VOR Vor Biopharma Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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VOR33
Acute myeloid leukemia (AML)
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Phase 1/2
Phase 1/2
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VCAR33
Acute myeloid leukemia (AML)
|
Phase 1/2
Phase 1/2
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Latest News
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Health Canada clears the CTA for VOR33 clinical trial
Vor to present on VOR33 manufacturing scale-up and in-depth genomic characterization at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
Successful completion of Initial Public Offering raising $203.4 million in gross proceeds
CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Vor Biopharma (NASDAQ:VOR) today reported financial results for the three-month period ended March 31, 2021, and provided an update on its business, including the announcement of the second regulatory authority to allow development of VOR33 in acute myeloid leukemia (AML) patients with the Canadian clinical trial application (CTA) clearance.
"The Vor team remains focused on delivering upon multiple clinical and operational milestones," said Robert Ang, MBBS, MBA, Vor's President and Chief Executive Officer. "We have established a strong foundation to generate several important catalysts over the next 12 to 24 months which will demonstrate the potential of Vor's novel platform. With respect to our lead clinical program, VOR33 in patients suffering from acute myeloid leukemia, we remain on track to enroll the first patient in our Phase 1/2a trial in the second quarter of 2021."
Corporate Highlights
- Health Canada clears the CTA for VOR33 clinical trial. In April 2021, the Company received a No Objection Letter from Health Canada for its CTA to conduct a clinical trial for Vor's lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33. This CTA clearance represents the second regulatory authority to allow development of VOR33 in AML patients following the U.S. Food and Drug Administration's clearance of Vor's investigational new drug (IND) application for VOR33 in AML in January 2021. The Canadian CTA utilizes the same protocol as cleared under the IND in the U.S. and data from these patients can be used with data from U.S.-treated patients to expedite development. Vor expects to initiate a first-in-human Phase 1/2a trial of VOR33 in AML patients in combination with MylotargTM (gemtuzumab ozogamicin) by enrolling the first patient in the second quarter of 2021. The Company believes VOR33, if successful, has significant potential to improve clinical outcomes for malignant cancers beyond AML.
- Vor to present on VOR33 manufacturing scale-up and in-depth genomic characterization at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place virtually May 11-14, 2021. On May 11, 2021 at 7:00 PM ET, the Company will conduct an oral presentation titled VOR33: A Clinic-Ready CRISPR/Cas9 Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia. The study outlined the pre-clinical journey of VOR33 and the manufacturing scale-up for its clinical development. Also on May 11, 2021, between 8:00 AM and 10:00 AM ET, the Company will have a poster presentation titled Rigorous Assessment of Off-Target Editing by CRISPR/Cas9 in VOR33, an Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia. The in-depth genomic characterization included evaluation of homology sequence based as well as sequence homology independent off-target assessment through deep sequencing of genome-edited DNA. These studies confirmed that CD33-edited HSCs, generated at clinical scale, had no significantly detectable genotoxic risk.
- Successful completion of Initial Public Offering raising $203.4 million in gross proceeds. In February 2021, the Company completed its IPO, raising $203.4 million in gross proceeds before deducting underwriting discounts, commissions and other offering expenses.
First Quarter 2021 Financial Results
- Cash and Cash Equivalents: Vor finished the first quarter of 2021 with $262.6 million in cash and cash equivalents, compared with $48.5 million as of December 31, 2020. Cash and cash equivalents on March 31, 2021 includes the $186.3 million in net proceeds from the Company's IPO completed in February 2021 as well as $45.4 million in net proceeds from the completion of its Series B preferred stock financing in January 2021.
- R&D Expenses: Research and development expenses increased to $8.9 million in the first quarter of 2021, up from $6.2 million in the first quarter of 2020. The increase in research and development expenses was primarily due to an increase in employee headcount necessary to support the growth of our research and development efforts and increases in facilities and other expenses.
- G&A Expenses: General and administrative expenses totaled $4.8 million in the first quarter of 2021, up from $1.7 million in the first quarter of 2020, primarily due to an increase in employee headcount, increased professional fees, and an increase in facilities and other expenses.
- Net Loss: Vor reported a net loss of $13.7 million in the first quarter of 2021, compared with a net loss of $7.8 million for the first quarter of 2020.
