Vor Bio Reports Third Quarter 2024 Financial Results and Provides Company Update
08 November 2024 - 8:05AM
Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering
company, today reported financial results for the three-month
period ended September 30, 2024, and provided a corporate
update.
“We are pleased with our operational and clinical execution this
quarter and look forward to sharing additional clinical data since
our September data update at the ASH annual meeting in December,”
said Dr. Robert Ang, Vor Bio’s President and Chief Executive
Officer.
Corporate Updates
Trem-cel + Mylotarg (VBP101) Clinical Trial
- 25 patients have
been dosed with trem-cel and 6 patients have now received the third
dose level of Mylotarg at 2 mg/m2.
- On September 5, 2024, the Company provided a clinical update on
18 patients treated with trem-cel in the VBP101 study. The data
demonstrated:
- Reliable engraftment, robust platelet recovery, and high CD33
editing efficiency with full myeloid chimerism at Day 28.
- Shielding of the blood system, with maintained neutrophil and
platelet counts across multiple Mylotarg doses of 0.5, 1, and 2
mg/m2.
- Broadened therapeutic index for Mylotarg with drug exposure
represented by AUC which is related to efficacy, consistent with
labeled Mylotarg doses, and with maximal concentrations, measured
by Cmax and related to veno-occlusive disease, well below
known toxic range.
- Early evidence suggesting patient benefit as measured by
relapse-free survival when compared to published high-risk AML
comparators1.
- The Company plans to share a further clinical data update in a
poster presentation at the ASH Annual Meeting on Sunday, December
8, from 6-8pm PT. (Poster Session 616. Acute Myeloid Leukemias:
Investigational Drug and Cellular Therapies. Publication number
2873). The poster will be made available on Vor Bio’s website when
the ASH embargo lifts at 9am PT on the day of the
presentation and the Company plans to host a webcast to review
the data on Monday, December 9th at 5am PT/8am ET.
Trem-cel is a shielded transplant in development for patients
with acute myeloid leukemia (AML) and myelodysplastic syndromes
(MDS), in which healthy transplant donor cells are genetically
engineered by removing CD33, with the potential to shield healthy
cells and enable targeted therapies post-transplant such as
Mylotarg and CAR-T therapy.
VCAR33ALLO (VBP301)
Clinical Trial
- Dosing is ongoing in the VBP301
study with continued strong enrollment.
- On September 5, 2024, the Company
announced encouraging in vivo CAR-T expansion data from three
patients treated to date, all at the lowest dose of 1 x 106 CAR+
cells/kg.
VBP301, a Phase 1/2, multicenter, open-label, first-in-human
study of VCAR33ALLO, is a transplant donor-derived anti-CD33 CAR-T
cell therapy for patients with AML who have relapsed following a
standard-of-care or trem-cel transplant.
Preclinical Research Updates
VADC45In September 2024, the Company announced
a new preclinical asset, VADC45, which has several potential
opportunities in oncology, gene therapy, and autoimmune disorders.
VADC45 is an antibody-drug conjugate (ADC) designed to target the
CD45 protein, a well-validated target for a wide variety of blood
cancers with clinical poof of concept. The linker-payload used in
VADC45 has also been clinically validated. Vor Bio is currently
progressing IND-enabling studies to enable future Phase 1
studies.
Preclinical data presentations further validate Vor
Bio’s robust platform
ESGCT 2024In October 2024, the Company
presented preclinical data at the European Society of Gene and Cell
Therapy Congress on the development and characterization of its
novel CD33/CLL-1-directed CAR-T approach in AML.
