– Complete B cell depletion, rapid reduction in autoantibodies and near-complete resolution of clinical symptoms in two of three refractory patients; all three patients remained off immunomodulators since infusion and are off or tapering steroids as of the data cut-off – – CAR T cell expansion in all three patients without preconditioning was similar to expansion across 30+ patients dosed with preconditioning in the other RESET™ trials – – Initial dose data support continued exploration of rese-cel without ...

Core Viewpoint - Adicet Bio Inc. has experienced a decline in share price following the announcement of an $80 million direct offering, despite positive early safety and efficacy data from its Phase 1 trial of ADI-001 for autoimmune diseases [1][7]. Financial Offering - The company has priced a registered direct offering of 70 million shares and pre-funded warrants for 10 million shares at $1.00 per share and $0.9999 per warrant, aiming for gross proceeds of approximately $80 million [1]. Clinical Trial Results - Initial safety and efficacy data from the first seven patients treated with ADI-001 showed promising results, with 100% of patients in the lupus nephritis cohort achieving a renal response, including three complete responses [3][4]. - All patients experienced rapid and sustained reductions in SLEDAI-2K and PGA scores, indicating the potential for a durable effect on a wide range of lupus symptoms [4]. Safety Profile - ADI-001 was generally well-tolerated, with no serious adverse events reported among the seven patients, suggesting a favorable safety profile that may allow for outpatient dosing [5]. Future Development Plans - The company plans to request a meeting with the FDA in Q1 2026 to discuss the design of a potentially pivotal Phase 2 trial, which is expected to start in Q2 2026 [6].

JADE201 builds on clinical proof-of-concept for BAFF-R targeting, adding half-life extension technology to provide extended receptor occupancy with the goal of delivering deeper, more durable B cell depletion with less frequent subcutaneous dosing JADE201’s high affinity binding and extended half-life enabled dose-dependent BAFF receptor occupancy and sustained B cell depletion in non-human primatesFirst-in-human trial in rheumatoid arthritis expected to begin in the first half of 2026; potential for broad ...

BOSTON, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that clinical data from the Phase 3 study in China evaluating telitacicept in adults with primary Sjögren’s disease, a study sponsored by Vor’s collaborator RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), will be presented as a late-breaking poster presentation at ACR Convergence 2025, being held October 24-29, 2025, at McCormick Place in Chi ...

BOSTON, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that clinical data from the Phase 3 study in China evaluating telitacicept in adults with primary Sjögren’s disease, a study sponsored by Vor’s collaborator RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), will be presented as a late-breaking poster presentation at ACR Convergence 2025, being held October 24-29, 2025, at McCormick Place in Chi ...

Dr. Khan brings decades of global medical affairs, commercial, and scientific leadership across multiple therapeutic areas including seven successful product launchesCAMBRIDGE, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company dedicated to transforming the treatment of autoimmune diseases, today announced the appointment of Navid Z. Khan, Ph.D., as Chief Medical Affairs Officer. Dr. Khan joins Vor Bio with over two decades of experience spanning medical ...

Core Viewpoint - RemeGen's telitacicept has shown promising results in treating generalized myasthenia gravis (gMG), with data from a 48-week open-label extension of a Phase III clinical study to be presented at the upcoming AANEM Annual Meeting, indicating its potential as a best-in-class therapy in this field [1][3]. Group 1: Clinical Study Results - The 24-week data from the Phase III study of telitacicept revealed that 98.1% of patients experienced a 3-point improvement in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score, while 87% achieved a 5-point improvement in the Quantitative Myasthenia Gravis (QMG) score [3]. - The complete 48-week data presentation is expected to further emphasize telitacicept's efficacy and safety in treating gMG [3]. Group 2: Licensing and Development - In June 2025, RemeGen out-licensed telitacicept to Vor Bio, which is currently advancing a global multicenter Phase III clinical trial for gMG, with patient recruitment ongoing across multiple regions including the US, Europe, South America, and Asia-Pacific [4]. Group 3: Product Information - Telitacicept is the first-in-class injectable recombinant dual-target fusion protein that inhibits the binding of BLyS and APRIL cytokines to B cell receptors, addressing autoimmune diseases [5]. - The drug has already received approval in China for treating systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG) [5]. Group 4: Market Context - Generalized myasthenia gravis is a rare autoimmune disorder affecting approximately 90,000 patients in the US, 140,000 in Europe, and 29,000 in Japan, highlighting a significant unmet clinical need for effective therapies [7].

