Jade Biosciences (NasdaqCM:JBIO) 2025 Conference November 17, 2025 10:30 AM ET Company ParticipantsAndrew King - Chief Scientific OfficerTom Frohlich - CEOConference Call ParticipantsAkash Tewari - Farm and Biotech AnalystAkash TewariAkash Tewari. I am a Farm and Biotech Analyst here at Jefferies. Day one of our London Healthcare Conference, and I got the pleasure of hosting the Jade Management Team. Why don't I hand it off to you for some intro remarks, and then we'll get started with Q&A?Tom FrohlichAbsol ...

Multiple Late-Stage Data Readouts Reinforce Telitacicept’s Broad Potential Across Autoimmune DiseasesExpansion 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 autoi ...

Summary of Vor Biopharma Conference Call Company Overview - **Company**: Vor Biopharma (NasdaqGS:VOR) - **Event**: First annual INI Summit held on November 12, 2025 Key Industry and Company Insights Mechanism of Action - Vor Biopharma's bispecific compound targets both BAFF and APRIL, enhancing the ability to modulate B cells, plasma cells, and long-lived plasma cells [2][5] - BAFF is a survival factor for early B cell populations, while APRIL supports plasmablast and early plasma cell lineages [2] - The dual blockade of BAFF and APRIL leads to a reduction in autoantibody production and a remodeling of the B cell repertoire towards a less autoreactive population [3][5] Clinical Efficacy - The drug has shown efficacy in reducing IgG, IgA, and IgM levels without significant immunosuppressive effects, with minimal infection signals reported after treating over 60,000 patients in China [4][5] - In a phase three study for Sjogren's syndrome, the drug demonstrated a 4.4 reduction in ESSDAI at the 160 mg dose and a 3-point reduction at the 80 mg dose, with a 75% responder rate compared to 21% in placebo [9][10] Market Potential - The myasthenia gravis (MG) market is projected to grow from $4 billion today to $10 billion by 2030, with Vor Biopharma aiming for a leading position [24] - The Sjogren's market is estimated to be between $8 billion to $12 billion, with a significant portion of patients being moderate to severe [28] Competitive Landscape - The company is positioned to follow Novartis in the Sjogren's market, leveraging their data and market awareness efforts [25][26] - Vor Biopharma's mechanism of action is distinct from competitors, which may provide a competitive edge in the MG market [24] Financial Position - Vor Biopharma raised approximately $315 million, providing a cash runway until mid-2027, sufficient to complete the phase three trial for MG and support commercial launch activities [33] Additional Important Points - The company is focused on maintaining a clean study population to control placebo effects in global trials [22] - The potential for a plateau in treatment efficacy is acknowledged, with some patients achieving low disease activity levels [16] - Vor Biopharma is currently not prioritizing additional indications beyond MG and Sjogren's, focusing on capital allocation and resource management [32]

Group 1 - Vor Biopharma Inc. has priced an underwritten public offering of 10 million shares at $10.00 per share, expecting gross proceeds of $100 million [1] - The company has granted underwriters a 30-day option to purchase up to an additional 1.5 million shares [1] Group 2 - Vor Bio's collaborator, RemeGen Co., Ltd, reported positive results for telitacicept in treating autoimmune disorders, achieving statistically significant benefits across all secondary endpoints [4] - In a Phase 3 clinical study in China, telitacicept achieved the primary endpoint of reducing proteinuria, with a significant reduction in 24-hour urine protein-to-creatinine ratio compared to placebo (-58.9% vs. -8.8%) [8] - The study enrolled 318 adult patients and demonstrated that telitacicept stabilized kidney function, with a decline in the placebo group compared to a stabilization in the telitacicept group [5][8] Group 3 - Telitacicept showed a favorable safety profile, with treatment-emergent adverse events occurring more frequently but mostly being mild or moderate; serious adverse events were less common with telitacicept than with placebo [7] - The drug demonstrated significant efficacy, with 61% of patients on telitacicept achieving a 24h-UPCR <0.8 g/g compared to 19.5% on placebo [6]

Company Overview - Vor Biopharma Inc. is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [5] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development [5] Offering Details - Vor Biopharma announced a public offering of 10,000,000 shares of its common stock at a price of $10.00 per share, expecting gross proceeds of $100 million [1] - The offering is set to close on or about November 12, 2025, subject to customary closing conditions [1] - Underwriters have a 30-day option to purchase an additional 1,500,000 shares at the public offering price [1] Underwriters - J.P. Morgan, Jefferies, Citigroup, and TD Cowen are acting as joint book-running managers for the offering [2] Regulatory Information - The shares are being offered pursuant to a shelf registration statement filed with the SEC, which was declared effective on March 31, 2025 [3] - A preliminary prospectus supplement and accompanying prospectus have been filed with the SEC and are available on their website [3]

