VOR
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Earnings documents stored for VOR.
Investor releaseQuarter not tagged2026-08-11Vor Bio Reports Second Quarter 2026 Financial Results and Provides Corporate Update
GlobeNewswire
Vor Bio Reports Second Quarter 2026 Financial Results and Provides Corporate Update
Enrollment on track for Phase 3 UPSTREAM MG trial of telitacicept in generalized myasthenia gravis patients with topline results anticipated in 1H27 Enrollment ongoing for Phase 3 UPSTREAM SjD of telitacicept in primary Sjögren’s disease Pro-forma cash and investment balance of $514.5 million expected to provide runway into early 2029 BOSTON, Aug. 11, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the second quarter ended June 30, 2026, and provided a corporate update. “We continued to make meaningful progress in 2026 with telitacicept receiving its fourth and fifth commercial approvals in China for the treatment of Sjögren’s disease and IgA nephropathy and its Phase 3 TELIGAN trial in IgA nephropathy being featured in The New England Journal of Medicine. Enrollment in our global studies, UPSTREAM MG and UPSTREAM SjD, remains on track, bringing us closer to demonstrating telitacicept’s potential as a first-in-class and best-in-disease BAFF/APRIL therapy in both indications. We are encouraged by our growing momentum, the caliber of talent joining Vor, and the opportunity ahead,” said Jean-Paul Kress, M.D., Chairman and Chief Executive Officer of Vor Bio.Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG Primary Sjögren’s Disease UPSTREAM SjD Corporate Updates Appointed David Zaccardelli, Pharm.D., former Chief Executive Officer and President of Verona Pharma, to its Board of Directors Announced China’s National Medicinal Products Administration (NMPA) approved telitacicept for the treatment of adult patients with SjD and IgA nephropathy (IgAN) Results from the Phase 3 TELIGAN trial evaluating telitacicept in IgAN in China were published in The New England Journal of Medicine Second Quarter 2026 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $466.1 million as of June 30, 2026, which together with the $48.4 million net proceeds from at-the-market sales during July 2026, are projected to fund operations into early 2029. Research & Development (R&D) Expenses: R&D expenses for the second quarter of 2026 were $25.9 million, c…Read full documentShow less
Enrollment on track for Phase 3 UPSTREAM MG trial of telitacicept in generalized myasthenia gravis patients with topline results anticipated in 1H27 Enrollment ongoing for Phase 3 UPSTREAM SjD of telitacicept in primary Sjögren’s disease Pro-forma cash and investment balance of $514.5 million expected to provide runway into early 2029 BOSTON, Aug. 11, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the second quarter ended June 30, 2026, and provided a corporate update. “We continued to make meaningful progress in 2026 with telitacicept receiving its fourth and fifth commercial approvals in China for the treatment of Sjögren’s disease and IgA nephropathy and its Phase 3 TELIGAN trial in IgA nephropathy being featured in The New England Journal of Medicine. Enrollment in our global studies, UPSTREAM MG and UPSTREAM SjD, remains on track, bringing us closer to demonstrating telitacicept’s potential as a first-in-class and best-in-disease BAFF/APRIL therapy in both indications. We are encouraged by our growing momentum, the caliber of talent joining Vor, and the opportunity ahead,” said Jean-Paul Kress, M.D., Chairman and Chief Executive Officer of Vor Bio.Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG Primary Sjögren’s Disease UPSTREAM SjD Corporate Updates Appointed David Zaccardelli, Pharm.D., former Chief Executive Officer and President of Verona Pharma, to its Board of Directors Announced China’s National Medicinal Products Administration (NMPA) approved telitacicept for the treatment of adult patients with SjD and IgA nephropathy (IgAN) Results from the Phase 3 TELIGAN trial evaluating telitacicept in IgAN in China were published in The New England Journal of Medicine Second Quarter 2026 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $466.1 million as of June 30, 2026, which together with the $48.4 million net proceeds from at-the-market sales during July 2026, are projected to fund operations into early 2029. Research & Development (R&D) Expenses: R&D expenses for the second quarter of 2026 were $25.9 million, compared to $261.5 million for the second quarter of 2025. The decrease of $235.6 million was primarily due to the $222.6 million expense incurred for the Telitacicept License Agreement in the second quarter of 2025, as well as costs incurred in connection with the termination of employees and the prior lease in the same quarter in 2025. These decreases were partially offset by the increase in spend for our new programs, telitacicept in gMG and SjD in the second quarter of 2026. General & Administrative (G&A) Expenses: G&A expenses for the second quarter of 2026 were $21.9 million, compared to $12.8 million for the second quarter of 2025. The increase of $9.1 million was primarily due to increases in stock-based compensation compared to the prior year period. The increase was also attributable to an increase in personnel-related expenses and commercial-related expenses. Net Loss: Net loss for the second quarter of 2026 was $62.8 million, compared to $1,573.7 million net loss for the second quarter of 2025. The decrease in loss of $1,510.9 million was primarily due to the change in fair value of the outstanding liability-classified warrants in the second quarter of 2026 compared to the change in fair value recognized in the second quarter of 2025. About TelitaciceptTelitacicept is a novel recombinant fusion protein designed to treat autoimmune diseases through dual inhibition of BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), generalized myasthenia gravis (gMG), IgA nephropathy (IgAN), and Sjögren’s disease (SjD). Vor Bio is advancing global development programs across major autoimmune indications, including a global Phase 3 trial in gMG and SjD, to support potential regulatory approvals in the United States, Europe, and Japan. About Vor BioVor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. For more information visit www.vorbio.com. Vor Bio routinely posts information that may be important to investors in the “Investors” section of its website. The Company encourages investors to consult that section of its website regularly. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “anticipate,” “continue,” “could,” “design,” “expect,” “initiate,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “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 telitacicept’s potential as a first-in-class and best-in-disease BAFF/APRIL therapy for gMG and SjD; Vor Bio’s projected cash runway; Vor Bio’s development and commercialization plans for telitacicept, including having topline data from the UPSTREAM-MG trial in the first half of 2027; 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 the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. 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. Statements regarding Vor Bio’s cash runway do not indicate when or if Vor Bio may access the capital markets. 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: Media & Investor Contacts: Carl Mauch [email protected]
