Lupus Vulgaris

Dive Brief:Royalty Pharma will pay biotechnology startup ImmuNext $525 million for the right to earn royalties and milestone payments from an autoimmune disease drug the privately held company licensed to Sanofi.Sanofi acquired the medicine from ImmuNext in 2017 and promised milestone payments worth as much as $500 million plus tiered royalties on potential sales. The French drugmaker moved the drug, known as frexalimab, into a Phase 3 trial after promising results in a mid-stage study of patients with relapsing multiple sclerosis released last year.As part of the deal with ImmuNext, Royalty will be in line for substantial milestone payments from Sanofi as well as all royalties on up to $2 billion of global sales for the drug. A minority of royalties on sales surpassing that threshold will be shared with ImmuNext shareholders. The tiered royalties range from high single- to low double-digits, Royalty said Thursday.Dive Insight:With frexalimab in its portfolio, Royalty will have 15 experimental therapies with the potential to generate combined peak annual royalties significantly greater than $1 billion, the company said. Approved products will add to the cash flow coming from currently marketed drugs including GSKs asthma treatment Trelegy and Roches spinal muscular atrophy medicine Evyrsdi.In 2017, ImmuNext and Sanofi envisioned frexalimab, then called INX-021, as a treatment for a range of diseases. Since then, Sanofi has seen both successes and failures. Last month, the company said it stopped development of the drug for Sjgrens Syndrome after disappointing study results. Research for two forms of MS, Type 1 diabetes and lupus continue, Sanofi said.The MS indication is the furthest along. In February, the mid-stage study results were published in The New England Journal of Medicine. Sanofi expects to file for approval of the medicine in 2027. In December, Sanofi told investors the medicine could generate annual sales of more than 5 billion euros, or about $5.4 billion. Royalty said it expects frexalimab to generate royalties through 2041.Royalty has been steadily building its portfolio through a variety of deals. In October, it scooped up a greater share of royalties on Evrysdi. Before that, Royalty ventured into gene therapy, agreeing to pay $300 million for a 5% royalty on a Ferring Pharmaceuticals cancer treatment. In March 2023, it bought a portion of royalty rights to Karuna Therapeutics experimental schizophrenia drug. '

Century Therapeutics, Inc. (NASDAQ: IPSC), an innovative clinical-stage biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immune-oncology, and FUJIFILM Cellular Dynamics, Inc., a leading global developer and manufacturer of human iPSCs and iPSC-derived cells, today announce a new worldwide license agreement and expansion of existing licenses for the development and commercialization of cell therapies derived from iPSCs for the treatment of autoimmune and inflammatory diseases, including type 1 diabetes, multiple sclerosis, lupus, and rheumatoid arthritis. There are up to 100 autoimmune conditions which collectively impact more than 24 million Americans,1 with studies indicating that both incidence and prevalence of autoimmune disease are rising for reasons that are poorly understood2. Treatment of autoimmune disease is currently limited to symptom management, and includes correcting resulting deficiencies in the body, along with reducing inflammation and the immune response. iPSCs have the capability to self-renew and differentiate into key cell types, providing regenerative and immune-modulating properties as a treatment modality. The new licenses expand on the companies’ existing collaboration initiated in 2018 for iPSC-derived cancer immunotherapeutics. Under the terms of the agreements, FUJIFILM Cellular Dynamics will be eligible to receive certain development and regulatory milestone payments as well as royalties related to products developed in connection with the companies’ agreements. “Leveraging our proprietary Allo-Evasion™ technology, we believe we are uniquely positioned to develop differentiated, effective cell therapies that can be redosed to potentially deliver effective levels of drug exposure and immunologic pressure not only for the treatment of cancer, but also for immunologic diseases with high unmet need. These agreements enable further evaluation of Century’s iPSC-derived NK, T cell, and monocyte/macrophage platforms,” said Hy Levitsky, M.D., president of research and development, Century Therapeutics. “We look forward to sharing at ASH later this year our first clinical and translational dataset from the ongoing Phase 1 ELiPSE-1 trial of CNTY-101, an iPSC-derived CD19-specific CAR-NK cell therapy in patients with relapsed or refractory B cell lymphomas, supporting the potential of our platform to overcome the limitations that have historically prevented the successful application of off-the-shelf allogeneic NK cell approaches.” “As a global leader in the field of iPSCs, we are pleased to expand our partnership with Century as they seek to develop new and transformative allogeneic cell therapies for the treatment of immune-mediated diseases,” said Tomoyuki Hasegawa, president and chief executive officer, FUJIFILM Cellular Dynamics, Inc. “FUJIFILM Cellular Dynamics supported Century Therapeutics in the manufacturing of CNTY-101 and we look forward to expanding our manufacturing partnership to address autoimmune and inflammatory diseases. We are committed to ensuring that iPSC-derived therapeutics realize their full potential, and we look forward to supporting Century Therapeutics’ progress.” Century Therapeutics (NASDAQ: IPSC) is harnessing the power of adult stem cells to develop curative cell therapy products for cancer and autoimmune and inflammatory diseases that we believe will allow us to overcome the limitations of first-generation cell therapies. Our genetically engineered, iPSC-derived cell product candidates are designed to specifically target hematologic and solid tumor cancers, with a broadening application to autoimmune and inflammatory diseases. We are leveraging our expertise in cellular reprogramming, genetic engineering, and manufacturing to develop therapies with the potential to overcome many of the challenges inherent to cell therapy and provide a significant advantage over existing cell therapy technologies. We believe our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer and autoimmune and inflammatory disease care. For more information on Century Therapeutics please visit www.centurytx.com. FUJIFILM Cellular Dynamics, Inc. is a leading developer and manufacturer of human iPSCs and iPSC-derived cells utilized in drug discovery, contract development and manufacturing services for cell therapies. For its partners, FUJIFILM Cellular Dynamics utilizes its iPSC platform to advance the progress of therapeutic candidates in the clinic and provides contract development and manufacturing (CDMO) services. In addition to cell therapy, FUJIFILM Cellular Dynamics also offers life science research tools including the company’s inventoried iCell® products, which are available in almost any cell type and are sourced from multiple cell lines which can be applied for target identification as well as toxicity testing. The company also offers custom cell services and cell banking. FUJIFILM Cellular Dynamics’ goal is to leverage the vast utility of iPSCs to advance human health and improve the quality of life for patients around the world. For more information, please visit: https://www.fujifilmcdi.com/ FUJIFILM Holdings Corporation, Tokyo, leverages its depth of knowledge and proprietary core technologies to deliver “Value from Innovation” in our products and services in the business segments of healthcare, materials, business innovation, and imaging. Our relentless pursuit of innovation is focused on providing social value and enhancing the lives of people worldwide. Fujifilm is committed to responsible environmental stewardship and good corporate citizenship. For more information about Fujifilm’s Sustainable Value Plan 2030, click here. For the year ended March 31, 2023, the company had global revenues of approximately 2.9 trillion yen (21 billion USD at an exchange rate of 134 yen/dollar). For more information, please visit: www.fujifilmholdings.com. 1 National Institute of Allergy and Infectious Diseases (NIAID). Autoimmune Diseases [Website]. National Institutes of Health (NIH): Bethesda, MA. Available at: https://www.niaid.nih.gov/diseases-conditions/autoimmune-diseases [accessed 24 Oct 2023]. 2 Dinse GE et al. 2020. Arthritis Rheumatol 72(6):1026-1035, PMID: 32266792, https://doi.org/10.1002/art.41214. The content above comes from the network. if any infringement, please contact us to modify.

