ADial Pharmaceuticals LLC, of Charlottesville, Va., said the FDA approved its plan to initiate Phase III trials of AD04 in alcohol use disorder in certain targeted genotypes. Patients will initially be screened for the targeted genotypes, and those with one or more of the targeted genotypes will be enrolled in the trial. Enrolled patients will then be randomized between the active drug and placebo. The endpoints for demonstrating the efficacy of AD04 in the trial are percent of heavy drinking

Inovio Pharmaceuticals Inc., of Blue Bell, Pa., said its SynCon universal H1N1 influenza vaccine generated protective antibody levels comparable to an FDA-approved seasonal influenza vaccine against a currently circulating flu strain. Data from the Phase I study in healthy volunteers were presented at the World Vaccine Congress in Washington. Northwest Biotherapeutics Inc., of Bethesda, Md., said that a patient treated with DCVax-L for glioblastoma multiforme in 2002 has celebrated his 10th

Achillion Pharmaceuticals Inc., of New Haven, Conn., said it started a Phase II trial of sovaprevir and ACH-3102, in combination with ribavirin, for the treatment of genotype 1 chronic hepatitis C virus patients who have not received prior therapy. The study will evaluate the all-oral, 12-week, interferon-free regimen for safety, tolerability and efficacy in up to 50 patients. Initially, 30 patients will be enrolled and randomized to receive either a combination of sovaprevir, ACH-3012 and

Advanced Cell Technology Inc., of Marlborough, Mass., treated the first patient in patient cohort 2a of its Phase I trials, consisting of patients with better vision, in its U.S. trial for Stargardt's Macular Dystrophy (SMD), a form of juvenile macular degeneration. The patient was injected with 100,000 human embryonic stem cell-derived retinal pigment epithelial cells. As the company announced on Jan. 22, patients with a visual acuity of 20/100 are eligible for enrollment in cohort 2a in the

Iroko Pharmaceuticals LLC, of Philadelphia, will present positive Phase III data for its nonsteroidal anti-inflammatory drug pipeline at two medical meetings summarizing studies of submicron indomethacin and submicron diclofenac for osteoarthritis pain. Both studies met the primary endpoint of significant pain relief compared to placebo. Data for indomethacin will be presented at the 29th Annual Meeting of the American Academy of Pain Medicine in Fort Lauderdale, Fla. Data for diclofenac will

Pacira Pharmaceuticals Inc., of Parsippany, N.J., reported Phase IV (EXCLAIM) results for Exparel (bupivacaine liposome injectable suspension) in postsurgical analgesia. Patients who received Exparel after abdominoplasty, breast augmentation, breast reduction or a combination procedure had low pain scores, high satisfaction with their pain control and minimal opioid use. Pacira presented the findings at The Aesthetic Meeting 2013.

Cardio3 BioSciences SA, of Mont-Saint-Guibert, Belgium, published results of its Phase II (C-CURE) trial in the Journal of the American College of Cardiology showing that therapy with C3BS-CQR-2 is feasible and safe with signals of benefit in chronic heart failure, and merits further evaluation. In the prospective, randomized study, patients with chronic heart failure secondary to ischemic cardiomyopathy received treatment with their own stem cells engineered to become progenitors of cardiac

BioLineRx Ltd., of Jerusalem, said it received regulatory approval in the U.S. to begin a Phase IIa trial of BL-8040, a selective antagonist of chemokine receptor CXCR4, in acute myeloid leukemia (AML). The open-label study under an investigational new drug application is designed to evaluate the safety and efficacy profile of repeated escalating doses of BL-8040 in adults with relapsed/refractory AML. The primary endpoints are safety and tolerability, with secondary endpoints including the

Reviva Pharmaceuticals Inc., of San Jose, Calif., reported top-line results from its Phase II REFRESH study of RP5063 in schizophrenia and schizoaffective disorder, with the dopamine serotonin stabilizer showing overall broad efficacy across the Positive and Negative Syndrome Scale total scores, as well as subscales: Positive, Negative and General Psychopathology. RP5063 also showed efficacy in the Clinical Global Impression Severity Scale and was well tolerated. The study enrolled 234 patients

