|Healthcare Review: GW Pharmaceuticals, Galena Biopharma, Isis Pharmaceuticals, Novartis, Alexion Pharmaceuticals|
|By Staff and Wire Reports|
|Tuesday, 17 June 2014 11:10|
GW Pharmaceuticals ($GWPH) reports preliminary data on 27 children and young adults with treatment-resistant epilepsy who have been treated with its investigational cannabidiol product candidate Epidiolex for a period of 12 weeks. Of the 27 patients, nine have Dravet syndrome and the remaining 18 have treatment-resistant epilepsies with convulsive and/or non-convulsive siezures.The mean overall reduction in seizure frequency compared to baseline was 44%. The median overall reduction was 42%.At the end of 12 weeks, 48% of the patients achieved at least a 50% reduction in seizure frequency compared to baseline. 41% of the patients achieved at least a 70% reduction. 22% of the patients achieved at least a 90% reduction and 15% of the patients were seizure-free.
Galena Biopharma ($GALE) completes enrollment of the full 45-patient cohort for the Phase 2a clinical trial for GALE-301 (Folate Binding Protein (FBP) peptide immunotherapy). The two arms of the trial consist of HLA-A2-positive patients treated with the FBP peptide plus granulocyte macrophage-colony stimulating factor (GM-CSF) and an observational control group of HLA-A2-negative patients. After an initial induction series of six vaccinations given once per month, patients will receive booster inoculations at two subsequent six-month intervals. The primary objectives are to measure the immune response, time to recurrence and overall survival between treatment arms.
Isis Pharmaceuticals ($ISIS) earns its first milestone payment from its neurological disorder collaboration with Biogen (BIIB). The $10M payment was triggered by the validation of an undisclosed target.
The FDA designates Alexion Pharmaceuticals' ($ALXN) Soliris (eculizumab) an Orphan Drug for the neurologic disorder Myasthenia Gravis (MG). The company is currently enrolling patients in a multinational placebo-controlled registration trial of eculizumab in patients with refractory generalized MG.
Novartis ($NVS) submits its BLA to the FDA for its Breakthrough Therapy-designated meningitis B vaccine Bexsero (Multicomponent Meningococcal Group B Vaccine [recombinant, absorbed]). The vaccine helps protect adolescents and young adults from ages 10 - 25 against invasive meningococcal disease caused by serogroup B (meningitis B).