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. Read More
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.
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. Read More
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. Read More
With new results from Wave Life Sciences Ltd., Structure Therapeutics Inc. and Ascletis Pharma Inc., obesity management drugs continue to move forward in producing weight loss and move the market. Read More
Ireland is taking stock of the implications Trump administration tariffs and drug pricing policies hold for its world-leading biomanufacturing sector, which has been largely built on the back of investment by U.S. pharma companies – and is hugely reliant on exports to the U.S. Read More
Fourteen therapies to treat moderate to severe psoriasis are expected to enter the Chinese market in the next two years, according to Clarivate and BioWorld reports. Eleven of them are being developed by domestic biopharmaceutical firms. Read More
GSK plc said in paperwork filed with the U.S. SEC that the potentially whopping deal inked in the summer of 2020 with Ideaya Biosciences Inc. has gone down the tubes. Read More
Mirum Pharmaceuticals Inc. is buying privately held Bluejay Therapeutics Inc., an $820 million purchase that will bring it Bluejay’s lead asset, brelovitug, for treating chronic hepatitis D virus. Read More
Viatris Inc. is padding its coffers with $400 million in cash through the sale of its equity stake in Biocon Biologics Ltd., which also involves $415 million in newly issued equity shares, for a total deal value of $815 million. Read More
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Ascentage, BMS, Vanda. Read More
.webp?t=1670008839 "Acute myeloid leukemia")
