Vor Bio announces clinical data on treating AML to be presented at ASH meeting

Vor Bio (VOR) announced that preclinical and clinical data supporting the Company’s novel platform and approach for treating acute myeloid leukemia, AML, will be presented at the 65th American Society of Hematology, ASH, Annual Meeting & Exposition, being held from December 9-12, 2023, in San Diego, CA. “These three oral and two poster presentations represent further preclinical and clinical validation of our platform and reflect the significant progress we continue to make as we develop truly novel next generation transplants that have the potential to improve the lives of patients with blood cancers,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. VBP101 Clinical Data Update: An abstract providing a clinical update from the VBP101 clinical trial, a Phase 1/2a multicenter, open-label, first-in-human study of trem-cel in patients with AML, was accepted by ASH for oral presentation. This data supports robust neutrophil engraftment of trem-cel and provides evidence of hematologic protection from MylotargTM, a CD33-targeted antibody drug conjugate.CD33CART Study Clinical Data Update: The Pediatric Transplantation and Cellular Therapy Consortium released data providing a clinical update on a Phase 1/2 study of CD33CART, an autologous CAR-T therapy targeting CD33 which uses the same CAR-T construct as VCAR33ALLO. Nineteen pediatric and young adult patients with relapsed/refractory AML with a median age of 16 years were infused in the Phase 1 portion of the study. This data shows that as of the cutoff date of June 1, 2023, 2 of 5 evaluable patients treated at the highest dose level achieved complete remission. Transient CD33CART expansion was detected in 11 subjects across all doses tested and in all 6 subjects evaluated at DL4, as of the cutoff date. Four out of 19 evauable patients treated had cytokine release syndrome greater than or equal to Grade 3. Additional ASH Presentations: The Company also released data from a single cell analysis studying molecular signatures from 28 AML patients in various stages of AML progression. This data is the most comprehensive analysis to date on AML profiling. This abstract was accepted by ASH for oral presentation. The preclinical collaboration between Vor Bio and Janssen (JNJ) yielded in vitro and in vivo xenotransplant data demonstrating that CD33-deleted allografts were synergistic with Janssen’s CD33 directed immunotherapy candidate, and maintained robust on-target cytotoxicity while reducing production of inflammatory cytokines associated with CRS. This abstract was accepted by ASH for poster presentation.