• Alnylam Pharmaceuticals Inc., of Cambridge, Mass., began dosing in a Phase I trial of ALN-TTR02 for TTR-mediated amyloidosis to evaluate the safety and tolerability of the drug in healthy volunteers. The study also will assess the activity of ALN-TTR02 by means of serum levels of TTR. The single-blind, randomized, placebo-controlled, single-ascending dose trial is being carried out in the U.K., and will enroll about 32 subjects.

• Convergence Pharmaceuticals Ltd., of Cambridge, UK, initiated a Phase II proof-of-concept study of CNV1014802 in pain associated with trigeminal neuralgia (TN). CNV1014802 is a small-molecule, state-dependent sodium channel blocker that exhibits potency and selectivity against the Nav1.7 sodium channel. The study is evaluating the efficacy and safety of orally administered CNV1014802 in patients with TN, a severe form of facial pain experienced in short bursts or attacks that affects about 50,000 people in the U.S. The company expects to report top-line results in mid-2013.

• Cytori Therapeutics Inc., of San Diego, said the European Journal of Surgical Oncology published data from the 71-patient RESTORE-2 study testing autologous adipose-derived regenerative cell (ADRC)-enriched fat grafting for reconstruction of the breast after cancer surgery. Key findings showed high rates of investigator (85 percent) and patient (75 percent) satisfaction with the overall treatment results at 12 months and high rates of investigator (87 percent) and patient (67 percent) satisfaction with overall breast deformity, based on functional and cosmetic outcomes) at 12 months. There also were no local cancer recurrences or serious adverse events related to the ADRC-enriched fat grafting procedure.

• Genmab A/S, of Copenhagen, Denmark, submitted a protocol amendment for its ofatumumab Phase III head-to-head study in diffuse large B-cell lymphoma (DLBCL). Patients in the study will receive ofatumumab plus chemotherapy or Rituxan (rituximab, Biogen Idec Inc. and Roche AG) plus chemotherapy. The amended protocol calls for both groups to receive the same chemotherapy regimen, and it will revise underlying timing assumptions, which could move the primary endpoint forward, as early as 2014.

• Hemispherx Biopharma Inc., of Philadelphia, published an article in the current issue of PLoS ONE with results of a Phase III trial of Ampligen in chronic fatigue syndrome (CFS). The trial met its primary endpoint of exercise tolerance, with an average improvement of 21 percent compared to placebo. The cohort receiving Ampligen also had reduced dependence on medications used to control CFS symptoms.

• Medivir AB, of Huddinge, Sweden, said it will file an application in Europe to start Phase I trials of MIV-711, a cathepsin K inhibitor aimed at treating bone disorders such as osteoporosis, osteoarthritis or bone metastases.

• Provectus Pharmaceuticals Inc., of Knoxville, Tenn., reported top-line data for its Phase II trial of PH-10 in mild to moderate plaque psoriasis. The trial compared the safety and efficacy of three dose levels (0.002 percent, 0.005 percent and 0.01 percent) of PH-10, an aqueous hydrogel formulation of Rose Bengal disodium for topical administration, against vehicle. Efficacy was assessed using the Psoriasis Severity Index (PSI), the Plaque Response Assessment and the Pruritus Self Assessment scale. Results showed improvement in psoriasis symptoms over the treatment interval, with the low dose of PH-10 providing uniformly consistent improvement, while the two higher doses showed reduced therapeutic activity. After 28 days of treatment with PH-10 at all strengths, 23 percent to 29 percent of subjects achieved complete or nearly complete resolution of PSI component symptoms (erythema, induration and desquamation), compared to no subjects in the vehicle arm.

• Rockwell Medical Inc., of Wixom, Mich., said the independent data safety monitoring board (DSMB) providing oversight for its Phase III SFP iron replacement therapy program reviewed cumulative safety data from the Phase III CRUISE studies and identified no safety concerns. The DSMB recommended continuing the studies with no modifications. SFP is in Phase III trials for use as an iron replacement therapy in patients with end-stage renal disease.

• Telormedix SA, of Bioggio, Switzerland, said it completed the safety portion of its Phase I/II trial of TMX-101 in bladder cancer. The open-label, dose-escalation trial enrolled patients with the nonmuscle-invasive form of the disease who also had undergone a complete transurethral resection. Patients received TMX-101 instilled into the bladder once a week for a total of six treatments. The first part of the study assesses safety, and that is now complete. In the second part, effective biological dose will be determined, and, in the third part, patients will be followed for one year to determine status of the disease and safety. Based on results of the first part, intravesical instillation of TMX-101 is safe at doses between 0.05 percent and 0.4 percent.

• VBL Therapeutics Inc., of Tel Aviv, Israel, said a Phase II substudy of VB-201 in moderate to severe psoriasis patients with cardiovascular risk achieved its primary endpoint, demonstrating a statistically significant reduction in vascular inflammation associated with atherosclerotic lesions, as measured by PET-CT imaging. In the overall Phase II study, VB-201 demonstrated safety and tolerability, with no treatment-related serious adverse events. The study also showed statistically significant improvements in the psoriasis efficacy endpoints, physician global assessment and patient global assessment (p = 0.019). VB-201 is an oral disease-modifying agent selected from the company's series of oxidized phospholipid analogues in the Lecinoxoid molecular class. The compound is being developed as an oral controller medicine for chronic immuno-inflammatory disease and atherosclerosis inflammation. The Phase II substudy results were presented at the American Academy of Dermatology meeting in San Diego.