Investor releaseQuarter not tagged2026-08-11Vor Biopharma Shares Drop After Q2 Results
MT Newswires
Vor Biopharma Shares Drop After Q2 Results
Vor Biopharma (VOR) shares were down 1.2% in Tuesday trading after the company reported its Q2 resul
Investor releaseQuarter not tagged2026-05-13Vor Bio Reports First Quarter 2026 Financial Results and Provides Corporate Update
GlobeNewswire
Vor Bio Reports First Quarter 2026 Financial Results and Provides Corporate Update
Enrollment on track for Phase 3 UPSTREAM MG trial of telitacicept in generalized myasthenia gravis patients with topline results anticipated in 1H27 Enrollment ongoing for Phase 3 UPSTREAM SjD of telitacicept in primary Sjögren’s disease Cash and investment balance of $491.5 million as of March 31, 2026 expected to provide runway into early 2029 BOSTON, May 13, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the first quarter ended March 31, 2026, and provided a corporate update. “We had another quarter of solid execution for our two global Phase 3 programs in generalized myasthenia gravis and primary Sjögren’s disease, both of which are progressing as planned,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Encouragingly, clinician feedback this quarter points to a shift in myasthenia gravis treatment toward broader, more durable disease control through upstream B-cell modulation and targeting multiple pathogenic immunoglobulins, which are central to the BAFF/APRIL mechanism. While efficacy benchmarks in myasthenia gravis are well established, many physicians see the potential for this approach moving earlier in the treatment paradigm, given its potential to change the disease trajectory. Our conviction in telitacicept continues to grow, and we believe it has the potential to become a foundational therapy in B-cell driven autoimmune diseases.” Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG Enrollment ongoing in global randomized, double-blind, placebo-controlled Phase 3 registrational trial with an open-label extension assessing the efficacy and safety of telitacicept in gMG Topline data anticipated in 1H 2027 Primary Sjögren’s Disease UPSTREAM SjD Enrollment ongoing in global randomized, double-blind, placebo-controlled Phase 3 registrational trial assessing the efficacy and safety of telitacicept in SjD First Quarter 2026 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $491.5 million as of March 31, 2026, which are projected to fund operations into early 2029. Researc…Read full documentShow less
Enrollment on track for Phase 3 UPSTREAM MG trial of telitacicept in generalized myasthenia gravis patients with topline results anticipated in 1H27 Enrollment ongoing for Phase 3 UPSTREAM SjD of telitacicept in primary Sjögren’s disease Cash and investment balance of $491.5 million as of March 31, 2026 expected to provide runway into early 2029 BOSTON, May 13, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the first quarter ended March 31, 2026, and provided a corporate update. “We had another quarter of solid execution for our two global Phase 3 programs in generalized myasthenia gravis and primary Sjögren’s disease, both of which are progressing as planned,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Encouragingly, clinician feedback this quarter points to a shift in myasthenia gravis treatment toward broader, more durable disease control through upstream B-cell modulation and targeting multiple pathogenic immunoglobulins, which are central to the BAFF/APRIL mechanism. While efficacy benchmarks in myasthenia gravis are well established, many physicians see the potential for this approach moving earlier in the treatment paradigm, given its potential to change the disease trajectory. Our conviction in telitacicept continues to grow, and we believe it has the potential to become a foundational therapy in B-cell driven autoimmune diseases.” Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG Enrollment ongoing in global randomized, double-blind, placebo-controlled Phase 3 registrational trial with an open-label extension assessing the efficacy and safety of telitacicept in gMG Topline data anticipated in 1H 2027 Primary Sjögren’s Disease UPSTREAM SjD Enrollment ongoing in global randomized, double-blind, placebo-controlled Phase 3 registrational trial assessing the efficacy and safety of telitacicept in SjD First Quarter 2026 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $491.5 million as of March 31, 2026, which are projected to fund operations into early 2029. Research & Development (R&D) Expenses: R&D expenses for the first quarter of 2026 were $17.6 million, compared to $26.7 million for the first quarter of 2025. The decrease of $9.1 million was primarily due to reduced spend on our previous programs, trem-cel and VCAR33, a decrease in personnel costs due to a reduction in headcount compared to the prior year period, and a decrease in various other research and development activities. These decreases were partially offset by the increase in spend for our new programs, telitacicept - gMG and telitacicept - SjD. General & Administrative (G&A) Expenses: G&A expenses for the first quarter of 2026 were $17.6 million, compared to $6.6 million for the first quarter of 2025. The increase of $11.0 million was primarily due to increases in stock-based compensation compared to the prior year period. The increase was also attributable to an increase in personnel-related expenses and commercial-related expenses. Net Loss: Net loss for the first quarter of 2026 was $219.6 million, compared to $32.5 million net loss for the first quarter of 2025. The increase of $187.1 million was primarily due to the change in fair value of the outstanding liability-classified warrants in the first quarter of 2026. About TelitaciceptTelitacicept is a novel recombinant fusion protein designed to treat autoimmune diseases through dual inhibition of BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). Additional regulatory filings in China are underway, including biologics license applications for primary Sjögren’s disease (SjD) and IgA nephropathy (IgAN). Vor Bio is advancing global development programs across major autoimmune indications, including a global Phase 3 trial in gMG and SjD, to support potential regulatory approvals in the United States, Europe, and Japan. About Vor BioVor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. 