First AML Patient Transplanted with Vor Bio’s Trem-cel Demonstrated Durable Engraftment through Multiple Mylotarg™ Cycles at Initial Dose Level

• Trem-cel exhibited robust engraftment five months post-transplant through three cycles of Mylotarg
• Mylotarg treatment enriched CD33-negative donor hematopoiesis
• Second patient successfully received trem-cel transplant and achieved neutrophil engraftment and platelet recovery

Excerpt from the Press Release:

CAMBRIDGE, Mass., Feb. 16, 2023 (GLOBE NEWSWIRE) — Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, presented clinical data from VBP101, its Phase 1/2a multicenter, open-label, first-in-human 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 level of 0.5 mg/m². A second patient has successfully received a trem-cel transplant and engrafted normally. These data were presented today by Miguel-Angel Perales, MD, Chief, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center in a late-breaking poster at the 2023 Tandem Meetings (Transplantation & Cellular Therapy Meetings of ASTCT^™ and CIBMTR^®) in Orlando, FL.

“These data continue to demonstrate the potential of trem-cel as a next-generation hematopoietic stem cell transplant, which could transform treatment for patients with blood cancers such as AML,” said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “We are also encouraged that a second patient successfully received a trem-cel transplant and look forward to learning more as we treat additional patients and dose escalate Mylotarg. In addition, we plan to file an IND for VCAR33^ALLO, a novel allogeneic CAR-T treatment which we believe could be more efficacious than Mylotarg in combination with a trem-cel transplant.”

Trem-cel Safety & Durability
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.

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