In a terse bulleted statement, Globeimmune Inc. disclosed what sounded like its death knell: dissolution of its license agreement with Gilead Sciences Inc. for hepatitis B virus (HBV) candidate GS-4774, wholesale resignation of the company's CEO and board and termination of its office and manufacturing space.
Gilead Sciences Inc. reported another late-stage pipeline setback after a pair of phase III trials evaluating JAK inhibitor momelotinib (formerly GS-0387) in myelofibrosis missed key endpoints. SIMPLIFY 1 and 2 were designed to compare momelotinib to Jakafi (ruxolitinib, Incyte Corp.) or best alternative therapy (BAT) in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. In the double-blind, active-controlled SIMPLIFY 1 study, 432 myelofibrosis patients who were not previously treated with a JAK inhibitor were randomized (1-to-1) to momelotinib or ruxolitinib for 24 weeks. In SIMPLIFY-2, 156 patients previously treated with but not refractory to ruxolitinib were randomized (2-to-1) to momelotinib or BAT for 24 weeks.
After quietly incubating for a year under the tutelage of co-investors Third Rock Ventures LLC and Atlas Venture, Magenta Therapeutics burst onto the cell therapy scene with a $48.5 million A round to advance its goal of transforming stem cell transplantation for patients with autoimmune diseases, genetic blood disorders and cancer.
The dollars were small but the trend was mighty. Such was the consensus of analysts on the first full quarter results for Acadia Pharmaceuticals Inc. following the launch of Nuplazid (pimavanserin) on May 31 to treat hallucinations and delusions associated with Parkinson's disease psychosis (PDP).
Biogen Inc. looked for a broader label in connection with potential approval of the spinal muscular atrophy (SMA) candidate, nusinersen, after reporting with Ionis Pharmaceuticals Inc. that the phase III CHERISH trial in children with later-onset disease met the primary endpoint at interim analysis and was stopped early.
Signaled by briefing documents posted by the FDA earlier in the week, the efficacy of solithromycin (soli) to treat community-acquired bacterial pneumonia (CABP) was not in dispute even before the agency's Antimicrobial Drugs Advisory Committee (AMDAC) met Friday to discuss new drug applications submitted by sponsor Cempra Inc. for the agent's oral and injectable formulations.
Per the annual pre-holiday tradition, abstracts poured out Thursday morning precisely one month prior to the start of the American Society of Hematology (ASH) annual meeting in San Diego. As usual, analysts and investors reacted quickly to the summaries, and many were less than enthused.
Avexis Inc. is preparing for a David-and-Goliath showdown with Biogen Inc. and its partner, Ionis Pharmaceuticals Inc., after the FDA green-lighted the design of a pivotal trial for the upstart's gene therapy candidate, AVXS-101, in infants and children with spinal muscular atrophy (SMA) type 1.
After advancing its proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor, bococizumab, into a global phase III development program that included six lipid-lowering and two cardiovascular (CVD) outcome trials (CVOTs), Pfizer Inc. blinked.
