Agios Pharmaceuticals Inc., of Cambridge, Mass., said a multiple ascending dose phase I trial of AG-348, an orally available activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency, a rare, hemolytic anemia, in healthy volunteers has started. It is designed to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of increasing doses of AG-348 for 14 days.

Elite Pharmaceuticals Inc., of Northvale, N.J., said the first subject was dosed in the treatment phase of a human abuse liability clinical study for ELI-200, an abuse-deterrent product for an undisclosed opioid. The randomized, double-blind, double-dummy, active and placebo-controlled five-way crossover study is comparing crushed ELI-200 capsules to a comparator product, with the primary objective of assessing the abuse potential of ground ELI-200 when administered intranasally relative to the crushed comparator. Secondary objectives include an assessment of relative bioavailability and product safety. Completion of dosing is expected in approximately two months, with results expected to report shortly thereafter. The company plans to file a new drug application through the 505(b)(2) pathway by year-end.

Ico Therapeutics Inc., of Vancouver, British Columbia, reported top-line results for the eight-month visual acuity (VA) primary endpoint for patients enrolled in its phase II IDEAL study of Ico-007 in diabetic macular edema (DME). Using two statistical analyses, the company found negative mean changes in VA measures in all four enrolled patient groups at months four and eight. Findings showed that losses in VA were greater at higher dosage levels. IDEAL, conducted in collaboration with JDRF, randomized 187 patients 18 years and older to explore whether various combinations and concentrations of Ico-007, alone or in combination, are safe and effective in improving visual acuity in patients with DME. The company said it will conduct additional data analysis at 12 months, including an evaluation of secondary endpoints, to determine the viability of Ico-007 in the indication. On Monday, Ico's shares (OTCQX:ICOTF) fell to a 52-week low, dropping 20 cents to close at 9 cents.

Isis Pharmaceuticals Inc., of Carlsbad, Calif., initiated a phase I study of ISIS-DMPKRx in myotonic dystrophy type I (DM1), earning a related $14 million milestone payment from Biogen Idec Inc., of Cambridge, Mass. ISIS-DMPKRx is designed to reduce the production of toxic dystrophia myotonica-protein kinase RNA in cells, including muscle cells. DM1 is the most common form of muscular dystrophy in adults, affecting approximately 150,000 patients in the U.S. and characterized by progressive muscle atrophy, weakness and muscle spasms. ISIS-DMPKRx is designed to improve the underlying genetic defect that causes DM1, which currently has no disease-modifying therapies that address more than one symptom. (See BioWorld Today, July 2, 2012.)

Karyopharm Therapeutics Inc., of Natick, Mass., initiated a phase II trial of its oral selective inhibitor of nuclear export, or SINE, compound selinexor (KPT-330) in patients with metastatic hormone-refractory prostate cancer (HRPC). The SHIP (Selinexor in Hormone Refractory Indications in Prostate Cancer) study is designed to enroll approximately 50 patients with metastatic HRPC following treatment with at least one recently approved agent, including enzalutamide (Xtandi, Medivation Inc./Astellas Pharma Inc.), abiraterone (Zytiga, Johnson & Johnson) or radium 223 (Xofigo, Bayer AG). Patients will receive 50 mg/m2 of Selinexor orally twice weekly over each 28-day cycle. The primary endpoint is disease control rate assessed using RECIST criteria and prevention of new bone lesions. The secondary endpoint is prostate-specific antigen response relative to baseline. The company presented phase I data for selinexor at the American Society of Clinical Oncology meeting in Chicago showing an 88 percent disease control rate in eight evaluable patients with heavily pretreated prostate cancer.

Quest Pharmatech Inc., of Edmonton, Alberta, reported that patient enrollment has been completed in the company's phase IIb trial of its lead immunotherapy product candidate, oregovomab, to treat ovarian cancer. The study is the first of three in conjunction with preclinical studies dissecting combinatorial immunotherapy. The objective is to confirm the specific immune response seen in the combination front-line setting in a population with more favorable surgical debulking. The enrolled patients will complete the treatment schedule and be followed up for long-term disease outcomes. Immunology data for all patients who completed the treatment phase will be analyzed and evaluated while the final survival data are being collected.