Vor Biopharma Inc. (VOR) announced encouraging clinical data from its ongoing Phase 1/2 VBP101 study for patients with relapsed/refractory acute myeloid leukemia (AML). The study involved administering trem-cel followed by Mylotarg, a drug developed by Pfizer Inc. (PFE) for newly diagnosed and relapsed/refractory AML.
The data showcased several key findings:
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Reliable Engraftment:
100% of patients achieved primary neutrophil engraftment (median 9 days) and robust platelet recovery (median 16.5 days) after receiving trem-cel.*
Shielding from Toxicity:
Trem-cel effectively protected the blood system from Mylotarg’s potential on-target toxicity, maintaining neutrophil and platelet counts across various Mylotarg dosages.*
Early Evidence of Patient Benefit:
Preliminary data suggests a positive impact on relapse-free survival compared to published data for high-risk AML patients.These findings have fueled optimism about the potential of trem-cel in combination with Mylotarg for treating AML, a disease with a high mortality rate even after transplantation.
“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,” stated Eyal Attar, Vor Bio’s Chief Medical Officer.
Encouraged by these results, Vor Bio plans to discuss a pivotal trial design for trem-cel + Mylotarg with the FDA by the end of the year.
The company also announced a new preclinical asset, VADC45, with potential applications in oncology, gene therapy, and autoimmune disorders.
As of June 30, 2024, Vor Bio had $85.9 million in cash, cash equivalents, and marketable securities, which is projected to fund operations into the second half of 2025.
Following the positive clinical data release, VOR stock surged 28.5% to $1.06 per share at the close of trading on Friday.
