Vor Biopharma (VOR) Oppenheimer 35th Annual Healthcare Life Sciences Conference 2025 summary

Scientific and clinical approach

• Developing gene-edited stem cell transplants (trem-cel) for AML by deleting CD33 to shield healthy cells while enabling targeted therapies to attack cancer cells.

• Approach leverages standard donor stem cell transplant methods with an added genetic modification step.

• CD33 deletion allows use of agents like Mylotarg or CAR-Ts with reduced toxicity and improved therapeutic window.

• Early clinical data show improved relapse-free survival compared to historical controls.

• Shielded grafts enable maintenance therapy with Mylotarg, maintaining stable blood counts.

Clinical trial progress and regulatory milestones

• Achieved recommended Phase 2 dose of Mylotarg (2 mg/m² monthly for 8 cycles) with positive safety and efficacy data.

• Median relapse-free survival not yet reached at 7.4 months follow-up, outperforming published controls.

• FDA Type C meeting confirmed safety profile and pivotal study design, including relapse-free survival as the primary endpoint.

• Pivotal Phase 3 trial will compare trem-cel plus Mylotarg to standard transplant, with data from a larger cohort expected in H2 2025.

• Plan to reduce Mylotarg administration window from 60 to 30 days post-transplant based on improved safety data.

Pipeline expansion and future directions

• Advancing a CD33 CAR-T program using healthy donor cells, with expansion and persistence data expected in H1 2025.

• Developing multi-headed CAR-Ts targeting both CD33 and CLL-1, alongside dual-antigen shielded transplants.

• Licensed a CD45-directed ADC for potential use in AML cytoreduction, conditioning, and rare disease gene therapy.

• Exploring epitope engineering to shield healthy cells from CD45-targeted therapies.

• Considering CD45 ADC for autoimmune diseases and fertility-preserving conditioning.