Geron Corp. executives highlighted a potential billion-dollar market opportunity for imetelstat on the back of positive phase III data for the telomerase inhibitor in patients with lower-risk myelodysplastic syndromes (MDS), news that sent shares (NASDAQ:GERN) rising 36% and could portend the first regulatory win for a scientific approach the company has pursued since the 1990s.
Magenta Therapeutics Inc., the developer of an antibody-drug conjugate intended to help prep certain relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome patients for stem cell transplant or gene therapy, has stopped dosing the drug in part of an ongoing trial after observing two cases of serious adverse events (SAEs) possibly related to the candidate.
Additional deaths believed to be associated with one of Foghorn Therapeutics Inc.'s lead candidates led the U.S. FDA to put a full clinical hold on its phase I study in relapsed and/or refractory acute myelogenous leukemia and myelodysplastic syndrome.
Ryvu Therapeutics SA aims to move its lead drug candidate RVU-120 into three phase III trials in the first half of next year, to evaluate its potential in acute myeloid leukemia and high-risk myelodysplastic syndrome, in low-risk MDS, and in a number of solid-tumor indications.
The Hail Mary pass by Syros Pharmaceuticals Inc. – shares of which have dwindled severely since Jan. 3 – foreseen by some on Wall Street, came in the form of a $130 million financing and the merger with Tyme Technologies Inc., bringing in-house pipeline assets as well as net cash in a deal that, after accounting for wind-down and transaction expenses, is worth about $60 million.
In the wake of a patient’s death, the U.S. FDA has placed a partial clinical hold on Foghorn Therapeutics Inc.’s phase I study of FHD-286 in treating relapsed and/or refractory acute myelogenous leukemia (r/r AML) and myelodysplastic syndrome.
A patient’s death has prompted the FDA to place a partial clinical hold on Curis Inc.’s phase I/IIa study of emavusertib in leukemia. The patient, who had relapsed or refractory acute myeloid leukemia, experienced several conditions. One of them was rhabdomyolysis, a dose-limiting toxicity of emavusertib. Rhabdomyolysis is a breakdown of muscle fibers in the blood.
While Aprea Therapeutics Inc.’s data disclosed July 21 from the phase II trial with eprenetapopt – also known as APR-246, a reactivator of mutant tumor suppressor protein p53 – plus azacitidine (AZA) whetted interest in taking aim at the “guardian of the genome,” the company’s fortune took an unexpected turn when the FDA smacked a partial clinical hold on work with the duo.
Advaxis Inc.’s long journey is ending happily by way of a merger with Biosight Ltd. that leaves the new firm with $50 million to help advance aspacytarabine (BST-236), a prodrug of cytarabine for acute myeloid leukemia and myelodysplastic syndromes.
San Diego-based Biotheryx Inc. has raised $92 million in series E financing led by Farallon Capital Management to advance multiple molecular glues, proteolysis-targeting chimeras and monovalent degraders toward the clinic. Its first molecular glue program, BTX-1188, is expected to enter the clinic by the end of this year.
