Summary of Vor Bio Conference Call on Telitacicept Phase 3 Results Company and Industry Overview - **Company**: Vor Bio - **Industry**: Biotechnology, specifically focusing on autoimmune diseases and therapies Key Points and Arguments Telitacicept and Clinical Results - Telitacicept is a fusion protein therapy targeting BAF and APRIL, in-licensed from RemeGen, and is the most advanced BAF-APRIL inhibitor globally [4][10] - The Phase 3 study in China for primary Sjögren's disease met all primary and secondary endpoints, indicating potential as a best-in-disease therapy [4][6] - The study demonstrated a statistically significant and clinically meaningful efficacy with a placebo-adjusted ESSDAI reduction of 3.8 and ESSPRI reduction of 1.5 [6][34] - By week 24, patients on telitacicept 160 mg improved by more than 4 points on ESSDAI compared to 1.4 for placebo, a seven-fold difference [35] - Over 50% of patients on telitacicept reached low disease activity (ESSDAI < 5) by week 48, compared to 12% on placebo [38] Safety and Tolerability - The safety profile of telitacicept was consistent with previous trials, showing no new safety signals and comparable event rates to placebo [42][47] - The only background therapy allowed in the trial was hydroxychloroquine, with no DMARDs or steroids, allowing for a clearer assessment of telitacicept's efficacy [34][54] Patient-Reported Outcomes - Patients reported a nearly 2.6 reduction in ESSPRI by week 48, indicating significant improvement in symptoms like fatigue, dryness, and pain [39] - The MFI-20 score showed a 12-point or 21% reduction, indicating a shift from moderate to mild fatigue, enhancing daily functioning [40] Market Opportunity - There are over 800,000 diagnosed Sjögren's patients in key global markets, with no approved therapies currently available, presenting a significant market opportunity for telitacicept [46] - The company aims to position itself as a leader in the Sjögren's market, leveraging the compelling efficacy and safety data presented [48] Future Directions - Vor Bio plans to expand clinical development globally, starting with myasthenia gravis and Sjögren's disease [10][49] - The company is preparing for a global Phase 3 study, learning from the data and experiences of other trials [56] Additional Important Information - Sjögren's disease is a multi-organ systemic autoimmune disease with significant impacts on quality of life, often underdiagnosed and undertreated [12][31] - The dual BAF-APRIL mechanism of telitacicept is designed to modulate B cells and decrease pathogenic autoantibodies, aiming for long-term disease modification [48] - The conference highlighted the importance of understanding the pathophysiology of Sjögren's disease to develop effective treatments [32][31] This summary encapsulates the critical insights from the Vor Bio conference call regarding the Phase 3 results of telitacicept, emphasizing its potential impact on the treatment landscape for Sjögren's disease and the company's strategic direction.

Global Science. One Purpose. 48-week Phase 3 Clinical Trial Data from China for Telitacicept in Primary Sjögren's Disease October 28, 2025 Forward Looking Statement This presentation (the "Presentation") contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. ("Vor," "Vor Bio" or the "Company"). The words "aim," "anticipate," "believe," "can," "could," "design," "enable" "estimate," "expect," "intend," "may," "ongoing," "plan," ...

Core Insights - Vor Bio is hosting a live webcast to present late-breaking 48-week Phase 3 clinical data for telitacicept in primary Sjögren's disease, conducted in collaboration with RemeGen Co., Ltd [1][2] - The webcast will feature Vor Bio management and Dr. Ronald van Vollenhoven, who will discuss key efficacy and safety results from the trial [2] Webcast Details - The webcast is scheduled for Tuesday, October 28, 2025, at 4:30 PM Eastern Time [3] - A replay of the webcast will be available on the investor section of Vor Bio's website approximately two hours after the conclusion and will remain accessible for 30 days [3] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, particularly through the development of telitacicept, a novel dual-target fusion protein [4] - The company aims to advance telitacicept through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions globally [4]

Core Insights - Vor Bio announced clinical data from Stage A of a Phase 3 study in China for telitacicept, targeting IgA nephropathy (IgAN), to be presented at ASN Kidney Week 2025 [1][2] - Telitacicept achieved a primary endpoint with a 55% reduction in 24-hour urine protein-to-creatinine ratio (UPCR) at 39 weeks compared to placebo, indicating significant efficacy [2] - RemeGen has submitted a Biologics License Application (BLA) for telitacicept in IgAN to the NMPA in China, which would mark its fifth approved indication in the country [3] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on advancing telitacicept, a dual-target fusion protein, for treating autoimmune diseases [4] - Telitacicept selectively inhibits BLyS (BAFF) and APRIL, two cytokines essential for B cell survival, thereby reducing autoreactive B cells and autoantibody production [5] - The drug is already approved in China for systemic lupus erythematosus, rheumatoid arthritis, and generalized myasthenia gravis, with ongoing global Phase 3 trials for further approvals [6] Industry Context - IgA nephropathy is a leading cause of chronic kidney disease and end-stage renal disease, with up to 40% of patients progressing to ESRD within 20 years of diagnosis, highlighting the need for effective therapies [7] - Current treatments primarily slow disease progression without addressing the underlying immunopathology, indicating a significant unmet medical need [7] - The overproduction of galactose-deficient IgA1 is recognized as a central driver of IgAN, with BAFF and APRIL promoting its production [8]

