Anti-CD 19 CAR-T Cell Therapy in Patients with ANCA Vasculitis: a Two-stage Interventional, Prospective, Open-label, Phase I/II Trial in Patients with Active, Treatment Refractory, ANCA-IgG-positive Vasculitis

The goal of this phase I/II clinical trial is to investigate anti-CD 19 chimeric antigen receptor T cell (CAR-T cell) therapy in patients with antineutrophil cytoplasmic antibodies (ANCA) immunoglobulin (IgG) positive ANCA associated vasculitis (AAV).
The main questions it aims to answer are:
* To assess the safety of anti-CD19 CAR-T cell therapy in subjects with active, treatment refractory, ANCA-IgG-positive AAV
* To assess the clinical efficacy of anti-CD19 CAR-T cell therapy in subjects with active, treatment refractory ANCA-IgG-positive AAV
* To assess the ANCA seroconversion rate in subjects with active, treatment refractory ANCA-IgG-positive AAV
Participants will receive a single dose of KYV-101 i.v., an autologous fully-human anti-CD19 CAR-T cell immunotherapy. Follow-up time is 52 weeks with regular visits at the site.

Start Date01 Jan 2025

Sponsor / Collaborator

Kyverna Therapeutics, Inc.Startup

NCT06475495 / Not yet recruitingPhase 1/2IIT

Comparison of B-cell Depletion by Rituximab and Anti-CD 19 CAR-T Therapy in Patients With Rheumatoid Arthritis. Two-stage Interventional, Prospective, Randomized, Controlled, Open Label, Parallel-group Phase I/II Trial in Patients With Active, ACPA-positive and Treatment Refractory Rheumatoid Arthritis

The goal of this phase I/II clinical trial is to compare B-cell depletion by rituximab and anti-CD 19 CAR-T therapy in patients with rheumatoid arthritis. The main questions it aims to answer are:
* To assess the safety of anti-CD19 CAR T cell therapy in subjects with active, ACPA positive and treatment refractory RA (Phase-I)
* To assess the safety of anti-CD19 CAR T cell therapy and of rituximab in subjects with active, ACPA positive and treatment refractory RA (Phase-II)
* To assess ACPA seroconversion after anti-CD19 CAR T cell or rituximab therapy in subjects with active, ACPA positive and treatment refractory RA (Phase-II)
Participants in the test-arm will receive a single dose of KYV-101 i.v., an autologous fully-human anti-CD19 CAR T-cell immunotherapy. In the comparator group patients will receive 2x1 g Rituximab i.v.
Follow-up time (both arms) is 52 weeks with regular visits at the site.

Start Date01 Nov 2024

Sponsor / Collaborator

Kyverna Therapeutics, Inc.Startup

NCT06588491 / RecruitingPhase 2

KYSA-8: A Phase 2 Open-Label, Single-Arm, Multicenter Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Subjects With Treatment Refractory Stiff Person Syndrome

A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects With Treatment Refractory Stiff Person Syndrome

Start Date25 Sep 2024

Sponsor / Collaborator

Kyverna Therapeutics, Inc.Startup

100 Clinical Results associated with KYV-101

100 Translational Medicine associated with KYV-101

100 Patents (Medical) associated with KYV-101

Literatures (Medical) associated with KYV-101

01 Oct 2024·Cytotherapy

Successful generation of fully human, second generation, anti-CD19 CAR T cells for clinical use in patients with diverse autoimmune disorders

Article

Author: Krönke, Gerhard ; Register, Ames ; Walker, Karen ; Schett, Georg ; Lorente, Mario ; Sengupta, Ranjita ; Kröger, Nicolaus ; Pendleton, Michael ; Bullinger, Lars ; Haghikia, Aiden ; Furie, Richard ; Gutman, Jonathan ; Dietrich, Sascha ; Bayer, Ruthee ; Mackensen, Andreas ; Gold, Ralf ; Distler, Jörg H.W. ; Podoll, Amber ; Schroers, Roland ; Mougiakakos, Dimitrios ; Distler, Jörg H W ; Heesen, Christoph