Company Overview - Vor Bio is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [1][2] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and commercialization [2][3] Clinical Study Details - Vor Bio announced 48-week clinical data from a Phase 3 study in China evaluating telitacicept in adults with generalized myasthenia gravis [1] - The study demonstrated a placebo-adjusted 4.83-point improvement in the Myasthenia Gravis Activities of Daily Living scale (MG-ADL) at 24 weeks, which is the primary endpoint of the trial [3] Product Information - Telitacicept selectively inhibits BLyS (BAFF) and APRIL, two cytokines essential for B cell and plasma cell survival, thereby reducing autoreactive B cells and autoantibody production [3] - Telitacicept is already approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG) [4] Market Context - Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder affecting approximately 90,000 people in the United States, 140,000 in Europe, and 29,000 in Japan [5] - There is a significant unmet need for new therapies that provide durable efficacy and a favorable safety profile for gMG patients [5] Upcoming Presentation - The clinical data will be presented as an oral presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting on October 29, 2025 [1][2]

Core Insights - Roivant and Priovant Therapeutics announced positive results from the Phase 3 VALOR study for brepocitinib in treating dermatomyositis (DM) [1][2] Study Results - Brepocitinib 30 mg achieved a week 52 mean Total Improvement Score (TIS) of 46.5 compared to 31.2 for placebo, with a statistically significant p-value of 0.0006 [2][5] - This study marks the first positive outcome for a 52-week placebo-controlled trial in DM and the first positive registrational trial for a targeted therapy in DM [2][3] - Brepocitinib demonstrated clinically meaningful and statistically significant improvements across all nine key secondary endpoints [3][5] Patient Outcomes - Approximately 75% of patients entered the study on background steroids, with a mean baseline dose of 12.2 mg/day for the brepocitinib group and 11.3 mg/day for placebo [4] - 62% of brepocitinib 30 mg patients achieved a steroid dose of ≤2.5 mg/day by the end of the study, compared to 34% for placebo [4] - More than two-thirds of brepocitinib 30 mg patients experienced at least a moderate response (TIS≥40), and nearly half experienced a major response (TIS≥60) [5][6] Safety Profile - The safety profile of brepocitinib 30 mg was consistent with previous clinical trials, with no increased frequency of adverse events of special interest compared to placebo [5][7] - The median time to a TIS≥40 response was approximately 8 weeks, indicating a rapid onset of clinical improvement [6] Future Plans - An NDA filing for brepocitinib in dermatomyositis is planned for the first half of 2026 [5][8] - Roivant will host an investor call to discuss these updates on September 17, 2025 [11] Background Information - Dermatomyositis is a debilitating autoimmune disease affecting approximately 50,000 adults in the U.S., characterized by muscle weakness and skin lesions [9] - The VALOR study is noted as the longest and largest interventional DM study ever conducted, enrolling 241 subjects globally [10]

Core Insights - Jeito Capital has made a significant investment in Odyssey Therapeutics, participating in an oversubscribed $213 million Series D financing round aimed at advancing treatments for autoimmune diseases with high unmet needs [1][2]. Company Overview - Odyssey Therapeutics, founded in 2021, is a clinical-stage biopharmaceutical company focused on transforming the standard of care for autoimmune and inflammatory diseases through targeted medicines [3][11]. - The company has developed comprehensive drug discovery and development capabilities in both the United States and Europe, achieving multiple clinical milestones in a short time frame [3][11]. Investment Details - Jeito Capital is the largest European contributor to the Series D financing, joining a group of new investors including Affinity Asset Advisors, Dimension Capital, Lightspeed Ventures, TPG Life Sciences Innovations, and Wedbush Healthcare Partners [2]. - The funds raised will be utilized to advance Odyssey's clinical and preclinical programs, particularly focusing on small-molecule therapies for complex autoimmune diseases [5][8]. Product Pipeline - Odyssey's lead compound, OD-07656, is an oral small-molecule RIPK2 scaffolding inhibitor targeting Inflammatory Bowel Disease (IBD), including ulcerative colitis and Crohn's disease, which affect millions globally [4]. - The second program involves an oral small molecule IRAK4 scaffolding inhibitor in preclinical development, aimed at treating various inflammatory diseases such as atopic dermatitis and osteoarthritis [5]. Strategic Importance - Jeito Capital's investment reflects its expertise in immunology and inflammation, building on previous successful investments in the sector, such as HI-BioTM, which was acquired by Biogen for up to $1.8 billion [6]. - The investment is expected to enhance Odyssey's capabilities in developing differentiated therapies that address significant treatment gaps in the autoimmune disease space [6][7].