Mr. Mahatme brings decades of experience in strategic finance, capital formation, and operational leadership to support company’s transformation and growth in autoimmune disease CAMBRIDGE, Mass., July 10, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced the appointment of Sandy Mahatme as Chief Financial Officer and Chief Business Officer, effective July 9, 2025. Mr. Mahatme joins Vor Bio with more than ...

Core Insights - Vor Bio has secured exclusive global rights (excluding Greater China) to develop and commercialize telitacicept, a dual-target recombinant fusion protein for autoimmune diseases [1][6] - RemeGen has received an initial payment of $125 million, which includes a $45 million upfront payment and $80 million in warrants, along with potential milestones exceeding $4 billion and tiered royalties [1][5] - Jean-Paul Kress, MD, has been appointed as the new CEO and Chairman of Vor Bio, bringing extensive experience in clinical development and commercialization [3][4] Company Developments - Vor Bio is focused on advancing telitacicept through Phase 3 clinical development to address serious autoantibody-driven conditions globally [7] - RemeGen is conducting a global Phase 3 clinical trial for telitacicept, with initial results expected in the first half of 2027 [2][6] - The strategic out-licensing of telitacicept's ex-China rights is aimed at maximizing its clinical and commercial potential on a global scale [5] Product Information - Telitacicept targets key immune pathways by inhibiting BlyS (BAFF) and APRIL, which are critical for B cell survival, thereby reducing autoreactive B cells and autoantibody production [2][5] - In a Phase 3 trial in China for generalized myasthenia gravis, telitacicept showed a 4.8-point improvement in the MG-ADL scale compared to placebo at 24 weeks [5]

Vera Therapeutics (VERA) FY 2025 Conference June 11, 2025 03:15 PM ET Speaker0 Okay. Good afternoon. We'll continue with the next session. I'm Paul Choi, and I cover the SpidCap biotech sector here at Goldman Sachs. It's my pleasure to have Vera Therapeutics here on stage with us. To my immediate left is CEO, Marshall Fortis, and to my far left, Chief Medical Officer Rob Brenner. Maybe what we'll do is let Marshall kick it off with maybe some high level comments or an introduction of Avera, just kind of wha ...

ALKIVIA data demonstrate significant improvement in muscle strength and physical function in myositis patients treated with efgartigimodRHO data show efgartigimod achieved sustained reduction in autoantibodies and improved functional outcomes in patients with Sjogren’s disease; program granted U.S. FDA Fast Track designationargenx committed to new therapeutic areas in rheumatology with ongoing Phase 3 studies in myositis (ALKIVIA) and Sjogren’s disease (UNITY) June 11, 2025, 12:01 AM CET Amsterdam, the Net ...

Summary of Key Points from the Conference Call Company Overview - CRISPR Therapeutics has been operational for approximately eleven years, with a focus on developing gene editing therapies, particularly in the areas of cardiovascular medicine and autoimmune diseases [1][2] Core Products and Pipeline - **KASJEVY**: Approved for sickle cell disease and thalassemia, currently ramping up commercial uptake with over 65 authorized treatment centers activated globally [2][8] - **Cardiovascular Medicine**: Focus on gene editing therapies targeting ANGPTL3, showing nearly 80% reduction in LDL and triglycerides from a single injection [2][3][20] - **Autoimmune Diseases**: Development of allogeneic CAR T therapies, with plans to expand indications beyond lupus [3][4] Key Data and Results - **ANGPTL3 Targeting**: Initial data indicates a significant reduction in triglycerides and LDL, outperforming expectations and existing therapies [20][21] - **Gentler Preconditioning Regimens**: Development of gentler conditioning methods could expand the addressable market for KASJEVY by 3-4 times [15][17] Market Dynamics - The launch of KASJEVY is compared to medical devices rather than traditional pharmaceuticals, indicating a unique commercialization strategy [10][11] - The cardiovascular space is evolving with multiple modalities, including siRNA and gene editing, with a focus on long-term patient outcomes and compliance [30][35] Competitive Landscape - The company believes that gene editing will provide a superior long-term solution compared to ongoing treatments like siRNA, which require continuous administration [34][36] - The potential for significant cost savings and improved patient compliance with a one-time gene editing therapy versus ongoing treatments [35][36] Future Expectations - Upcoming data releases are anticipated to further validate the efficacy of ANGPTL3 and Lp targeting therapies, with a focus on biomarker-based approvals rather than traditional outcome studies [24][37] - The company is exploring strategic partnerships, particularly in the cardiovascular and autoimmune spaces, as interest in cell and gene therapies increases [52][53] Additional Insights - The company is also working on regenerative medicine for type one diabetes, with ongoing trials for both encapsulated and unencapsulated islet cells [51] - The allogeneic CAR T platform is being optimized, with promising data expected mid-year [45][48] This summary encapsulates the critical aspects of CRISPR Therapeutics' current status, product pipeline, market positioning, and future outlook based on the conference call.

Artiva Biotherapeutics (ARTV) 2025 Conference June 04, 2025 09:20 AM ET Speaker0 Good morning, everyone. Thank you for attending Jefferies Healthcare Conference. My name is Kelly Xu, one of the equity analysts here on the biotech team. We are very pleased to have Doctor. Fred Aslan, CEO of Ativa Biotherapeutics join us today. Welcome, Fred. Speaker1 Thank you, Kelly. Speaker0 And maybe let's first start with a big picture question. Many cell therapies in the autoimmune space originated from oncology and Ati ...

Core Viewpoint - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of VYVGART® (efgartigimod alfa) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adult patients, marking a significant advancement in treatment options for this rare autoimmune disease [1][4]. Company Overview - argenx SE is a global immunology company focused on developing innovative treatments for severe autoimmune diseases, aiming to address significant unmet medical needs [2][9]. - The company has developed VYVGART, the first targeted IgG Fc-antibody fragment for CIDP, which, if approved, would be the first novel treatment for CIDP in Europe in over 30 years [2][5]. Clinical Trial Insights - The CHMP recommendation is based on positive results from the ADHERE clinical trial, which is the largest study of CIDP patients to date, involving 322 participants [3][5]. - In the ADHERE trial, 66.5% of patients treated with VYVGART showed clinical improvement, with a primary endpoint met demonstrating a 61% reduction in the risk of relapse compared to placebo [3][5]. - The trial also indicated significant functional improvements in various clinical assessment tools, with 99% of participants opting to continue in the open-label extension of the study [3][5]. Market Implications - The CHMP's positive opinion serves as a scientific recommendation for marketing authorization, with the European Commission expected to make a decision within approximately two months [4][5]. - If approved, VYVGART will be available for subcutaneous injection, providing a new treatment option for CIDP patients across all 27 EU member states, as well as Iceland, Norway, and Liechtenstein [4][5]. Disease Context - CIDP is a rare autoimmune disease affecting the peripheral nervous system, leading to symptoms such as fatigue, muscle weakness, and loss of sensation, which can significantly impair daily functioning [7]. - There are an estimated 31,413 individuals living with CIDP in the European Union, highlighting the need for effective treatment options [7].