Core Insights - Vor Bio announced significant results from a Phase 3 study of telitacicept for systemic lupus erythematosus (SLE), achieving a primary endpoint with 67.1% of patients responding compared to 32.7% with placebo [1][2] - The dual inhibition of BAFF and APRIL is validated as a transformative approach for B-cell targeting, indicating telitacicept's potential as a disease-modifying therapy for SLE [1][2] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, particularly through the development of telitacicept [8] - The company aims to advance telitacicept through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions globally [8] Study Details - The Phase 3 study was conducted at 42 hospitals in China, involving 335 patients with active SLE despite standard therapy [2] - The study met its primary endpoint with a statistically significant improvement in disease activity, as measured by the modified SLE Responder Index-4 (SRI-4) response at week 52 [1][2] Efficacy and Safety Findings - Telitacicept demonstrated improvements across multiple secondary measures, including a 70.1% reduction in SELENA-SLEDAI score compared to 40.5% for placebo [4] - The safety profile of telitacicept was consistent with previous studies, with serious adverse events occurring less frequently compared to placebo [3] Mechanism of Action - Telitacicept is designed to selectively inhibit BAFF and APRIL, two cytokines essential for B cell and plasma cell survival, thereby reducing autoreactive B cells and autoantibody production [6] - This dual-target mechanism is believed to restore immune balance in patients, potentially reducing the burden of lupus [2][6] Regulatory Status - Telitacicept is already approved in China for SLE, rheumatoid arthritis, and generalized myasthenia gravis, with ongoing global Phase 3 trials to support potential approval in the United States, Europe, and Japan [7]

Core Insights - Vor Bio announced positive results from a Phase 3 study of telitacicept in primary Sjögren's disease, achieving significant improvements in disease activity compared to placebo [1][2][4] - The study demonstrated a favorable safety profile and sustained efficacy over 48 weeks, indicating potential for telitacicept to be a leading treatment option in this area [2][4] - The company is considering the timing for a global Phase 3 clinical study to expand the treatment's availability [2] Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, particularly through the development of telitacicept [9] - Telitacicept is designed to inhibit key cytokines involved in B cell survival, addressing the underlying causes of autoimmune conditions rather than just managing symptoms [7][9] - The company has previously achieved approval for telitacicept in China for conditions such as systemic lupus erythematosus and rheumatoid arthritis, and is currently conducting a global Phase 3 trial for generalized myasthenia gravis [8] Study Details - The Phase 3 trial was randomized, double-blind, and placebo-controlled, involving 381 patients with active, anti-SSA-positive primary Sjögren's disease [2][3] - Key endpoints included changes in ESSDAI and ESSPRI at multiple time points, with significant improvements noted at both 24 and 48 weeks [3][4] - At week 24, 71.8% of patients receiving telitacicept 160mg achieved a ≥3-point reduction in ESSDAI compared to 19.3% on placebo, with similar results at week 48 [1][4] Disease Context - Primary Sjögren's disease is a chronic autoimmune condition characterized by inflammation and damage to moisture-producing glands, leading to symptoms such as dry eyes and mouth, fatigue, and systemic complications [5][6] - The disease is underdiagnosed, with a significant unmet need for effective therapies, as current treatments primarily focus on symptom management [6] - Telitacicept's dual-target mechanism may provide a novel approach to treating this condition by reducing autoreactive B cells and autoantibody production [7]

Group 1: Reviva Pharmaceuticals Holdings Inc. (RVPH) - RVPH experienced a significant after-hours rally, increasing 18.27% to $0.58 after a 35.1% surge during regular trading, with a trading volume of over 40.5 million shares, nearly nine times its daily average [2][3] - The company has a market cap of $47 million and is focused on its lead candidate brilaroxazine, which targets neuropsychiatric and respiratory conditions [3] Group 2: Penumbra Inc. (PEN) - PEN surged 7.97% in after-hours trading to $273.69, following a 1.55% gain during the regular session, with a market cap nearing $9.9 billion [4][5] - The company has increased its revenue guidance for FY25 to a range of $1.355 billion - $1.370 billion, indicating a growth of 13% to 15% over 2024 [6] Group 3: Palisade Bio Inc. (PALI) - PALI jumped 10% in after-hours trading to $1.87, extending a 29.8% surge during the regular session, driven by a $138 million public offering [7][8] - The company has a market cap of $15.8 million and is gaining attention due to recent clinical updates and licensing agreements [8] Group 4: IO Biotech Inc. (IOBT) - IOBT rose 5.08% in after-hours trading to $0.4571, following a 22.3% surge during the regular session, with heavy trading volume of 16.7 million shares [9] - The company announced plans to design a new registrational study for its cancer vaccine candidate Cylembio after receiving regulatory feedback from the FDA [10] Group 5: Rallybio Corp. (RLYB) - RLYB increased 11.2% in after-hours trading to $0.5688, reversing a modest dip during the regular session, with a market cap of $21.4 million [11][12] - The company recently completed dosing in its Phase 1 study for RLYB116 and received a $12.5 million milestone payment for progress on the REV102 program [12] Group 6: Vor Biopharma Inc. (VOR) - VOR rose 3.6% in after-hours trading to $38.00, recovering from a 6.6% decline during the regular session, with a market cap of $251 million [13][14] - The company is focusing on its trem-cel platform and CD33-directed therapies for acute myeloid leukemia, alongside new executive appointments [14]

