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 July 27, 2015

Aerpio Therapeutics reported results of a phase IIa study of AKB-9778 in diabetic macular edema (DME). The proof-of-concept study evaluated 144 DME patients randomized equally to AKB-9778 as monotherapy or in combination with Lucentis compared with Lucentis alone for three months. The combination of AKB-9778 (dosed at 15mg BID) and Lucentis (ranibizumab injection) provided a clinically significant benefit in reduction of central subfield thickness (CST) compared to Lucentis alone (p=0.008). In association with this improvement in CST, a positive trend also showed that more patients receiving the combination of AKB-9778 and Lucentis achieved greater than or equal to three lines of visual acuity compared to Lucentis alone. The trial additionally included a third treatment arm of patients receiving AKB-9778 alone, dosed at 15mg BID for three months, and this arm did not show a reduction in CST. The company is in the process of planning a follow-on clinical study with combination therapy.

FORUM Pharmaceuticals released results of a phase II study of encenicline in schizophrenia patients with cognitive impairment. The trial was a 12-week, double-blind, randomized, placebo-controlled, parallel-design, multinational study conducted in 317 patients. Randomized patients with schizophrenia or schizoaffective disorder treated with atypical antipsychotics were administered once daily doses of 0.27mg or 0.9mg of encenicline, or placebo. Results demonstrated statistically significant and clinically meaningful effects on both cognitive impairment and realworld functioning, including the primary endpoint of global cognitive function, as measured by the CogState Overall Cognition Index computerized battery test, for all encenicline-treated patients versus placebo, with the most effective dose being 0.27mg (p=0.034). The MATRICS Consensus Cognitive Battery (MCCB) also showed a strong trend for improvement (p=0.069) in the 0.9mg treatment group. Significant effects in the secondary endpoint of clinical function were observed with encenicline treatment as measured by the Schizophrenia Cognition Rating Scale (SCoRS) Interviewer Rating. The 0.9mg dose group showed a statistically significant improvement (p=0.011) over placebo after three months of dosing. Improvement also was seen in the secondary endpoint assessing negative symptoms of schizophrenia, as measured by the Negative Subscale of the Positive and Negative Syndrome Scale (PANSS). The 0.9mg dose group showed a statistically significant decrease in negative symptoms (p=0.028) compared to the placebo group after three months of dosing, with the 0.27mg group also demonstrating a positive trend. Two phase III registration multicenter clinical trials (COGNITIV SZ) investigating the use of encenicline for cognitive impairment in schizophrenia have recently completed enrollment. Encenicline for the treatment of cognitive impairment in schizophrenia has received Fast Track designation by the FDA.

Regeneron Pharmaceuticals and Sanofi reported results of a phase III trial of Praluent (alirocumab) Injection for low-density lipoprotein cholesterol (LDL-C). ODYSSEY JAPAN evaluated Praluent (n=144) compared to placebo (n=72), on top of standard care, in Japanese patients with hypercholesterolemia, with either HeFH or at high CV risk, and who could not reach their LDL-C treatment goal as defined by the JAS guidelines despite lipid-lowering treatments that included statins. The mean LDL-C value at baseline was 141.2mg/dL. Patients initially were randomized to receive either Praluent 75mg every two weeks administered as a single 1mL injection, or placebo. At week 24, 97% of patients in the Praluent group reached their LDL-C treatment goal, compared to 10% for placebo (p<0.0001). Ninety-nine percent of patients who received Praluent at week eight remained on the initial 75mg dose, while 1% of patients had their dose adjusted to receive 150mg every two weeks, also as a single 1mL injection. The most common adverse events (occurring in at least 5% of patients in the Praluent group) were nasopharyngitis, injection site reaction and back pain. The FDA has recommended the approval of Praluent.

Relypsa issued results of a phase II study of Patiromer for Oral Suspension (Patiromer FOS) in chronic kidney disease (CKD) and mild or moderate hyperkalemia. AMETHYST-DN was a multicenter, randomized, open-label, dose-ranging trial that included 306 patients with CKD stages 3 to 5, as well as type 2 diabetes. All patients were taking RAAS inhibitors to treat their CKD prior to and during study treatment and had mild-to-moderate hyperkalemia. Mild hyperkalemia was defined as potassium levels >5–5.5 mEq/L and moderate hyperkalemia as >5.5–<6 mEq/L. Study participants received one of three randomized total daily starting doses of Patiromer FOS: 8.4g, 16.8g or 25.2g for those with mild hyperkalemia, and 16.8g, 25.2g or 33.6g for those with moderate hyperkalemia. Through week 52, significant mean decreases in potassium from baseline at each monthly visit were observed (p<0.001) and the vast majority of patients with mild (83.1-92.7%) or moderate hyperkalemia (77.4-95.1%) had potassium levels within the target range (3.8-5mEq/L). Significant reductions in mean potassium were seen at the first post-baseline assessment, approximately 48 hours after Patiromer FOS initiation, in both mild and moderate hyperkalemia patients (p<0.001). After discontinuing treatment with Patiromer FOS, significant increases in mean potassium levels were observed by day 3 in both patient groups (mild hyperkalemia: 0.25 [0.19-0.31] mEq/L; moderate hyperkalemia: 0.33 [0.20-0.46] mEq/L; p<0.001). The most common SAE was worsening of CKD (2%).