BACKGROUNDB-cell targeting chimeric antigen receptor (CAR) T-cell therapies, which lead to profound B-cell depletion, have been well-established in hematology-oncology. This deep B-cell depletion mechanism has prompted the exploration of their use in B-cell driven autoimmune diseases. We herein report on the manufacturing of KYV-101, a fully human anti-CD19 CAR T-cell therapy, derived from patients who were treated across a spectrum of autoimmune diseases.METHODSKYV-101 was manufactured from peripheral blood-derived mononuclear cells of 20 patients across seven autoimmune disease types (neurological autoimmune diseases, n = 13; rheumatological autoimmune diseases, n = 7). Patients ranged from 18 to 75 years of age. Duration of disease ranged from <1 to 23 years since diagnosis. Patients were heavily pretreated, and most were refractory to prior immunosuppressive treatments. Apheresis was collected across nine sites, cryopreserved, and shipped to the manufacturing facility. Healthy donor apheresis samples were collected for manufacturing comparison. Manufacturing was performed using the CliniMACS Prodigy system. Cells were enriched for CD4⁺/CD8⁺ T cells, transduced with a third generation lentiviral vector encoding the CAR, expanded in vitro, and harvested. Percent cell viability, T-cell purity, cellular expansion, and transduction efficiency were assessed. Activity was assessed using cytokine release assays for KYV-101 CAR T cells co-cultured with different CD19^+/- target cell lines.RESULTSKYV-101 was successfully manufactured for 100% of patients. Transduced cell populations were highly viable, with expansion ranging from 11 to 66 fold at Day 8, and were comparable across disease types. Healthy donor-derived controls displayed similar expansion ranges. High CAR expression and transduction rates were observed, ranging between 37 and 77% with low variation in transgene copy number (two to four per cell). Cell viability of the final KYV-101 drug product ranged from 87 to 97%. KYV-101 displayed robust CD19-dependent and effector dose-related release of the pro-inflammatory cytokine IFN-γ.CONCLUSIONSKYV-101 manufacturing yielded a CAR T-cell product with high viability and consistent composition and functionality, regardless of disease indication, pre-treatment, and heterogeneity of the incoming material. Cryopreservation of the apheresis and final drug product enabled widespread distribution. These results support the robustness of the manufacturing process for the fully human KYV-101 anti-CD19 CAR T-cell therapy drug product for patients across diverse autoimmune disease types.

25 Jun 2024·Proceedings of the National Academy of Sciences

Successful use of anti-CD19 CAR T cells in severe treatment-refractory stiff-person syndrome

Article

Author: Gold, Ralf ; Sgodzai, Melissa ; Motte, Jeremias ; Mougiakakos, Dimitrios ; Faissner, Simon ; Borie, Dominic ; Haghikia, Aiden ; Schroers, Roland ; Geis, Christian

Treatment with autologous chimeric antigen receptor (CAR) T cells has emerged as a highly effective approach in neuroimmunological disorders such as myasthenia gravis. We report a case of successful anti-CD19 CAR T cell use in treatment-refractory stiff-person syndrome (SPS). To investigate clinical and immunological effects of anti-CD19 CAR T cell use in treatment-refractory SPS, a 69-y-old female with a 9-y history of treatment-refractory SPS with deteriorating episodes of stiffness received an infusion of autologous anti-CD19 CAR T cells (KYV-101) and was monitored clinically and immunologically for more than 6 mo. CAR T cell infusion resulted in reduced leg stiffness, drastic improvement in gait, walking speed increase over 100%, and daily walking distance improvement from less than 50 m to over 6 km within 3 mo. GABAergic medication (benzodiazepines) was reduced by 40%. KYV-101 CAR T cells were well tolerated with only low-grade cytokine release syndrome. This report of successful use of anti-CD19 CAR T cells in treatment-refractory SPS supports continued exploration of this approach in SPS and other B cell–related autoimmune disorders.