ASH 2024Additional analyses from the most
comprehensive single cell AML atlas known to date (more than
400,000 cells from 26 AML patients and 10 healthy donors) were
accepted for presentation at the ASH 2024 annual meeting in
December. These data provide an unprecedented view of AML
immunophenotypic heterogeneity, both across patients and within
patients across clinical timepoints.Abstract
Title: Multimodal Atlas of Paired Diagnosis and
Relapse AML Reveals Surface Antigens for Multi-Specific
ImmunotherapyFormat: Poster
presentationSession Name: Acute Myeloid Leukemias:
Biomarkers and Molecular Markers in Diagnosis and
Prognosis Session date and
time: Saturday, Dec 7, 5:30-7:30pm PT
New Chief Financial Officer
appointed Han Choi, M.D., LL.M., was appointed as
Vor Bio’s new Chief Financial Officer. Dr. Choi brings more than 25
years of experience in investment management, business development,
and corporate strategy within the pharmaceutical and biotechnology
sectors to Vor Bio. His extensive expertise in forming corporate
partnerships, managing complex transactions, and developing capital
markets strategies will be pivotal as the Company advances its
clinical pipeline and strengthens its leadership in cell and genome
engineering.
Upcoming Milestones
- Trem-cel + Mylotarg clinical data update planned at ASH 2024
annual meeting
Third Quarter 2024 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $62.8 million as of September 30, 2024,
which is projected to fund operations into the second half of
2025.
- Research & Development (R&D)
Expenses: R&D expenses for the third quarter of
2024 were $21.8 million, compared to $27.6 million for the third
quarter of 2023. The decrease of $5.8 million was due to a decrease
in license payments, preclinical expenses, and manufacturing
starting materials, offset in part by an increase in clinical trial
costs to support our trem-cel and VCAR33ALLO programs.
- General & Administrative (G&A)
Expenses: G&A expenses for the third quarter of
2024 were $6.7 million, compared to $7.7 million for the third
quarter of 2023. The decrease of $1.0 million was primarily due to
a decrease in professional fees and stock-based compensation
expense.
- Net Loss: Net loss for the third quarter
of 2024 was $27.6 million, compared to $33.2 million for the third
quarter of 2023.
Condensed
Consolidated Balance Sheet Data (Unaudited) |
|
(in
thousands) |
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Cash, cash equivalents and marketable securities |
$ |
62,809 |
|
|
$ |
137,175 |
|
|
Total
assets |
|
|
115,991 |
|
|
|
198,126 |
|
|
Total
liabilities |
|
|
43,036 |
|
|
|
47,402 |
|
|
Total
stockholders' equity |
|
|
72,955 |
|
|
|
150,724 |
|
|
|
|
|
|
|
|
Condensed
Consolidated Statement of Operations (Unaudited) |
|
(in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
Three Months
Ended |
|
|
|
September 30, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Operating
expenses: |
|
|
|
|
|
Research and development |
|
$ |
21,817 |
|
|
$ |
27,606 |
|
|
General and administrative |
|
|
6,696 |
|
|
|
7,710 |
|
|
Total operating expenses |
|
$ |
28,513 |
|
|
$ |
35,316 |
|
|
Loss from
operations |
|
$ |
(28,513) |
|
|
$ |
(35,316) |
|
|
Other
income: |
|
|
|
|
|
Interest income |
|
|
954 |
|
|
|
2,126 |
|
|
Total other income |
|
|
954 |
|
|
|
2,126 |
|
|
Net
loss |
|
$ |
(27,559) |
|
|
$ |
(33,190) |
|
|
|
|
|
|
|
|
Net loss per
share attributable to common stockholders, basic and diluted |
|
$ |
(0.40) |
|
|
$ |
(0.49) |
|
|
Weighted-average common shares outstanding, basic and diluted |
|
|
68,465,801 |
|
|
|
67,607,713 |
|
|
|
|
|
|
|
|
About Vor BioVor 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 StatementsThis 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 and dosing in clinical trials and
the availability of data therefrom, 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 cash runway and expected capital requirements, and
other statements that are not historical fact. 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; uncertainties regarding 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 and Vor
Bio’s ability to continue as a going concern. 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 & MediaSarah Spencer +1
857-242-6076sspencer@vorbio.com
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