InDex Pharmaceuticals AB, of Stockholm, Sweden, completed enrollment for its Phase III COLLECT study of Kappaproct. The multinational, randomized, double-blind, placebo-controlled study reached the recruitment goal of 120 patients with treatment-refractory ulcerative colitis. InDex is expecting final study results in mid-2014. The primary endpoint of the study is the induction of clinical remission at week 12. The patients will be followed for a total of 52 weeks. Kythera Biopharmaceuticals

Arena Pharmaceuticals Inc., of San Diego, initiated dosing in a Phase I trial of APD334, an oral drug candidate targeting the sphingosine 1-phosphate subtype 1 receptor, for the potential treatment of autoimmune diseases. The randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability and pharmacokinetics of single-ascending doses of APD334 in up to 64 healthy adult volunteers. Emergent BioSolutions Inc., of Rockville, Md., expanded the protocol for its ongoing

Aastrom Biosciences Inc., of Ann Arbor, Mich., enrolled its first patients in a Phase IIb trial of ixCELL-DCM to assess safety and efficacy of ixmyelocel-T in advanced heart failure that is caused by ischemic dilated cardiomyopathy. The randomized, double-blind, placebo-controlled trial will enroll 108 patients at about 30 sites in the U.S. to receive ixymyelocel-T through a catheter. The primary endpoint will be the average number of events per patient, over a 12-month period. Aragon

Advanced Cell Technology Inc., of Santa Monica, Calif., treated patients in a third cohort of each of its two Phase I trials of human embryonic stem cell-(hESC) derived retinal pigment epithelial cells for macular degeneration. The patients received 150,000 cells, compared with the 100,000-cell dose used in the second cohort. The trials will assess safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with Stargardt's macular dystrophy and dry age

Phosphagenics Ltd., of Melbourne, Australia, said it achieved all the endpoints of its Phase I trial of its oxymorphone patch by successfully delivering the opioid into the bloodstream via its TPM transdermal patch. The company will proceed with Phase II trials in the second half of 2013.

Benitec Biopharma Ltd., of Sydney, Australia, selected the Duke Clinical Research Unit in Durham, N.C., as a site for its upcoming Phase I/II first-in-human trial of TT-034 in hepatitis C virus (HCV). The open-label, dose escalation study will evaluate the safety and activity of single doses of TT-034 in patients with chronic HCV genotype 1 infection who failed previous treatments. The trial is expected to involve 14 patients in 5 sequential dose cohorts, although additional consolidation

Affinium Pharmaceuticals Inc., of Toronto, said its Phase IIa trial evaluating oral AFN-1252 in acute bacterial skin and skin structure infections (ABSSSI) demonstrated efficacy and safety, marking a proof-of-concept milestone for Affinium's first-in-class antibiotic targeted against bacterial fatty acid biosynthesis inhibition. The study in 103 patients confirmed the efficacy, safety and tolerability of 200 mg of oral AFN-1252 dosed twice daily for five to 14 days in patients with ABSSSIs due

LEO Pharma UK, of Princes Risborough, UK, reported that data published in the Journal of the American Medical Association Dermatology demonstrated the long-term effectiveness of Picato (ingenol mebutate) in treating actinic keratosis (AK), a leading cause of nonmelanoma skin cancer. The data showed that about 45 percent of patients who achieved clearance after the initial two- or three-day treatment experienced sustained clearance of AK lesions a year later. Patients in the overall study

Teva Pharmaceutical Industries Ltd., of Jerusalem, and H. Lundbeck A/S, of Copenhagen, Denmark, said the ANDANTE (Add oN to Dopamine AgoNists in the TrEatment of Parkinson's disease), testing Azilect (rasagiline tablets) as an add-on treatment to dopamine agonists compared to placebo met its primary endpoint. Results showed that the addition of Azilect 1 mg/day provided a statistically significant improvement in total Unified Parkinson's Disease Rating Scale score from baseline to week 18 in

Acorda Therapeutics Inc., of Ardsley, N.Y., said data from a Phase I study showed that a single dose of 20-mg Diazepam Nasal Spray had comparable plasma bioavailability to 20 mg of diazepam rectal gel. Diazepam Nasal Spray is being developed for treating people with epilepsy who experience cluster seizures, also known as acute repetitive seizures. Data were presented at the American Academy of Neurology meeting in San Diego. Acorda plans to submit a new drug application under the 505(b)(2