Roche heralded the late-stage success of two big trials for its multiple sclerosis drug ocrelizumab today, which they say puts them on a straight road to regulatory filings for the injectable in early 2016. But even under the best-case scenario for the future, the Big Pharma player will face a tough task squaring off against the new oral drugs that have been divvying up this big market recently.
DBV Technologies has exited its end-of-Phase II meeting with FDA with a plan for the next steps of development of its peanut allergy vaccine. And with Aimmune Therapeutics breathing down its neck, the French biotech is keen to get its pivotal trial up and running before the end of the year.
Baxalta won't formally debut as an independent Baxter drug spinoff for a few more days, but the company isn't waiting to discuss an early peek at efficacy results from a Phase I/II study of their long-term gene therapy for hemophilia.
A couple of years ago, as Vical struggled to overcome a Phase III flop for its cancer vaccine, the biotech said it would hustle ahead with a Phase I/II study of a herpes vaccine as part of its strategy to calm jittery investors. But after the markets closed Monday, the San Diego-based company was forced to concede that their herpes strategy had misfired as well, with their vaccine failing to perform as well as a placebo.
Portola Pharmaceuticals has laid out the last batch of Phase III data on its "breakthrough" anti-anticoagulant andexanet alfa, filling in the numbers that will be reviewed by regulators considering the biotech's upcoming pitch for marketing approval. And right alongside that announcement comes the latest numbers from Boehringer Ingelheim's breakthrough program for idarucizumab, which is already under review as an antidote to its blockbuster Pradaxa.
Three years ago, BioMarin top scientist Henry Fuchs pitched BMN-111 as one of the company's most promising early-stage drug prospects, with stellar mouse data to suggest that it was on track to develop a therapy to correct the genetic mutation behind dwarfism. On Wednesday evening, the San Rafael, CA-based biotech outlined Phase II data from 26 children that provided ample proof-of-concept human data that Fuchs was well justified in his belief.
Last spring, as Aerie Pharmaceuticals execs rushed to man the ramparts as its share price tanked following the failure of its glaucoma drug Rhopressa, the company insisted that a slight change to its late-stage program would have been enough to get them over the goal line. So this morning, in a head-snapping turnaround, shares jumped 50% as the FDA proved amenable to making some changes that might let another Phase III trial succeed.
AstraZeneca says that its experimental gout drug lesinurad failed to hit the primary biomarker target for the dose that it's now seeking regulatory approval for in the U.S. and Europe. And this latest Phase III--the last of a trio--continues to demonstrate that the pharma giant can't land evidence that the drug provides a clear benefit to patients in the time allotted for the study.
Agios Pharmaceuticals' lead cancer drug extended its streak of promising clinical results, racking up remissions in an expanded Phase I trial as the company prepares for late-stage testing.
Genmab has moved a step closer to seeing daratumumab approved in double refractory multiple myeloma. A rolling submission for the breakthrough cancer therapy is now underway, raising hopes that the product will be on the market early next year.