• First patient dosed in company’s second clinical program with initial data expected in second half of 2024
  • Cash runway extended into second half of 2025 with clinical trials on track

CAMBRIDGE, MA, USA I January 17, 2024 I Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, announced today it has dosed the first patient in VBP301, its Phase 1/2, multicenter, open-label, first-in-human study of VCAR33ALLO. The Company has extended its cash runway into the second half of 2025.

“We are pleased to start 2024 with strong execution and we look forward to sharing initial clinical data from the VBP301 trial later this year,” said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “We are excited to bring to AML patients, for the first time, a healthy transplant donor-derived CAR-T developmental therapy which may overcome shortfalls seen with either autologous or allogeneic off-the-shelf approaches.”

VBP301 Clinical Trial/VCAR33ALLO
The first patient with relapsed/refractory acute myeloid leukemia (AML) has been dosed with VCAR33ALLO in the VBP301 clinical trial, a significant milestone demonstrating that VCAR33ALLO can be successfully manufactured in Vor Bio’s in-house manufacturing facility.

VCAR33ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes.

While this first patient had relapsed after a standard-of-care transplant, patients who have relapsed after a trem-cel transplant are also eligible to enroll in the VBP301 protocol and to receive VCAR33ALLO. The ability to treat relapsed trem-cel transplant patients with VCAR33ALLO may provide valuable early insights into the potential of the Company’s trem-cel + VCAR33 Treatment System, which pairs VCAR33 after trem-cel to reduce the risk of relapse or treat evidence of relapse. Initial data from VBP301 is expected in the second half of 2024.

Corporate Update
The Company has further extended its cash runway into the second half of 2025 through an internal review and prioritization process and will continue to invest in its platform and clinical programs while strategically prioritizing late-stage programs and managing employee growth to preserve cash.

About AML
AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting 20,000 newly diagnosed patients each year in the United States. Approximately half of patients with AML who receive a hematopoietic cell transplant suffer a relapse of their leukemia where their two-year survival rates are less than 20%. Transplanted hematopoietic stem cells are fragile following transplant, preventing the use of potentially curative treatment options.

About VCAR33ALLO
VCAR33ALLO is a CD33-directed CAR-T cell therapy made from healthy cells obtained from the same donor from which the patient was previously transplanted. VBP301 is a Phase 1/2, multicenter, open-label, first-in-human (FIH) study of VCAR33ALLO in patients with relapsed or refractory AML after standard-of-care transplant or a trem-cel transplant. The trial is designed to test the hypothesis that a CD33-targeted CAR-T derived from a healthy donor can be safely administered to a patient with AML who has relapsed after transplant and that the CAR-T can demonstrate anti-leukemia activity. For more information, visit: https://www.clinicaltrials.gov/study/NCT05984199.

About Vor Bio
Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com.

SOURCE: Vor Bio