Vor Biopharma Inc.

Previous wording

Leveraging our expertise in HSC biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as HCTs to patients. Once these cells engraft into bone marrow, the patient’s healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as VCAR33 ALLO, a chimeric antigen receptor (“CAR”)-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.

Current wording

Leveraging our expertise in hematopoietic stem cell ("HSC") biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as hematopoietic cell transplants (“HCTs”) to patients. Once these cells engraft into bone marrow, the patient’s healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as antibody drug conjugates (“ADCs”) or VCAR33 (previously called VCAR33 ALLO), a chimeric antigen receptor (“CAR”)-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.

Previous wording

We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data for this trial most recently in December 2023. The data showed that primary neutrophil engraftment occurred in all eight patients treated with trem-cel. Three out of three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity typically seen with Mylotarg treatment. We expect to release additional engraftment and hematologic protection data in the second half of 2024. If successful, this trial will provide important validating evidence of the potential of trem-cel and our broader approach.

Current wording

We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML, MDS and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. The latest data update from VBP101 was presented in a poster at the American Society of Hematology (ASH) Annual Meeting on December 8, 2024, which was also presented in an encore presentation at the TANDEM Meetings (Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR) on February 15, 2025.

Previous wording

VCAR33 ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33 ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33 ALLO in VBP301 and expect to treat multiple additional patients in the first half of 2024. We expect to report initial data in the second half of 2024.

Current wording

VCAR33 is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell therapy that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024, we dosed the first patient with VCAR33 in VBP301 and are continuing to dose patients in this study. In September 2024, we announced encouragingin vivo CAR-T expansion data from the first three patients treated, all at the lowest dose of 1 x 106 CAR+ cells/kg. We anticipate sharing additional data from the study in the first half of 2025.

Previous wording

We believe that the combination of trem-cel followed by treatment with VCAR33 ALLOin the post-transplant setting, which we refer to as the trem-cel + VCAR33 Treatment System, may transform patient outcomes and offer the potential for cures for patients that have limited treatment options. The trem-cel + VCAR33 Treatment System would utilize the same healthy donor allogenic cell source for both trem-cel and VCAR33 ALLO. We plan to collect initial data on trem-cel from the VBP101 clinical trial and initial clinical data from the VCAR33 ALLO program prior to IND submission for the trem-cel + VCAR33 Treatment System. However, the VBP301 protocol allows for patients who have received a trem-cel transplant on the VBP101 study to enroll in VBP301 and receive VCAR33 ALLO. This may provide valuable early insights into the potential of the trem-cel + VCAR33 Treatment System to enable a more potent therapy and durable responses post-transplant.

Current wording

We believe that the combination of a trem-cel transplant followed by VCAR33 in the maintenance setting, which we refer to as the trem-cel+VCAR33 Treatment System, may transform patient outcomes and offer the potential for cures for patients that have limited treatment options. The trem-cel+VCAR33 Treatment System would utilize cells from the same healthy donor for both trem-cel and VCAR33. The VBP301 protocol allows for patients who have received a trem-cel transplant on the VBP101 study to enroll in VBP301 and receive VCAR33. This may provide valuable early insights into the potential of the trem-cel+VCAR33 Treatment System to enable a more potent therapy and durable responses post-transplant.

Previous wording

We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support development of our shielded transplants and CAR-T therapeutic candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates and preparing for clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through December 31, 2023, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.0 million.

Current wording

We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support the development of our our potentially transformative product candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for clinical manufacturing of trem-cel and our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates, preparing for and initiating clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through December 31, 2024, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $517.1 million.

Previous wording

We have incurred significant operating losses since inception, including net losses of $117.9 million and $92.1 million for the years ended December 31, 2023 and 2022, respectively. As of December 31, 2023, we had an accumulated deficit of $340.1 million.

Current wording

We have incurred significant operating losses since inception, including net losses of $116.9 million and $117.9 million for the years ended December 31, 2024 and 2023, respectively. As of December 31, 2024, we had an accumulated deficit of $457.0 million.

Previous wording

As of December 31, 2023, we had cash, cash equivalents and marketable securities of $137.2 million. We expect that our cash, cash equivalents and marketable securities at December 31, 2023 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.

Current wording

As of December 31, 2024, we had cash, cash equivalents and marketable securities of $91.9 million. We expect that our cash, cash equivalents and marketable securities at December 31, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2026.

Previous wording

Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our consolidated financial statements as prepaid expenses or accrued research and development expenses. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

Current wording

We expense research and development costs as incurred. We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our consolidated financial statements as prepaid expenses or accrued research and development expenses. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

Previous wording

A significant portion of our research and development costs have been external costs, which we track by stage of development, preclinical or clinical. However, we do not track our internal research and development expenses on a program specific basis because these costs are deployed across multiple projects and, as such, are not separately classified.

Current wording

A significant portion of our research and development costs have been external costs, which we track by program.

Previous wording

Other income increased by $6.8 million for the year ended December 31, 2023, compared to the year ended December 31, 2022. The increase in interest income was due to increases in interest received from our cash, cash equivalents and marketable securities.

Current wording

Other income decreased by $3.9 million for the year ended December 31, 2024, compared to the year ended December 31, 2023. The decrease in other income was due to decreases in interest received from our cash, cash equivalents and marketable securities.

Previous wording

Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.0 million as of December 31, 2023.

Current wording

Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $517.1 million as of December 31, 2024.

Previous wording

In order to fund our future operations, including our planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of December 31, 2023, $274.8 million remains available under this Shelf Registration Statement, including $120.1 million reserved for at-the market offerings discussed below.

Current wording

In order to fund our future operations, including our ongoing and planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of December 31, 2024, $274.4 million remained available under the shelf registration statement, including $119.7 million reserved for at-the market offerings discussed below. This universal shelf registration statement expired on March 18, 2025. We intend to file a new universal shelf registration statement on March 20, 2025, to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof, to replace the expired registration statement.

