Think "rare, pediatric diseases" and you tend to think "drug cocktail that changes over time" and "almost impossible to get enough investors, given the risk vs. return, not to mention the long development period."
Is it possible, by tweaking the only molecule approved for movement disorders in Huntington's disease, to come up with a new chemical entity that's safer and dosed less often, thanks to a much longer half-life?
Poised to wade into a crowded field of drugs for hereditary angioedema (HAE), Santarus Inc. and partner Pharming Group NV said the pivotal Phase III study with the C1 esterase inhibitor Ruconest for acute HAE attacks met the primary endpoint of time to beginning of symptom relief.
With safety and efficacy endpoints met last week in the Phase III trial testing Biogen Idec Inc.'s long-lasting rFVIIIFc (recombinant Factor VIII Fc fusion protein) for hemophilia A, investors were scrutinizing the candidate's odds in a market led by Advate, the serum-free, recombinant Factor VIII therapy from Baxter Healthcare Corp.
At least a year shy of the next milestone payment from partner Astellas Pharma Inc., Ambit Biosciences Inc. raised $50 million from existing investors to advance quizartinib in acute myelogenous leukemia (AML).
With a year's worth of progress on the would-be competition targeting cholesterol by way of a new mechanism, Regeneron Pharmaceuticals Inc. and partner Sanofi SA set forth on another Phase III trial – this time measuring cardiovascular (CV) outcomes – with SAR236553/REGN727.
Not every day do you see a company, in the course of developing its drug, sign a deal with two other firms that are working on a therapy in the same class. Even more unusual: The deal is designed to help come up with a reversing agent for both compounds, and others.
By entering a potential $186 million research pact with Ensemble Therapeutics Inc. for synthetic macrocycles, Boehringer Ingelheim GmbH became the latest to place its bet on a newish, challenging approach that could combine the all-in-a-pill possibility of small molecules with the biological bull's-eye talent of large ones.
With safety and efficacy endpoints met in the Phase III trial testing Biogen Idec Inc.'s long-lasting rFVIIIFc (recombinant Factor VIII Fc fusion protein) for hemophilia A, investors were scrutinizing the candidate's odds in a market led by Advate, the serum-free, recombinant Factor VIII therapy from Baxter Healthcare Corp.
I was on the phone last week with Rick Andrews, president and CEO of Thrasos, about acute kidney injury (AKI), and how it’s no longer called “acute renal failure,” the latter phrase having been scrapped in recent years. Cynics might claim it’s something the drug companies invented, so they can pump more chemicals into people earlier. But the idea, Andrews said, is to express that kidney trouble appears along a continuum, and pre-shutdown symptoms need to be handled promptly, because they create big woes on their own. Woes that might be lucratively attacked by the likes of Thrasos, with its...
