Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
An earlier-than-intended release of abstracts for the American Society of Hematology annual meeting spilled multiple market-moving updates Nov. 4, though with no apparent shocks so far. Shares of Allogene Therapeutics Inc. sunk 8% on Nov. 4 over some metered disappointment around initial data for ALLO-715, a potential medicine for relapsed/refractory (r/r) multiple myeloma. By contrast, shares of Global Blood Therapeutics Inc. climbed nearly 15%, buoyed by new data supporting the long-term use of its sickle cell disease therapy, Oxbryta (voxelotor).
Dren Bio Inc. has closed on a $60 million series A to push its two lead programs through early clinical development. The company’s DR-01 is an antibody-based therapy for treating rare leukemias, lymphomas and specific phenotypes of autoimmune disorders. The company said it is initially targeting neglected hematology-oncology indications.
Cyclerion Therapeutics Inc.’s phase II blowup with sickle cell disease (SCD) candidate olinciguat ended its development, and attention turned to the Cambridge, Mass.-based firm’s earlier-stage effort with IW-6463, a drug in the same class for central nervous system (CNS) disorders.
A phase III failure of Swedish Orphan Biovitrum AB's oral thrombopoietin receptor agonist avatrombopag to effectively outperform a placebo in treating chemotherapy-induced thrombocytopenia, or low platelet counts, sent company shares down 17.9% on Oct. 9.
A revised U.S. label for Glaxosmithkline plc's Nucala (mepolizumab) has expanded the first-in-class anti-IL-5 treatment's approval to hypereosinophilic syndrome (HES), a group of rare disorders associated with persistent eosinophilia. It's the new therapy approved for Americans with HES in nearly 14 years, according to the FDA. An EMA filing in HES is expected later this year.
The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments.
LONDON – Lava Therapeutics BV has raised $83 million in a series C round, enabling it to begin clinical trials of its bispecific gamma-delta T-cell engager technology, in both a hematologic and a solid tumor indication in 2021.
DUBLIN – Versant Ventures, the founding investor of Crispr Therapeutics AG, is teaming up with one of the academic founders of that company, Stanford University’s Matt Porteus, to start a new gene editing firm, Graphite Bio Inc., which is focused on targeted integration of DNA to achieve a therapeutic benefit.
As Constellation Pharmaceuticals Inc. gears up to start the pivotal phase III study of CPI-0610 in myelofibrosis (MF), Wall Street is mulling how likely are the stars to align for the Cambridge, Mass.-based company with its small-molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
RSS
