The FDA issued a draft guidance Thursday to tackle some of the practical and ethical challenges of using placebos and blinding in clinical trials testing therapies for hematological malignancies and other cancers. The draft recommends placebo-controlled designs only in selected circumstances, such as when surveillance is the standard of care or when the trial uses an add-on design. Read More
Obalon Therapeutics Inc., of San Diego, said it entered into an agreement to sell shares of its common stock for aggregate gross proceeds of $10 million in a private placement. The company will sell approximately 5.5 million shares of common stock at $1.82 each. It plans to use the proceeds for commercial operations, working capital and general corporate purposes. Read More
Innovate Biopharmaceuticals Inc., of Raleigh, N.C., said it signed an agreement with Amarex Clinical Research to provide data management and biostatistics for its planned phase III larazotide trial in celiac disease. Larazotide acetate has a mechanism of action that renormalizes the dysfunctional intestinal barrier by decreasing intestinal permeability and reducing antigen trafficking, such as gliadin fragments in celiac disease, and bacterial toxins and immunogenic antigens in nonalcoholic steatohepatitis. Read More
A phase III test combining Novartis AG's phosphoinositide 3-kinase (PI3K) inhibitor, BYL-719 (alpelisib), with fulvestrant to treat a genetically-defined subset of advanced breast cancer patients found that it improved progression-free survival in those individuals, as compared to treatment with fulvestrant alone. The top-line success set the stage for the company to move closer to filing global regulatory applications for the drug, which Novartis oncology chief Samit Hirawat called the first of its kind "to show potential increased benefit and acceptable tolerability for patients." Read More
Rakuten Aspyrian Inc.'s $150 million from its series C round will fuel a near-term phase III trial and more with ASP-1929, a light-activated antibody drug conjugate (ADC), CEO Miguel Garcia-Guzman told BioWorld. "Probably by mid-next year, we'll be running ASP-1929 in head and neck cancer plus three additional [oncology] indications," he said, adding that the new capital "will not be able to finance everything toward the market," and the ambitious firm will seek more as needed. Read More
As it so often is, Thursday's U.S. Senate Health, Education, Labor and Pensions (HELP) Committee hearing on NIH research was a showcase of innovation and the realization of what once seemed the realm of science fiction. But the show-and-tell session wasn't limited to the wonders springing from what NIH Director Francis Collins likes to call the "National Institutes of Hope." Read More
Bluebird Bio Inc. and privately held Gritstone Oncology Inc. said they plan to collaborate in a broad cancer cell therapy R&D and commercialization pact involving 10 tumor-specific targets across multiple tumor types and, in certain cases, T-cell receptors (TCRs) directed to those targets. Read More
LONDON – The U.K. government has told pharma companies to build a six-week stockpile and prepare to import drugs that have a short shelf life by air rather than by road, as part of contingency planning for a no-deal Brexit in March 2019. Read More
A little over one year after European regulators gave their nod, regulators in the U.S. cleared for marketing Milan-based Dompé farmaceutici SpA's Oxervate (cenegermin) eye drops for neurotrophic keratitis. Read More
Following its first FDA approval, for Inveltys (loteprednol etabonate ophthalmic suspension 1 percent) to treat inflammation and pain following ocular surgery, developer Kala Pharmaceuticals Inc. said it plans to hire a specialty sales force of about 60 reps to target 80 percent of likely prescribers in preparation to launch the ophthalmic suspension product early next year. Read More