Core Viewpoint - Vor Biopharma Inc. has announced a public offering of 10,000,000 shares at a price of $10.00 per share, aiming to raise gross proceeds of $100 million before expenses [1] Group 1: Offering Details - The offering is expected to close on or about November 12, 2025, subject to customary closing conditions [1] - Vor Biopharma has granted underwriters a 30-day option to purchase an additional 1,500,000 shares at the public offering price [1] - The shares are being offered under a shelf registration statement declared effective by the SEC on March 31, 2025 [3] Group 2: Company Overview - Vor Biopharma is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [5] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development [5]

Core Points - Vor Biopharma Inc. has initiated an underwritten public offering of $100 million in common stock, with an additional option for underwriters to purchase up to $15 million more [1] - The offering is subject to market conditions and there is no guarantee on its completion or terms [1] - The shares are being offered under a shelf registration statement effective since March 31, 2025, and a preliminary prospectus will be filed with the SEC [3] Company Overview - Vor Biopharma is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases [5] - The company is advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development for serious autoantibody-driven conditions [5]

Core Viewpoint - Vor Biopharma Inc. has initiated a public offering of $100 million in common stock, with an option for underwriters to purchase an additional $15 million, aimed at advancing its clinical-stage biotechnology efforts in treating autoimmune diseases [1][2]. Company Overview - Vor Biopharma is a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, particularly through its lead product, telitacicept, which is in Phase 3 clinical development [5]. Offering Details - The public offering consists of $100 million in common stock, with a 30-day option for underwriters to acquire an additional $15 million [1]. - The offering is subject to market conditions and other factors, with no guarantee on completion or terms [1]. - The shares are being offered under a shelf registration statement effective since March 31, 2025, and will be made available through a prospectus supplement [3].

Core Insights - Telitacicept achieved a 55% reduction in 24-hour urine protein-to-creatinine ratio (24h-UPCR) at 39 weeks compared to placebo, demonstrating significant clinical benefits in treating IgA nephropathy (IgAN) [1][3] - The Phase 3 clinical study in China met its primary endpoint and showed statistically significant improvements across all key secondary endpoints, indicating telitacicept's potential as a foundational therapy for B-cell mediated diseases [2][4] Group 1: Clinical Study Results - The Phase 3 study was a multicenter, randomized, double-blind, placebo-controlled trial involving 318 adult patients with IgAN at high risk of progression [2][5] - Telitacicept demonstrated a significant reduction in 24h-UPCR (-58.9% vs. -8.8%, p<0.0001) and stabilized kidney function, with a change in estimated glomerular filtration rate (eGFR) showing a decline in the placebo group (-0.77) compared to stabilization in the telitacicept group (-0.10) [3][5] - 61% of patients on telitacicept achieved 24h-UPCR <0.8 g/g compared to 19.5% on placebo, indicating a lower risk of disease progression [5] Group 2: Safety Profile - Telitacicept exhibited a favorable safety profile, with treatment-emergent adverse events occurring in 89.3% of patients compared to 78.6% in the placebo group; however, serious adverse events were less frequent with telitacicept (2.5% vs. 8.2%) [6] Group 3: Regulatory and Market Potential - RemeGen has submitted a Biologics License Application (BLA) to the National Medical Products Administration (NMPA) in China for telitacicept in treating IgAN, which could mark its fifth approved indication in the country [7][10] - Vor Bio is focused on advancing telitacicept through Phase 3 clinical development to address serious autoantibody-driven conditions globally [8][9]

Core Insights - Telitacicept has shown significant improvement in quality of life for patients with generalized myasthenia gravis (gMG), with 100% of patients achieving a ≥2-point improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) after 48 weeks [1][2] - The drug demonstrated sustained efficacy and a favorable safety profile, suggesting it may have a global best-in-disease profile for gMG [1][2] Study Results - In the 48-week open-label extension (OLE) study, patients on telitacicept achieved a mean reduction of -7.5 points in MG-ADL, while those who crossed over from placebo achieved a -6.3 point reduction [6] - At week 48, 96.2% of continuous telitacicept patients and 90.2% of crossover patients reached a ≥3-point improvement in MG-ADL [6] - The mean change in Quantitative Myasthenia Gravis (QMG) was -9.8 for continuous patients and -9.3 for crossover patients, with 94.2% and 90.2% achieving a ≥5-point improvement, respectively [6] Mechanism of Action - Telitacicept is a 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 [5] Regulatory Status - Telitacicept is already approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG) [7] - A global Phase 3 clinical trial for gMG is currently ongoing across 14 countries, aiming for potential approval in the United States, Europe, and Japan [7] Disease Context - Generalized myasthenia gravis (gMG) is a rare autoimmune neuromuscular disorder affecting approximately 90,000 people in the United States, 140,000 in Europe, and 29,000 in Japan, characterized by muscle weakness due to autoantibodies [8]