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 telitacicept’s mechanism to change the trajectory of MG; telitacicept’s potential to become a foundational therapy in B-cell driven autoimmune diseases; telitacicept’s potential to become a best-and first-in-class therapy for gMG and SjD; Vor Bio’s projected cash runway; Vor Bio’s development and commercialization plans for telitacicept, including having topline data from the UPSTREAM-MG trial in the first half of 2027; 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 the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. 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. Statements regarding Vor Bio’s cash runway do not indicate when or if Vor Bio may access the capital markets. 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: Media & Investor Contacts: Carl Mauch [email protected]
Investor releaseQuarter not tagged2026-03-30Vor Bio Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Corporate Update
GlobeNewswire
Vor Bio Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Corporate Update
First patient dosed in UPSTREAM SjD, a global Phase 3 clinical trial assessing telitacicept in primary Sjögren’s disease $75 million private placement strengthens balance sheet and supports telitacicept global clinical development Pro-forma cash and investment balance of $530.2 million as of December 31, 2025 expected to provide runway into early 2029 BOSTON, March 30, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a corporate update. “Over the past six months, Vor Bio repositioned the company around telitacicept and moved quickly to build momentum. We are seeing strong engagement from key opinion leaders and principal investigators, which is critical as we advance the global development of telitacicept. In the first quarter, we initiated our global Phase 3 trial in primary Sjogren’s disease and subsequently dosed our first patient within weeks. For myasthenia gravis, we anticipate topline data from the global trial in the first half of 2027 which will prove to be a significant catalyst for both the company and MG landscape,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Importantly, the promising Phase 3 results seen from telitacicept in China across multiple indications, including potential best-in-disease profiles in generalized myasthenia gravis and primary Sjögren’s disease, provide an invaluable foundation as we work to bring a meaningful new treatment option to patients living with these serious autoimmune diseases.” Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG (formerly RemeMG) Enrollment ongoing globally in randomized, double-blind, placebo-controlled Phase 3 registrational trial with an open-label extension assessing the efficacy and safety of telitacicept in gMG Topline data anticipated in 1H 2027 Primary Sjögren’s Disease UPSTREAM SjD Initiated enrollment and dosed first patient in global randomized, double-blind, placebo-controlled Phase 3 registrational trial assessing the efficacy and safety of telitacicept in SjD Corporate Updates Appo…Read full documentShow less
First patient dosed in UPSTREAM SjD, a global Phase 3 clinical trial assessing telitacicept in primary Sjögren’s disease $75 million private placement strengthens balance sheet and supports telitacicept global clinical development Pro-forma cash and investment balance of $530.2 million as of December 31, 2025 expected to provide runway into early 2029 BOSTON, March 30, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a corporate update. “Over the past six months, Vor Bio repositioned the company around telitacicept and moved quickly to build momentum. We are seeing strong engagement from key opinion leaders and principal investigators, which is critical as we advance the global development of telitacicept. In the first quarter, we initiated our global Phase 3 trial in primary Sjogren’s disease and subsequently dosed our first patient within weeks. For myasthenia gravis, we anticipate topline data from the global trial in the first half of 2027 which will prove to be a significant catalyst for both the company and MG landscape,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Importantly, the promising Phase 3 results seen from telitacicept in China across multiple indications, including potential best-in-disease profiles in generalized myasthenia gravis and primary Sjögren’s disease, provide an invaluable foundation as we work to bring a meaningful new treatment option to patients living with these serious autoimmune diseases.” Program Highlights Telitacicept: a potential best- and first-in-class dual BAFF/APRIL inhibitor in development for generalized myasthenia gravis (gMG) and primary Sjögren’s disease (SjD) Generalized Myasthenia Gravis UPSTREAM MG (formerly RemeMG) Enrollment ongoing globally in randomized, double-blind, placebo-controlled Phase 3 registrational trial with an open-label extension assessing the efficacy and safety of telitacicept in gMG Topline data anticipated in 1H 2027 Primary Sjögren’s Disease UPSTREAM SjD Initiated enrollment and dosed first patient in global randomized, double-blind, placebo-controlled Phase 3 registrational trial assessing the efficacy and safety of telitacicept in SjD Corporate Updates Appointed Andrew Levin, M.D., Ph.D., Partner at RA Capital Management, and Wouter Joustra, General Partner at Forbion, to its Board of Directors Announced a $75 million private placement with TCGX on March 27, 2026, to advance the clinical development of telitacicept, including the ongoing global Phase 3 clinical trials for gMG and SjD Fourth Quarter and Full Year 2025 Financial Results Cash Position: Pro-forma cash, cash equivalents and marketable securities were $530.2 million as of December 31, 2025, including the $75.0 million of gross proceeds from the March 2026 private placement, which are projected to fund operations into early 2029. Research & Development (R&D) Expenses: R&D expenses for the fourth quarter of 2025 were $19.2 million, compared to $25.3 million for the fourth quarter of 2024. The decrease of $6.1 million was primarily due to lower stock-based compensation and personnel costs as the Company had lower headcount following the implementation of the Restructuring Plan and reduced spend on its previous