It proved a quick nod for Novartis AG's Entresto (sacubitril/valsartan), a combination ACE/neprilysin inhibitor that demonstrated an ability to reduce the rate of cardiovascular death and hospitalization related to heart failure in pivotal trials.
Vertex Pharmaceuticals Inc.'s Orkambi won FDA approval Thursday, as largely expected, expanding the firm's reach into the cystic fibrosis (CF) market and serving as a "great example of what precision medicine can do and will do in the future," said Chairman, President and CEO Jeff Leiden.
A phase IIb failure of sodium channel Nav1.7 inhibitor TV-45070 in osteoarthritis (OA) of the knee should have no readthrough to an ongoing phase IIb study of the topically applied small molecule in postherpetic neuralgia (PHN), executives of Xenon Pharmaceuticals Inc. assured investors Wednesday morning, citing distinct pain mechanisms between OA and neuropathic pain indications.
Executives at Amag Pharmaceuticals Inc. were already hinting at future acquisitions on Monday's conference call even as investors were digesting news of the $700 million cash purchase of Cord Blood Registry (CBR), a newborn stem cell collection and storage firm.
Results from Alcobra Ltd.'s phase II study of MDX (metadoxine extended release) in the rare neurogenetic disorder fragile X syndrome (FXS) may point in a positive direction for the program, even if the firm's stock went the other way Wednesday.
Three months after regaining global rights to EZH2 inhibitor tazemetostat, Epizyme Inc. presented updated phase I data demonstrating a deepening response rate over time in patients with advanced B-cell non-Hodgkin lymphoma (NHL) and reiterated plans to advance into phase II testing in NHL and in solid tumors that are INI1-deficient.
PHILADELPHIA – With biotech enjoying a spurt of tremendous growth – increasing market caps and widening profit margins – another trend has emerged, one that’s not quite so enjoyable, at least for company execs: increasingly confrontational shareholder activity.
PHILADELPHIA – Kicking off the last panel of this year's BIO International Convention, radio and television host David Brancaccio referenced the scene from The Graduate, when a family friend tells Dustin Hoffman's character, "There's a great future in plastics." Brancaccio asked a group of panelists during the Scientific American Worldview session what word or phrase signifies to each of them a great future in today's world.
PHILADELPHIA – Interest – with perhaps a bit of confusion – abounds in the microbiome space, as evidenced by the fact that there were two panels at this year's BIO International Convention, compared to the single panel last year.
PHILADELPHIA – Anything an antibody can do, only better: That's the big idea behind Cytomx Inc.'s Probody platform, which, despite its early stage, has landed a couple of potentially lucrative big pharma deals.
