In a potential $1 billion-plus deal that Cowen and Co. analyst Eric Schmidt described as "a necessary and favorable solution" for Merrimack Pharmaceuticals Inc.'s financial straits, the company is selling its approved pancreatic cancer drug Onivyde and its generic version of Doxil candidate to Ipsen SA.
Regulatory progress in a little-understood blood disease with no established treatment put juice into shares of Stemline Therapeutics Inc., which nailed down a swift pathway for phase II, interleukin 3 receptor-targeting SL-401.
Analysts split on what the positive, pooled-groups phase II data in pancreatic cancer might mean for Halozyme Therapeutics Inc.'s larger, ongoing phase III trial, but the company sounded upbeat in a conference call on the top-line results from HALO 202 with PEGPH20 (pegylated recombinant human hyaluronidase).
Scott Wasserman, Amgen Inc.'s vice president of global development, told BioWorld Today that a new, 5,000-patient observational study will "cast a more focused light on the unmet need" in knocking down levels of low-density lipoprotein cholesterol (LDL-C), as researchers will take "multiple snapshots" over a three-year period. "It's meant to help the medical community get a pulse on how we're doing in terms of treating these high-risk patients," he said.
Although doing so isn’t necessary for the a 505(b)(2) regulatory pathway, Ardelyx Inc. is conducting phase II and phase III trials with its potassium binder to win a package insert that competes well, President and CEO Mike Raab told BioWorld Today.
A weight-losing outlier in the phase III trial's placebo group "should have been picked up earlier or should have been thrown out before we unblinded," Opko Health Inc.'s vice president, Steven Rubin, told BioWorld Today, and that anomaly, with potentially others, changed an otherwise-positive outcome with long-acting human growth hormone hGH-CTP in adults with growth hormone deficiency.
Given the frets of an advisory committee, the FDA’s complete response letter to Cempra Inc. for ketolide antibiotic solithromycin (soli) wasn’t much of a surprise – but a request by the agency for a pre-approval safety study that would enroll about 9,000 patients was, and Wall Street reacted accordingly.
Much talked-about at this year's ASH meeting, the CD33-targeting antibody-drug conjugate SGN-CD33A (33A) from Seattle Genetics Inc. faltered as the FDA put clinical holds on phase I and phase I/II trials in acute myeloid leukemia, though enrollment in the phase III CASCADE study continues.
The "beautiful, eloquent" research that led to emicizumab for hemophilia A will "dramatically change the way they live," Nancy Valente with Roche Holding AG unit Genentech told BioWorld Today. "No one's ever used this approach to treat this disease."
