Politicians, scientists, health officials and advocates working to improve the lots of an estimated 5.7 million Americans living with Alzheimer's disease met in Washington Tuesday for a Senate Special Committee on Aging hearing on legislation promoting better management of the condition. If enacted, The Building Our Largest Dementia (BOLD) Infrastructure for Alzheimer's Act, first introduced late last year, would create a nationwide framework in which to implement Alzheimer's interventions focused on public health issues, such as increasing early detection and diagnosis, reducing risk, and preventing avoidable hospitalizations. With 35 co-sponsors, it's moving through the Senate Health Committee.
Building on positive phase II data in an ultra-rare form of pediatric epilepsy, Marinus Pharmaceuticals Inc. is kicking off a pivotal phase III test of oral ganaxolone, its GABA A receptor agonist and sole clinical candidate. The drug, which like many candidates failed at first in another indication, appears to have the potential to help treat cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder, an early-onset condition that mostly affects girls. The study has a treatment period of 17 weeks and will measure percent reduction in seizures as its primary endpoint. If successful, ganaxolone would become the first drug approved for the indication.
Updated interim data on an experimental spinal muscular atrophy (SMA) therapy co-developed by PTC Therapeutics Inc., Roche Holding AG and the SMA Foundation show that more than 90 percent of the 21 babies enrolled in the first part of an open-label study experienced a greater than four-point increase on a test designed to measure motor milestone development of patients with SMA type 1.
Stridebio Inc., a company developing adeno-associated viral (AAV) vectors for in vivo gene therapies, has raised $15.7 million in an oversubscribed series A financing, money the company said will enable it to move multiple programs toward the clinic, with the first likely to reach proof of concept within the next six to nine months.
An oligonucleotide-based TLR antagonist developed by Idera Pharmaceuticals Inc. to treat adults with the rare inflammatory disease dermatomyositis (DM) has failed to best a placebo on a common measure of disease severity, missing the phase II trial's primary endpoint. The shortfall for Idera's IMO-8400 opens the field for the mid-stage cannabinoid receptor type 2 agonist, lenabasum, in development at Corbus Pharmaceuticals Holdings Inc., and other DM programs as Idera turns its attention more fully to advancing its lead program, tilsotolimod, for melanoma and other tumor types, and its proposed merger with Biocryst Pharmaceuticals Inc.
Vical Inc. is quitting development of VCL-HB01, its Vaxfectin-formulated plasmid DNA vaccine against herpes simplex virus type 2 (HSV-2), after the candidate missed what company President and CEO Vijay Samant called a "very rigorous" primary endpoint of annualized recurrence rate in a phase II trial. Company shares (NASDAQ:VICL) fell about 26.4 percent to $1.20 by Monday's market close, marking the second big drop this year after a phase III cytomegalovirus vaccine the company had partnered with Astellas Pharma Inc. missed its endpoints in January.
BOSTON – Digital health platforms and advanced computational techniques are finding increasing use in big pharma, both improving and, at times, complicating the enterprise. Though varied efforts are taking shape across the world, Rachel Sha, vice president of digital business development at Sanofi SA shed light on her company's approach at BIO International Convention.
BOSTON – U.S. bioscience firms directly employ 1.74 million people, according to a new report published by BIO, with average annual wages for a U.S. bioscience worker reaching $98,961, at least as of 2016. But despite the industry’s positive impact, there remains room to grow, and energy to be invested in broadening the industry’s reach, two new efforts show.
BOSTON – Following the Senate Health, Education, Labor and Pensions Committee's overwhelming approval to reauthorize the Pandemic and All Hazards Preparedness Act, which is set to expire in September, the House starts to examine renewal options for health emergency preparedness programs this week. It helps fund U.S. response to future threats and beef up the nation's medical countermeasure enterprise. But ultimately, some of the projects its funds flow to also support civilian biopharma ventures.