01 Jun 2024·Med

CD19-targeted chimeric antigen receptor T cell therapy in two patients with multiple sclerosis

Article

Author: Heesen, Christoph ; Badbaran, Anita ; Gagelmann, Nico ; Seibel, Johan ; Fehse, Boris ; Kuhle, Jens ; Friese, Manuel A ; Reinhardt, Stefanie ; Borie, Dominic ; Rathje, Kristin ; Berger, Susanna Carolina ; Richter, Johanna ; Pfeffer, Lena Kristina ; Ayuk, Francis ; Kröger, Nicolaus ; Fischbach, Felix

BACKGROUNDProgressive multiple sclerosis (MS) is characterized by compartmentalized smoldering neuroinflammation caused by the proliferation of immune cells residing in the central nervous system (CNS), including B cells. Although inflammatory activity can be prevented by immunomodulatory therapies during early disease, such therapies typically fail to halt disease progression. CD19 chimeric antigen receptor (CAR)-T cell therapies have revolutionized the field of hematologic malignancies. Although generally considered efficacious, serious adverse events associated with CAR-T cell therapies such as immune effector cell-associated neurotoxicity syndrome (ICANS) have been observed. Successful use of CD19 CAR-T cells in rheumatic diseases like systemic lupus erythematosus and neuroimmunological diseases like myasthenia gravis have recently been observed, suggesting possible application in other autoimmune diseases.METHODSHere, we report the first individual treatment with a fully human CD19 CAR-T cell therapy (KYV-101) in two patients with progressive MS.FINDINGSCD19 CAR-T cell administration resulted in acceptable safety profiles for both patients. No ICANS was observed despite detection of CD19 CAR-T cells in the cerebrospinal fluid. In case 1, intrathecal antibody production in the cerebrospinal fluid decreased notably after CAR-T cell infusion and was sustained through day 64.CONCLUSIONSCD19 CAR-T cell administration in progressive MS resulted in an acceptable safety profile. CAR-T cell presence and expansion were observed in the cerebrospinal fluid without clinical signs of neurotoxicity, which, along with intrathecal antibody reduction, indicates expansion-dependent effects of CAR-T cells on CD19⁺ target cells in the CNS. Larger clinical studies assessing CD19 CAR-T cells in MS are warranted.FUNDINGBoth individual treatments as well the generated data were not based on external funding.

News (Medical) associated with KYV-101

14 Nov 2024

·www.prnewswire.com

Kyverna Therapeutics Announces New Patient Data Highlighting Potential of KYV-101 for Treatment of Lupus Nephritis in Symposium at ACR Convergence 2024

