Scleroderma Research Foundation

Dr. Gordon will have oversight over the SRF's global clinical trial program CONQUEST and will contribute to the research program across the SRF portfolio SAN FRANCISCO, Jan. 25, 2024 /PRNewswire/ -- The Scleroderma Research Foundation (SRF)— the nation's largest non-profit funder of scleroderma research—announces the appointment of Gregory Gordon, MD, JD as its first Chief Medical Officer (CMO). As CMO, Dr. Gordon will provide both strategic management and operations oversight for the recently announced global clinical trial program CONQUEST, which will involve 130 medical centers in 22 countries. He will also liaise with CONQUEST's two pharmaceutical partners—Sanofi and Boehringer Ingelheim—as well as any other partners that opt to bring innovative therapies to the CONQUEST platform. Dr. Gordon's contributions are expected to add focus to the ongoing work of SRF—in particular regarding its efforts to bring new and innovative therapeutic alternatives to scleroderma patients and the scleroderma community worldwide. Continue Reading Dr. Gregory Gordon joins Scleroderma Research Foundation as Chief Medical Officer "Dr. Gordon, with his impressive track record spanning over fifteen years in pivotal leadership roles in the biotechnology industry, brings a unique and vital perspective to the Scleroderma Research Foundation. His diverse experience is an invaluable asset to the SRF and the entire scleroderma community. Among his contributions, he will enhance our already extensive community outreach and patient engagement initiatives," said Joanne Gold, Executive Director of SRF. "I'm excited to be part of an organization with a long history of funding and supporting innovative research for the scleroderma community," said Dr. Gordon, who officially started his role in January. "I am pleased to join the SRF at this pivotal time to help realize the SRF's vision of building a global community tuned to rapid and efficient clinical development with CONQUEST." "With CONQUEST, the Scleroderma Research Foundation is deepening its commitment and support for research that addresses the high unmet needs of scleroderma patients. We are very excited to welcome Dr. Gordon to the SRF team with his deep experience and proven capabilities and his ability to drive the CONQUEST program forward. CONQUEST represents the first platform approach to clinical trials in rare autoimmune disease more generally and in particular to scleroderma," said Luke Evnin, Ph.D., Chairman of the SRF. Dr. Gordon joined the SRF after leading a team of physicians overseeing the development of potential treatments for multiple neurologic and pediatric diseases at PTC Therapeutics, where he was Vice President of Clinical Development. Originally a primary care physician at Harvard Vanguard Medical Associates, he became a well-respected figure in pharmaceutical development, working with teams responsible for successfully bringing multiple treatments to patients, including linaclotide (Linzess™), darvadstrocel (Alofisel™), and setmelanotide (Imcivree™). He has previously headed clinical development and operations at Rhythm Pharmaceuticals, served as the Head of US Medical at TiGenix, and served as the Global Clinical Affairs Lead at Nestlé Health Science. Before attending medical school at the State University of New York at Stony Brook, Dr. Gordon earned a J.D. from Columbia Law School and worked as an attorney in New York City. Greg is a native of Larchmont, New York. He now lives in Brookline, just outside Boston, with his wife and two sons. About the Scleroderma Research Foundation The Scleroderma Research Foundation (SRF), a 501(c)(3) organization, was established in 1987 with a mission to fund and facilitate the most promising, highest-quality research aimed at new treatments and, ultimately, a cure for scleroderma. Led by a Scientific Advisory Board comprised of some of the most highly regarded scientists in the nation, the SRF's research program actively seeks out the leading scientific minds from disparate fields including autoimmunity, immunology, genetics, and fibrosis to join the scleroderma research community. In addition to its core research program, the SRF recently announced the initiation of CONQUEST, a global platform clinical trial. The SRF has also led the formation of two other large-scale projects aimed at accelerating scleroderma research: The CONQUER Registry, the first nationwide longitudinal registry for scleroderma patients, and The GRASP Project, which examines scleroderma in the African American community. The SRF is also dedicated to educating people living with scleroderma and their caregivers as they learn about how to best manage the challenges of the disease. About CONQUEST Using a model first created over a decade