Although Jefferies analyst Michael Yee noted that the "market is somewhat skeptical" of neurodegenerative disease research at the moment, Prothena Corp. plc CEO Gene Kinney told BioWorld that he expects there will be more, not fewer, of the deals like the potential $2.2 billion pact that his firm struck with Celgene Corp. "We're seeing a high level of interest in the space right now," he said, a contrast from the slacking caused years ago by some well-publicized blowups.
To say whether U.S. regulators will require heart monitoring after the first dose of Arena Pharmaceuticals Inc.'s etrasimod for ulcerative colitis (UC) would be "prognosticating a bit in terms of what the FDA is going to choose to do," said Chief Medical Officer (CMO) Preston Klassen, who refused such guesswork.
Given the timing of the company's earnings call last week, investors might have expected phase II data with etrasimod in ulcerative colitis (UC) from Arena Pharmaceuticals Inc., but eagerly awaited results from the 157-patient phase experiment testing the oral sphingosine-1-phosphate (S1P) receptor modulator will not roll out until later this month.
As PDUFA dates near for the antisense drug inotersen and a competing therapy in hereditary transthyretin amyloidosis, or hATTR, Ionis Pharmaceuticals Inc.'s potential $1.7 billion deal with spinout Akcea Therapeutics Inc.was designed to prepare for launch, but not everybody was impressed – at first, anyway.
The surprise thumbs-down recently by the FDA for Celgene Corp.'s ozanimod to treat relapsing multiple sclerosis (MS) echoed the disappointment last year with mongersen (GED-0301) for Crohn's disease (CD), but apparently didn't affect late-stage work with the former compound in ulcerative colitis (UC) and CD.
Though it provided what CEO Craig Thompson called "key information" to inform a second trial, Anthera Pharmaceuticals Inc.'s narrow topline phase III SOLUTION miss with recombinant pancreatic enzyme replacement therapy (PERT) Sollpura (liprotamase) in cystic fibrosis (CF) patients who have exocrine pancreatic insufficiency (EPI) sent shares of the firm (NASDAQ:ANTH) reeling 63 percent, or $1.27, to close at 74 cents.
VBL Therapeutics Inc. CEO Dror Harats cautioned investors about jumping to conclusions regarding the late-stage ovarian cancer bid with anti-angiogenic gene therapy VB-111 after the candidate failed in top-line data from the phase III trial against recurrent glioblastoma (GBM).
By signing the potential $548.8 million deal for the oral, small-molecule glucagon receptor antagonist LGD-6972 with Ligand Pharmaceuticals Inc., Roivant Sciences Inc. brings aboard a type 2 diabetes candidate that "should surpass blockbuster status" if approved, in the view of one analyst.
Glycomimetics Inc. CEO Rachel King said "a unique constellation of benefits" brought by GMI-1271 to relapsed/refractory acute myeloid leukemia (AML) led the company to decide on a phase III endpoint of overall survival (OS) to capture the widest upside clinically.