Upcoming Milestones
- Enroll first patient in VOR33 Phase 1/2a trial in the second quarter of 2021
- Initial VOR33 human engraftment and protection data expected in late 2021 or first half of 2022
- Initial VCAR33 monotherapy clinical proof-of-concept data expected in 2022, depending on investigator's timing of data release
- File IND for VOR33/VCAR33 Treatment System in the second half of 2022, following data from our VOR33 clinical trial and the National Marrow Donor Program's Phase 1/2 clinical trial studying the VCAR33 construct
About Vor Biopharma
Vor Biopharma is a cell therapy company that aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells. For more information please visit www.vorbio.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning the Private Securities Litigation Reform Act of 1995. The words "believe," "continue," "could," "estimate," "expect," "intend," "may," "milestones," "plan," "potential," "project," "should," "target," "upcoming," "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 statements regarding the Company's initiation the VOR33 Phase 1/2a clinical trial by enrolling the first patient in the second quarter of 2021, the release of data from such trial, the submission of an IND for the VOR33/VCAR33 Treatment System and the timing of the release of data from the VCAR33 clinical trial. The Company 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 the Company'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 and availability of funding sufficient for the Company's 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 the Company's most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission (SEC), and in other filings that the Company make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company 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.
Investor:
Chris Brinzey
Westwicke, an ICR Company
+1 339-970-2843
[email protected]
Media:
Rebecca Spalding
Ten Bridge Communications
[email protected]Condensed Consolidated Balance Sheets
(in thousands, unaudited)March 31, December 31, 2021 2020 Cash and cash equivalents $ 262,567 $ 48,539 Total assets 292,939 75,908 Total liabilities 25,752 27,637 Convertible preferred stock - 107,336 Total stockholders' equity (deficit) 267,187 (59,065 ) Condensed Consolidated Statement of Operations
(unaudited)Three Months Ended March 31, (in thousands, except share and per share amounts) 2021 2020 Operating expenses: Research and development $ 8,941 $ 6,171 General and administrative 4,789 1,707 Total operating expenses 13,730 7,878 Loss from operations (13,730 ) (7,878 ) Other income (expense): Interest income 7 29 Total other income 7 29 Net loss and comprehensive loss $ (13,723 ) $ (7,849 ) Cumulative dividends on redeemable convertible preferred stock (1,228 ) (715 ) Net loss attributable to common stockholders $ (14,951 ) $ (8,564 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.67 ) $ (59.06 ) Weighted-average common shares outstanding, basic and diluted 22,263,994 145,016 
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CAMBRIDGE, Mass., April 28, 2021 (GLOBE NEWSWIRE) -- Vor Biopharma (NASDAQ:VOR) is pleased to announce that Vor scientists will give two presentations documenting the development, manufacturing, and validation of its engineered hematopoietic stem cell (eHSC) clinical candidate VOR33 at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place virtually May 11-14, 2021.
"As we ready VOR33 for a Phase 1/2a trial, we are pleased to share the first consolidated review of the comprehensive research that underpins this cell therapy candidate and our wider eHSC platform," said John Lydeard, PhD, scientific lead on the VOR33 program and Head of Target Discovery at Vor. "This tremendous progress with Vor's genome engineering process and manufacturing scale-up brings us one step closer to potentially realizing the benefits of our powerful platform to treat patients with acute myeloid leukemia."