Positive Sustained Efficacy and Durability at >6-month Follow-Up Observed in Patients With Severe Lupus Nephritis (LN) Treated With KYV-101 Target Dose KYV-101 Treatment Continues to Demonstrate Robust Safety and Tolerability With No High-Grade CRS or ICANS Observed EMERYVILLE, Calif., Nov. 14, 2024 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, announces today that it will present updated clinical data from LN patients treated with KYV-101 in ongoing Kyverna-sponsored KYSA-1 and KYSA-3 Phase 1/2 studies and named patient treatments. Kyverna, alongside leading academic collaborators, will highlight all six patients treated with the target dose of 1×108 CD19 CAR T cells, four of which have at least six months of follow-up. All patients at six months of follow-up after treatment at the target dose continue to display sustained efficacy and durability across numerous key clinical measures. These updates will be presented at a company symposium titled, "KYV-101 Anti-CD19 CAR T-Cell Therapy: The Future of Autoimmune Disease Treatment," to be held at 5:45 pm ET on November 18, 2024. Slides from the presentation will be posted to the company website following the symposium. "Lupus nephritis patients encounter a tremendous burden of disease associated with high morbidity and mortality, with up to 30% of patients ultimately experiencing end-stage renal disease that requires dialysis or kidney transplant," said Prof. Georg Schett, M.D. from the Friedrich-Alexander-University in Erlangen, Germany, and one of the presenters. "The data reinforce that treatment with KYV-101 drives deep B cell depletion in patients with LN, and appears to reset the immune system, stabilize eGFR, preserve kidney function and enable clinical improvement in SLE activity. Notably, this clinical benefit is occurring while also eliminating immunosuppressants and reducing glucocorticoids to physiologic levels with a manageable safety profile." "As our KYV-101 clinical datasets mature, we are increasingly able to focus on the right patients, treated at the right dose, with the right protocol. We are excited to share these new data, which continue to reinforce KYV-101's potential for durable and life-changing outcomes in lupus nephritis patients, including those with high chronicity and disease severity," said Warner Biddle, Chief Executive Officer at Kyverna. Additional Updates: Also at ACR Convergence 2024, Kyverna will present data on next-generation approaches, most notably with a poster on Ingenui-T, the Company's preclinical 3-day manufacturing process using autologous whole blood as starting material. Ingenui-T is designed to improve the patient experience by eliminating apheresis, leading to a potential for improved convenience, access and overall cost reduction. As the poster highlights, drug product from the Ingenui-T process manufactured with whole blood from patients with SLE or healthy donors displayed product characteristics similar to KYV-101. In addition, results from collaborative work on the molecular mechanisms underlying immune reset through deep B-cell depletion with CD19 CAR T-cell therapy performed in collaboration with Verily Life Sciences, an Alphabet precision health company, and the University of Erlangen will be shared as an oral presentation. The posters and slides from the oral presentation at ACR Convergence 2024 will be available on the publications page of Kyverna's website. About Lupus Nephritis (LN) Lupus nephritis (LN) is a serious complication of systemic lupus erythematosus (SLE), more commonly known as lupus. Approximately 40 percent of adults diagnosed with lupus eventually develop LN and 60 percent or more of LN patients will fail standard of care and approved treatments1,2. Aside from modest efficacy, current treatments expose these young adults to the well-demonstrated detrimental consequences of chronic treatment with corticosteroids and other powerful immunosuppressants. Up to 30 percent of patients with LN will develop kidney failure, requiring dialysis or a kidney transplant to stay alive3 . About KYV-101 KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health (NIH) to improve tolerability and tested in a 20-patient Phase 1 trial in oncology. Results were published by the NIH in Nature Medicine4. KYV-101 is currently being evaluated in sponsored, open-label, Phase 1/2 and Phase 2 trials in the United States and Germany across two broad areas of autoimmune diseases, rheumatologic and neuroinflammatory, as well as in investigator-initiated trials for multiple indications in multiple geographies. The clinical experience to date with KYV-101 in both oncological and autoimmune diseases highlights the differentiated properties of KYV-101 and the potential to treat autoimmune patients. About Kyverna Therapeutics Kyverna Therapeutics, Inc. (Nasdaq: KYTX) is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases. Our lead CAR T-cell therapy candidate, KYV-101 is advancing through clinical development with sponsored clinical trials across two broad areas of autoimmune disease: rheumatology and neurology, including Phase 2 trials for stiff-person syndrome, multiple sclerosis and myasthenia gravis, a Phase 1/2 trial for systemic sclerosis, and two ongoing multi-center Phase 1/2 trials in the United States and Germany for patients with lupus nephritis. Kyverna's pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats with properties intended to be well suited for use in B cell-driven autoimmune diseases. For more information, please visit . Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements." The words, without limitation, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release include, without limitation, those related to: the potential impact of the clinical outcomes from the ongoing clinical programs; the potential impact of the new data on the treatment efficacy and safety profile of KYV-101; the potential that the results of the ongoing trials could drastically change the treatment landscape for the targeted autoimmune diseases; Kyverna's goals to develop certain paradigm-shifting treatment options; the potential for KYV-101 to provide durable, immunosuppressant-free remission for autoimmune disease patients; Kyverna's beliefs about the differentiated properties of KYV-101; and Kyverna's clinical trials and named-patient activities. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties related to market conditions, and other factors discussed in the "Risk Factors" section of Kyverna's most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q that Kyverna has filed or may subsequently file with the U.S. Securities and Exchange Commission. Any forward-looking statements contained in this press release are based on the current expectations of Kyverna's management team and speak only as of the date hereof, and Kyverna specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Contact: Consort Partners for Kyverna: [email protected] Investors: [email protected] Media: [email protected] 1Anders et al., Nat Rev Dis Primers. 2020; 6:7. 2Parodis et al., Autoimmun Rev. 2024; 23:103418. 3Lateef and Petri, Arthritis Res & Ther. 2012; 14(Suppl 4):S4. 4Brudno et al., Nature Medicine. 2020; 26:270-280. SOURCE Kyverna Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyPhase 1Phase 2ImmunotherapyClinical Result

