With a phase III trial bearing an identical endpoint and dosing schedule already under way, Alnylam Pharmaceuticals Inc. reported favorable data from the phase II open-label extension (OLE) study with patisiran, the RNAi therapy for transthyretin (TTR)-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP).
With a global, confirmatory phase III trial under way as of June with Translarna (ataluren) in nonsense mutation cystic fibrosis (nmCF), plus a phase III experiment in nonsense mutation Duchenne muscular dystrophy (nmDMD) fully enrolled as of last month, PTC Therapeutics Inc. is raising $108.75 million through the sale of 3 million shares at $36.25 each.
With encouraging data from a phase I trial in amyotrophic lateral sclerosis (ALS) and a phase II trial under way testing NSI-566, Neuralstem Inc. has begun – with the same candidate – the first human neural stem cell study to be given the FDA's nod for chronic spinal cord injury.
The stellar top-line data unveiled in January from Intercept Pharmaceuticals Inc.'s phase IIb obeticholic acid (OCA) trial known as FLINT in nonalcoholic steatohepatitis (NASH) helped the shares to a near 300 percent gain and whetted investor appetite for the full results, but it looks like they'll have to wait longer than next month's annual meeting of the American Academy for the Study of Liver Diseases (AASLD).
By now I’ve learned to keep my flu shot habit a secret. Every year about this time, since I was 18 years old, I get vaccinated. But long ago I stopped telling people about it, so I don’t have to hear: “Oh, you know the flu vaccines are made of antifreeze [mercury, aluminum, formaldehyde], right?” or “That’s a crazy thing to do, because the shot itself can make you sick!” or – this one’s fairly new – “You don’t really need it if you’ve got a healthy immune system. Anyway flu shots cause Alzheimer’s [in the old] and autism [in...
The modified intent to treat (mITT) phase III results with Alcobra Ltd.'s non-stimulant metadoxine extended release (MDX) for attention deficit hyperactivity disorder (ADHD) in adults gave investors the jitters, but they may have been distracted from the real meaning of top-line data by the exclusion of four high responders to placebo.
Investors hailed Esperion Therapeutics Inc.'s success with ETC-1002, the firm's dual-action cholesterol-lowering pill, sending upward the Ann Arbor, Mich.-based firm's stock as onlookers sought to gauge the drug's chances in a market that already includes generic therapies and could soon include proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors.
Visterra Inc.'s $30 million series B financing brought in more than expected and leaves the company with "a clear path for the next couple of years" to pursue therapies in dengue fever and influenza, said CEO Brian Pereira.
Would-be therapies in respiratory syncytial virus (RSV) took center stage with Johnson & Johnson's (J&J) plan to take over Alios Biopharma Inc. for $1.75 billion, gaining a pipeline that includes the phase II oral nucleoside analogue AL-8176 in RSV and a uridine nucleotide (nuke) analogue, AL-335, for hepatitis C virus (HCV) – another, less loudly touted driver for the deal.
BOSTON – The head of Pfizer Inc.'s Center for Therapeutic Innovation (CTI) called Boston "a wildly synergistic hotbed" for new ideas, during a panel on the final day of EBD Group's BioPharm America, designed to hook up would-be partners for talks around drug development deals.
