Gene therapies command avid attention due to their promise of providing a cure for some terrible conditions along with the prospect of a 7-figure price tag. But some prominent investigators at the University of Pennsylvania reported that their "cure" for blindness only worked for one to three years before patients' vision began to fade again. And their decline could rattle some of the hot biotechs in the group.
Zostavax is the only shingles vaccine approved in the U.S. and the EU, but GlaxoSmithKline is one step closer to changing that. On Tuesday, the British pharma announced Phase III results for its shingles candidate, HZ/su, reporting an overall efficacy of 97.2% compared with placebo.
Two months ago Aerie Pharmaceuticals CEO Vicente Anido was boasting to Reuters that his late-stage therapy for glaucoma, Rhopressa, was on track to an approval with a clear shot at earning $1 billion a year. On Thursday evening, he was forced to mount a defense of the drug after conceding defeat in a key Phase III study and watching shares plunge 66% in a rout.
Amgen and its partner AstraZeneca are winding up to deliver U.S. and European applications for their big blockbuster hopeful brodalumab for plaque psoriasis by midyear. But their last shot to push ahead with the drug in asthma ran into defeat, with Amgen announcing Tuesday afternoon that it pulled the plug on a Phase IIb following a futility assessment.
Two years after going down in flames in a Phase III study for CD22-positive non-Hodgkin's lymphoma, Pfizer has helped resurrect hopes for top drug prospect inotuzumab ozogamicin, saying the drug hit its first primary endpoint for spurring hematologic remission in patients with CD22-positive acute lymphoblastic leukemia.
Shares of Ampio Pharmaceuticals were crushed this morning after the biotech acknowledged that its lead drug flunked a Phase III test for osteoarthritis. And the news comes just days after one of its drug subsidiaries was absorbed into a new biotech that will be called Aytu.
Buoyed by the success of its PAH franchise, Switzerland's Actelion says it has begun a Phase III study of its in-house drug ponesimod for multiple sclerosis.
The litany of clinical trial disasters that has played out at Targacept over the past three years continues this morning with the news that its last remaining pipeline asset failed a Phase I/II study for diabetic gastroparesis. And as has happened so many times before, the biotech's share price took another nasty tumble, dropping 22%.
Pharmalink has stopped a Phase IIb trial of its primary IgA nephropathy treatment early after it met its primary endpoint in a planned interim analysis. The success makes Pharmalink a rare example of a private biotech with an unpartnered, Phase III-ready asset, characteristics that inevitably lead to talk of IPOs and deals.
Given the varying ways that individual ALS patients progress, gradually growing paralyzed and dying in 3 to 5 years, he reasoned, the results from 8 patients after only 12 weeks of treatment, with 4 patients reserved for the placebo arm, couldn't provide a reliable look at efficacy.