Shattuck Labs Inc. opted, as one analyst put the matter, to do “the right thing early” by ending the clinical program with phase I-stage SL-172154 and shift resources to SL-325, a death receptor 3 antagonist, initially for inflammatory bowel disease, where TL1A/DR3-blocking antibodies have shown compelling monotherapy efficacy.
Shwachman-Diamond syndrome is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, cytopenia, growth restriction and skeletal abnormalities, and for which primary treatment is allogeneic hematopoietic stem cell transplantation, which is associated with significant toxicity.
China’s National Medical Products Administration cleared Ascentage Pharma Group Corp. Ltd. to begin a registrational phase III trial of its BCL-2 selective inhibitor lisaftoclax (APG-2575) in combination with azacitidine for first-line treatment of newly diagnosed patients with higher-risk myelodysplastic syndrome.
China’s National Medical Products Administration cleared Ascentage Pharma Group Corp. Ltd. to begin a registrational phase III trial of its BCL-2 selective inhibitor lisaftoclax (APG-2575) in combination with azacitidine for first-line treatment of newly diagnosed patients with higher-risk myelodysplastic syndrome.
Data from an interim analysis have convinced Syros Pharmaceuticals Inc. to stop enrollment in its phase II study of treating newly diagnosed acute myeloid leukemia (AML) and retinoid alpha receptor agonist gene overexpression. Analysts seemed unconcerned about the repercussions for Syros’ phase III study in myelodysplastic syndrome.
Bluesphere Bio Inc. has received FDA clearance of its IND application for BSB-1001 for patients with relapsed or refractory acute myeloid leukemia (AML), acute lymphocytic leukemia and myelodysplastic syndromes, in conjunction with allogeneic hematopoietic stem cell transplantation (alloHSCT).
After hearing two conflicting presentations of the safety and efficacy of Geron Corp.’s imetelstat, the U.S. FDA’s Oncology Drugs Advisory Committee (ODAC) voted 12-2 March 14 that the drug’s benefit outweighed its risks as a treatment for transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes in patients who have failed or no longer respond to erythropoiesis stimulating agents (ESAs), or who are not eligible for ESA treatment.
Although Geron Corp.’s imetelstat met its primary and key secondary endpoints in a phase III study, the U.S. FDA is questioning the magnitude and durability of the effect of the first-in-class telomerase inhibitor as a second-line treatment of transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes. The agency’s concerns resulted in more than an 12% stock tumble March 12 after the FDA released its briefing document two days ahead of an Oncology Drugs Advisory Committee meeting, in which the panel will be asked to vote on whether imetelstat’s benefits outweigh its risks.
Kling Biotherapeutics BV has presented preclinical data on an antibody, KBA-1413, which recognizes a specific glycoform of CD43 that is shared among acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and solid tumor cells.