programs, trem-cel and VCAR33, partially offset by the increases in spend on telitacicept – gMG and telitacicept – SjD, as the Company began research and development activities for the new programs. R&D expenses for the year ended December 31, 2025 were $321.5 million, compared to $93.3 million for the year ended December 31, 2024. The $228.2 million increase was primarily attributable to the expense incurred in 2025 for the purchase of the telitacicept license and the increased spend for telitacicept – gMG and telitacicept – SjD, partially offset by decreases in personnel costs due the lower headcount following the implementation of the Restructuring Plan, and decreased spend on the Company’s previous programs. General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2025 were $16.8 million, compared to $6.0 million for the fourth quarter of 2024. The increase of $10.8 million was primarily due to increases in stock-based compensation, personnel costs and professional service costs. G&A expenses for the year ended December 31, 2025 were $50.1 million, compared to $27.9 million for the year ended December 31, 2024. The increase of $22.2 million was primarily due to increases in stock-based compensation, personnel costs and professional service costs. Net Income/Loss: Net income for the fourth quarter of 2025 was $1,722.8 million, compared to $30.7 million net loss for the fourth quarter of 2024. The increase of $1,753.5 million was primarily due to the gain on change in fair value of the outstanding liability-classified warrants in the fourth quarter of 2025. Net loss for the year ended December 31, 2025 was $696.0 million, compared to $116.9 million net loss for the year ended December 31, 2024. The $579.1 million increase in loss was primarily due to the loss on change in fair value of the outstanding liability-classified warrants and the purchase of the telitacicept license. About Telitacicept Telitacicept is a novel recombinant fusion protein designed to treat autoimmune diseases through dual inhibition of BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). Additional regulatory filings in China are underway, including biologics license applications for primary Sjögren’s disease (SjD) and IgA nephropathy (IgAN). Vor Bio is advancing global development programs across major autoimmune indications, including a global Phase 3 trial in gMG and SjD to support potential regulatory approvals in the United States, Europe, and Japan. About Vor Bio Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. 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 telitacicept’s potential to have best-and first-in-class profile; Vor Bio’s projected cash runway; Vor Bio’s development and commercialization plans for telitacicept, including having topline data from the UPSTREAM-MG trial in the first half of 2027; Vor Bio’s goal to bring a meaningful new treatment option to patients living with serious autoimmune diseases; 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 the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. 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. The results of the clinical trial described in this press release are based on information reported by RemeGen; Vor Bio has not independently verified this data. Statements regarding Vor Bio’s cash runway do not indicate when or if Vor Bio may access the capital markets. 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: Media & Investor Contacts: Carl Mauch [email protected]
Investor releaseQuarter not tagged2025-11-14Vor Bio Reports Third Quarter 2025 Financial Results and Provides Corporate Update
GlobeNewswire
Vor Bio Reports Third Quarter 2025 Financial Results and Provides Corporate Update
Multiple Late-Stage Data Readouts Reinforce Telitacicept’s Broad Potential Across Autoimmune Diseases Expansion of Executive Leadership and Board Strengthens Global Development Capabilities Expected gross proceeds of $115 million raised in the November 2025 underwritten public offering, including the underwriters’ full exercise of the option to purchase additional shares BOSTON, Nov. 13, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the third quarter ended September 30, 2025, and provided a corporate update. “This has been a pivotal quarter for Vor Bio, as we continue to redefine success in autoimmune disease. Across multiple late-stage programs, telitacicept has now demonstrated consistent results on multiple efficacy endpoints, durable benefit, and a favorable safety profile, a rare combination in our field. We are especially pleased with the recent Phase 3 results in Sjögren’s disease in China where we saw sustained efficacy and a favorable safety profile through 48 weeks, supporting a potential best-in-disease profile,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “With proof of concept in five autoimmune indications and a global Phase 3 clinical trial underway in generalized myasthenia gravis, we are delivering on our vision to make telitacicept the most advanced BAFF/APRIL inhibitor globally and a true pipeline-in-a-product capable of transforming care for patients with serious autoimmune conditions.” Recent Corporate and Clinical Highlights Telitacicept: A Potential Best-in-Class Dual BAFF/APRIL Inhibitor Generalized Myasthenia Gravis (gMG) In October 2025, Vor Bio and its collaborator, RemeGen Co., Ltd., announced new 48-week open-label extension (OLE) data from the Phase 3 study in China evaluating telitacicept in generalized myasthenia gravis (gMG). Results were featured in an oral presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting (AANEM) on October 29, 2025. 96.2% of patients treated with telitacicept for 48 weeks achieved ≥ 3-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living), with a mean reduction of 7.5 points. 94.2% of patients achieved ≥ 5-point improvement in QMG (Quantitative Myasthenia Gravis), with a mean…Read full documentShow less