KYV-101 is a novel, fully human CD19 CAR T-cell therapy designed for use in patients with B cell-driven autoimmune diseases Phase 1 open-label clinical trial in the U.S. is actively recruiting patients with LN at multiple sites Patients in treatment with KYV-101 for multiple indications across the U.S. and Europe EMERYVILLE, Calif., June 29, 2023 /PRNewswire/ -- Kyverna Therapeutics ("Kyverna"), a cell therapy company with the mission of engineering a new class of therapies for serious autoimmune diseases, today announced the enrollment of the first patient in its Phase 1 clinical trial of KYV-101 for the treatment of lupus nephritis (LN). KYV-101 is a novel, fully human anti-CD19 chimeric antigen receptor (CAR) T-cell therapy for use in B cell-driven autoimmune diseases such as LN and represents an innovative approach to fighting autoimmune diseases by harnessing the power of the body's immune system. "We are immensely proud to have reached this significant milestone in our journey to transform the lives of patients battling life-altering ailments such as LN. Our KYV-101 cell therapy has the potential to revolutionize treatment for individuals suffering from autoimmune diseases, with patients currently being treated for multiple indications across the U.S. and Europe. We look forward to advancing our clinical programs so we can bring a new option to patients as quickly as possible," said Peter Maag, Ph.D., chief executive officer of Kyverna Therapeutics. The open label, dose escalation, multi-center clinical trial is designed to assess the safety of KYV-101 in patients with refractory LN. The study is targeted to enroll approximately 9-to-12 patients with LN across the U.S. CAR T-cell therapy involves modifying a patient's immune T cells to recognize and remove B cells in the patient's body. Kyverna's anti-CD19 CAR T-cell therapy, KYV-101, specifically targets CD19, a protein expressed on the surface of B cells, which are involved in various types of malignancies, including autoimmune diseases. This experimental treatment may offer new hope to patients who have exhausted conventional treatment options. As more patients commence their journey with KYV-101, Kyverna continues to explore additional indications for this therapy, as well as develop a robust pipeline of promising immunotherapies aimed at addressing unmet medical needs. 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 of LN patients will fail standard of care and approved treatments1. 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 10 percent of patients with LN and 40 percent with diffuse LN (class IV) will ultimately develop kidney failure, requiring dialysis or a kidney transplant to stay alive2. About KYV-101 KYV-101 is an autologous version of a novel, fully human clinical-stage anti-CD19 chimeric antigen receptor T-cell (CAR T) construct with properties well suited for use in B cell-driven autoimmune diseases such as lupus nephritis and other B-cell driven autoimmune diseases. In a 20-patient Phase 1/2 study in oncology, expected anti-lymphoma activity was associated with a significant reduction of cytokines released that translated into a strong reduction of cytokine-driven side effects such as the rate of immune effector cells-associated neurotoxicity syndrome (ICANS)3. The fully human anti-CD19 CAR also translated into reduced immunogenicity that favorably impacted cell persistence at one month. Kyverna recognized that these properties singled out KYV-101 as a product ideally poised for use in autoimmune disease patients, and the company obtained exclusive, worldwide licenses from the National Institutes of Health (NIH) to use this CD19 construct in both autologous and allogeneic CAR T-cell therapies. About Kyverna Therapeutics Kyverna Therapeutics is a cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. Kyverna's pipeline includes next-generation chimeric antigen receptor T-cell (CAR T) therapies in both autologous and allogeneic formats with properties well suited for use in B cell-driven autoimmune diseases. By offering more than one mechanism for taming autoimmunity, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated. For more information, please visit . 1 E. Carter et al., Nature Reviews Rheumatology, 12, Oct. 2016, 605-620. 2 Adv Chronic Kidney Dis. 2019;26(5):313. 3 Brudno et al., Nature Medicine 2020; 26:270-280. SOURCE Kyverna Therapeutics