Lupus Vasculitis, Central Nervous System

WASHINGTON, Dec. 1, 2022 /PRNewswire/ -- The Lupus Foundation of America is pleased to announce the recipients of the 2022 Gary S. Gilkeson Career Development Award (CDA). The two-year grant supports early career scientists studying important areas of lupus research, while providing mentorship and guidance, helping continued professional growth that builds the lupus research field. The 2022 recipients are: Tala El Tal, MD, Hospital for Sick Children Yevgeniya Gartshteyn, MD, Columbia University Rufei Lu, MD, PhD, The Regents of the University of California, San Francisco Rachel Randell, MD, Duke University "The Lupus Foundation of America's Gary S. Gilkeson Career Development Award continues to play an important role in growing the number of lupus-focused researchers by supporting them with funding and mentorship during this pivotal time in their career. These early career scientists bring a fresh perspective and drive to lupus research that have the promise to lead to the next breakthrough, a better understanding of lupus and innovative treatments," shared Karen H. Costenbader, MD, MPH, Chair of the Lupus Foundation of America's Medical-Scientific Advisory Council. This year's grantees will focus their research on pediatric lupus, neuropsychiatric lupus and cognitive challenges, and potential new treatment pathways. Each grantee works with an established clinical scientist as a mentor. "The Lupus Foundation of America's Gary S. Gilkeson Career Development Award encourages young investigators to engage in lupus research early in their careers, setting the stage for their long-term commitment in the field," shared Laura Schanberg, MD, professor of pediatrics, Duke University Medical Center, one of this year's grantee mentors. "The grant not only provides the opportunity and funding for young scientists to pursue research, but it ensures they receive the support and guidance needed from experienced mentors." The 2022 Career Development Award recipients are committed to advancing the field by studying the following important areas of lupus research: Tala El Tal, MD, will study cognitive impairment in children and adolescents with lupus. The study will help improve the understanding of possible links between other lupus factors and cognitive impairment in pediatric patients, and how it affects children and adolescents over time as their brains develop. Yevgeniya Gartshteyn, MD, PhD, will study T-cell subtypes as a potential treatment pathway for lupus. While many lupus treatments target T-Cells, part of the immune system that normally fights infection, this study will look at subtypes to better understand how they work to be able to design better treatments. Rufei Lu, MD, PhD, will be looking at the potential causes of neurological lupus symptoms, such as confusion and anxiety disorders. The research aims to create an enhanced understanding of how unhealthy immune cells attack the cells in the brain. This could pave the way for future studies and the development of potential therapies for people with lupus with neurological complications. Rachel Randell, MD, aims to improve medication taking behaviors among children and adolescents with lupus. The study will help further the understanding of factors associated with taking medications correctly, as well as the feasibility of using an app to measure medication taking as prescribed to improve patient health over time. Each research study by the CDA grantees will strategically advance the Foundation's research agenda, which includes serving as the leader in supporting childhood lupus research. Established in 2006, the LFA's Michael Jon Barlin Childhood Lupus Research Program is the first and only national childhood lupus research program, and calls on some of the greatest minds in the field to address the most urgent challenges in this area of medicine. Learn more about the Gary S. Gilkeson Career Development Award and its 2022 grantees, here. About Lupus Lupus is an unpredictable and misunderstood autoimmune disease that ravages different parts of the body. It is difficult to diagnose, hard to live with and a challenge to treat. Lupus is a cruel mystery because it is hidden from view and undefined, has a range of symptoms, strikes without warning, and has no known cause and no known cure. Its health effects can range from a skin rash to a heart attack. Lupus is debilitating and destructive