Sage Therapeutics Inc. sought to emphasize the lessons learned and increased understanding from the top-line readout of its first phase III trial, which assessed brexanolone (SAGE-547) in patients with super-refractory status epilepticus, or SRSE. The highly anticipated trial missed its primary endpoint of comparing the candidate's success in weaning of third-line agents and resolution of potentially life-threatening status epilepticus vs. placebo (43.9 percent vs. 42.4 percent; p=0.8775) when added to standard of care. Read More
Hopes that Onxeo SA's doxorubicin-loaded nanoparticle, Livatag, would finish a pivotal trial and be ready for filing as a new treatment for challenging cases of hepatocellular carcinoma were dashed as the drug failed to outperform standard-of-care therapies, such as oxaliplatin, gemcitabine or tyrosine kinase inhibitors. News of the results sent Onxeo shares (PARIS:ONXEO) down more than 50 percent and turned the company's attentions to finding a partner to advance Livatag, as it focuses on other programs in its pipeline. Read More
SHANGHAI – For the first time in its 12-year history, the International Conference for Rare Diseases and Orphan Drugs (ICORD) convened its annual event in China. The event marks a milestone for the local event partner, the Chinese Organization for Rare Disorders (CORD), a patient advocacy non-governmental organization that has managed to thrive in China's harsh civil society climate. Read More
MADRID, Spain – The global disparity of available and affordable medicines is not a topic on the forefront of many oncologists' minds when an ailing patient sits in front of them. Read More
PERTH, Australia – New listings of higher-priced innovative drugs on Australia's government-subsidized Pharmaceutical Benefit Scheme (PBS) illustrate the government's commitment to list new innovative drugs, and pharma companies will see improved commercial opportunities in Australia, according to a recent BMI Research report. Read More
DUBLIN – Proqr Therapeutics NV has spun out a startup firm, Amylon Therapeutics NV, to take forward an RNA-based drug development program focused on rare beta-amyloid-related disorders. The new company has raised an undisclosed level of seed funding from a group of institutional and private investors and has appointed Thomas de Vlaam, formerly therapeutic area lead for CNS indications at Proqr, as CEO. Read More
The FDA released a draft guidance providing nonclinical study recommendations for the development of microdose radiopharmaceutical diagnostic drugs. The draft is intended to provide recommendations for a pathway to marketing authorization, the FDA said in a notice slated for publication in Wednesday's Federal Register. Comments should be submitted by Nov. 12. Read More
India Globalization Capital Inc., of Bethesda, Md., reported in vitro data compiled from genetically engineered cell lines showing that, at varying concentrations of tetrahydrocannabinol (THC), the production of amyloid beta 40 protein decreases by as much as 50 percent over a 48-hour period. Read More
Amgen Inc., of Thousand Oaks, Calif., and UCB SA, of Brussels, Belgium, disclosed detailed results from the phase III Arch study showing that 12 months of sclerostin binder Evenity (romosozumab) followed by alendronate was superior in reducing new vertebral, clinical, nonvertebral and hip fracture risk in postmenopausal women with osteoporosis at high risk for fracture, compared to alendronate alone. Read More
Synthetic Biologics Inc., of Rockville, Md., said it closed a privately placed stock purchase transaction for the sale of redeemable convertible preferred stock to an affiliate of MSD Partners LP for aggregate gross proceeds of $12 million. Read More
Targovax ASA, of Oslo, Norway, presented results from an observational clinical study with RAS peptide vaccine TG-01 in nonresectable pancreatic cancer patients, showing that 14 of 25 patients (56 percent) had a positive delayed type hypersensitivity (DTH) skin reaction test by week 10. Read More