13 Nov 2024

·www.prnewswire.com

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

13 Nov 2024

·www.prnewswire.com

Kyverna Therapeutics Provides Business Update and Reports Third Quarter 2024 Financial Results

Multiple Key Leadership Appointments, Including Warner Biddle as CEO, Bringing Clinical, Commercial and Strategic Expertise in Cell Therapy Continuing to Advance Broad KYSA Clinical Development Program: Presented Clinical Data Highlighting Potential for KYV-101 in SPS, MG and MS at ECTRIMS and Plan to Share Updated Clinical Data in LN at Company Symposium at ACR Convergence 2024 Strong Financial Position; Ended the Quarter With $321.6 Million in Cash, Cash Equivalents and Marketable Securities EMERYVILLE, Calif., Nov. 13, 2024 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today reported its business highlights and financial results for the third quarter ended September 30, 2024. "With KYV-101 advancing towards later stages of development, we are scaling Kyverna to bring the transformative impact of our differentiated CAR T therapies to patients with a range of B cell-driven autoimmune diseases. Since I joined the company in September, we have made significant progress, both clinically and operationally, to maintain Kyverna's leadership position," said Warner Biddle, Chief Executive Officer of Kyverna. "At ECTRIMS, we shared promising clinical data highlighting KYV-101's potential to reset the immune system and durably improve symptoms of neuroinflammatory diseases. At ACR Convergence 2024 later this week, we will share the latest clinical data from lupus nephritis patients demonstrating the potential for durable treatment effect at the target clinical dose." Mr. Biddle continued, "In order to deliver on the long-term opportunity for KYV-101, we are working to sharpen our focus, leveraging our clinical datasets and academic partnerships, and continue executing our KYSA clinical trial programs. In 2025 we will share our long-term plan in both neuroinflammatory and rheumatologic diseases." Third Quarter 2024 and Recent Business Highlights KYV-101 Clinical Data Updates: KYV-101 Clinical Data in Neuroinflammatory Indications Presented at ECTRIMS: In September 2024, in a company-sponsored symposium at the 40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), Kyverna presented data from 11 patients with stiff-person syndrome, myasthenia gravis and multiple sclerosis reinforcing KYV-101's broad potential in B cell-driven neuroinflammatory diseases. Numerous patients demonstrated ongoing durability at one year and beyond. KYV-101 Clinical Data in Lupus Nephritis to be Presented at the American College of Rheumatology (ACR) Convergence 2024: At a company-sponsored symposium at ACR Convergence 2024, being held in Washington, DC from November 14 - 19, 2024, Kyverna will share clinical data from lupus nephritis patients with at least six months of follow-up after treatment with KYV-101 at its selected target dose. The Company expects to share efficacy, safety and durability data, and highlight KYV-101's impact on clinically relevant pillars of disease: preservation of kidney function, symptom improvement in underlying disease and elimination of immunosuppressive therapy. Also at ACR Convergence 2024, Kyverna will present one oral and two poster presentations outlining its approach to understanding mechanisms of immune reset and detailing Kyverna's next-generation CAR T-cell therapies, including preclinical data on Ingenui-T, the Company's 3-day manufacturing process using autologous whole blood as starting material. KYV-101 Clinical Development Program Updates: As of November 2024, Kyverna initiated Phase 2 dosing with KYV-101, received RMAT designation in two indications and received Orphan Drug Designation in three indications. Stiff-Person Syndrome: Kyverna anticipates initiating dosing by end-of-year in KYSA-8, the Company's Phase 2 study in Stiff-Person Syndrome, for which KYV-101 has been granted an RMAT designation and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). Myasthenia Gravis: Kyverna initiated dosing in KYSA-6, the Company's Phase 2 study in myasthenia gravis, for which KYV-101 has been granted an RMAT designation and Orphan Drug Designation by the U.S. FDA. Additionally, in November 2024, the European Medicines Association (EMA) granted Orphan Drug Designation to KYV-101 for treatment of myasthenia gravis. Multiple Sclerosis: Kyverna's academic partners at Stanford and UCSF initiated dosing in Phase 1 investigator-initiated trials (IITs) for the use of KYV-101 in multiple sclerosis. Kyverna intends to leverage these clinical insights and collaborate with FDA to design a potential registration-enabling study for KYSA-7 in multiple sclerosis. Lupus Nephritis: Kyverna completed the dose-escalation cohort of KYSA-1 and is currently treating patients at the target dose in both KYSA-1 (US) and KYSA-3 (EU), the Company's Phase 1/2 studies in lupus nephritis. Systemic Sclerosis: Kyverna initiated dosing in KYSA-5, the Company's Phase 1/2 study in systemic sclerosis. In