Vor Bio Announces Q2 2024 Financial Results and Company Update

Vor Bio, a clinical-stage company specializing in cell and genome engineering, released its financial results for the quarter ending June 30, 2024, along with a corporate update. The company, listed on Nasdaq under the ticker VOR, is optimistic about the progress made in its clinical trials for the trem-cel + Mylotarg and VCAR33ALLO therapies, with significant updates expected in the latter half of 2024.

Dr. Robert Ang, Vor Bio's President and CEO, expressed satisfaction with the enrollment pace for these trials. He emphasized the potential of their shielded transplant approach, which aims to provide new treatment options for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who relapse post-transplant. Currently, these patients have limited safe and effective treatment choices.

Corporate Developments

Trem-cel Trial Progress:
The trem-cel trial is advancing well, with 21 patients dosed so far. Patients are now receiving the third dosage level of Mylotarg at 2.0 mg/m². The company plans to release data on engraftment, hematologic protection, and additional pharmacokinetic analyses for Mylotarg in the second half of 2024. Notably, some patients have been monitored for over a year, and a clinical update on these individuals is anticipated.

Trem-cel, designed for AML and MDS patients, involves genetically engineering healthy transplant donor cells by removing CD33. This modification aims to protect healthy cells and enable targeted treatments post-transplant, such as Mylotarg and CAR-T therapies.

VCAR33ALLO Advancement:
The VCAR33ALLO study, known as VBP301, is a Phase 1/2, multicenter, open-label, first-in-human trial. It is investigating an anti-CD33 CAR-T cell therapy derived from transplant donors for AML patients who have relapsed after standard-of-care or trem-cel transplants. Enrollment is ongoing, with several patients already dosed. Preliminary data is expected in the second half of 2024.

Key Upcoming Milestones

- A clinical data update for the trem-cel trial is anticipated in the second half of 2024.
- An update on the VCAR33ALLO clinical trial is also expected during the same period.

Financial Performance for Q2 2024

Cash Position:
As of June 30, 2024, Vor Bio had $85.9 million in cash, cash equivalents, and marketable securities. This is projected to support operations into the second half of 2025.

Research & Development Expenses:
R&D expenses for Q2 2024 were $21.8 million, down from $23.9 million in Q2 2023. The decrease is attributed to the timing of procurement for manufacturing materials for the VCAR33ALLO program and a reduction in preclinical expenses. However, clinical trial costs for both the trem-cel and VCAR33ALLO programs increased.

General & Administrative Expenses:
G&A expenses for Q2 2024 were $7.2 million, a decrease from $8.3 million in Q2 2023. This reduction was primarily due to lower consulting and legal expenses, partially offset by higher personnel costs.

Net Loss:
Vor Bio reported a net loss of $27.8 million for Q2 2024, compared to $30.0 million for Q2 2023.

Financial Summary

Balance Sheet Data (Unaudited)

- Cash, cash equivalents, and marketable securities: $85.9 million (June 30, 2024) vs. $137.2 million (December 31, 2023).
- Total assets: $141.6 million (June 30, 2024) vs. $198.1 million (December 31, 2023).
- Total liabilities: $43.5 million (June 30, 2024) vs. $47.4 million (December 31, 2023).
- Total stockholders' equity: $98.1 million (June 30, 2024) vs. $150.7 million (December 31, 2023).

Statement of Operations (Unaudited)

- R&D expenses: $21.8 million (Q2 2024) vs. $23.9 million (Q2 2023).
- G&A expenses: $7.2 million (Q2 2024) vs. $8.3 million (Q2 2023).
- Total operating expenses: $29.0 million (Q2 2024) vs. $32.2 million (Q2 2023).
- Net loss: $27.8 million (Q2 2024) vs. $30.0 million (Q2 2023).
- Net loss per share (basic and diluted): $(0.41) (Q2 2024) vs. $(0.45) (Q2 2023).

Vor Bio continues to focus on developing innovative treatments for blood cancers by engineering hematopoietic stem cells. The company's ongoing clinical trials and financial strategies aim to bring new hope to patients with AML and MDS.

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