First to market with an all-oral first-line therapy for adults with Gaucher disease type 1, the most common form, is the Genzyme unit of Paris-based Sanofi SA, which gained FDA marketing clearance for Cerdelga (eliglustat), giving patients an option to the infused enzyme replacement therapy (ERT) Cerezyme (imiglucerase), also developed by Genzyme.
Globavir Biosciences Inc. formally threw its hat into the Ebola ring, making known the firm's plan to develop the preclinical candidate GBV006 as a potential remedy for the virulent outbreak in West Africa.
Biogen Idec Inc. is keeping its injectable bases covered in relapsing multiple sclerosis (RMS) with the approval Friday of Plegridy (peginterferon beta-1a), given by an auto-injector pen or prefilled syringe every two weeks, a significant half-life advantage over the firm's Avonex (interferon beta-1a) for RMS, given weekly.
Among the founders of cystic fibrosis (CF) player Proqr Therapeutics BV, which has filed to raise $75 million in an initial public offering (IPO), is a familiar name: Henri Termeer, former CEO of Genzyme Corp.
Word of another Captisol-based deal, the phase III failure of an ongoing royalty source and the forecast – from short-positioned Lemelson Capital Management – of financial doom because of new convertible debt complicated Ligand Pharmaceuticals Inc.'s picture.
A morsel of phase IIa data from Arrowhead Research Corp. left investors wanting more and sparked analyst speculation about the potential of the firm's ARC-520, an RNAi-based candidate for hepatitis B virus (HBV).
Warning shareholders that the strategy to handle $620 million in debt due in 2016 could result in their ending up with "little or no ownership," Dendreon Corp. reported second-quarter earnings that showed the prostate cancer (PC) drug Provenge (sipuleucel-T) gaining 12 percent in sales year over year and 19 percent quarter over quarter, reaching $82 million.
No longer left to its own devices in putting Afrezza inhaled insulin on the market, Exubera-haunted Mannkind Corp. landed an eagerly awaited commercialization deal with Sanofi SA that brings $150 million up front and as much as $775 million in regulatory, development and sales milestone payments for the second-generation product.
With a multi-cyclin-dependent kinase (CDK) small-molecule inhibitor working its way toward phase III trials next year in acute myeloid leukemia (AML), Tolero Pharmaceuticals Inc. raised $14.2 million in series B money that will also help advance the Salt Lake City-based firm's significant preclinical pipeline.
The outbreak of Ebola virus in West Africa and the experimental treatment of two afflicted Americans at Emory University Hospital have brought to the fore ethical questions regarding the wise use of previously untried therapies, as well as a debate about the patients who should get them.
