Aerpio Therapeutics Inc., of Cincinnati, said data from its phase IIa study of lead candidate AKB-9778 for the treatment of diabetic macular edema (DME), reported at the American Academy of Ophthalmology meeting in Las Vegas, showed the combination of AKB-9778 and Lucentis (ranibizumab, Roche AG) provided a clinically significant benefit in reduction of macular edema, as measured by central subfield thickness (CST), compared to Lucentis alone at month two (p = 0.02) and at month three (p = 0.008). In association with the improvement in CST, the combination therapy showed a trend toward improved visual acuity when compared to Lucentis alone. Systemically administered AKB-9778 also demonstrated the ability to improve underlying retinopathy by two or more steps on the diabetic retinopathy severity scale in eyes with and without DME. In regard to the safety profile, there were no clinically significant differences in the percentage of patients that experienced ocular or non-ocular adverse events across the three study arms. AKB-9778 is a small molecule designed to inhibit human protein tyrosine phosphatase beta, which acts as a negative regulator of the Tie2 receptor.

Arqule Inc., of Burlington, Mass., and the National Human Genome Research Institute reported enrolling the first patient in the first clinical trial for people with Proteus syndrome, a rare condition that involves atypical growth of the bones, skin, head and a variety of other symptoms. The partners hope the biodynamic dose-finding phase I study of ARQ 092 will help to identify a safe and effective dose of the orally available, selective pan-AKT inhibitor.

Cidara Therapeutics Inc., of San Diego, said its antifungal drug candidate, CD101 intravenous (I.V.) proved well tolerated systemically and at infusion sites across entire dose ranges during a phase I single ascending-dose study in healthy volunteers. The company reported seeing no serious or severe adverse events and no dose response in adverse events, no clinical chemistry, hematology or ECG safety concerns were observed, and pharmacokinetics were consistent with preclinical data and supportive of once-weekly dosing. Cidara plans to initiate a phase II trial of CD101 I.V. in candidemia in the first half of 2016.

Infinity Pharmaceuticals Inc., of Cambridge, Mass., said it reached target enrollment of 300 patients in DUO, a randomized, phase III, monotherapy study of duvelisib compared to Arzerra (ofatumumab, Genmab A/S), an anti-CD20 antibody, in patients with relapsed or refractory chronic lymphocytic leukemia. The primary endpoint is progression-free survival. Duvelisib, an oral, dual inhibitor of PI3K-delta and PI3K-gamma, is jointly being developed with North Chicago-based Abbvie Inc.

Ocera Therapeutics Inc., of Palo Alto, Calif., said its phase I study showed that the oral formulation of OCR-002 (ornithine phenylacetate) in healthy subjects exhibited encouraging extended-release properties, demonstrated a desirable pharmacokinetic (PK) profile and was well tolerated. The open-label, single-dose, five-treatment, five-period crossover study evaluated the PK, safety and tolerability of three prototype, extended-release oral formulations of OCR-002 compared to an immediate-release oral solution of OCR-002 and the ammonia-lowering agent Ravicti (glycerol phenylbutyrate, Horizon Pharma plc), a pre-pro-drug of phenylacetate, a component of OCR-002. Ocera is currently conducting a phase IIb trial, STOP-HE, testing intravenously administered OCR-002 in resolving neurocognitive symptoms of acute hepatic encephalopathy in hospitalized patients with elevated ammonia. The company expects to complete enrollment in that trial in the second half of 2016.

Vectura Group plc, of Chippenham, UK, said Novartis AG, of Basel, Switzerland, reported results from the phase III FLAME head-to-head trial examining the rate of chronic obstructive pulmonary disease (COPD) exacerbations. Once-daily Ultibro Breezhaler (indacaterol/glycopyrronium) 110/50 mcg met its primary endpoint of noninferiority and also demonstrated superiority to twice-daily Seretide (salmeterol/fluticasone, Glaxosmithkline plc) 50/500 mcg in reducing the rate of all COPD exacerbations (mild/moderate/severe) over one year of treatment.

Viking Therapeutics Inc., of San Diego, said it submitted an investigational new drug application to the FDA to conduct a phase II study of VK2809, an orally available small-molecule thyroid receptor agonist, in patients with hypercholesterolemia and fatty liver disease. The randomized, double-blind, parallel-group, placebo-controlled trial is designed to evaluate the efficacy, safety and tolerability of VK2809 in about 100 patients with elevated LDL-cholesterol and fatty liver disease, with a primary endpoint defined as the effect of treatment on LDL-C after 12 weeks compared to placebo. Secondary and exploratory endpoints will assess changes in liver fat content, triglycerides and inflammatory markers. Viking expects to initiate the trial by the end of 2015 and complete the study in 2016.