Pediatrics

Keywords: Allergy; Vaccines

Dynavax Technologies and UCB Pharma have reported positive results of their phase I pediatric trial of AIC, their investigational ragweed allergy vaccine. Results have indicated that the drug is well tolerated in children and adolescents. The dose-escalating, open-label study enrolled 24 children between the ages of 9 and 17, all of whom had documented ragweed allergy; subjects were divided into three dosing cohorts, which each received increasing doses of AIC. Overall safety and tolerability were excellent, with only minor, localized, injection-site reactions reported and no serious adverse incidents. Based on these data, the companies announced plans to extend their clinical investigations into the use of AIC as a prophylactic against ragweed-allergy-induced sequelae, including asthma and chronic sinusitis.

Neurology

Keywords: Parkinson's Disease; Intracerebral Hemorrhage (ICH)

Amgen announced negative results of their phase II trial of their drug GDNF (glial cell line-derived neurotrophic factor), for the treatment of Parkinson’s disease (PD). The drug, while safe and well tolerated, failed to meet its primary efficacy endpoint, the improvement of symptom severity score on the Unified Parkinson’s Disease Rating Scale (UPDRS). The double-blinded study enrolled 34 subjects with advanced PD, who received direct, continuous, intra-putamen infusions of either GDNF or placebo for six months. No significant difference in UPDRS score was noted between the two groups, despite measurable changes in brain function. Amgen announced that all subjects were enrolled in an open label extension study, in the hopes of resolving these results.

Novo Nordisk has announced positive results of their phase IIb study of NovoSeven for the treatment of intracerebral hemorrhage (ICH). NovoSeven is currently approved as a treatment for acute bleeding in hemophilia. Results have shown that the drug led to a significant reduction in hematoma growth, compared to placebo, and significantly improved neurological and functional outcomes following an ICH incident. The double-blind, dose-response study enrolled a total of 400 subjects in 20 countries; subjects with spontaneous ICH confirmed by CT scan within three hours of symptom onset randomized to receive either NovoSeven or placebo, in addition to standard therapy. Follow-up CT scans, performed at 24 and 72 hours, found that NovoSeven significantly and dose-dependently reduced hematoma expansion, and observations at 15 and 90 days found significant improvement in severity score on neurological function tests in the NovoSeven group. Novo Nordisk announced plans to file supplementary regulatory submissions based upon this data.

Endocrinology

Keywords: Obesity

Nastech reported positive results of their phase I c trial of PYY3-36, their intranasal-spray formulation of peptide YY 3-36, for the treatment of obesity. Trial data have indicated that administration of the drug significantly reduced caloric intake and yielded significant weight loss over the course of the study, compared to placebo. The double blind study enrolled 37 men and women between the ages of 20 and 55, with a mean BMI of 33.3 (a BMI > 30.0 is classified as medically obese). All subjects received thrice-daily intranasal sprays of placebo for two days, and then were randomized to receive placebo or 1, 2, or 3 pre-meal doses of PYY3-36 per day for 6 days. Subjects receiving the drug demonstrated significant dose-per-day-dependent reduction in caloric intake, lost an average of 1.3 pounds, and had a 6% reduction in serum cholesterol over the course of the study.

Infectious Diseases

Keywords: Pneumonia; Acquired Immune Deficiency (AIDS) and AIDS-Related
Infections

Immtech International reported positive preliminary results of their phase II trial investigating DB289, for the treatment of Pneumocystis carinii pneumonia (PCP) in AIDS patients. Data from the first cohort of subjects have indicated that investigational doses of the drug have been safe and efficacious in treating PCP infection. The first cohort in this open label study received a pilot dosing regimen of 50 mg of DB289 twice daily for 21 days. A second study cohort is currently being treated with a 100 mg twice-daily regimen of the drug, and preliminary data from this group show a dose dependent improvement in efficacy and a reduction in time to return to normal lung function over the pilot group. The study is being conducted is being conducted in PCP-treatment-refractory AIDS patients in Peru.