Ambition: Curing Blood Cancers through cell and genome engineering
March 2024
Disclaimer
This presentation (the "Presentation") contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. ("Vor," "Vor Bio" or the "Company"). The words "aim," "anticipate," "believe," "can," "could," "design," "enable" "estimate," "expect," "intend," "may," "ongoing," "plan," "potential," "project," "should," "target," "towards," "will," 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 Presentation include those regarding the feasibility of a trem-cel (formerly VOR33) transplant to be successfully manufactured, to engraft normally, to maintain blood counts following treatment with Mylotarg following allogeneic hematopoietic cell transplant and to be well tolerated, the potential of VCAR33ALLO 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-targetedCAR-Ts, the potential of Vor Bio's platform, Vor Bio's plans, strategies, expectations and anticipated milestones for its preclinical and clinical programs, including the availability and timing of
results from preclinical studies and clinical trials, the timing of regulatory filings, and the timing of dosing patients, Vor Bio's expectations regarding expedited regulatory review of its product candidates as a result of Fast Tracks designation or otherwise, the expected safety profile of Vor Bio's product candidates, cash runway and expected capital requirements, Vor Bio's expectations regarding addressable patient population and reimbursement rates for its product candidates, if approved, and its plans and expectations related to the Company's manufacturing and facilities. 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, completion of, and availability and timing of results from, preclinical studies and
clinical trials and clinical development of Vor Bio's product candidates; whether preclinical data or interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; the uncertainty of 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. The interim data for trem-cel presented in this Presentation is based on eight patients and future results for these patients or additional patients may not produce the same or consistent results. 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 Presentation speak only as of the date of this Presentation, 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.
Certain information contained in this Presentation relates to or is based on studies, publications, surveys and other data obtained from third party sources and Vor Bio's own internal
estimates and research. While we believe these third-party sources to be reliable as of the date of this Presentation, we have not independently verified, and make no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third party sources. In addition, there can be no guarantee as to the accuracy or reliability of
any assumptions or limitations that may be included in such third-party information. While we believe our own internal research is reliable, such research has not been verified by any independent source.
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Our Vision: Cure Blood Cancers Through Cell and Genome Engineering
Unique Approach
Shielded stem cell transplants enabling targeted therapy
VCAR33ALLO | Trem-cel |
CD33-directed | Positive POC demonstrated |
novel transplant donor | in AML with shielded |
CAR-T in clinic with initial | CD33-deleted transplant |
data in 2H 2024 | Additional data in 2H 2024 |
$137M as of Dec. 31, 2023 Cash runway into 2H 2025
In-house GMP
Clinical Manufacturing
Reliable process with rapid
release time
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AML is a Common Leukemia with High Unmet Need
20,000 new cases/year (US)2
(10% of new blood cancer cases)2
Standard of Care is Transplantation
Replace Diseased Bone Marrow with Healthy Donor Cells
Relapse
10,000 deaths/year (US)2
(20% of blood cancer deaths)2
Relapse Despite Transplantation
is still common in AML patients
0.8Post-transplant Incidence of Relapse1Measurable Residual Disease (MRD) positive
3 | 3 | ||
1 | 2 | 3 | 4 |
Conditioning | Harvest | Transplant | Watchful Waiting |
Incidence of
0.6
0.4
Active disease
MRD negative
Toxic therapies | Mobilize and collect | Infuse stem cells | Monitor for relapse; |
to kill existing | stem cells from | into patient to | any follow-up |
bone marrow | matched healthy | restore the blood | treatment will damage |
donor | system | the transplant |
Cumulative
0.2
0.0
0 | 1 | 2 |
Years Since HCT
HCT, Hematopoietic Cell Transplant.
- 1 Araki et al, JCO 2016. 2 LLS 2023
Pipeline Generating Multiple Clinical Readouts over Next 12 Months
Description | Preclinical | Clinical | |||||
Anticipated Milestones | |||||||
Program / Trial | Modality | Indication | Discovery/ | IND- | Phase 1/2 | Phase | |
Validation | Enabling | 2/3 | |||||
VCAR33ALLO | AML | Initial data expected in the | |||||
(healthy transplant donor | CD33-directed transplant donor CAR-T | Post- | |||||
second half of 2024 | |||||||
CAR-T) / VBP301 | transplant | ||||||
AML | Clinical update in second half of | ||||||
Trem-cel + Mylotarg / | Shielded CD33-deleted transplant + | 2024 | |||||
VBP101 | CD33-directed ADC | ||||||
MDS | |||||||
Trem-cel + VCAR33 | Shielded CD33-deleted transplant + | AML | IND filing following initial | ||||
Treatment System | CD33-directed transplant donor CAR-T | trem-cel and VCAR33ALLO data | |||||
CD33-CLL1 Treatment | Multi-specificCAR-T | AML | |||||
System | Multiplex-edited shielded transplant | AML | |||||
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Cell Therapies Designed to Synergize to Potentially Cure AML
VCAR33ALLO
Healthy transplant donor CD33-directed
CAR-T
Trem-cel
Shielded CD33-deleted
stem cell transplant
Vor Bio Treatment
System
A shielded healthy marrow
co-existing with a
potent, persistent CAR-T
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VCAR33ALLO: Healthy Transplant Donor CAR-T
VCAR33ALLO
Healthy transplant donor
CD33-directed
CAR-T
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A New Way of Generating CAR-T Therapy
Traditional Approaches
Autologous cells | Allogeneic cells |
(derived from patient) | (off-the-shelf) |
Exhausted, depleted T cells | Poor expansion and persistence |
High manufacturing failure | Poorer clinical durability |
Vor Bio Approach
Transplant Donor Cells
T cells exactly matched to patient's new immune system, more likely to persist
Stem-like,CAR-T cells more likely to expand and less prone to exhaustion
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Vor Bio's T Cell Manufacturing Process Preserves Stemness
T Cell Phenotype from VCAR33ALLO Process
100
90 | Effector (TEM, TEMRA) | |
80 | ||
70 | Central Memory (TCM) | |
60 | Stem-like, | |
50 | ||
long-lived | ||
40 | ||
Stem Memory (TSCM) | cells capable | |
30 | ||
of self- | ||
20 | renewal | |
10 | ||
0 | ||
CD4+ | CD8+ |
Rapid ~7-day process to preserve stemness
Transplant donor cells | VCAR33ALLO |
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VBP301: VCAR33ALLO Phase 1/2 Clinical Trial
Patient Journey | Enroll | VCAR33ALLO Infusion |
MRD+ or relapsed AML | Arm A: Blasts ≥ 5% | |
following SOC transplant or | ||
Lymphodepletion | ||
trem-cel transplant | Arm B: MRD+ |
3x3 dose escalation starting at 1 x 106 CAR+ cells/kg
Prior TransplantDonor | Consent | Collect | VCAR33ALLO |
Manufacturing | |||
~7-day process | |||
First patient treated in Jan 2024 with initial data expected in 2H 2024
Day 28 Follow-up
2nd transplant
if required
Key Endpoints
01 Safety
02 Expansion, persistence
03 Disease control/response
10 Most clinical trial sites overlap with VBP101 trem-cel study
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Vor Biopharma Inc. published this content on 20 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 20 March 2024 20:31:00 UTC.