Thursday, Vor Biopharma Inc. ( VOR ) announced new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory acute myeloid leukemia (AML) receiving trem-cel followed by Mylotarg.

Pfizer Inc’s Mylotarg is indicated for newly diagnosed CD33-positive AML in adults and relapsed or refractory AML CD33-positive acute myeloid leukemia in adults and pediatric patients two years and older.

The data demonstrated reliable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of patient benefit.

“We are encouraged by this data and the potential benefit that trem-cel in combination with Mylotarg may offer to patients in a disease that has extremely poor outcomes even after transplant,” said Eyal Attar, Vor Bio’s Chief Medical Officer. “With this data, we plan to explore a registrational trial while we continue to pursue other synergistic opportunities for Vor Bio’s platform such as VCAR33ALLO and VADC45.”  

The data released included 18 patients treated with trem-cel of which ten had received Mylotarg as of the data cut-off date of July 19, 2024. The data demonstrated:

Reliable engraftment, with 100% of patients achieving primary neutrophil engraftment​ (median 9 days) and robust platelet recovery (median 16.5 days)​.

Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2.

Early evidence suggests patient benefit as measured by relapse-free survival compared to published high-risk AML comparators.

Vor Bio plans to approach the FDA to discuss a pivotal trial design for trem-cel + Mylotarg by around the end of the year.

Vor Bio announced a new preclinical asset, VADC45, which has several potential opportunities in oncology, gene therapy, and autoimmune disorders.

The company said cash, cash equivalents, and marketable securities were $85.9 million as of June 30, 2024, projected to fund operations into the second half of 2025.

Price Action: VOR stock is up 28.5%at $1.06 at last check Friday.

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