September 2024, KYV-101 was granted Orphan Drug Designation from the U.S. FDA for the treatment of systemic sclerosis. Corporate, Operational & Manufacturing Updates Kyverna Leadership Updates: Kyverna strengthened its management team and Board of Directors with key leadership appointments to support the company's evolution in preparation for its next phase of growth, including: Warner Biddle as Chief Executive Officer (CEO) and a member of the Board of Directors, bringing over 30 years of global experience in commercial, product planning and franchise leadership, including successful launches of several CAR-T products. Christi Shaw and Mert Aktar to the Board of Directors, bringing decades of industry leadership in corporate strategy and manufacturing expertise, including for gene and cell therapy. Cara Bauer as Chief Human Resources Officer (CHRO), bringing expertise in human resources strategy, culture building and talent development to support scaling global organizations. In addition, Kyverna announced today that James Chung, M.D., Ph.D., the Company's Chief Medical Officer, will step down from his position to pursue external opportunities, effective November 22, 2024. Kyverna has initiated a search for an external candidate to replace Dr. Chung. Expanded Manufacturing Capacity to Support Advancing Clinical Development: In October 2024, Kyverna signed an agreement with ElevateBio as a second-source supplier for KYV-101. Publication Highlights Manufacturing Capabilities with KYV-101 in Autoimmune Disease: In October 2024, Kyverna and collaborators published "Successful Generation of Fully Human, Second Generation, Anti-CD19 CAR T Cells for Clinical Use in Patients with Diverse Autoimmune Disorders" in Cytotherapy. The publication details Kyverna's 100% manufacturing success rate for KYV-101 in 20 patients, demonstrating a robust and consistent process for manufacturing and delivering its fully human anti-CD19 CAR T cell therapy across various autoimmune indications. Financial Results for the Quarter Ended September 30, 2024 For the quarter ended September 30, 2024, the company reported a net loss of $34.3 million, or a net loss per common share of $0.80, compared to a net loss of $15.5 million, or a net loss per common share of $23.27, for the same period in 2023. During the nine months ended September 30, 2024, net cash used in operating activities was $77.2 million, compared to $33.8 million for the same period in 2023. Kyverna reported $321.6 million in cash, cash equivalents, and available-for-sale marketable securities as of September 30, 2024. About Kyverna Therapeutics Kyverna Therapeutics, Inc. (Nasdaq: KYTX) is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases. Our lead CAR T-cell therapy candidate, KYV-101, is advancing through clinical development across two broad areas of autoimmune disease: rheumatology and neurology, including Phase 2 trials for Stiff-Person Syndrome, myasthenia gravis, and multiple sclerosis, a Phase 1/2 trial for systemic sclerosis, and two ongoing multi-center Phase 1/2 trials in the United States and Germany for patients with lupus nephritis. Kyverna's pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats with properties intended to be well suited for use in B cell-driven autoimmune diseases. For more information, please visit . Forward-looking Statements This press release contains forward looking statements that are based on management's beliefs and assumptions and on information currently available to management of Kyverna Therapeutics, Inc. ("Kyverna", "we", "our," or the "Company"). All statements other than statements of historical facts contained in this press release are forward looking statements. Forward looking statements include, but are not limited to, statements concerning: the Company's future results of operations and financial position, business strategy, drug candidates, planned preclinical studies and clinical trials, results of preclinical studies and named patient activities, ongoing clinical trials, research and development costs, plans for manufacturing, regulatory approvals, timing and likelihood of success, as well as plans and objectives of management for future operations. These forward-looking statements are subject to risks and uncertainties, including the factors described under the Risk Factors section of the Company's Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC on March 26, 2024 and any subsequent Quarterly Reports on Form 10-Q filed by the Company. Actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. When evaluating Kyverna's business and prospects, careful consideration should be given to these risks and uncertainties. These statements speak only as of the date of this press release, and Kyverna undertakes no obligation to update or revise these statements. For more information, please contact: Precision AQ on behalf of Kyverna Therapeutics Investors: [email protected] Media: [email protected] SOURCE Kyverna Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyOrphan DrugPhase 1Phase 2Financial Statement