and can be fatal, yet research on lupus remains underfunded relative to diseases of similar scope and devastation. About the Lupus Foundation of America The Lupus Foundation of America is the national force devoted to solving the mystery of lupus, one of the world's cruelest, most unpredictable and devastating diseases, while giving caring support to those who suffer from its brutal impact. Through a comprehensive program of research, education, and advocacy, we lead the fight to improve the quality of life for all people affected by lupus. Learn more about the Lupus Foundation of America at lupus.org. Media Contact Mike Donnelly [email protected] SOURCE Lupus Foundation of America

Benlysta is now the first and only biologic approved for adults and children who have lupus or lupus nephritis LONDON--(BUSINESS WIRE)-- GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Benlysta (belimumab) for the treatment of children aged 5 to 17 with active lupus nephritis (LN) who are receiving standard therapy. Lupus nephritis is a serious inflammation of the kidneys caused by lupus, which can lead to end-stage kidney disease, requiring dialysis or a kidney transplant.i The approval extends the current indication in the US to include both lupus and active LN for the intravenous formulation in the pediatric patient population. Please see US Prescribing Information for Benlysta. This is the first FDA-approved treatment for pediatric LN, which remains a driving factor in increased complications, hospitalizations and mortality rates in childrenii. Prior to this, treatment options for children were mainly limited to use of non-selective immunosuppressants and corticosteroids. “Active lupus nephritis is a potential serious complication in children with lupus, with most cases occurring within the first two years after their initial lupus diagnosis,” said Stevan W. Gibson, President and CEO, Lupus Foundation of America. “This approval marks a significant step forward in providing treatment options to these children at risk of incurring kidney damage early on in life.” “The long-term goal of lupus nephritis management in adults and children is to preserve renal function while minimizing treatment-related toxicities and associated morbidity,” said Herson Quinones, VP, Specialty and Pipeline US Medical Affairs, GSK. “This Benlysta approval highlights GSK’s commitment to bring treatment options to children living with lupus nephritis. This is another example of how GSK continues to get ahead of this burdensome disease by focusing on science and being grounded in over a decade of clinical experience.” Through ongoing research, GSK is committed to improving the lives of those living with lupus and researching ways to prevent organ and kidney damage in adults and children living with lupus and active LN. About lupus nephritis (LN) Systemic lupus erythematosus (SLE), the most common form of lupus, is a chronic, incurable, autoimmune disease associated with a range of symptoms that can fluctuate over time including painful or swollen joints, extreme fatigue, unexplained fever, skin rashes and organ damage. In lupus nephritis (LN), SLE causes kidney inflammation (swelling or scarring) of the small blood vessels that filter wastes in your kidney (glomeruli) and sometimes the kidneys, by attacking them like they would attack a disease.ii LN can lead to end-stage kidney disease, which requires kidney dialysis or a transplant. Despite improvements in both diagnosis and treatment over the last few decades, LN remains an indicator of poor prognosis.iii,iv Manifestations of LN include proteinuria, elevations in serum creatinine and the presence of urinary sediment. About BENLYSTA (belimumab) BENLYSTA, a BLyS-specific inhibitor, is a human monoclonal antibody that binds to soluble BLyS. BENLYSTA does not bind B cells directly. By binding BLyS, BENLYSTA inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells. First approved in 2011, it is the first and only approved biologic for both SLE and LN in more than 50 years. The following information is based on the US Prescribing Information for BENLYSTA in licensed indications only. Please consult the full Prescribing Information for all the labelled safety information for BENLYSTA. INDICATION BENLYSTA is indicated for patients aged ≥5 with active systemic lupus erythematosus (SLE) or active lupus nephritis who are receiving standard therapy. BENLYSTA is not recommended in patients with severe active central nervous system lupus. IMPORTANT SAFETY INFORMATION CONTRAINDICATION Previous anaphylaxis with BENLYSTA. WARNINGS AND PRECAUTIONS Serious Infections: Serious and sometimes fatal infections have been reported and occurred more frequently with BENLYSTA. Use caution in patients with severe or chronic infections, and consider interrupting therapy in patients with a new infection. Progressive Multifocal Leukoencephalopathy (PML): Cases of JC virus-associated PML resulting in neurological deficits, including fatal cases, have been reported. If PML is confirmed, consider stopping immunosuppressant therapy, including BENLYSTA. Hypersensitivity Reactions (Including Anaphylaxis): Acute hypersensitivity reactions, including anaphylaxis and death, and infusion-related reactions have been reported. Generally, reactions occurred within hours of the infusion but may occur later, including in patients who have previously tolerated BENLYSTA. Non-acute hypersensitivity reactions (eg, rash, nausea, fatigue, myalgia, headache, and facial edema) typically occurred up to a week after infusion. Monitor patients during and after treatment and be prepared to manage anaphylaxis and infusion-related reactions. Be aware of the risk of hypersensitivity reactions, which may present as infusion-related reactions. Discontinue immediately in the event of a serious reaction. With intravenous administration, if an infusion reaction develops, slow or interrupt the infusion. Depression and Suicidality: Depression and suicidality were reported in patients receiving BENLYSTA. Before adding BENLYSTA, assess patients’ risk of depression and suicide and monitor them during treatment. Instruct patients/caregivers to contact their HCP if they experience new/worsening depression, suicidal thoughts/behavior, or other mood changes. Malignancy: There is an increased risk of malignancies with the use of immunosuppressants. The impact of BENLYSTA on the development of malignancies is unknown. Immunization: Live vaccines should not be given for 30 days before or concurrently with BENLYSTA as clinical safety has not been established. Use With Biologic Therapies: BENLYSTA has not been studied and is not recommended in combination with other biologic therapies, including B-cell targeted therapies. ADVERSE REACTIONS The most common serious adverse reactions in adult SLE clinical trials were serious infections; some were fatal. The most common adverse reactions (≥5%) were nausea, diarrhea, pyrexia, nasopharyngitis, bronchitis, insomnia, pain in extremity, depression, migraine, pharyngitis, and injection site reactions (subcutaneous injection). Adverse reactions reported in clinical trials with SLE pediatric patients (≥5 years) and adult patients with lupus nephritis were consistent with those observed in adult SLE trials. USE IN SPECIFIC POPULATIONS Pregnancy: There are insufficient data in pregnant women to establish whether there is drug-associated risk for major birth defects or miscarriage. After a risk/benefit assessment, if prevention is warranted, women of childbearing potential should use contraception during treatment and for ≥4 months after the final treatment. Pregnancy Registry: HCPs are encouraged to register patients and pregnant women are encouraged to enroll themselves by calling 1-877-681-6296. About GSK GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com/company Cautionary statement regarding forward-looking statements GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the Company's Annual Report on Form 20-F for 2021, GSK’s Q1 Results for 2022 and any impacts of the COVID-19 pandemic. Registered in England & Wales: No. 3888792 Registered Office: 980 Great West Road Brentford, Middlesex TW8 9GS i National Institute of Diabetes and Digestive and Kidney Diseases. Lupus and Kidney Disease (Lupus Nephritis). Available at ii Ardoin, S.P., Daly, R., Merzoug, L. et al. Research priorities in childhood-onset lupus: results of a multidisciplinary prioritization exercise. Pediatr Rheumatol 2019; 17, 32. iii National Kidney Foundation, Lupus and Kidney Disease (Lupus Nephritis). Available at iv Gordon C, Hayne D, Pusey C, et al. European Consensus Statement on the Terminology used in the Management of Lupus Glomerulonephritis. Lupus 2009;18:257-26. v Waldman M and Appel GB. Update of the Treatment of Lupus Nephritis. Kidney International 2006;70:1403-1412.

July 14, 2022 11:05 UTC Data show correlation between targeted activity in the brain and improvement in motor speed and executive function, providing further validation of Akili’s technology platform and its potential to improve select cognitive impairments across different indications Data also offer preliminary evidence of Akili’s EVO™ Monitor’s ability to provide rapid mobile assessment of cognitive function BOSTON--(BUSINESS WIRE)-- PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage biotherapeutics company noted that its Founded Entity, Akili Interactive Labs, Inc. (“Akili”), a leading digital medicine company, today announced the publication of full data from a randomized, unblinded study conducted by National Jewish Health and the University of Colorado School of Medicine Departments of Neurology, Psychiatry and Rheumatology that evaluated the ability of Akili’s digital therapeutic AKL-T01 to improve cognitive dysfunction in patients diagnosed with Systemic Lupus Erythematosus (SLE). Data from the study show that AKL-T01 resulted in significant improvement in motor speed and executive functions. The study results were published in the medical journal Lupus. In the randomized, unblinded study of 60 SLE patients aged 18-65, participants showed a high adherence to the 4-week AKL-T01 treatment and showed significant improvement in visuomotor speed (Trail Making A, p=0.025) and cognitive flexibility/sequencing (Trail Making B, p=0.018) compared to the no contact control group. Further, the study investigated the ability of the product EVOTM Monitor1, built on the same technology platform, to serve as a rapid mobile assessment of cognitive function. At baseline, the multitasking threshold (reaction time difference between single tasking and multitasking) from EVOTM Monitor was associated with performance on tasks of cognitive flexibility and psychomotor speed (Trail Making B, r=-0.37, p=0.001 and WAIS-IV coding, r=0.30, p=0.02). At follow up, the treatment group also demonstrated significant improvement in EVOTM Monitor compared to the control group (p=0.001). No additional between groups differences were found in other neuropsychological, behavioral, or health outcomes. The full text of the announcement from Akili is as follows: The Journal Lupus Publishes Investigator-Initiated Study Results Demonstrating Improved Executive Function in Patients with Systemic Lupus Erythematosus Following Use of Akili’s AKL-T01 Product Candidate Data show correlation between targeted activity in the brain and improvement in motor speed and executive function, providing further validation of Akili’s technology platform and its potential to improve select cognitive impairments across different indications Data also offer preliminary evidence of EVO™ Monitor’s ability to provide rapid mobile assessment of cognitive function. BOSTON, Mass. – July 14, 2022 – Akili Interactive Labs, Inc. (“Akili”), a leading digital medicine company, today announced the publication of full data from a randomized, unblinded study conducted by National Jewish Health and the University of Colorado School of Medicine Departments of Neurology, Psychiatry and Rheumatology that evaluated the ability of Akili’s digital therapeutic AKL-T01 to improve cognitive dysfunction in patients diagnosed with Systemic Lupus Erythematosus (SLE). Data from the study show that AKL-T01 resulted in significant improvement in motor speed and executive functions. The study results were published in the medical journal Lupus. Approximately 1.5 million people in the United States are living with SLE.2 Cognitive dysfunction occurs in 20% to 80% of patients with SLE3, twice the prevalence of the general population,4 and substantially impacts their quality of life5. In the randomized, unblinded study of 60 SLE patients aged 18-65, participants showed a high adherence to the 4-week AKL-T01 treatment and showed significant improvement in visuomotor speed (Trail Making A, p=0.025) and cognitive flexibility/sequencing (Trail Making B, p=0.018) compared to the no contact control group. Further, the study investigated the ability of the product EVOTM Monitor1, built on the same technology platform, to serve as a rapid mobile assessment of cognitive function. At baseline, the multitasking threshold (reaction time difference between single tasking and multitasking) from EVOTM Monitor was associated with performance on tasks of cognitive flexibility and psychomotor speed (Trail Making B, r=-0.37,p=0.001 and WAIS-IV coding, r=0.30, p=0.02). At follow up, the treatment group also demonstrated significant improvement in EVOTM Monitor compared to the control group (p=0.001). No additional between groups differences were found in other neuropsychological, behavioral, or health outcomes. “Cognitive difficulties such as attention and executive function are linked to a number of autoimmune diseases, yet there are limited assessments and few interventions to support them,” said Anil S. Jina M.D., Chief Medical Officer of Akili. “The results of this study in patients with SLE are consistent with the cognitive improvements seen in other studies after using our digital therapeutic. We are excited to see this continued validation of our technology platform as we advance our pipeline to deliver clinically meaningful cognitive and quality-of-life improvements across patient populations.” AKL-T01 is delivered through an action video game experience. The technology presents specific sensory stimuli and simultaneous motor challenges designed to target and activate the neural systems that play a key role in attention function while using adaptive algorithms to personalize the treatment experience for each individual patient. AKL-T01 is built on Akili’s Selective Stimulus Management Engine (SSME™), its most advanced proprietary therapeutic engine, designed to target cognitive impairment at its source in the brain. SSME has been clinically validated across more than 20 research, proof-of-concept and pivotal clinical studies in a number of different disease areas to validate the efficacy and safety of Akili’s digital therapeutic solutions for the treatment of cognitive impairments, including ADHD, multiple sclerosis (MS), and Autism Spectrum Disorder (ASD). Study Design This was a randomized, unblinded study of 60 SLE patients aged 18-65. Study participants completed baseline neuropsychological tests (of attention, psychomotor speed, and executive function), a tablet-based digital assessment (EVOTM Monitor), and biobehavioral measures. The patients were randomized into treatment SLE (n=30) or no contact control SLE (n=30) groups, and returned four weeks later for follow-up cognitive, EVO Monitor, and biobehavioral testing. The SLE treatment group was trained on a tablet-based digital treatment (AKL-T01) and were instructed to complete 5 sessions at least 5 days per week for 4-weeks for a total of approximately 25 minutes of gameplay per day. About Akili Akili is pioneering the development of cognitive treatments through game-changing technologies. Our approach of leveraging technologies designed to directly target the brain establishes a new category of medicine – medicine that is validated through clinical trials like a drug or medical device, but experienced like entertainment. Akili’s platform is powered by proprietary therapeutic engines designed to target cognitive impairment at its source in the brain, informed by decades of research and validated through rigorous clinical programs. Driven by Akili’s belief that effective medicine can also be fun and engaging, Akili’s products are delivered through captivating action video game experiences. For more information, please visit . Forward-Looking Statements This communication may contain certain forward-looking statements within the meaning of the federal securities laws. These forward-looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result,” and similar expressions and include statements regarding Akili’s expectations for EndeavorRx® and digital medicine and the benefits expected therefrom. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this communication, including but not limited to (i) the ability of Akili to successfully commercialize EndeavorRx® and continue to advance its clinical development pipeline, (ii) the ability of Akili to maintain relationships with customers and suppliers and retain its management and key employees, (iii) the evolution of the markets in which Akili competes, (iv) the ability of Akili to defend its intellectual property and satisfy regulatory requirements, (v) the impact of the COVID-19 pandemic on Akili’s business, (vi) Akili’s expectations regarding its market opportunities and (vii) the risk of downturns and a changing regulatory landscape in the highly competitive industry in which Akili operates. The foregoing list of factors is not exhaustive. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Akili assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. Akili does not give any assurance that it will achieve its expectations. About PureTech Health PureTech is a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, including inflammatory, fibrotic and immunological conditions, intractable cancers, lymphatic and gastrointestinal diseases and neurological and neuropsychological disorders, among others. The Company has created a broad and deep pipeline through the expertise of its experienced research and development team and its extensive network of scientists, clinicians and industry leaders. This pipeline, which is being advanced both internally and through PureTech's Founded Entities, is comprised of 27 therapeutics and therapeutic candidates, including two that have received both U.S. FDA clearance and European marketing authorization, as of the date of PureTech's most recently filed Annual Report and corresponding Form 6-K. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points based on unique insights in immunology and drug development. For more information, visit or connect with us on Twitter @puretechh. Cautionary Note Regarding Forward-Looking Statements This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation those statements that relate to Akili’s and PureTech's future prospects, development plans, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption “Risk Factors” in our Annual Report on Form 20-F for the year ended December 31, 2021 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise. 1 EVO™ Monitor incorporates a proprietary multi-tasking assessment into a state-of-the-art mobile video game-like platform, which deploys modern video game graphics, engaging reward loops, and real-time adaptive mechanics to dynamically personalize difficulty in order to assess the user's ability. 2 . 3 Hanly, JG, Harrison, MJ. Management of neuropsychiatric lupus. Best Pract Res Cl Rh 2005; 19: 799–821. 4 Ainiala, H, Loukkola, J, Peltola, J. The prevalence of neuropsychiatric syndromes in systemic lupus erythematosus. Neurology 2001; 57: 496–500. 5 Mendelsohn S, Khoja L, Alfred S et al. Cognitive impairment in systemic lupus erythematosus is negatively related to social role participation and quality of life: a systematic review. Lupus 2021, 30:1617-1630