The details of the presentations are below:
Oral Presentation
Title: VOR33: A Clinic-Ready CRISPR/Cas9 Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia
Presenter: John Lydeard
Session Title: Advances in Ex Vivo Modified Cell Therapies
Session Date/Time: Tuesday May 11, 2021 5:30 PM - 7:15 PM ET
Presentation Time: 7:00-7:15 PM ET
Abstract Number: 7Poster Presentation
Title: Rigorous Assessment of Off-Target Editing by CRISPR/Cas9 in VOR33, an Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia
Session Title: Cell Therapy Product Engineering, Development or Manufacturing
Session Date/Time: Tuesday May 11, 2021 8:00 AM - 10:00 AM ET
Abstract Number: 858About VOR33
VOR33 is Vor's lead product candidate, consisting of eHSCs that we have engineered to lack the protein CD33, and is designed to replace the standard of care in transplant settings for patients suffering from AML and potentially other hematologic malignancies. Once the VOR33 cells have engrafted, we believe that patients can be treated with anti-CD33 therapies, such as Mylotarg® or, if approved by the FDA, Vor's in-licensed CD33 chimeric antigen receptor T-cell (CAR-T) therapy candidate, with limited on-target toxicity, leading to durable anti-tumor activity and potential cures. In preclinical studies, we have observed that the removal of CD33 provided robust protection of VOR33 eHSCs from the cytotoxic effects of CD33-directed therapies, yet had no deleterious effects on the differentiation or function of hematopoietic cells.About Vor Biopharma
Vor Biopharma is a cell therapy company that aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells.Forward Looking Statements
This press release contains forward-looking statements within the meaning the Private Securities Litigation Reform Act of 1995. The words "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "project," "should," "target," "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 statements regarding Vor's (the Company) platform's potential to treat patients suffering from acute myeloid leukemia. The Company 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 the Company'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 and availability of funding sufficient for the Company's 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 the Company's Annual Report on Form 10-K for the year ended December 31, 2020, which is on file with the Securities and Exchange Commission (SEC), and in other filings that the Company make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company 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.Contacts:
Investor:
Constantine Davides, CFA
Westwicke
+1 339-970-2846
[email protected]Media:
Dan Quinn
Ten Bridge Communications
[email protected] -
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IND accepted for VOR33, Vor's lead eHSC product candidate for treatment of AML
Completed initial public offering raising $203.4 million in total gross proceeds
CAMBRIDGE, Mass., March 25, 2021 (GLOBE NEWSWIRE) -- Vor Biopharma (NASDAQ:VOR) (the Company), a cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today reported financial results for the year ended December 31, 2020, and provided an update on its business.
"We are very pleased with our continued exceptional scientific, clinical and financial progress," noted Robert Ang, MBBS, MBA, Vor's President and Chief Executive Officer. "Earlier this year, the FDA accepted our IND for our lead eHSC product candidate VOR33, and with the completion of our successful initial public offering, Vor is now in a strong financial position to change the paradigm around targeted oncology treatment and hematopoietic stem cell transplants."
Corporate Highlights
- Investigational new drug (IND) application accepted for VOR33, Vor's lead eHSC product candidate for treatment of acute myeloid leukemia (AML). The Company is developing VOR33 as an eHSC product candidate to potentially replace the standard of care in the transplant setting. Vor intends to initiate a first-in-human Phase 1/2a trial of VOR33 in AML patients in combination with MylotargTM (gemtuzumab ozogamicin) by enrolling the first patient in the second quarter of 2021. The Company believes VOR33, if successful, has significant potential to improve clinical outcomes for malignancies beyond AML.
- Successfully completed an initial public offering (IPO). In February 2020, the Company completed its IPO, raising $203.4 million in gross proceeds before deducting underwriting discounts and commissions and other offering expenses.
- Vor strengthens platform with CAR-T license and collaborations. To complement VOR33 and lay groundwork for the VOR33/VCAR33 Treatment System, Vor licensed a CD33-targeted CAR-T construct, VCAR33, from the National Institutes of Health. The Company also announced collaborations with two next-generation gene-editing companies, Arbor Biotechnologies and Metagenomi.
Full Year 2020 Financial Results
- Cash and Cash Equivalents: Vor ended 2020 with $48.5 million in cash and cash equivalents, compared with $6.5 million as of December 31, 2019. Cash at December 31, 2020 does not include the $186.3 million in net proceeds from our IPO completed in February 2021 and $45.4 million in net proceeds from the completion of our Series B preferred stock financing in January 2021.
- R&D Expenses: Research and development expenses increased to $31.6 million for the year ended December 31, 2020, up from $6.2 million for the prior year. The increase in research and development expenses was primarily due to an increased level of expenses associated with external preclinical studies, consulting fees, laboratory supplies costs and increased employee headcount.
- G&A Expenses: General and administrative expenses totaled $11.7 million for the year ended December 31, 2020, up from $4.2 million for the prior year, primarily due to an increase in employee headcount, increased professional fees, and an increase in facilities and other expenses.
- Net Loss: Vor reported a net loss of $43.3 million for the year ended December 31, 2020, compared with a net loss of $10.8 million for the prior year.
