Calling the top-line phase Ia/IIb data with the company’s antisense drug for spinal muscular atrophy (SMA) in children “achingly important” and the benefit in a subset “shocking,” Isis Pharmaceuticals Inc. CEO Stanley Crooke said intrathecal ISIS-SMNRx has proven safe so far, too.

Not surprising but hardly well taken by investors was the second setback with Onconova Therapeutics Inc.’s intravenous (I.V.) rigosertib for higher-risk myelodysplastic syndromes (MDS), which failed to meet its primary endpoint of overall survival (OS) in the phase III trial known as ONTIME.

With a launch expected in the second half of this year, Chelsea Therapeutics International Ltd. is firming up other plans for Northera (droxidopa), just approved by the FDA for neurogenic orthostatic hypotension (NOH), or a sudden drop in blood pressure when the patient stands up.

With phase IIb experiments under way in nondialysis chronic kidney disease patients with anemia, Akebia Therapeutics Inc. filed for an initial public offering (IPO) that would raise as much as $75 million, as plans also firm up for a phase II study in dialysis patients, slated to begin in the first half of this year.

The long, hazard-fraught road for Chelsea Therapeutics International Ltd. with Northera (droxidopa) – including unfavorable briefing documents, followed by a positive advisory committee (adcom) vote – came to a happy end with approval of the drug by the FDA for neurogenic orthostatic hypotension (NOH), or a sudden drop in blood pressure when the patient stands up.

Steadily declining hopes for tivozanib, the tyrosine kinase inhibitor of vascular endothelial growth factor (VEGF) receptors, hit bottom with Astellas Pharma Inc.’s decision to quit its deal with Aveo Oncology Inc., which brought $125 million up front in 2011 and the promise of more than $1 billion in potential milestone payments.

Though winter storm Pax put the freeze on air and ground traffic in the Northeast U.S. after ravaging the South, biotech funders seemed to feel little of the chill, with PTC Therapeutics Inc. and Macrogenics Inc. pricing public offerings for $109.7 million and $109.5 million, respectively.

The question of whether ordinary equity offerings are better than the sale of convertible notes as a financing method found differing answers from thriving Ironwood Pharmaceuticals Inc. and troubled intravenous-iron specialist Amag Pharmaceuticals Inc., both of which raised $175 million.

Fueled by $45 million in Series A money, gene therapy player Voyager Therapeutics Inc. is expected to make public today the details of its adeno-associated virus (AAV) approach in central nervous system (CNS) disorders.

The ability to seamlessly switch patients from an intravenous (I.V.) to an oral version of the same, broad-spectrum antibiotic could give Melinta Therapeutics Inc. a leg up in the field, and the company raised $70 million to help find out whether phase III-stage fluoroquinolone delafloxacin will make the grade.