Vor Bio Q3 2024 Financial Results and Company Update

Vor Bio, a company focused on cell and genome engineering, has released its financial results for the third quarter ending September 30, 2024, and provided an update on its corporate activities. The company reported significant progress in its operations and clinical trials, particularly in the development of therapies for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Dr. Robert Ang, President and CEO of Vor Bio, expressed satisfaction with the company's performance and announced plans to present further clinical data at the American Society of Hematology (ASH) 2024 annual meeting in December.

**Clinical Trials and Research**

Vor Bio is advancing its clinical trials with several key updates. In the VBP101 study, which combines trem-cel with Mylotarg, 25 patients have been dosed with trem-cel, and six patients have received the third dose level of Mylotarg at 2 mg/m2. The trial aims to assess the efficacy and safety of this combination therapy in AML patients. Results from 18 patients showed reliable engraftment, robust platelet recovery, and high CD33 editing efficiency. Additionally, the trial demonstrated the ability to shield the blood system while maintaining neutrophil and platelet counts across various Mylotarg doses. Early evidence suggested potential patient benefits as measured by relapse-free survival.

The company plans to provide a further clinical update in a poster presentation at the ASH Annual Meeting on December 8, 2024. Trem-cel is being developed as a shielded transplant for AML and MDS patients by genetically engineering donor cells to remove CD33, thus allowing targeted therapies post-transplant.

In another clinical trial, VBP301, Vor Bio is investigating VCAR33ALLO, an anti-CD33 CAR-T cell therapy derived from transplant donors. This Phase 1/2 study is focused on AML patients who have relapsed following standard-of-care or trem-cel transplants. Initial data from three patients treated at the lowest dose showed promising CAR-T expansion in vivo.

**Preclinical Research**

Vor Bio is also making strides in preclinical research. In September 2024, the company introduced a new preclinical asset, VADC45, an antibody-drug conjugate (ADC) targeting the CD45 protein, which is associated with various blood cancers. The company is progressing with IND-enabling studies for potential Phase 1 trials.

The company presented additional preclinical data at the European Society of Gene and Cell Therapy Congress in October 2024. This included the development and characterization of a novel CD33/CLL-1-directed CAR-T approach in AML. They also plan to present further analyses at the ASH 2024 annual meeting, offering deep insights into AML immunophenotypic heterogeneity.

**Corporate Leadership and Financials**

Vor Bio has appointed Dr. Han Choi as its new Chief Financial Officer. Dr. Choi, a seasoned healthcare institutional investor, brings over 25 years of experience in investment management, business development, and corporate strategy within the pharmaceutical and biotechnology sectors.

Financially, Vor Bio reported a cash position of $62.8 million as of September 30, 2024, expected to fund operations into the second half of 2025. Research and development expenses for the third quarter were $21.8 million, a decrease from $27.6 million in the same period in 2023. General and administrative expenses also decreased to $6.7 million from $7.7 million. The net loss for the third quarter was $27.6 million, compared to $33.2 million in the previous year.

Vor Bio continues to make significant progress in its clinical and research endeavors, aiming to improve treatment options for patients with blood cancers. The upcoming ASH 2024 annual meeting will be a crucial platform for presenting their latest clinical data and advancements.