Novartis AG’s monoclonal antibody, ianalumab (VAY-736), when added to standard-of-care eltrombopag, extended disease control of primary immune thrombocytopenia (ITP) by 45%, according to data presented Dec. 9 during a late-breaker abstract session at the 67th American Society of Hematology’s annual meeting in Orlando, Fla.
Exicure Inc.’s buyout early this year of GPCR Therapeutics Inc. is paying off in a big way with data from the finished phase II trial testing burixafor (GPC-100). The agent is used with propranolol and granulocyte colony-stimulating factor to mobilize hematopoietic progenitor cells in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplant.
Impressive results of a potential second-line combination treatment for multiple myeloma from the Majestec-3 trial of teclistamab plus daratumumab raised eyebrows at the American Society of Hematology’s 67th annual meeting, with the combination showing an 83.4% rate of progression-free survival at three years vs. 29.7% for standard of care.
On the heels of mixed phase III data from Agios Pharmaceuticals Inc. with mitapivat to treat sickle cell disease (SCD), Fulcrum Therapeutics Inc. wowed investors by way of initial results from the ongoing 20-mg dose cohort in the phase Ib Pioneer trial testing oral, once-daily fetal hemoglobin inducer pociredir.
Targeted therapies and immunotherapies continue to show better results than chemotherapy in investigator-initiated and company-sponsored cancer trials, and newer options demonstrate improvements over older ones, supporting potential shifts in how patients are treated.
While several targeted therapies are approved for acute myeloid leukemia, a 2023 U.K. study found that median survival following diagnosis was only about seven months, highlighting the need not only for new therapies, but for a faster regulatory strategy. At the American Society of Hematology’s 67th annual meeting in Orlando Dec. 6, researcher Jesse Tettero presented data supporting the use of a measurable residual disease (MRD) surrogate endpoint in acute myeloid leukemia (AML) research.
Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS (UNAIDS) described “a global system in shock” by sharply reduced funding from the U.S. and other wealthy nations. Scientifically, for now, progress is ongoing. To mark World AIDS Day, Nature published three independent studies on HIV.
The U.S. FDA said postmarketing reports of neutralizing antibodies to ADAMTS13, including one reported patient death, have prompted an investigation into Takeda Pharmaceutical Ltd.’s Adzynma (apadamtase alfa), a recombinant protein product approved in 2023 for use in adults and pediatric patients with congenital thrombotic thrombocytopenic purpura.
“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints.
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