100 Deals associated with KYV-101

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis	Phase 2	-	Kyverna Therapeutics, Inc.Startup	01 Jan 2025
Rheumatoid Arthritis	Phase 2	-	Kyverna Therapeutics, Inc.Startup	01 Nov 2024
Ischiopatellar Dysplasia	Phase 2	US	Kyverna Therapeutics, Inc.Startup	25 Sep 2024
Stiff-Person Syndrome	Phase 2	US	Kyverna Therapeutics, Inc.Startup	25 Sep 2024
Multiple Sclerosis, Primary Progressive	Phase 2	US	Kyverna Therapeutics, Inc.Startup	01 Sep 2024
Multiple Sclerosis, Secondary Progressive	Phase 2	US	Kyverna Therapeutics, Inc.Startup	01 Sep 2024
Myasthenia Gravis	Phase 2	DE	Kyverna Therapeutics, Inc.Startup	28 Aug 2024
Myasthenia Gravis	Phase 2	US	Kyverna Therapeutics, Inc.Startup	28 Aug 2024
Scleroderma, Diffuse	Phase 2	US	Kyverna Therapeutics, Inc.Startup	06 Aug 2024
Lupus Nephritis	Phase 2	DE	Kyverna Therapeutics, Inc.Startup	01 Dec 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06342960 \| NCT05938725 (KYSA-3, PRNewswire) Manual	Phase 1/2	Lupus Nephritis	6	KYV-101	(wzgrzhcsvh) = No High-Grade CRS or ICANS Observed. hpazdpktdq (wvocvcmawk ) View more	Positive	14 Nov 2024
Company_Website Manual	Not Applicable	Multiple Sclerosis \| Myasthenia Gravis \| Neuroinflammatory Diseases \| Stiff-Person Syndrome	41	KYV-101	(ejefjokork) = In stiff-person syndrome, two patients treated with KYV-101 have demonstrated improved mobility and/or walking speed and reduced autoantibody titers, including one patient at greater than one year post-infusion. A third patient demonstrated reduced autoantibody titers but does not yet have mobility scores available. In myasthenia gravis, three patients treated with KYV-101 have shown reduced pathogenic anti-AChR antibody titers and sustained disease control, including two patients at more than one year post-infusion and all three at greater than 8 months post-infusion. In multiple sclerosis, five patients treated with KYV-101 who failed prior anti-CD20 medications have shown a significant and unprecedented average reduction in oligoclonal bands in the central nervous system (CNS), a potential surrogate biomarker for reduced disease progression. tztasswyao (zamnefitbj ) View more	Positive	18 Sep 2024
PRNewswire Manual	Not Applicable	Stiff-Person Syndrome	1	KYV-101	wgdkzurxih(cnxmywjnzm) = iwwhddrnxy dckwkgbaam (dkengetwoa )	Positive	17 Jun 2024
PRNewswire Manual	Not Applicable	Myasthenia Gravis	1	KYV-101	(bffqvcwamy) = pqyyidgsia voosrmdlzd (wmqsqrmsoa ) View more	Positive	15 Nov 2023

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis