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.
Biomarin Pharmaceutical Inc.’s complete response letter (CRL) for Roctavian (valoctocogene roxaparvovec; Valrox) gene therapy for severe hemophilia A shocked the company, its investors and analysts mere days before its Aug. 21 PDUFA date. Now an approval and launch for what would have been the first approved hemophilia gene therapy is likely pushed back roughly two years.
LONDON – Varmx BV is poised to demonstrate its modified recombinant factor X acts a universal antidote to reverse the effects of direct oral anticoagulants in patients suffering severe spontaneous bleeding or needing emergency surgery, after closing a €32 million (US$36.1 million) series B round.
DUBLIN – Gene therapy developer Freeline Therapeutics Ltd. added $80 million in new investment to take its series C round to $120 million in total. The new cash will help to fund a pivotal trial of its lead gene therapy program in hemophilia B, enable it to continue a phase I/II trial of a gene therapy in Fabry disease and allow it to progress its preclinical programs in Gaucher disease and hemophilia A, while also making ongoing investments in its adeno-associated virus (AAV) gene therapy technology and its manufacturing platform.
HONG KONG – Astellas Pharma Inc. reported positive results from phase III of its DOLOMITES study comparing the efficacy and safety of roxadustat to that of darbepoetin alfa in treating anemia in nondialysis-dependent, stage 3 to 5 chronic kidney disease (CKD) patients.
Arca Biopharma Inc., of Westminster, Colo., is developing AB-201, a selective inhibitor of tissue factor (TF), as a treatment for COVID-19-associated coagulopathy, abnormal blood clotting and related inflammatory response. The move gave the company new life Thursday.