• VBP101 clinical data update planned at European Hematology Association (EHA) 2023
• VCAR33^ALLO IND submission on-track for 1H 2023

CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended March 31, 2023, and provided a business update. 

“We are pleased with the progress we made during the quarter. Our focus continues to be on actively enrolling and treating additional patients in our VBP101 study and submission of the VCAR33^ALLO IND, which is on-track for the first half of 2023,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. “We look forward to sharing additional data from VBP101 at EHA in June, initiating enrollment in VBP301, and treating our first patient with VCAR33^ALLO once the IND clears.”

Corporate Updates

Trem-cel clinical trial (VBP101) continues to actively enroll patients; next data update expected at EHA 2023

• The company plans to provide further clinical updates on patients treated in the VBP101 study at the EHA2023 Congress, June 9-11, 2023, in Frankfurt, Germany, presented by Dr. Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida.
• The study continues to see strong investigator enthusiasm with robust enrollment.
• Additional trem-cel engraftment and hematologic protection data updates are expected by year-end 2023.
  

Trem-cel is a genome-edited allogeneic hematopoietic stem cell transplant (HSCT) that is lacking the CD33 protein. It is designed to replace standard of care transplants for patients suffering from acute myeloid leukemia (AML) and potentially other blood cancers. Trem-cel has the potential to enable powerful targeted therapies in the post-transplant setting including Mylotarg and CD33-targeted CAR-Ts.

VCAR33^ALLO IND on-track for 1H 2023 submission to U.S. Food & Drug Administration (FDA)

• VCAR33^ALLO is planned to be studied in the VBP301 clinical trial, which will focus on patients who have relapsed following allogeneic stem cell transplant where T cells harvested from the original donor are used as starting material for the drug product.
• The majority of the planned clinical trial sites for VBP301 overlap with VBP101, which should optimize clinical trial site initiation and enrollment.

VCAR33^ALLO is a T-cell therapy derived from allogeneic healthy donors using a chimeric antigen receptor (CAR) specifically binding to CD33.

cGMP qualification of in-house clinical manufacturing facility nearly complete

Current Good Manufacturing Practices (cGMP) qualification activities at the Company’s new in-house manufacturing facility have nearly completed, and the Company is ready to initiate clinical manufacturing of VCAR33^ALLO upon IND clearance. The Company plans to commence trem-cel production at the in-house facility in 2023.    

Upcoming Milestones

• VBP101 data update at EHA in June 2023
• VCAR33^ALLO IND submission expected in 1H 2023
• Additional trem-cel engraftment and hematologic protection data updates expected by year-end 2023
• In-house manufacturing of trem-cel operational by year-end 2023    

First Quarter 2023 Financial Results

• Cash Position: Cash, cash equivalents and marketable securities were $210.2 million as of March 31, 2023, which is projected to fund operations into the first quarter of 2025.
  

• Research & Development (R&D) Expenses: R&D expenses for the first quarter of 2023 were $21.9 million, compared to $15.3 million for the first quarter of 2022. The increase in R&D expenses was primarily due to an increase in clinical, manufacturing and consulting expenses as a result of the ongoing trem-cel clinical trial and the development of the VCAR33 program, an increase in personnel expenses, and an increase in facility costs from the Company’s laboratory and cGMP manufacturing facility expansion.
  

• General & Administrative (G&A) Expenses: G&A expenses for the first quarter of 2023 were $8.5 million, compared to $7.5 million for the first quarter of 2022. The increase in G&A expense was primarily due to increased personnel expenses, including an increase in stock-based compensation expense, and an increase in professional fees.
  

• Net Loss: Net loss for the first quarter of 2023 was $28.4 million, compared to $22.7 million for the first quarter of 2022.

About Vor Bio
Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, the timing and pace of patient enrollment in clinical trials and the availability of data therefrom, the timing of regulatory filings, the expected safety profile of its product candidates, the potential cGMP qualification of its manufacturing facility and the success and timing of manufacturing clinical supply for its product candidates, its intentions to use VCAR33^ALLO in combination with trem-cel as a Treatment System, the potential of trem-cel to enable targeted therapies in the post-transplant setting including Mylotarg and CD33-targeted CAR-Ts, its potential upcoming milestones, its intended use of proceeds from capital raising activities, and its cash runway and expected capital requirements. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; the success of Vor Bio’s in-house manufacturing capabilities and efforts; and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.

Contact:
Investors & Media
Sarah Spencer
+1 857-242-6076
sspencer@vorbio.com