Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Pfizer Inc. has positive phase III data for its hemophilia treatment as it wades deeper into an indication that already has plenty of competition and at least one company with earnings of more than $1 billion. Pfizer’s marstacimab in treating hemophilia could lead to the first once-weekly subcutaneous treatment for hemophilia B and could end up being the first treatment administered as a flat dose for treating hemophilia A or B.
Varmx BV has raised €30 million (US$32 million) in an extension to its series B to fund preparations for the pivotal trial of VMX-C001, a modified recombinant factor X that acts as a universal antidote to reverse the effects of direct oral anticoagulants. The drug is intended for use in patients suffering severe spontaneous bleeding or needing emergency surgery.
After long years of endeavor, Scarlet Therapeutics Ltd. believes it is poised to realize the potential of red blood cells (RBCs) as drug delivery vehicles. The company recently closed a seed round and is now laying the ground for its first clinical trials in which cultured and genetically engineered RBCs will be used to deliver enzyme replacement therapies in the treatment of rare inherited forms of two metabolic disorders.
After Keros Therapeutics Inc.’s first-quarter earnings report, the Wall Street spotlight turned its beam toward additional data due soon from the phase II studies with KER-050, an ActRIIA-Fc fusion protein in myelodysplastic syndrome (MDS) and myelofibrosis.
Swedish Orphan Biovitrum (Sobi) AB is offering a hefty 95% premium to shareholders in CTI Biopharma Corp., as it tables a $9.10-per-share cash offer for the company, which implies a total equity valuation of $1.7 billion. Shares in Seattle-based CTI (NASDAQ:CTIC) had closed May 9 at $4.82 but surged 85% to close at $8.93 during trading May 10. The premium calculation is based on CTI’s 30-day volume-weighted average trading price of $4.67 prior to the deal announcement.
Fibrogen Inc. and its co-development partners for roxadustat, Astrazeneca plc and Astellas Pharma Inc., were dealt a major blow May 5 as the oral hypoxia-inducible factor prolyl hydroxylase inhibitor failed to meet its primary efficacy endpoint in a phase III trial in patients with anemia caused by transfusion-dependent lower-risk myelodysplastic syndromes (MDS).
By the end of this year, it will become apparent whether Morphosys AG has executed one of the biotechnology industry’s boldest pivots in recent years or has instead blown $1.7 billion of investor cash on a dud. The day of reckoning is coming a little sooner than expected for the Planegg, Germany-based firm, as it has completed recruitment in a phase III trial of pelabresib in first-line myelofibrosis ahead of schedule. Top-line data from the study are now expected before year-end, instead of early 2024.
Incyte Corp.’s bid for a once-daily vs. twice-daily version of the Janus kinase inhibitor Jakafi (ruxolitinib) was foiled, at least temporarily, by a complete response letter (CRL) from the U.S. FDA.
The U.S. FDA has approved the priority BLA for Sanofi SA’s hemophilia A treatment nearly a week before its Feb. 28 PDUFA date. The approval is for efanesoctocog alfa, a recombinant factor VIII (rFVIII) therapy – the company has managed to partially incorporate rFVIII into the drug’s brand name, Altuviiio. The price per dose was not released by the company.
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