Crowned by a potential cure for severe hemophilia A, that could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society's ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements.
LONDON – Freeline Therapeutics Ltd. believes it has found the dose at which FLT-180a, its gene therapy for hemophilia B, will provide a functional cure, promoting expression of factor IX (FIX) blood clotting factor within the normal range.
Emendo Biotherapeutics Inc. CEO David Baram told BioWorld his firm’s allele-specific gene-editing approach offers such an advantage over previous methods that “we decided to take the challenge of curing diseases that require the highest precision possible,” and the New York-based firm bears an impressive list of partners. “Doors opened immediately and collaborations formed very fast,” sometimes “even faster than we could digest,” he said.
Ten days after its JAK1 inhibitor, itacitinib, failed a phase III trial in acute graft-vs.-host-disease (GVHD), Incyte Corp. has finalized a deal that could provide an alternative growth path. It is paying Morphosys AG $750 million up front, investing $150 million in its stock and is on the hook for up to $1.1 billion in milestones for a 50% interest in U.S. rights to the CD19-targeting antibody tafasitamab (MOR-208) and for 100% of the rights in all other territories.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”
Nearly four years after its start, a phase III trial of Gamida Cell Ltd.'s ex vivo expanded cord blood candidate, omidubicel, for hematologic malignancies is fully enrolled, the company said.
DUBLIN – Gene therapy firm Freeline Therapeutics Ltd. secured the first $40 million tranche of an $80 million series C round from its founding investor and principal shareholder Syncona plc to generate further data from its two clinical-stage programs, in hemophilia B and Fabry disease, to fund expansion of its team and to continue the ongoing buildout of its manufacturing operations in Munich.
Syros Pharmaceuticals Inc. and Global Blood Therapeutics Inc. (GBT) agreed to a discovery, development and commercialization deal to treat sickle cell disease (SCD) and beta-thalassemia. GBT, of South San Francisco, Calif., will pay Syros $20 million up front and fund up to $40 million in preclinical research for at least three years, with the goal of identifying targets and discovering drugs to induce fetal hemoglobin.
ORLANDO, Fla. – The world’s biggest and certainly most lavish hematology gathering, the 61st American Society of Hematology conference, just ended in Orlando, having brought 30,024 people from 25 countries to glory in Florida sunshine, if they got outside, but mostly to bask in the discipline’s most up-to-the-minute data. The amount of research was staggering, with 5,978 abstracts available for review. Key themes included work aimed at overcoming obstacles to CAR T therapy, new progress in preventing and treating venous thromboembolism, moves to address health care disparities and new developments in the care of sickle cell diseases. Late-breakers highlighted new data on Blincyto (blinatumomab, Amgen Inc.), Sanofi SA's sutimlimab, azacitidine and Darzalex (daratumumab, Janssen Biotech Inc.).
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