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.
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