Vor Bio is currently conducting two clinical trials for AML and MDS

VBP101 AML/MDS Clinical Trial

VBP101, a Phase 1/2a clinical trial for patients with CD33-positive AML or MDS who are at high risk of Leukemia relapse following hematopoietic cell transplantation, is actively enrolling patients. Participants (18-70 years old) of the clinical trial must have a diagnosis of CD33+ AML or MDS, an HLA-matched (8/8) related or unrelated donor, and be a candidate for a hematopoietic stem cell transplant (HCT). The primary goals of the clinical trial for AML or MDS are to evaluate tolerability and feasibility of the trem-cel stem cell transplant, with a focus on confirming that trem-cel can engraft normally.

Following engraftment, patients will be eligible for treatment with Mylotarg, a CD33-directed targeted therapy, to potentially extend leukemia-free survival and to provide evidence that transplant with trem-cel protects normal blood cells from the count-suppressing side effects of Mylotarg.

More information on the VBP101 study, including participating centers, can be found here: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment with Mylotarg, for Patients with CD33+ AML or MDS.

VBP301 AML Clinical Trial

The FDA has cleared Vor Bio’s Investigational New Drug (IND) application for VCAR33, a CAR-T cell therapy derived from allogeneic healthy donors that targets the CD33 protein. VCAR33 is being studied in the VBP301 AML clinical trial, which will focus on patients aged 18 and older with relapsed or refractory Acute Myeloid Leukemia (AML) following an allogeneic hematopoietic stem cell transplant, including patients who have received trem-cel. This AML clinical trial uses T cells harvested from the original donor as the starting material for the drug product.

Mylotarg, an Antibody Drug Conjugate (ADC), is currently the only anti-CD33 therapy approved by the FDA. We believe that our CAR-T product candidate, VCAR33, may ultimately be a better targeted therapy due to higher expected potency and longer expected persistence.

More on the VBP301 study, including participating centers, can be found here: Donor-Derived Anti-CD33 CAR T Cell Therapy (VCAR33) in Patients With Relapsed or Refractory AML After Allogeneic Hematopoietic Cell Transplant.

Vor Bio is working with clinical trial investigators at leading transplant and cancer centers in the U.S. and Canada with close oversight from the U.S. Food & Drug Administration (FDA) and Health Canada, as well as an independent data safety monitoring committee. Together, these groups provide rigorous supervision to ensure patient and donor safety.

Joining a clinical trial

Clinical trials are highly regulated by the US Food and Drug Administration and other governmental regulatory agencies for the safety of patients and for the validity of the data. Vor Bio cannot help you in any way get into a trial. We can, however, direct you to information about the trial and to the clinical investigators who are managing the clinical trial at the various transplant centers.

Talk to your doctor about eligibility

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below.

For general information:

Expanded access policy:

Expanded access, sometimes called “compassionate use,” provides a pathway for patients to gain access to investigational treatments for serious diseases or conditions. Expanded access refers to the use of an investigational therapy (one that has not been approved by the U.S. Food & Drug Administration (FDA) or other applicable regulatory authorities) outside of a clinical trial that is conducted under an Investigational New Drug (IND) application.

When considering expanded access, it is important to consider guidelines from the U.S. FDA as well as guidelines from regulatory agencies outside of the U.S. According to the FDA, expanded access may be appropriate when all of the following apply:

• Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
• There is no comparable or satisfactory alternative therapy to treat the disease or condition;
• Patient enrollment in a clinical trial is not possible;
• Potential patient benefit based on available safety and efficacy information justifies the potential risks of treatment;
• Providing the investigational medical product will not interfere with, or delay, investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

Currently, Vor Bio does not offer an expanded access program. Our goal is to provide patients with access to the engineered hematopoietic stem cell therapy programs that we are developing as safely and quickly as possible. We believe the most appropriate way to achieve this goal is through participation in our clinical trials. Vor Bio may revise this policy at any time.

If you have questions about Vor Bio’s expanded access policy, please contact us. We plan to respond within five (5) business days of receiving your questions.