Previous wording

In December 2022, we terminated the Jefferies ATM Facility and entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the “Stifel ATM Facility”). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 1,016,662 and 10,000 shares of common stock under the Stifel ATM Facility during the years ended December 31, 2023 and 2022 at a weighted average price per share of $4.75 and $6.67, respectively, for aggregate net proceeds of $4.7 million and less than $0.1 million, respectively after deducting commissions and issuance costs payable by us. As of December 31, 2023, $120.1 million remained available to be sold under the Stifel ATM Facility.

Current wording

In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the “Stifel ATM Facility”). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 249,096 and 1,016,662 shares of common stock under the Stifel ATM Facility during the years ended December 31, 2024 and 2023, respectively at a weighted average price per share of $1.64 and $4.75, respectively, for aggregate net proceeds of $0.4 million and $4.7 million, respectively, after deducting commissions. As of December 31, 2024, $119.7 million remained available to be sold under the Stifel ATM Facility.

Previous wording

As of December 31, 2023, we had cash, cash equivalents and marketable securities of $137.2 million. We will need to raise additional capital in the future to fund our future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding, will be obtainable on terms satisfactory to us. In the event that we are unable to obtain sufficient additional funding, there can be no assurance that we will be able to continue as a going concern.

Current wording

As of December 31, 2024, we had cash, cash equivalents and marketable securities of $91.9 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.

Previous wording

We expect that our existing cash, cash equivalents and marketable securities at December 31, 2023 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

Current wording

Based on our current business plan and current capital resources, management has concluded that there is substantial doubt regarding our ability to continue as a going concern for a period of 12 months from the date of issuance of the accompanying consolidated financial statements. We expect that our existing cash, cash equivalents and marketable securities at December 31, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2026. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect. The accompanying financial statements have been prepared on a going concern basis and do not include any adjustments to the carrying amounts and classification of assets and liabilities that may be necessary if we were unable to continue as a going concern.

Previous wording

Net cash used in operating activities was $85.1 million for the year ended December 31, 2022, reflecting a net loss of $92.1 million and net cash use of $9.8 million for operating assets and liabilities, which were offset by non-cash charges of $16.8 million. The non-cash charges primarily consisted of stock-based compensation expense of $10.7 million, non-cash lease expense of $3.6 million and depreciation expense of $2.5 million.

Current wording

Net cash used in operating activities was $99.7 million for the year ended December 31, 2024, reflecting a net loss of $116.9 million, offset by non-cash charges of $17.3 million. The non-cash charges primarily consisted of stock-based compensation expense of $9.8 million, non-cash lease expense of $5.0 million and depreciation expense of $3.5 million, offset by non-cash interest accretion of $1.1 million.

Previous wording

The $15.1 million increase in net cash used in operating activities for the year ended December 31, 2023 compared to the year ended December 31, 2022 was primarily due to an increase in research and development expenses, including clinical and manufacturing expenses as a result of the ongoing trem-cel clinical trial and the start of our VCAR33 clinical timing, and differences in the timing of payments for research and development expenses incurred during each respective period.

Current wording

The $0.6 million decrease in net cash used in operating activities for the year ended December 31, 2024 compared to the year ended December 31, 2023 was primarily due to a decrease in research and development expenses offset by differences in the timing of payments for costs incurred during each respective period.

Previous wording

Net cash provided by investing activities was $71.0 million for the year ended December 31, 2023, which consisted of purchases of $74.9 million of marketable securities and $1.1 million of property and equipment offset by proceeds of $147.0 million from the maturity of marketable securities. Net cash used in investing activities was $94.1 million for the year ended December 31, 2022, which consisted of purchases of $123.2 million of marketable securities and $8.5 million of property and equipment offset by proceeds of $37.6 million from the maturity of marketable securities.

Current wording

Net cash provided by investing activities was $96.9 million for the year ended December 31, 2024, which consisted of purchases of $9.9 million of marketable securities and $0.2 million of property and equipment offset by proceeds of $107.0 million from the maturity of marketable securities. Net cash provided by investing activities was $71.0 million for the year ended December 31, 2023, which consisted of purchases of $74.9 million of marketable securities and $1.1 million of property and equipment offset by proceeds of $147.0 million from the maturity of marketable securities.

Previous wording

Net cash provided by financing activities was $2.9 million for the year ended December 31, 2023, which consisted of proceeds of $4.6 million from the sale of common stock under the Stifel ATM Facility and proceeds of $0.3 million from the exercise of stock options and purchases of common stock under our ESPP, offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $1.2 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $117.1 million for the year ended December 31, 2022, which consisted of proceeds of $111.5 million from the sale of common stock under the public offering and concurrent private placement, before deducting commissions and issuance costs payable by us, proceeds of $4.5 million received from the sale of common stock under the Jefferies ATM Facility and the Stifel ATM Facility, and net proceeds of $1.2 million from the exercise of stock options and purchases of common stock under our ESPP.

Current wording

Net cash provided by financing activities was $53.4 million for the year ended December 31, 2024, which consisted of $55.6 million of proceeds from the private placement, $0.3 million of proceeds from the sale of common stock under the Stifel ATM Facility, and proceeds of $0.2 million from the exercise of stock options and purchases of common stock under our ESPP. These amounts were offset by the payment of $2.3 million of issuance costs related to the private placement and $0.3 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.9 million for the year ended December 31, 2023, which consisted of proceeds of $4.6 million from the sale of common stock under the Stifel ATM Facility and proceeds of $0.3 million from the exercise of stock options and purchases of common stock under our ESPP, offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $1.2 million of taxes paid related to net share settlement of equity awards.