ago to accelerate oncology drug development, the SRF platform clinical trial is the first of its kind in autoimmune diseases. In its initial iteration, CONQUEST will focus on Interstitial Lung Disease secondary to Scleroderma (SSc-ILD) which manifests in patients as lung fibrosis. In the future, the CONQUEST platform will be expanded to address other manifestations of scleroderma. The ground-breaking effort, conceived and led by the SRF, will enroll patients across more than 130 centers in more than 22 countries. CONQUEST allows multiple therapies to be evaluated under a common trial infrastructure with a common control arm. CONQUEST will initially enroll approximately 400 patients but with the advantages of the platform design, only approximately 134 patients will be randomized to the placebo arm (to receive standard-of-care). Accordingly, the design is very patient-centric since patients generally enroll in a trial to get access to novel medicines. However, the trial retains high statistical power for analysis of efficacy endpoints since the data from the placebo patients from all concurrently enrolled sub-protocols (e.g. Sanofi and Boehringer Ingelheim) will be pooled for efficacy analysis. This efficiency is not available in traditional trials and is one of the features that underscore the uniqueness of CONQUEST. By assembling a global network of high-performing centers that are dedicated to treating scleroderma, the SRF and its pharmaceutical partners expect to make enduring contributions to the scleroderma community by creating an ecosystem designed to enable success in new drug development. Sanofi and Boehringer Ingelheim are the inaugural partners for CONQUEST, which was first announced on August 1, 2023. Media Contact: Julie Richter | [email protected] | 480-818-8022 SOURCE Scleroderma Research Foundation

FDA clears IND to launch CONQUEST trial platform with initial focus on Interstitial Lung Disease Secondary to Scleroderma (SSc-ILD) SCLERODERMA RESEARCH FOUNDATION (SRF) holds the IND as trial Sponsor Sanofi and Boehringer Ingelheim share the platform; patient enrollment to begin early in 2024 SAN FRANCISCO, Dec. 19, 2023 /PRNewswire/ -- The Scleroderma Research Foundation (SRF), the nation's largest non-profit funder of scleroderma research, today announced that the FDA has cleared its Investigational New Drug application (IND) to launch the CONQUEST clinical trial platform and begin enrolling patients. Sanofi and Boehringer Ingelheim will each contribute an experimental agent to CONQUEST, and as such they are the first pharma partners to commit to this protocol designed to efficiently advance new scleroderma treatments through clinical development. The first two nominated investigational drugs will be evaluated for safety and efficacy in scleroderma patients burdened by interstitial lung disease (SSc-ILD). The Sanofi agent is amlitelimab, a non-depleting monoclonal antibody that binds to OX40-Ligand; the Boehringer Ingelheim agent is BI -1015550, an oral PDE-4B inhibitor. Both investigational agents have shown preliminary evidence of efficacy in prior clinical trials for non-scleroderma indications in modifying disease processes. Luke Evnin, Ph.D., Chairman of the Scleroderma Research Foundation, commented, "Both Sanofi and Boehringer Ingelheim have a rich history of innovation in the immunologic and fibrotic disease arena, and they are bringing their considerable expertise to CONQUEST. With these two partners, CONQUEST is well positioned to ensure that the platform will animate the SRF vision of building a global community tuned to rapid and efficient clinical development of promising novel therapeutic agents for the high unmet needs of scleroderma patients. I am proud and excited to see two promising experimental agents for SSc-ILD advancing, and I have high hopes that these agents will prove safe and efficacious in our patients." Boehringer Ingelheim's BI - 1015550 is an orally-administered phosphodiesterase 4B (PDE4B) inhibitor that is being investigated as a potential treatment for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). Preclinical studies have shown that BI 1015550 has anti-inflammatory and antifibrotic properties. The FIBRONEER™ global program includes two Phase III studies – FIBRONEER-IPF (NCT05321069) in patients with IPF and FIBRONEER-ILD (NCT05321082) in people living with other progressive fibrosing interstitial lung diseases (ILDs) and was initiated in 2022. The FDA granted BI - 1015550 Breakthrough Therapy Designation for the treatment of IPF in February 2022 and Orphan Drug Designation for BI -1015550 in IPF in October 2022. According to Ioannis Sapountzis, Global Head of Therapeutic Areas, Boehringer Ingelheim, "As a global innovation leader, we remain steadfast in our commitment