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.
With positive early phase III data in hand, Clearside Biomedical Inc. plans to file an NDA in the fourth quarter of this year and is probing partnership options outside the U.S. for marketing CLS-TA, a drug-device combo designed to treat patients with macular edema associated with non-infectious uveitis.
"We want to build the Genentech of small molecules, and we want to do it San Diego," Vividion Therapeutics Inc. CEO Diego Miralles told BioWorld, as the company made public a deal that brought $101 million up front from Celgene Corp., which included an equity investment as part of the amount.
The clinical failure-strewn world of Alzheimer's disease (AD) awaits top-line, part A results due in April from Vtv Therapeutics Inc.'s phase III study called Steadfast with azeliragon, an antagonist of the receptor for advanced glycation end products (RAGE), and many hope the new approach can break through the development wall that other drug candidates have splattered themselves against.
