Word has trickled down from scientists to investors to patients that gene therapy may offer a potentially curative approach for crippling blood disorders, such as sickle cell disease (SCD) and hemophilia, that involve the mutation of a single gene. That growing awareness is leading to a wholesale effort to handicap the field of noncancer hematology agents and propelling heightened – some would say naïve – expectations to produce tomorrow's therapies today.
Eric Devroe, founder, CEO and – for now – the only full-time employee of Opsonix Inc., is well aware of multiple failed efforts by drug developers to thwart the often deadly effects of sepsis. Many of those shortfalls, he maintained, focused on approaches that attempted to temper the immune response, which occurs downstream in the sepsis cascade.
The glass slipper fit. In short, that's how the founders of X4 Pharmaceuticals Inc. managed to in-license a platform, corral a $37.5 million series A round and secure FDA clearance of an investigational new drug (IND) application to move lead candidate, X4P-001, into a phase Ib/IIa study in patients with refractory clear cell renal cell carcinoma (ccRCC), expected to begin enrolling in the first quarter of 2016.
Baxalta Inc. rang up the second U.S. approval since its official split from Baxter International Inc. with the FDA's nod to Vonvendi (von Willebrand factor [recombinant]).
The pick-up of Synageva Biopharma Corp. provided an early payback for Alexion Pharmaceuticals Inc., which saw the FDA green-light Kanuma (sebelipase alfa) as the first approved U.S. therapy to treat patients with lysosomal acid lipase deficiency.
Ten days after disappointing investors in what appeared a messy mistake in the European phase III trial of Xilonix in symptomatic, advanced colorectal cancer, Xbiotech Inc. turned its story around by reporting that the interleukin-1 alpha ligand inhibitor met the primary study endpoint of response rate compared to placebo.
The annual meeting of the American Society of Hematology (ASH) in early December serves as the traditional curtain closer for the year's clinical development efforts. In 2015, it's fair to say, the FDA upstaged the ASH bash by approving a bevy of important drugs targeting blood disorders – many of them just weeks before the meeting.
Zafgen Inc. apparently learned that waiting only delays the inevitable after its earlier two-day silence following a patient death in its phase III study, bestPWS, evaluating beloranib, a twice-weekly subcutaneously administered drug targeting the methionine aminopeptidase 2a (MetAP2) pathway, in Prader-Willi syndrome (PWS).
The saga that unfolded during last week's Peripheral and Central Nervous System Drugs Advisory Committee (adcom) not only left Biomarin Pharmaceutical Inc. twisting in the wind as the company awaits the FDA's decision on Kyndrisa (drisapersen), but also raised a bigger question for companies pursuing therapies to treat DMD that link their utility to an effect on the protein dystrophin.
Let’s face it, Star Wars references are unavoidable in this year’s holiday gift guide. Not convinced? I find your lack of faith disturbing. Several members of our intrepid staff and respondents to our annual poll gave a nod to the awakening force. Jennifer Boggs, BioWorld’s managing editor, was lamenting about holiday gifts like toasters that are practical but, let’s just say, not very exciting when she discovered “there are some cool toasters after all. I stand corrected.” Her find was the Star Wars Darth Vader toaster, a true likeness of the famously redeemed villain, although you’ll have to look for...
