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.
Due to launch in the second half of this year, Rallybio Inc.’s phase I/II study testing RLYB-211 for prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) will begin with experiments in men, said Steve Uden, one of the company’s three founders. “The good news is, we can confirm the safety and the pharmacology of this concept in male volunteers before having to start a clinical trial in pregnant mothers,” he told BioWorld.
HONG KONG – South Korea’s Hanmi Pharmaceutical Co. Ltd. has filed a new drug approval application for Rolontis (eflapegrastim) with the country’s Ministry of Food and Drug Safety (MDFS).
Alexion Pharmaceuticals Inc., of Boston, will diversify its portfolio beyond its top-selling Soliris (eculizumab) and C5 inhibitor Ultomiris (ravulizumab) by acquiring Portola Pharmaceuticals Inc. for $1.4 billion. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Andexxa is the only FDA-approved factor Xa inhibitor reversal agent in severe and uncontrolled bleeding.
Keros Therapeutics Inc. CEO Jasbir Seehra told BioWorld that he plans to use at his new company lessons learned as co-founder of Acceleron Pharma Inc., where work with receptors in the TGF-beta superfamily “taught me the potential of the biology and those molecules, but also the limitations” with regard to safety that need to be surmounted.
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