Vor Bio Initiates VCAR33ALLO Therapy in AML Patient and Extends Cash Runway

Vor Bio, a clinical-stage cell and genome engineering firm, has reached a significant milestone in its development of a novel cell therapy for acute myeloid leukemia (AML). The company has commenced dosing in its Phase 1/2 clinical trial, VBP301, which is evaluating the safety and efficacy of VCAR33ALLO, a CD33-directed CAR-T cell therapy. This therapy is derived from the lymphocytes of a patient's healthy transplant donor, ensuring an exact match to the recipient's blood system.

The initiation of the VBP301 trial marks a crucial step forward in Vor Bio's mission to enhance the standard of care for blood cancer patients. The treatment is designed to overcome the limitations associated with autologous and allogeneic off-the-shelf approaches, offering a new option for AML patients who have relapsed or are refractory to standard treatments.

VCAR33ALLO's unique manufacturing process involves using cells from the patient's transplant donor, which results in a CAR-T cell product with a stem-like phenotype. This characteristic is believed to enhance the therapy's potential for expansion, persistence, and effectiveness against leukemia. The trial is open to patients who have relapsed after a standard transplant or a trem-cel transplant, providing an opportunity to explore the combination of trem-cel and VCAR33ALLO in reducing the risk of relapse or treating relapse.

Vor Bio's Chief Medical Officer, Dr. Eyal Attar, expressed optimism about the trial's commencement and the potential of VCAR33ALLO to make a significant impact on AML patients. The company anticipates sharing initial clinical data from the VBP301 trial in the latter half of 2024.

In addition to the clinical progress, Vor Bio has successfully extended its financial resources, ensuring that it can continue to invest in its platform and clinical programs. The company has strategically prioritized its late-stage programs and managed its growth to maintain a cash runway into the second half of 2025.

AML is a devastating disease, with over 20,000 new cases diagnosed annually in the United States alone. The prognosis for patients who suffer a relapse after a hematopoietic cell transplant is particularly grim, with two-year survival rates below 20%. The fragility of transplanted stem cells post-transplant has historically limited the use of curative treatments.

Vor Bio's approach to cell and genome engineering aims to change this landscape by engineering hematopoietic stem cells to enable targeted therapies post-transplant. The company's commitment to innovation in blood cancer treatment is evident in its ongoing clinical trials and its focus on developing therapies that can offer hope to patients with limited options.

The VBP301 trial and the development of VCAR33ALLO represent a significant advancement in the field of cell therapy for blood cancers. As the trial progresses, the medical community and patients alike will be watching closely for the initial data expected later this year, which could potentially pave the way for new treatment paradigms in the management of AML.