) v. 5.3% (1/19) in placebo-treated patients]; of the 11 patients treated with Nplate with oropharyngeal pain, streptococcal pharyngitis (n=2), allergic rhinitis (n=2), gastroesophageal reflux (n=1) and serum sickness from IVIg (n=1) were also reported. No oropharyngeal pain AEs were serious or considered treatment-related. No patients died and none withdrew due to AEs. Serious adverse events (SAEs) were seen in 23.8% of Nplate patients and 5.3% of placebo patients. SAEs seen in the Nplate arm included epistaxis, contusion and headache (n=2 each), bronchiolitis, nausea, petechiae, epilepsy, fever, thrombocytosis, urinary tract infection and vomiting (n=1 each). One subject with treatment-related SAEs experienced headache and thrombocytosis, which did not recur when romiplostim was restarted. There were no thrombotic events reported in the study. The company plans to work with regulatory authorities while seeking approval for Nplate for pediatric patients.

Merck & Co. reported results of a phase III trial of Zepatier (elbasvir and grazoprevir) 50mg/100mg tablets in chronic hepatitis C (HCV) patients with inherited blood disorders (C-EDGE IBLD). C-EDGE IBLD is a randomized, double-blind, placebo-controlled study. Patients were randomized in a 2:1 ratio to either an immediate treatment group (ITG; 12 weeks of Zepatier) or deferred treatment group (DTG; 12 weeks of placebo as control arm, followed by 12 weeks of open-label Zepatier). The primary efficacy endpoint for the study was the proportion of patients in the ITG who achieved SVR12. Safety and tolerability were evaluated by comparing subjects receiving Zepatier in the ITG (n=107) to those receiving placebo in the DTG (n=52). Following 12 weeks of treatment with Zepatier, 93% of patients in the ITG (100/107) achieved SVR12 (virologic cure). Six patients relapsed following 12 weeks of treatment; five of these patients (3 GT1a, 1 GT1b and 1 GT4) had detectable NS5A resistance-associated polymorphisms at baseline. The study did not evaluate other Zepatier-based dosage regimens or durations. Zepatier was approved by the FDA in January 2016, based in part on prior studies from the phase III program.

Novartis released results from a phase III study of Afinitor (everolimus) for reduced treatment-resistant seizures associated with tuberous sclerosis complex (TSC). The three-arm, randomized, double-blind, placebo-controlled study enrolled male and female participants (ages 2.2 to 56.3). In the study, 366 patients with TSC and treatment-resistant seizures were randomized to receive targeted concentrations of everolimus titrated to Low Exposure (LE; 3-7 ng/mL; n=117) or High Exposure (HE, 9-15 ng/mL; n =130), or placebo (n=119). The percentage reduction from baseline in seizure frequency was significantly greater among patients randomized to everolimus LE (29.3%, p=0.003; confidence interval [CI]=95%) and HE (39.6%, p<0.001; CI=95%) v. placebo (14.9%; CI=95%). Seizure response rate (50% reduction) was also significantly greater with everolimus LE (28.2%, p=0.008; CI=95%) and HE (40.0%, p<0.001; CI=95%) v. placebo (15.1%; CI=95%). The most common (20%) adverse events (AEs) reported with everolimus LE/HE v. placebo included stomatitis (28.2%/30.8% v. 3.4%), mouth ulceration (23.9%/21.5% v. 4.2%), and diarrhea (17.1%/21.5% v. 5.0%). Serious AEs reported were 13.7%/13.8% v. 2.5%.