Vor Biopharma (VOR) Stifel 2024 Healthcare Conference summary

Strategic focus and clinical progress

• Targeting acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) with shielded stem cell transplants to enable safer, more effective anti-cancer therapies.

• Phase 1/2 VBP101 trial of trem-cel transplantation plus Mylotarg shows promising early results, with increased patient numbers and longer follow-up now available.

• Median follow-up for the latest data set is over seven months, with 25 patients treated and more robust relapse-free survival data expected.

• Additional MDS patient enrollment is ongoing, with updates planned and potential for future integration into pivotal trials.

• Manufacturing capacity currently meets trial needs, with scalable processes ready for rapid expansion ahead of phase III.

Data updates and regulatory plans

• New data at ASH will include more patients at higher Mylotarg doses, with focus on safety, pharmacokinetics, and efficacy.

• Regulatory feedback from FDA is expected by year-end, guiding phase III trial design and requirements.

• Current thinking favors a randomized controlled phase III trial with standard of care as the control arm, aiming for rigorous and expedient enrollment.

• Phase III eligibility will focus on high-risk, post-transplant relapse patients, excluding those with active high-blast disease.

• Plans to allow earlier Mylotarg dosing (as early as day 30) in phase III to prevent early relapses.

Pipeline expansion and innovation

• VCAR33 (ALLO), a CD33-targeting CAR-T, is advancing in a separate protocol, with dose escalation ongoing and plans to integrate with trem-cel.

• In-licensed a CD45-targeting ADC, with potential as a conditioning agent and for broader blood cancer applications.

• Multiplex gene editing and dual-targeted CAR-T approaches are in development, with updates anticipated.