• Aquinox Pharmaceuticals Inc., of Vancouver, British Columbia, began dosing patients in a Phase II trial of its lead clinical candidate, AQX-1125, a SHIP1 activator, for the treatment of bladder pain syndrome/interstitial cystitis (BPS/IC). The trial, known as the LEADERSHIP study, is evaluating the safety and efficacy of AQX-1125 in about 70 BPS/IC patients. The company recently received a "no-objection" letter to its clinical trial application from Health Canada and expects to finish the trial in the second half of 2014.

• Arrowhead Research Corp., of Pasadena, Calif., said it started dosing in a Phase I trial of ARC-520, its candidate for treating chronic hepatitis B virus (HBV) infection. The objectives are to characterize the drug's safety profile, determine the maximum tolerated dose and evaluate pharmacokinetics in healthy volunteers. The study is expected to be completed in the fourth quarter, with a Phase IIa study in chronic HBV patients set to start in 2014. ARC-520 is the first candidate to use Arrowhead's Dynamic Polyconjugate delivery platform and includes two distinct siRNA sequences that have pan-genotypic coverage for 99.6 percent of HBV GenBank sequences.

• Cytokinetics Inc., of South San Francisco, amended the protocol for BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS), a Phase IIb study testing tirasemtiv, a fast skeletal muscle troponin activator, in patients with amyotrophic lateral sclerosis (ALS). The company said it had been informed by its data management vendor that a programming error in the electronic data capture system caused 58 patients initially randomized to and treated with tirasemtiv to receive placebo at a certain study visit and for the remainder of the study. The company, which remains blinded to the data, amended the study design to allow for increased enrollment, to about 680 patients total, and to update the statistical methods section, with the aim of maintaining the originally intended statistical power. The changes are expected to increase direct clinical trial costs by about $5 million in 2013 and 2014.

• Evofem Inc., of San Diego, reported the early completion of enrollment in its 3,200-subject Phase III registration study of Amphora nonhormonal contraceptive gel. The global trial compares Amphora to Conceptrol (nonoxynol-9, Personal Products Co.), the only spermicidal gel currently approved by the FDA.

• Mast Therapeutics Inc., of San Diego, disclosed results of its thorough QT/QTc study of MST-188, an investigational agent being developed to treat serious or life-threatening diseases with significant unmet needs, such as sickle cell disease and acute limb ischemia. The study met its primary endpoint and demonstrated that, based on analysis of electrocardiograms, MST-188 did not have an adverse effect on cardiac repolarization, as measured by prolongation of the QT interval. MST-188 was generally well tolerated at both therapeutic and supratherapeutic doses.

• Prana Biotechnology Ltd., of Parkville, Australia, said it completed its Phase II Reach2HD trial testing PBT2 in patients with early to midstage Huntington's disease. Results from the 109-patient study are expected in October. The primary outcome is safety and tolerability, with secondary outcomes including measures from the cognitive, motor and behavioral domains affected in Huntington's disease.

• Protalex Inc., of Summit, N.J., said the independent data safety monitoring committee completed a planned safety interim review, and the company is continuing enrollment in its Phase Ib dose-escalation study of PRTX-100 in combination with methotrexate or leflunomide in adults with active rheumatoid arthritis. PRTX-100 incorporates a highly purified form of Staphylococcal protein A.

• Verastem Inc., of Cambridge, Mass., said it confirmed the complete response in an ongoing trial of VS-6063 (defactinib) in combination with weekly paclitaxel. Preliminary results described a normalization of CA-125, a biomarker of ovarian cancer progression, and an initial radiologic complete response for one of the patients in the trial. The Phase I dose-escalation portion in six patients showed the drug was well tolerated at all dose levels, with no exacerbations of the well-known adverse effects of paclitaxel. Verastem recently started the expansion Phase Ib stage of the study, expected to enroll an additional 15 patients with ovarian cancer. The endpoints are safety, tolerability and efficacy as determined by RECIST and proprietary cancer stem cell biomarkers.