Multiple Late-Stage Data Readouts Reinforce Telitacicept’s Broad Potential Across Autoimmune Diseases Expansion of Executive Leadership and Board Strengthens Global Development Capabilities Expected gross proceeds of $115 million raised in the November 2025 underwritten public offering, including the underwriters’ full exercise of the option to purchase additional shares BOSTON, Nov. 13, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today reported financial results for the third quarter ended September 30, 2025, and provided a corporate update. “This has been a pivotal quarter for Vor Bio, as we continue to redefine success in autoimmune disease. Across multiple late-stage programs, telitacicept has now demonstrated consistent results on multiple efficacy endpoints, durable benefit, and a favorable safety profile, a rare combination in our field. We are especially pleased with the recent Phase 3 results in Sjögren’s disease in China where we saw sustained efficacy and a favorable safety profile through 48 weeks, supporting a potential best-in-disease profile,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “With proof of concept in five autoimmune indications and a global Phase 3 clinical trial underway in generalized myasthenia gravis, we are delivering on our vision to make telitacicept the most advanced BAFF/APRIL inhibitor globally and a true pipeline-in-a-product capable of transforming care for patients with serious autoimmune conditions.” Recent Corporate and Clinical Highlights Telitacicept: A Potential Best-in-Class Dual BAFF/APRIL Inhibitor Generalized Myasthenia Gravis (gMG) In October 2025, Vor Bio and its collaborator, RemeGen Co., Ltd., announced new 48-week open-label extension (OLE) data from the Phase 3 study in China evaluating telitacicept in generalized myasthenia gravis (gMG). Results were featured in an oral presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting (AANEM) on October 29, 2025. 96.2% of patients treated with telitacicept for 48 weeks achieved ≥ 3-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living), with a mean reduction of 7.5 points. 94.2% of patients achieved ≥ 5-point improvement in QMG (Quantitative Myasthenia Gravis), with a mean reduction of 9.8 at week 48. Safety was favorable and consistent with previous studies across indications; no new safety signals were observed. These results reinforce telitacicept’s potential to set a new standard for durable disease control in gMG. Sjögren’s Disease (SD) In October 2025, Vor Bio’s and its collaborator, RemeGen Co., Ltd, reported positive top-line Phase 3 results from the study of telitacicept in Sjögren’s disease in China. The results were also featured as a late-breaking poster presentation at the 2025 ACR Convergence Meeting on October 28, 2025. Telitacicept met the primary and all secondary endpoints, with placebo adjusted 3.8 points reduction of ESSDAI and 1.52 points reduction in ESSPRI, ~71.8% of patients on 160mg achieving ≥ 3-point reduction in ESSDAI at 24 weeks versus ~19.3% on placebo. The benefit was durable through 48 weeks with a favorable safety profile. These results support a potential best-in-disease profile in Sjögren’s disease. Systemic Lupus Erythematosus (SLE) In October 2025, The New England Journal of Medicine published results from the Phase 3 trial of telitacicept in patients with systemic lupus erythematosus (SLE) in China, reinforcing its potential as a disease-modifying therapy. 67.1% of patients treated with telitacicept achieved a modified SRI-4 response at Week 52, compared with 32.7% on placebo (p < 0.001). Telitacicept also demonstrated improvements across multiple secondary endpoints, including higher rates of SELENA-SLEDAI reduction (70.1% vs. 40.5%), extended time to flare, and greater steroid-dose reductions. The treatment was well tolerated, with a safety profile consistent with previous studies across autoimmune indications. IgA Nephropathy (IgAN) At the American Society of Nephrology (ASN) Kidney Week 2025, Vor Bio and its collaborator, RemeGen Co., Ltd., reported positive Phase 3 results from the study evaluating telitacicept in adults with IgA nephropathy (IgAN) in China. Telitacicept achieved the primary endpoint, showing a 55% reduction in 24-hour urine protein-to-creatinine ratio (24h-UPCR) at 39 weeks compared with placebo (p < 0.0001). Treatment resulted in deep, sustained, and statistically significant reductions in proteinuria with stabilization of kidney function and a favorable safety profile. Across all key secondary endpoints, telitacicept significantly: Preserved kidney function (GMR of eGFR relative to baseline: –1.0% (95% CI, -3.2% to 1.2%) vs –7.7% (95% CI, -9.9% to -5.4%) for placebo, Reduced the proportion of patients with a ≥ 30% decline in eGFR (6.3% vs 27.0%), and 61% versus 19.5% of patients achieved 24h-UPCR <0.8 g/g, 42.1% versus 7.5% of patients achieved <0.5 g/g, and 24.5% versus 0.6% of patients achieved <0.3 g/g, thresholds linked to low risk of disease progression. Adverse events were mostly mild to moderate, and serious adverse events occurred less often with telitacicept than with placebo (2.5% vs 8.2%). No unexpected safety findings were observed. Leadership and Governance Updates Vor Bio significantly expanded its Leadership Team and Board in the third quarter to support late-stage development and commercial readiness: Leadership Team Jeremy Sokolove, M.D. appointed Chief Medical Officer (November 2025) Adi Osovsky, S.J.D. appointed General Counsel (September 2025) Navid Z. Khan, Ph.D. appointed Chief Medical Affairs Officer (September 2025) Dallan Murray appointed Chief Commercial Officer (August 2025) Sandy Mahatme appointed Chief Financial Officer and Chief Business Officer (July 2025) Qing Zuraw, M.D. appointed Chief Development Officer (July 2025) Board of Directors Alexander (Bo) Cumbo, Michel Detheux, Ph.D. and Sarah Reed joined the Board of Directors, bringing deep commercial and biopharma development expertise (July and August 2025) Third Quarter 2025 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $170.5 million as of September 30, 2025, which, together with the proceeds from at-the-market sales during October 2025 and the public offering in November 2025, are projected to fund operations into the second quarter of 2027. Research & Development (R&D) Expenses: R&D expenses for the third quarter of 2025 were $14.1 million, compared to $21.8 million for the third quarter of 2024. The decrease of $7.7 million was primarily due to lower stock-based compensation and personnel costs as the Company had lower headcount following the implementation of the Restructuring Plan and reduced spend on its previous programs, trem-cel and VCAR33, partially offset by the $13.1 million increase in spend on telitacicept - gMG, as the Company began research and development activities for the new program. General & Administrative (G&A) Expenses: G&A expenses for the third quarter of 2025 were $14.0 million, compared to $6.7 million for the third quarter of 2024. The increase of $7.3 million was primarily due to an increase in stock-based compensation expense. Net Loss: Net loss for the third quarter of 2025 was $812.7 million, compared to $27.6 million for the third quarter of 2024. The decrease of $785.1 million was primarily due to the loss on change in fair value of the outstanding liability-classified warrants. About Telitacicept Telitacicept is a novel, investigational recombinant fusion protein designed to treat autoimmune diseases by selectively inhibiting BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia-Pacific to support potential approval in the United States, Europe, and Japan. About Vor Bio Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. 