Alnylam Pharmaceuticals Inc.'s eleventh-hour settlement with Tekmira Pharmaceuticals Corp. leaves Alnylam $65 million lighter but still in strong financial shape, without losing rights to access the Lipid NanoParticle (LNP) delivery technology important for its RNAi work.

Poised to wade into a crowded field of drugs for hereditary angioedema (HAE), Santarus Inc. and partner Pharming Group NV said last week 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.

Top-line data from a Phase III trial with Abraxane plus gemcitabine in pancreatic cancer by Celgene Corp.'s subsidiary Celgene International Sàrl suggested a benefit over the chemotherapy agent alone in overall survival (OS).

It wasn't the first time that a deal by GlaxoSmithKline plc left Wall Street unsatisfied and ultimately fizzled, but the severing of XenoPort's tie with the pharma giant leaves the biotech in fairly good shape: full rights to Horizant (gabapentin enacarbil) and $20 million in new equity money.

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.