Potential Upcoming Milestones
- Enroll first patient in VOR33 Phase 1/2a trial in the second quarter of 2021
- Initial VOR33 human engraftment and protection data expected in late 2021/first half of 2022
- Initial VCAR33 monotherapy clinical proof of concept data expected in 2022, depending on investigator's timing of data release
- File IND for VOR33/VCAR33 Treatment System in the second half of 2022, following data from our VOR33 clinical trial and the National Marrow Donor Program's Phase 1/2 clinical trial studying the VCAR33 construct
About Vor Biopharma
Vor Biopharma is a cell therapy company that aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells. For more information please visit www.vorbio.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning the Private Securities Litigation Reform Act of 1995. The words "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "project," "should," "target," "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 statements regarding the Company's initiation the VOR33 Phase 1/2a clinical trial by enrolling the first patient in the second quarter of 2021, the release of data from such trial, the submission of an IND for the VOR33/VCAR33 Treatment System and the timing of the release of data from the VCAR33 clinical trial. The Company 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 the Company'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 and availability of funding sufficient for the Company's 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 the Company's Annual Report on Form 10-K for the year ended December 31, 2020, which is on file with the Securities and Exchange Commission (SEC), and in other filings that the Company make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company 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.
Investor:
Constantine Davides, CFA
Westwicke
+1 339-970-2846
[email protected]Media:
Mary Carmichael
Ten Bridge Communications
+1 617-413-3543
[email protected]Consolidated Balance Sheet Data
(in thousands)December 31, 2020 2019 Cash and equivalents $ 48,539 $ 6,466 Total assets 75,908 9,826 Total liabilities 27,637 2,186 Convertible preferred stock 107,336 25,069 Total stockholders' equity (deficit) (59,065 ) (17,429 ) Consolidated Statement of Operations
Twelve Months Ended
December 31,(in thousands, except share and per share amounts) 2020 2019 Operating expenses: Research and development $ 31,618 $ 6,200 General and administrative 11,748 4,217 Total operating expenses 43,366 10,417 Loss from operations (43,366 ) (10,417 ) Other income (expense): Interest income 29 154 Interest expense related to convertible notes - (608 ) Change in fair value of derivative liabilities - 32 Total other income (expense) 29 (422 ) Net loss and comprehensive loss $ (43,337 ) $ (10,839 ) Cumulative dividends on redeemable convertible preferred stock (5,925 ) (1,773 ) Net loss attributable to common stockholders $ (49,262 ) $ (12,612 ) Net loss per share attributable to common stockholders,
basic and diluted$ (230.57 ) $ (109.70 ) Weighted-average common shares outstanding, basic and diluted 213,658 114,961 -
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CAMBRIDGE, Mass., March 05, 2021 (GLOBE NEWSWIRE) -- Vor Biopharma (NASDAQ:VOR), a cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today announced that the Company will be participating in the Barclays Global Healthcare Conference, which will take place virtually on March 9-11, 2021.
Robert Ang, MBBS, MBA, Vor's President and Chief Executive Officer, and Nathan Jorgensen, PhD, Chief Financial Officer, are scheduled to present on Thursday, March 11, 2021, at 3:00 p.m. E.T. A live webcast of the presentation can be accessed via the Investors section of the Company's website at www.vorbio.com. An archived replay of the webcast will be available on the Company's website following the conference.
About Vor Biopharma
Vor Biopharma is a cell therapy company that aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells.
Contacts:
Investor:
Constantine Davides, CFA
Westwicke
+1 339-970-2846
[email protected]Media:
Mary Carmichael
Ten Bridge Communications
+1 617-413-3543
[email protected] -
View Full Article Hide Full Article
CAMBRIDGE, Mass., March 01, 2021 (GLOBE NEWSWIRE) -- Vor Biopharma (NASDAQ:VOR), a cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today announced that the Company will be participating in the Cowen 41st Annual Health Care Conference, which will take place virtually on March 1-4, 2021. Robert Ang, MBBS, MBA, Vor's President and Chief Executive Officer, and Nathan Jorgensen, PhD, Chief Financial Officer, are scheduled to present on Thursday, March 4, 2021, at 1:30 p.m. E.T.
About Vor Biopharma
Vor Biopharma is a cell therapy company that aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells.
Contacts:
Investor:
Constantine Davides, CFA
Westwicke
+1 339-970-2846
[email protected]Media:
Mary Carmichael
Ten Bridge Communications
+1 617-413-3543
[email protected]