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 telitacicept to be disease-modifying; the potential of telitacicept in Sjögren’s disease to have a best-in-class profile; Vor Bio’s vision to make telitacicept the most advanced BAFF/APRIL inhibitor globally and a true pipeline-in-a-product capable of transforming care for patients with serious autoimmune conditions; Vor Bio’s expected proceeds from the November 2025 public offering; Vor Bio’s projected cash runway; Vor Bio’s development and commercialization plans for telitacicept; 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 the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. 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. The results of the clinical trial described in this press release is based on information reported by RemeGen; Vor Bio has not independently verified this data. 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. Media & Investor Contacts: Carl Mauch [email protected] Sarah Spencer [email protected]
Investor releaseQuarter not tagged2025-10-14Telitacicept Demonstrates Clinically Meaningful and Statistically Significant Impact on ESSDAI Compared to Placebo in Late-Breaking China Phase 3 Results in Primary Sjögren’s Disease at ACR 2025
GlobeNewswire
Telitacicept Demonstrates Clinically Meaningful and Statistically Significant Impact on ESSDAI Compared to Placebo in Late-Breaking China Phase 3 Results in Primary Sjögren’s Disease at ACR 2025
Telitacicept met primary and all secondary endpoints, demonstrating clinically meaningful improvements in disease activity versus placebo ~71.8% of patients receiving telitacicept 160mg achieved ≥3-point ESSDAI (EULAR Sjögren’s Syndrome Disease Activity Index) reduction vs 19.3% on placebo at 24 weeks Sustained efficacy and favorable safety profile through 48 weeks support potential best-in-disease profile in primary Sjögren’s disease (pSD) Company evaluating timing of global Phase 3 clinical study in primary Sjögren's disease Vor Bio to host a conference call on Tuesday, October 28, 2025 at 4:30PM ET BOSTON, Oct. 14, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), reported positive 48-week results from its Phase 3 study conducted in China evaluating telitacicept in primary Sjögren’s disease. The study met its primary endpoint of change from baseline in ESSDAI at week 24, as well as all secondary endpoints, with the telitacicept 160mg dose achieving highly significant p values (p<0.0001) for every endpoint at week 24 and 48 compared to placebo. The results will be presented in the late-breaking poster session at the American College of Rheumatology (ACR) Convergence 2025 on October 28, 2025 from 10:30am to 12:30pm CT in Chicago, Illinois. “With today’s Phase 3 results in primary Sjögren’s disease, we are thrilled to announce that telitacicept is demonstrating disease-modifying potential in a condition that has long lacked any approved treatment. We believe these are clear data which can help pave a path towards a brighter future for this deserving community. The consistency of benefit through 48 weeks, together with a reassuring safety profile, supports telitacicept’s potential to become the first treatment that addresses the root biology of Sjögren’s disease rather than managing symptoms alone,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Based on these promising results, we are evaluating the timing of a global Phase 3 clinical study in primary Sjögren’s disease, which represents a significant opportunity to expand into and bring telitacicept’s benefits to patients worldwide.” “Primary Sjögren’s disease represents a substantial unmet need in rheumatolog…Read full documentShow less
Telitacicept met primary and all secondary endpoints, demonstrating clinically meaningful improvements in disease activity versus placebo ~71.8% of patients receiving telitacicept 160mg achieved ≥3-point ESSDAI (EULAR Sjögren’s Syndrome Disease Activity Index) reduction vs 19.3% on placebo at 24 weeks Sustained efficacy and favorable safety profile through 48 weeks support potential best-in-disease profile in primary Sjögren’s disease (pSD) Company evaluating timing of global Phase 3 clinical study in primary Sjögren's disease Vor Bio to host a conference call on Tuesday, October 28, 2025 at 4:30PM ET BOSTON, Oct. 14, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), reported positive 48-week results from its Phase 3 study conducted in China evaluating telitacicept in primary Sjögren’s disease. The study met its primary endpoint of change from baseline in ESSDAI at week 24, as well as all secondary endpoints, with the telitacicept 160mg dose achieving highly significant p values (p<0.0001) for every endpoint at week 24 and 48 compared to placebo. The results will be presented in the late-breaking poster session at the American College of Rheumatology (ACR) Convergence 2025 on October 28, 2025 from 10:30am to 12:30pm CT in Chicago, Illinois. “With today’s Phase 3 results in primary Sjögren’s disease, we are thrilled to announce that telitacicept is demonstrating disease-modifying potential in a condition that has long lacked any approved treatment. We believe these are clear data which can help pave a path towards a brighter future for this deserving community. The consistency of benefit through 48 weeks, together with a reassuring safety profile, supports telitacicept’s potential to become the first treatment that addresses the root biology of Sjögren’s disease rather than managing symptoms alone,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of Vor Bio. “Based on these promising results, we are evaluating the timing of a global Phase 3 clinical study in primary Sjögren’s disease, which represents a significant opportunity to expand into and bring telitacicept’s benefits to patients worldwide.” “Primary Sjögren’s disease represents a substantial unmet need in rheumatology, with patients facing years of fatigue, pain, and systemic complications without a truly effective therapy,” said Ronald van Vollenhoven, M.D., Ph.D., Professor of Rheumatology at Amsterdam University Medical Center. “I am impressed how these data show that dual BAFF/APRIL inhibition with telitacicept could offer a clear impact on both disease activity and patient-reported outcomes.” The China Phase 3 trial was a randomized, double-blind, placebo-controlled trial in patients with active, anti-SSA-positive primary Sjögren’s disease. A total of 381 patients