Despite challenges tossed at the phase III study of mitapivat from Agios Pharmaceuticals Inc. by COVID-19, top-line data showed the oral therapy hit its primary endpoint in treating adults with pyruvate kinase (PK) deficiency who don’t receive regular transfusions.
PERTH, Australia – Melbourne-based Telix Pharmaceuticals Ltd. is acquiring Scintec Diagnostics GmbH subsidiary Therapharm GmbH in a deal worth AU$33 million (US$24.24 million) plus royalties.
Zug, Switzerland-based Therapharm has developed a portfolio of radiolabeled diagnostic and therapeutic products, and the deal brings Telix a new targeting asset in hematology, Telix CEO Chris Behrenbruch told analysts during a Dec. 1 conference call.
Positive top-line data from the pivotal phase III study of AAV5-based etranacogene dezaparvovec by Uniqure NV, shows 54 patients met the primary endpoint in treating severe to moderate hemophilia B.
The FDA has issued Sanofi SA a complete response letter (CRL) regarding its complement pathway inhibitor sutimlimab, citing deficiencies found during a pre-license inspection of a third-party manufacturing facility.
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.
RSS
