VCAR-33 is under clinical development by Vor BioPharma and currently in Phase II for Refractory Acute Myeloid Leukemia. According to GlobalData, Phase II drugs for Refractory Acute Myeloid Leukemia have a 23% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how VCAR-33’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

VCAR-33 overview

VCAR-33 is under development for the treatment of relapsed and refractory acute myeloid leukemia (AML) in children and adolescents/young adults. It is administered via parenteral route. It is developed based on eHSC technology. The drug candidate constitutes of autologous T cells genetically engineered to express chimeric antigen receptor targeting CD33. It acts by targeting cells expressing CD33.

Vor BioPharma overview

Vor BioPharma is a clinical stage cell therapy company that develops therapies for treatment of hematological malignancies. The company’s pipeline products include VOR33, an engineered hematopoietic stem cells (eHSC) designed to replace the standard of care in transplant settings; and VCAR33, a CD33-directed chimeric antigen receptor T cell (CAR-T) therapy. It is investigating VCAR33 for relapsed or refractory acute myelogenous leukemia (AML). Vor BioPharma’s multi-targeted treatment system is comprised of VCAR33-CLL1 multi-specific CAR-T therapy and VOR33-CLL1 multiplex-edited eHSC therapy to treat patients with AML using eHSC platform. Vor BioPharma is headquartered Cambridge, Massachusetts, the US.

For a complete picture of VCAR-33’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.