were randomized to receive weekly subcutaneous injections of telitacicept 160mg, telitacicept 80mg, or placebo for 48 weeks, in addition to standard therapy. During weeks 24-48, participants with inadequate response to treatment in the placebo group could switch to telitacicept 160mg or telitacicept 80mg at a ratio of 1:1 under blind conditions. The primary endpoint of the study was change from baseline in ESSDAI at week 24, with secondary endpoints including changes in ESSDAI and ESSPRI (EULAR Sjögren’s Syndrome Patient Reported Index) at 12, 24, 36, and 48 weeks, as well as the proportion of patients achieving clinically meaningful improvements (≥3-point decrease in ESSDAI and achievement of low disease activity [ESSDAI <5]) at 24 and 48 weeks. Key Findings from the 48-Week Results Mean change in ESSDAI: At week 24, -4.4 (160mg), -3.0 (80mg), and -0.6 (placebo); at week 48, -4.6 (160mg), -3.2 (80mg), and -0.4 (placebo), demonstrating durable, dose-dependent improvement in systemic disease activity. Mean change in ESSPRI: At week 24, -1.88 (160mg), -1.31 (80mg), and -0.36 (placebo); at week 48, -2.56 (160mg), -1.74 (80mg), and -0.41 (placebo), showing sustained symptomatic benefit in dryness, fatigue, and pain. ≥3-point ESSDAI improvement: At week 24, 71.8% (160mg), 47.1% (80mg), and 19.3% (placebo); at week 48, 73.0% (160 mg), 49.1% (80mg), and 16.5% (placebo). Participants with ESSDAI <5 (low disease activity): At week 24, 49.6% (160mg), 28.8% (80mg), and 10.9% (placebo); at week 48, 55.0% (160mg), 32.7% (80mg), and 12.2% (placebo). Participants with ≥1-point or ≥15% ESSPRI reduction: At week 24, 86.2% (160mg), 63.0% (80mg), and 32.2% (placebo); at week 48, 89.1% (160mg), 75.4% (80mg), and 33.3% (placebo). Change from baseline in MFI-20 total (fatigue): At weeks 24 and 48, telitacicept 160mg produced a statistically significant and clinically meaningful reduction in fatigue versus 80mg and placebo, with improvements sustained through the open-label extension. Telitacicept demonstrated a favorable safety profile comparable to placebo and consistent with prior studies across other autoimmune indications, including systemic lupus erythematosus, rheumatoid arthritis, myasthenia gravis, and IgA nephropathy. No new safety signals were observed. Most adverse events were mild to moderate in severity. About Sjögren’s Disease (formerly known as Sjögren’s Syndrome) Sjögren’s disease is a chronic autoimmune condition in which overactive B cells drive inflammation, damaging moisture-producing glands and, in many cases, other organs. Hallmark symptoms include dry eyes and dry mouth, alongside fatigue, pain, and systemic complications affecting the skin, lungs, kidneys, and nervous system. About one-third of patients develop significant extraglandular involvement, and the disease carries an elevated lymphoma risk, often leading to substantial impairment in daily life. One of the most common rheumatic autoimmune diseases, Sjögren’s remains underdiagnosed, with roughly half of cases unrecognized and women comprising the vast majority of patients. Despite its prevalence and burden, no systemic disease-modifying therapies exist; current care focuses on symptom management with incomplete relief. About Telitacicept Telitacicept is a novel, investigational recombinant fusion protein designed to treat autoimmune diseases by selectively inhibiting BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. In a Phase 3 clinical trial in generalized myasthenia gravis in China, telitacicept demonstrated a placebo adjusted 4.83-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living scale) at 24 weeks, the primary endpoint of the trial. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia-Pacific to support potential approval in the United States, Europe, and Japan. About Vor Bio Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. 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 telitacicept in primary Sjögren’s disease, including the potential to have a best-in-class profile, to be disease-modifying, to become the first treatment that addresses the root biology of Sjögren’s disease rather than managing symptoms alone, and offer a clear impact on both disease activity and patient-reported outcomes; our belief that the Phase 3 Primary Sjögren’s data are clear data which can help pave a path towards a brighter future for this deserving community; telitacicept’s market opportunity in primary Sjogren’s disease; the possibility of Vor Bio initiating a global Phase 3 clinical study in primary Sjögren’s disease; the timing of presentation of clinical data; Vor Bio’s development and commercialization plans for telitacicept; 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 the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. 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. The results of the clinical trial described in this press release is based on information reported by RemeGen; Vor Bio has not independently verified this data. 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: Media & Investor Contacts: Carl Mauch [email protected] Sarah Spencer [email protected]
Investor releaseQuarter not tagged2025-03-21Vor Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Company Update
GlobeNewswire
Vor Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Company Update
VCAR33 clinical data update planned for first half of 2025 and trem-cel + Mylotarg clinical data update planned for second half of 2025 Anticipate initiation of the first trem-cel+VCAR33 Treatment System clinical trial in second half of 2025 $55.6 million private placement completed in December 2024 CAMBRIDGE, Mass., March 20, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2024, and provided a business update. “We continue to make significant strides in advancing our novel cell and genome engineering platform. The continued progress in our clinical trials reinforces our confidence in the potential of trem-cel and VCAR33 to transform the treatment landscape,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. “With a strengthened financial position from recent investment by new investor Reid Hoffman and existing investor RA Capital Management, we are well-positioned to drive our mission forward and deliver meaningful impact to patients.” Corporate Updates Trem-cel + Mylotarg (VBP101) Clinical Trial Trem-cel is a shielded transplant in development for patients with AML and MDS, in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to enable targeted therapies such as Mylotarg and CD33-targeted CAR-T therapy post-transplant, while avoiding on-target toxicities. The latest data update from VBP101, the Phase 1/2a clinical trial of trem-cel + Mylotarg, was an encore presentation of data presented at ASH 2024 and took place at the TANDEM Meetings of ASTCT and CIBMTR on February 15, 2025. The data released included 25 patients treated with trem-cel of which 15 had received Mylotarg (six at the 2 mg/m2 dose) as of the data cut-off date of November 1, 2024. The data demonstrated durable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of improved relapse-free survival compared to published high-risk AML comparators1. Patients are now being treated in this study at the recommended Phase 2 dose of Mylotarg at 2 mg/m2. Patients receiving a trem-cel transplant who become measurable residual disease (MRD) positive or relapse have the option to receive Mylotarg or enroll in VBP301 and receive V…Read full documentShow less