for people living with scleroderma. Mounting our agents into CONQUEST is a critical step to help bring forward this next generation of treatment to those in need as quickly as possible." Sanofi's amlitelimab is a fully human non-depleting monoclonal antibody that binds to OX40-ligand, a key immune regulator, and has the potential to be a first-in-class treatment for a range of immune-mediated diseases and inflammatory disorders, including moderate-to-severe atopic dermatitis, asthma and hidradenitis suppurativa. By targeting OX40-ligand, amlitelimab aims to restore balance between pro-inflammatory and regulatory T cells. According to Naimish Patel, M.D., Head of Global Development, Immunology and Inflammation, Research & Development, Sanofi, "We look forward to initiating the amlitelimab development program in scleroderma, an underserved condition with high morbidity and mortality rates and limited treatment options. With our knowledge about the underlying biology of the disease and amlitelimab's unique mechanism of action, we hypothesize that amlitelimab could have benefits on ILD and dermal fibrosis endpoints of scleroderma. To date, amlitelimab's potential has been encouraging and we look forward to further advancing this asset as part of our commitment to delivering best-in-class treatments that improve the lives of people living with chronic inflammatory diseases." Amlitelimab is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. The CONQUEST platform will be open to adding additional investigational agents starting in 2025. One of the key features of the platform is that new drugs can be added at any time with minimal regulatory overhead and an opportunity to take maximum benefit of the infrastructure of the platform. In addition, the anonymized patient-by-patient data from the placebo arm will be owned by the SRF and become an invaluable resource for the entire community. About CONQUEST Using a model first created over a decade ago to accelerate oncology drug development, the SRF platform clinical trial is the first of its kind in autoimmune diseases. In its initial iteration, CONQUEST will focus on Interstitial Lung Disease secondary to Scleroderma (SSc-ILD) which manifests in patients as lung fibrosis. In the future, the CONQUEST platform will be expanded to address other manifestations of scleroderma. The ground-breaking effort, conceived and led by the SRF, will enroll patients across more than 130 centers in more than 22 countries. CONQUEST allows multiple therapies to be evaluated under a common trial infrastructure with a common control arm. CONQUEST will initially enroll approximately 400 patients but with the advantages of the platform design, only approximately 134 patients will be randomized to the placebo arm (to receive standard-of-care). Accordingly, the design is very patient-centric since patients generally enroll in a trial to get access to novel medicines. However, the trial retains high statistical power for analysis of efficacy endpoints since the data from the placebo patients from all concurrently enrolled sub-protocols (e.g. Sanofi and Boehringer Ingelheim) will be pooled for efficacy analysis. This efficiency is not available in traditional trials and is one of the features that underscore the uniqueness of CONQUEST. By assembling a global network of high-performing centers that are dedicated to treating scleroderma, the SRF and its pharmaceutical partners expect to make enduring contributions to the scleroderma community by creating an ecosystem designed to enable success in new drug development. Sanofi and Boehringer Ingelheim are the inaugural partners for CONQUEST, which was first announced on August 1, 2023. About Scleroderma Scleroderma is a systemic autoimmune disease that affects approximately 100,000 patients in the US with high morbidity and mortality rates and for which there are limited treatment options. The disease presents multiple challenges for patients with a spectrum of symptoms that prominently include vascular disease and internal organ fibrosis. About the Scleroderma Research Foundation The Scleroderma Research Foundation (SRF), a 501(c)(3) organization, was established in 1987 with a mission to fund and facilitate the most promising, highest-quality research aimed at new treatments and, ultimately, a cure for scleroderma. Led by a Scientific Advisory Board comprised of some of the most highly regarded scientists in the nation, the SRF's research program actively seeks out the leading scientific minds from disparate fields including autoimmunity, immunology, genetics, and fibrosis to join the scleroderma research community. In addition to its core research program, the SRF has also led the formation of two other large-scale projects aimed at accelerating scleroderma research: The CONQUER Registry, the first nationwide longitudinal registry for scleroderma patients, and The GRASP Project, which examines scleroderma in the African American community. The SRF is also dedicated to educating people living with scleroderma and their caregivers as they learn about how to best manage the challenges of the disease. About Boehringer Ingelheim Boehringer Ingelheim is working on breakthrough therapies that transform lives, today and for generations to come. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. More than 52,000 employees serve over 130 markets in the two business areas, Human Pharma and Animal Health. Learn more at . For more information about the FIBRONEER™ program, please visit ClinicalTrials.gov. BI -1015550 is an investigational therapy, which has not been approved for any proposed indication by any regulatory authority including the U.S. Food and Drug Administration (FDA). Boehringer Ingelheim is currently investigating its efficacy and safety profile. Shari Annes Annes Associates Cell 650 888 0902 370254@email4pr.com View original content to download multimedia: SOURCE Scleroderma Research Foundation

The investigational therapy will be used in Kyverna's KYSA-5 Phase 1/2 open-label, multicenter study to evaluate KYV-101 in adult patients with diffuse cutaneous systemic sclerosis (scleroderma) This is the third IND clearance for KYV-101, a novel, fully human CD19 CAR T-cell therapy designed for use in patients with B cell-driven autoimmune diseases EMERYVILLE, Calif., Oct. 11, 2023 /PRNewswire/ -- Kyverna Therapeutics ("Kyverna"), a clinical-stage cell therapy company with the mission of engineering a new class of therapies for serious autoimmune diseases, today announced the clearance of the third Investigational New Drug (IND) application for KYV-101 by the U.S. Food and Drug Administration (FDA). This will allow Kyverna to initiate a Phase 1/2 open-label, multicenter study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor (CAR) T cell therapy for the treatment of diffuse cutaneous systemic sclerosis (scleroderma). The trial, named KYSA-5, adds to the ongoing Phase 1 KYSA-1 trial in the U.S. and the Phase 1/2 KYSA-3 trial in Germany where KYV-101 is currently investigated in adults with active lupus nephritis. "We welcome Kyverna's enthusiasm and interest to push forward with their CAR T approach in scleroderma patients with the KYSA-5 trial," said Luke Evnin, chairman of the Scleroderma Research Foundation. "We await the results with optimism that CAR-T driven B cell ablation may ultimately deliver efficacy with adequate safety for a broader range of our patients than immune-ablative chemotherapy alone." "We are immensely proud of being able to bring KYV-101 to patients suffering from scleroderma," said Peter Maag, Ph.D., chief executive officer of Kyverna Therapeutics. "We are keen to initiate our KYSA-5 trial in this new patient population and generate data to support our KYV-101 design goals. With the deep B cell depletion from KYV-101 treatment, patients with scleroderma may have a full reset of their immune system to stop the vicious cycle of their overactive immune system." About KYV-101 KYV-101 is an autologous version of a novel, fully human clinical-stage anti-CD19 chimeric antigen receptor (CAR) T cell construct with properties well suited for use in B cell-driven autoimmune diseases such as lupus nephritis, scleroderma, 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 cell-associated neurotoxicity syndrome (ICANS).1 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 patients with autoimmune diseases, 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 Systemic Sclerosis Systemic sclerosis (scleroderma) is a rare, chronic autoimmune disorder that affects the skin and internal organs and can have life-threatening complications.2,3 Severe scleroderma is associated with widespread pain, discomfort, and stiffness due to skin and joint involvement. Organ involvement may include interstitial lung disease, pulmonary hypertension, heart complications, kidney dysfunction, and gastrointestinal issues and may significantly reduce life expectancy. Managing severe scleroderma is complex and often requires a combination of medications, including immunosuppressants and disease-modifying drugs, as well as specialized care. About Kyverna Therapeutics Kyverna Therapeutics is a clinical-stage 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 (CAR) T-cell 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  . Kyverna Media Contact: Christian Pflaumer +1 (917) 841-4525 christian.pflaumer@ruderfinn.com References: 1. Brudno et al., Nature Medicine 2020; 26:270-280. 2. Bergamasco et al., Clin Epidemiol 2019; 11:257-273 3. Adigun et al. Systemic Sclerosis. [Updated 2022 May 8].