In a fresh sign that the big biotech IPO wave that triggered a long lineup of dazzling debuts may have crested, two new California contenders have had to significantly scale down their expectations and a third has decided to bow out for now.
Winter's coming, along with warnings that new cases of H7N9 bird flu in China warrant a global pandemic alert. While the most recent pandemic alarms have fizzled out without any major outbreaks of a lethal virus, the federal government has been supporting work on new vaccine technologies.
After a scathing staff review, Sanofi walked away from an FDA panel with mixed messages on its long-delayed multiple sclerosis drug, as agency advisers said the injection wasn't too risky to approve but took issue with the drugmaker's trial design.
Theravance CEO Rick Winningham put out the word today that its drug TD-9855 flunked a mid-stage study for attention deficit/hyperactivity disorder, adding that the biotech planned to scrap the program while continuing a separate study for fibromyalgia.
The FDA has come through with an early approval of the BTK drug ibrutinib, a likely new cancer blockbuster from Pharmacyclics (and Johnson & Johnson.
Shares of Sarepta plunged 64% by the end of the day Tuesday, wiping out hundreds of millions of dollars of market valuation in a brutal reaction to the company's admission that the FDA had closed the door to an early approval of eteplirsen, its promising mid-stage therapy for Duchenne muscular dystrophy
Floundering Transcept Pharmaceuticals is cutting 43% of its workforce and shuffling its board, looking to cut costs as it scouts for a buyer.
So far this year the FDA has approved 27 new drugs, fewer than last year's total. But a greater focus on real medical breakthroughs will make this year's crop far more valuable than the harvest of 2012, according to EP Vantage.
Waltham, MA-based GeNO is looking to raise $50 million to back its work on a new, patient-friendly approach for delivering inhaled nitric oxide on the go. And a Dutch biotech, uniQure, is also positioning itself to go public after pioneering the world's first approved gene therapy.
Merck KGaA is strengthening its embrace of China's BeiGene, adding a second cancer drug development alliance to their collaboration along with an extra $232 million package of new milestone payments.
Fresh off a $46 million funding round, Germany's immatics has inked a deal to collaborate with Roche on cancer therapies, signing an end-to-end agreement that could bring in more than $1 billion in milestones.
Liquidia Technologies has spun out a new biotech company in Research Triangle Park, NC, which boasts an early-stage ophthalmology program and $25 million in venture backing from some high-profile investors.
Oxford-based PsiOxus Therapeutics came through with a slate of mostly upbeat results from its mid-stage study of its lead drug MT-102, an experimental therapy designed to combat potentially lethal weight loss among cancer patients.
Troubled Dendreon is again cutting its payroll, planning to shed about 200 employees in an effort to water down its cash burn rate and finally turn a profit. But, with a disappointing prostate cancer drug and a dim outlook for the future, the Seattle drugmaker may need more than lighter books to reverse its fortunes or find an acquirer.
The drug--designed to correct the circadian rhythms of the blind to ensure normal sleep patterns--worked as billed in the late-stage program for non-24 hour disorder, he wrote, and, given that there were no serious adverse events to consider, ought to be endorsed during an upcoming expert panel review and then approved by the agency.
In what has been one of the most hotly contested issues among biotech investors this year, many had bet big that the FDA would push through an accelerated approval for eteplirsen based on some very promising results from a mid-stage study. The study, though, only included a dozen boys, making the prospect of an early approval problematic at best.
The pharma giant reported that the drug did not produce a statistically significant improvement in the risk of heart attack, stroke or death, though it added that "greater reductions" for some of the secondary endpoints warranted careful follow-up review as investigators considered its potential.
The FDA has approved Sunovion's Aptiom (eslicarbazepine), an add-on therapy designed to prevent epilepsy seizures. "Some patients with epilepsy do not achieve satisfactory seizure control from existing treatments," said Dr. Eric Bastings, acting director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research.
GlaxoSmithKline has racked up a slate of drug approvals this year, an impressive accomplishment for a company that had to endure a lengthy fallow period in R&D after it reorganized its extensive research operations.
Over the past 5 years, the main arteries of drug R&D have pointed straight to the world's biggest biopharma hubs. As hubs have grown, outlying R&D operations have been steadily trimmed away.