• Amicus Therapeutics Inc., of Cranbury, N.J., reported positive results from clinical and preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease at the Lysosomal Disease Network World Symposium. The results from a Phase II study established human proof-of-concept that co-administration of AT2220 just prior to infusing ERT (Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle tissue compared to ERT alone. Based on those results, the company plans to initiate a repeat-dose clinical study in the third quarter of 2013 to evaluate an intravenous formulation of AT2220 co-administered with Myozyme/Lumizyme. Objectives of the study are to characterize safety and PK for later evaluation of infants and special populations.

• Argos Therapeutics Inc., of Durham, N.C., reported updated results from its Phase II study of AGS-003, its most advanced product candidate for the treatment of metastatic renal cell carcinoma (mRCC). The results, which will be presented during a poster session on Feb. 16, at the 2013 ASCO Genitourinary Cancers Symposium in Orlando, Fla., continue to show prolonged survival in patients with unfavorable risk mRCC, with one-third of patients still alive after nearly four years or longer. The open-label study followed 21 patients with unfavorable risk mRCC, with an expected survival of approximately 15 months. Treatment consisted of standard six-week cycles of sunitinib plus AGS-003, which was administered every three weeks for five doses, and then every 12 weeks until progression. The final median overall survival was 30.2 months. The company is currently enrolling patients in an ADAPT Phase III study for AGS-003 – a randomized, multicenter, open-label clinical trial that is expected to enroll 450 patients in approximately 120 global sites, mostly in North America. The study is designed to examine the potential for AGS-003 plus standard targeted drug therapy to extend overall survival vs. standard therapy alone in newly diagnosed patients with unfavorable risk mRCC. Secondary endpoints include progression-free survival, safety, overall response and immune response.

• BioAlliance Pharma SA, of Paris, said the FDA approved a U.S. extension of its Phase II trial of Validive (clonidine Lauriad) for the prevention of radio/chemotherapy-induced oral mucositis in patients with head and neck cancer. The extension will enable the company to increase the number of centers involved in the randomized, double-blind trial. To date, almost 50 percent of planned patients have been enrolled in about 30 European centers. BioAlliance expects to complete trial recruitment in early 2014 and report results later that year.

• Celldex Therapeutics Inc., of Needham, Mass., presented final results, at the American Society for Blood and Marrow Transplantation 2013 BMT Tandem Meetings, from a Phase I study of CDX-301 (recombinant human Flt3L; rhuFlt3L) in 30 healthy volunteers that strongly supported the initiation of a pilot study in hematopoietic stem cell transplantation later this year. CDX-301 is a cytokine that stimulates the expansion and differentiation of hematopoietic progenitor and stem cells.

• Dompe Group, of Milan, Italy, said the first patient has been enrolled in a Phase I/II REPARO study evaluating the ophthalmological use of a topical solution of recombinant human nerve growth factor (rhNGF) for the treatment of moderate to severe neurotrophic keratitis (NK). The study is randomized, double-masked, placebo-controlled trial that will enroll patients, from nine countries, with grade 2 (persistent epithelial defect) or grade 3 lesions (corneal ulcer) NK who do not respond to currently available medications. The primary objective of the trial is to evaluate the safety and efficacy of two doses of a rhNGF (10 µg/ml and 20 µg/ml) solution administered topically in the form of eye drops. It is planned to enroll at least 174 patients, who will be randomized to one of the two doses or placebo during the eight-week controlled treatment period. Follow-up will last at least 48 weeks. The secondary objectives of the trial are to evaluate the duration of corneal healing, improvement in visual acuity and corneal sensitivity.

• Ferring Pharmaceuticals SA, of Saint Prex, Switzerland, said results of a subanalysis of data from the study called MOTUS were presented at the Congress of the European Crohn's and Colitis Organization, showing that a once-daily 4-g dose of mesalazine (Pentasa granules) was noninferior to the twice-daily 2-g dose in inducing remission in patients with left-sided active ulcerative colitis. The objective of the MOTUS study was to show noninferiority of once-daily mesalazine vs. twice-daily dosing for the induction of remission in active ulcerative colitis. Since many patients with ulcerative colitis experience left-sided disease, the aim of the new subanalysis was to assess specifically the efficacy in patients with that form.

• Gamida Cell Ltd., of Jerusalem, disclosed results of the Phase I/II study of its second pipeline product NiCord, umbilical cord derived stem cells expanded using the company's NAM technology. Study data were presented during an oral at the 2013 BMT Tandem Meetings in Salt Lake City. Eleven patients, 21-61, with high-risk hematological malignancies received NiCord and an unmanipulated graft of umbilical cord blood (UCB). Eight patients engrafted with NiCord, and their median time to neutrophil engraftment was 10.5 (7-18) days for those engrafting with NiCord. Two patients engrafted with the unmanipulated UCB, and one patient experienced primary graft failure. There were no cases of Grade III/IV acute graft-vs.-host disease. No safety concerns surrounding the use of NiCord were raised. With a median follow-up of eight months, the progression-free and overall survival are both 90 percent.

• Neovacs SA, of Paris, said Arthritis & Rheumatism published an article on active immunization with IFN-alpha-Kinoid in patients with lupus. The article, co-authored by Bernard Lauwerys of the Catholic University of Louvain/University Clinics of Saint-Luc in Belgium and the Neovacs scientific team, concluded that active immunization with the Kinoid is a potentially effective approach in treating lupus. The article is based on the results of a Phase I/II study of IFN-alpha-Kinoid in lupus reported in November 2011 during the annual meeting of the American College for Rheumatology.

• Progenics Pharmaceuticals Inc., of Tarrytown, N.Y., reported positive data from a completed Phase I study of PSMA ADC, an antibody-drug conjugate (ADC) designed to deliver chemotherapy selectively to cells that express prostate-specific membrane antigen (PSMA). The open-label, dose-escalation trial was conducted in 52 men with hormone-refractory prostate cancer that had progressed despite prior treatment with taxane-based chemotherapy regimens. Patients were dosed at nine levels, and significant antitumor activity was observed across doses ranging from 1.8 mg/kg to 2.8 mg/kg. PSMA ADC was generally well tolerated in patients at doses up to and including 2.5 mg/kg, the maximum-tolerated dose. The most commonly reported adverse events were anorexia and fatigue. The findings were presented at the 2013 Genitourinary Cancers Symposium in Orlando, Fla.