VCAR33 clinical data update planned for first half of 2025 and trem-cel + Mylotarg clinical data update planned for second half of 2025 Anticipate initiation of the first trem-cel+VCAR33 Treatment System clinical trial in second half of 2025 $55.6 million private placement completed in December 2024 CAMBRIDGE, Mass., March 20, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2024, and provided a business update. “We continue to make significant strides in advancing our novel cell and genome engineering platform. The continued progress in our clinical trials reinforces our confidence in the potential of trem-cel and VCAR33 to transform the treatment landscape,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. “With a strengthened financial position from recent investment by new investor Reid Hoffman and existing investor RA Capital Management, we are well-positioned to drive our mission forward and deliver meaningful impact to patients.” Corporate Updates Trem-cel + Mylotarg (VBP101) Clinical Trial Trem-cel is a shielded transplant in development for patients with AML and MDS, in which healthy transplant donor cells are genetically engineered by removing CD33, with the potential to enable targeted therapies such as Mylotarg and CD33-targeted CAR-T therapy post-transplant, while avoiding on-target toxicities. The latest data update from VBP101, the Phase 1/2a clinical trial of trem-cel + Mylotarg, was an encore presentation of data presented at ASH 2024 and took place at the TANDEM Meetings of ASTCT and CIBMTR on February 15, 2025. The data released included 25 patients treated with trem-cel of which 15 had received Mylotarg (six at the 2 mg/m2 dose) as of the data cut-off date of November 1, 2024. The data demonstrated durable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of improved relapse-free survival compared to published high-risk AML comparators1. Patients are now being treated in this study at the recommended Phase 2 dose of Mylotarg at 2 mg/m2. Patients receiving a trem-cel transplant who become measurable residual disease (MRD) positive or relapse have the option to receive Mylotarg or enroll in VBP301 and receive VCAR33. The Company expects to report further follow-up data from patients receiving Mylotarg in the the second half of 2025. 1 Araki et al. JCO 2016; Jentzsch et al. Blood Cancer Journal 2022. VCAR33 (VBP301) Clinical Trial VBP301, a Phase 1/2, multicenter, open-label, first-in-human study of VCAR33, 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. Dosing is ongoing in the VBP301 study with continued strong enrollment, and the next clinical data update is planned for first half of 2025. The Company previously 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. Trem-cel+VCAR33 Treatment System The combination of a trem-cel transplant followed by VCAR33 in the maintenance setting has the potential to transform patient outcomes in AML and establish a new standard of care for patients with high risk of relapse. The trem-cel+VCAR33 Treatment System would utilize cells from the same healthy donor for both trem-cel and VCAR33, potentially prolonging persistence and optimizing CAR-T expansion. The Company anticipates initiating a Phase 1 clinical trial with the trem-cel+VCAR33 Treatment System in the second half of 2025. $55.6 Million Financing Completed and New Board Member Appointed In December 2024 the Company entered into a securities purchase agreement for a private investment in public equity financing (PIPE) that resulted in gross proceeds of approximately $55.6 million. The PIPE was led by new investor, Reid Hoffman, and included participation from existing investor and Vor Bio’s largest stockholder, RA Capital Management. In addition, as part of the transaction, life sciences and technology investor and entrepreneur Mr. Erez Kalir was appointed to a newly created seat on Vor Bio’s Board of Directors. Mr. Kalir brings a wealth of experience as an investor, entrepreneur, and thought leader with a deep focus on life sciences and technology. Upcoming Milestones VCAR33 clinical data update expected in the first half of 2025 Trem-cel clinical data update expected in the second half of 2025 Anticipate initiation of a Phase 1 trem-cel+VCAR33 Treatment System clinical trial in the second half of 2025 Fourth Quarter and Full Year 2024 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $91.9 million as of December 31, 2024, which is projected to fund operations into the first quarter of 2026. Research & Development (R&D) Expenses: R&D expenses for the fourth quarter of 2024 were $25.3 million, compared to $20.9 million for the fourth quarter of 2023, and for the year ended December 31, 2024, were $93.3 million, compared to $94.3 million for the year ended December 31, 2023. The quarter over quarter increase in R&D expenses was primarily attributable to an increase in clinical trial costs to support our trem-cel and VCAR33 programs, offset in part by a decrease in preclinical activities. General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2024 were $6.0 million, compared to $7.2 million for the fourth quarter of 2023, and for the year ended December 31, 2024, were $27.9 million, compared to $31.7 million for the year ended December 31, 2023. The quarter over quarter decrease in G&A expenses was primarily attributable to a decline in stock-based compensation and legal and consulting fees. Net Loss: Net loss for the fourth quarter of 2024 was $30.7 million, compared to $26.3 million for the fourth quarter of 2023, and for the year ended December 31, 2024, was $116.9 million, compared to $117.9 million for the year ended December 31, 2023. 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 and dosing in clinical trials and the availability of data therefrom, plans for a registrational trial of trem-cel, 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 & Media Sarah Spencer +1 857-242-6076 [email protected]

