The up front is only $10 million, but Arcturus Therapeutics Inc. stands to collect up to $1.56 billion more from Ultragenyx Pharmaceutical Inc. in a rare disease research collaboration and license deal to discover and develop messenger RNA (mRNA) therapeutics using its unlocked nucleomonomer agent (UNA) oligomer chemistry and Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR) nanoparticle delivery platform.
Look out, big pharma. The first generation of scientists who came of age during the genomics revolution is transitioning from the classroom to the boardroom, and they're seeking to shake up the industry.
FDA acceptance of the investigational new drug (IND) application for SM-88, a metabolic inhibitor that targets cancer cells, gave the green light to developer Tyme Inc., through wholly owned subsidiary Tyme Technologies Inc., to begin enrolling patients in a multicenter phase II study targeting breast cancer.
Months after closing a heavily oversubscribed series A extension and expanding its cash haul to $89 million, Crispr Therapeutics AG found itself opening a much larger cash drawer.
Shares of Spectrum Pharmaceuticals Inc. (NASDAQ:SPPI) fell to a one-year low of $4.89 Friday morning after the company said it received a complete response letter (CRL) from the FDA on Evomela (melphalan). The high-dose intravenous formulation of Captisol-enabled (CE) melphalan is designed as a conditioning treatment prior to autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM).
With trailblazer 23andme Inc. re-emerging in the personal genomics space with a $115 million series E to reinvigorate its direct-to-consumer efforts and the price point for whole genome analysis falling below the important $1,000-per-person barrier, precision medicine (PM) has made its way into the everyday vernacular.
SAN FRANCISCO – Data on the cost and efficiency of clinical trials are downright depressing. The ratio of dollars invested in R&D to the return on investment has declined about threefold over the past 15 years, according to a study by McKinsey & Co. cited by Lawrence Klein, an associate partner at the consulting firm, who led a lively discussion at the BIO Investor Forum examining methods and tools that could lower costs while speeding drug development.
SAN FRANCISCO – This year has been paved with more legislative and regulatory twists and turns for biopharma than the yellow brick road, and the end is not yet in sight, according to a panel of legal, business and regulatory experts at the 2015 BIO Investor Forum in San Francisco.
