VOR

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...

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...

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...

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...

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...