Vor Biopharma's Leukemia Cell Exhibits Favorable Engraftment Five Months Post-Transplant

  • Vor Biopharma Inc VOR presented clinical data from VBP101, its Phase 1/2a study of trem-cel (previously VOR33) in patients with acute myeloid leukemia (AML). 
  • In the first patient, trem-cel maintained hematopoiesis through three cycles of Mylotarg (gemtuzumab ozogamicin), which was well-tolerated at the initial dose of 0.5 mg/m2. 
  • Patient 1 maintained neutrophil and platelet counts approximately five months (147 days) after transplantation with trem-cel. 
  • Due to detectable, measurable residual disease (MRD), Patient 1 was moved to other therapies following administration of the third dose of Mylotarg, subsequently relapsed, and remains on study for long-term follow-up.
  • A second patient has received a trem-cel transplant and engrafted normally. 
  • Patient 2 showed robust cell recovery with neutrophil engraftment occurring at Day 11 and platelet recovery on Day 17. 
  • Trem-cel was well tolerated in both patients, with no related and no unexpected adverse events (AEs) reported.
  • In Patient 1, neutrophil and platelet cell counts were maintained following three sequential Mylotarg doses at 0.5 mg/m2, suggesting potential protection from Mylotarg-related hematotoxicity.
  • Additionally, the company plans to file an IND for VCAR33ALLO, a novel allogeneic CAR-T treatment that it believes could be more efficacious than Mylotarg in combination with a trem-cel transplant.
  • Price Action: VOR shares are up 2.19% at $5.84 on the last check Thursday.
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