• Aeterna Zentaris Inc., of Quebec City, said the Veterans Affairs Medical Center in Houston began a Phase IIa trial of AEZS-130 (macimorelin) in patients with cancer cachexia. The randomized trial will test different doses of the drug in 18 to 26 patients with cancer cachexia. The primary objective is safety and efficacy of repeated oral doses at different daily doses for one week.

• Alexion Pharmaceuticals Inc., of Cheshire, Conn., said targeted enzyme replacement candidate asfotase alfa was shown to improve skeletal abnormalities, pulmonary and physical function and cognitive development in a Phase II study of infants and young children with hypophosphatasia, an ultra-rare, genetic metabolic disease. The therapy, which Alexion acquired through its recent $1 billion acquisition of Montreal-based Enobia Pharma Corp., met the primary endpoint in the study, with 90 percent of patients showing substantial skeletal healing at 24 weeks. Key secondary endpoints, including improvement in cognitive development and motor and pulmonary functions, also were achieved. Findings were published in the New England Journal of Medicine. (See BioWorld Today, Jan. 3, 2012.)

• AMAG Pharmaceuticals Inc., of Lexington, Mass., reported preliminary results from a Phase III study of Feraheme (ferumoxytol) for iron-deficiency anemia. Treatment with Feraheme was compared to treatment with I.V. iron sucrose. In 605 patients, Feraheme achieved noninferiority in two primary efficacy endpoints. There was a mean increase in hemoglobin at week five of 2.7 g/dL, compared to 2.4 g/dL in the I.V. iron sucrose group. And a greater than 2 g/dL increase in hemoglobin at any time from baseline to week five was reached in 84 percent of patients treated with Feraheme, compared to 81 percent treated with I.V. iron sucrose.

• Apogenix GmbH, of Heidelberg, Germany, said the Phase II trial of lead compound APG101 met its primary endpoint of six-month rate of progression-free survival (PFS) in second-line treatment of glioblastoma multiforme. APG101 is a fully human fusion protein combining the extracellular domain of the CD95 receptor and the Fc portion of IgG. In the randomized, open-label trial, 83 patients were treated with APG101 and radiotherapy or with radiotherapy alone. Apogenix said the trial exceeded its primary objective, which was to increase the percentage of patients reaching six-month PFS by 100 percent. No drug-related adverse effects occurred. The company plans to present full findings at upcoming cancer conferences in the U.S. and Europe.

• BioMarin Pharmaceutical Inc., of Novato, Calif., said it completed enrollment for its pivotal Phase III trial of N-acetylgalactosamine 6-sulfatase (GALNS or BMN-110) in the lysosomal storage disorder mucopolysaccharidosis Type IVA, or Morquio A syndrome. The study is exploring doses of 2 mg/kg/week and 2 mg/kg/every other week over 24 weeks. The primary endpoint is the six-minute walk test, and secondary endpoints are the three-minute stair climb test and urine keratan sulfate concentration. The company expects to report results in the fourth quarter and to submit regulatory filings in the first quarter of 2013. (See BioWorld Today, Feb. 2, 2011.)

• Circassia Ltd., of Oxford, UK, reported Phase II findings for its ragweed allergy vaccine, ToleroMune. Results of the randomized trial showed that patients with a moderate level of symptoms at baseline achieved a 97 percent greater reduction than placebo (p less than or equal to 0.05) on the T-cell vaccine's optimal regimen. The treatment was safe and well tolerated in all groups. The findings were presented at the American Academy of Allergy, Asthma and Immunology meeting in Orlando, Fla.

• Cytheris SA, of Paris, said a Phase IIa trial (INSPIRE-2) of CYT107 (recombinant interleukin-7) for reconstitution of CD4 T cells in chronic HIV-1 immunological nonresponder patients showed that the drug expanded those cells in the gut mucosa. The companys said it believes the drug may be able to restore the immune system in chronic HIV infection.

• Gilead Sciences Inc., of Foster City, Calif., said full Phase III results from Study 102 showed that Quad (once-daily elvitegravir/cobicistat/emtricitabine/tenofovir disoproxil fumarate) for HIV-1 infection, was noninferior to Atripla (efavirenz/emtricitabine/tenofovir disoproxil fumarate) after 48 weeks of therapy in treatment-naive adults. The results were presented at the Conference on Retroviruses and Opportunistic Infections in Seattle. Gilead, which reported top-line data from Study 102 in August 2011, submitted a new drug application for Quad late last year. (See BioWorld Today, Aug. 16, 2011.)

• Palatin Technologies Inc., of Cranbury, NJ, completed enrollment in a Phase IIb trial of bremelanotide in female sexual dysfunction. It enrolled more than 400 premenopausal women with female sexual arousal disorder, hypoactive sexual desire disorder or both, for 16 weeks of treatment. Patients were randomized into one of four double-blind treatment groups to receive placebo or one of three doses of the drug. The goal of the trial is to evaluate safety and efficacy of subcutaneous bremelanotide for on-demand use. The trial also will assess pharmacokinetics.

• Sangamo BioSciences Inc., of Richmond, Calif., said data from Phase I studies testing SB-728-T in HIV-infected subjects showed that viral load initially increased as expected during the treatment interruption (TI) of HAART therapy in all six subjects, followed by a 0.8 to > 2.0-log reduction in viral load from peak observed in three of six patients with the highest estimated circulating levels of cells with biallelic modification of their CCR5 genes. In one subject, viral load decreased to undetectable levels such as the subject was aviremic at the end of the TI period. SB-728-T is an autologous CD4 T-cell product in which the gene for CCR5 is modified via ZFN-mediated genome editing to disrupt the CCR5 protein. Those and other data were presented at the Conference on Retroviruses and Opportunistic Infections in Seattle.

• Sosei Co. Ltd., of Tokyo, said it started a Japanese Phase I trial to evaluate the pharmacokinetics and safety of antifungal agent SO-1105 in healthy adults. The product, which is administered as a muco-adhesive buccal tablet, originally was developed by Paris-based BioAlliance Pharma SA and is sold in Europe, the U.S. and South Korea under the trade names Loramyc/Oravig for oropharyngeal candidiasis in immunocompromised adults.

• Ultragenyx Pharmaceutical Inc., of Novato, Calif., completed a Phase I study evaluating the pharmacokinetics and safety of UX001 in 28 patients with hereditary inclusion body myopathy (HIBM) and said it would report the data within several months. UX001 is an extended-release formulation of sialic acid designed as a substrate replacement therapy for HIBM, a neuromuscular disease caused by sialic acid deficiency. The FDA has granted the compound orphan drug designation in HIBM.