Core Insights - Several biotech and healthcare stocks experienced significant after-hours gains on October 1, driven by technical momentum and recent clinical updates rather than new disclosures [1] Company Summaries - Zhongchao Inc. (ZCMD) saw a 14.04% increase in after-hours trading, rising to $1.30 from $1.14, despite no new filings or press releases [2] - AbCellera Biologics Inc. (ABCL) rose 11.41% in after-hours trading, closing at $6.64, following an 18.49% gain during the regular session, attributed to recent clinical trial progress [3][4] - Vor Biopharma Inc. (VOR) surged 9.53% in after-hours trading, recovering from a 19.42% drop during the regular session, with no new updates since a recent announcement regarding clinical data presentation [5] - Incannex Healthcare Inc. (IXHL) gained 6.48% in after-hours trading, partially recovering from a 6.94% drop, with the latest update showing positive Phase 2 trial results for PSX-001 [6] - Ovid Therapeutics Inc. (OVID) shares rose 8.03% in after-hours trading, building on a 5.38% gain during the regular session, with recent earnings report indicating a narrower net loss and higher revenue [7] - Bullfrog AI Holdings Inc. (BFRG) advanced 3.44% in after-hours trading, following a 3.57% rise during the regular session, after announcing an expansion of its sales and marketing team [8]

Core Insights - Vor Bio is advancing telitacicept, a novel treatment for autoimmune diseases, with a Phase 3 study in China focusing on primary Sjögren's disease [1][3] - The clinical data will be presented at ACR Convergence 2025, highlighting the efficacy and safety of telitacicept [2] - Telitacicept has already received approvals in China for systemic lupus erythematosus, rheumatoid arthritis, and generalized myasthenia gravis [5] Company Overview - Vor Bio is a clinical-stage biotechnology company dedicated to transforming the treatment landscape for autoimmune diseases [3] - The company is focused on the rapid advancement of telitacicept through clinical development and commercialization [3] Product Information - Telitacicept is a dual-target fusion protein that inhibits BLyS and APRIL, which are crucial for B cell survival, thereby reducing autoreactive B cells and autoantibody production [4] - In a Phase 3 trial for generalized myasthenia gravis, telitacicept showed a placebo-adjusted improvement of 4.83 points in the MG-ADL scale at 24 weeks [4] Disease Context - Sjögren's disease is a chronic autoimmune condition characterized by overactive B cells leading to inflammation and damage to moisture-producing glands [6] - The disease is often underdiagnosed, with significant impacts on patients' quality of life and an elevated risk of lymphoma [7]

Core Insights - Vor Bio is advancing telitacicept for the treatment of autoimmune diseases, with a focus on primary Sjögren's disease, and will present clinical data from a Phase 3 study at ACR Convergence 2025 [1][2] Company Overview - Vor Bio is a clinical-stage biotechnology company dedicated to transforming the treatment of autoimmune diseases, particularly through the development of telitacicept [3] - The company aims to rapidly advance telitacicept through Phase 3 clinical development and commercialization to address serious autoantibody-driven conditions globally [3] Product Information - Telitacicept is a novel investigational recombinant fusion protein that selectively inhibits BLyS (BAFF) and APRIL, two cytokines crucial for B cell and plasma cell survival, thereby reducing autoreactive B cells and autoantibody production [4] - The drug has shown a placebo-adjusted improvement of 4.83 points in the MG-ADL scale at 24 weeks in a Phase 3 trial for generalized myasthenia gravis [4] Regulatory Progress - RemeGen, a collaborator of Vor Bio, announced the acceptance of its Biologics License Application (BLA) for telitacicept for primary Sjögren's disease by the National Medical Products Administration (NMPA) in China, marking its fourth approved indication in the country [2][5] Disease Context - Sjögren's disease is a chronic autoimmune condition characterized by overactive B cells that lead to inflammation and damage to moisture-producing glands, with symptoms including dry eyes, dry mouth, fatigue, and systemic complications [6][7] - The disease is underdiagnosed, with approximately half of the cases unrecognized, and predominantly affects women [7]