The trem-cel + VCAR33 Treatment System has the potential to transform clinical outcomes for patients suffering with AML

The trem-cel + VCAR33 Treatment System is a novel and comprehensive approach that has the potential to transform clinical outcomes and establish a new standard of care for patients suffering with AML.

We believe trem-cel could unlock the potential of anti-CD33 therapies that are much more potent than Mylotarg™ such as VCAR33^ALLO, which could be a highly potent anticancer therapy that, when combined with trem-cel, is not associated with severe myeloablative toxicities.

An attractive feature of this Treatment System is that it uses the same allogeneic source of cells for both the eHSC and CAR-T. In this scenario, the apheresis product from the healthy donor can be processed to serve as starting materials for both products. One advantage of this approach is that donor derived T cells should not recognize CAR-T cells as foreign, potentially prolonging persistence. In addition, sourcing T cells from healthy donors may provide a healthier, more abundant cell source, allowing for optimizations and efficiencies in the manufacturing process that are not possible with autologous sources.

Trem-cel + VCAR33 Treatment System—Clinical Development

We intend to submit an IND application with the FDA and conduct a clinical trial of the trem-cel + VCAR33 Treatment System after we obtain the initial results from our trem-cel Phase 1/2a clinical trial and our planned VCAR33^ALLO Phase 1/2 monotherapy clinical trial. We believe demonstration of disease clearance activity by VCAR33^ALLO would provide a fundamental rationale for further development in a non-relapse/refractory population which is still high risk, including patients with poor prognostic molecular markers and/or MRD positivity.

We would evaluate VCAR33^ALLO in a post-trem-cel transplant setting to reduce the risk of recurrence or treat evidence of early relapse. Through use of trem-cel, we believe VCAR33^ALLO could be used in a post-transplant maintenance setting since CD33 negative hematopoiesis established by the trem-cel graft would be protected from eradication. The objective of this trial would be to assess the safety and initial clinical efficacy of the trem-cel + VCAR33 Treatment System.