Dystonia, Dopa-Responsive

·More than 530 million people worldwide living with diabetes are at risk for developing diabetic retinal disease (DRD), the leading cause of blindness in working-age adults. 1·DRD is a growing epidemic that is expected to increase as the incidence of diabetes escalates across the globe.2,3 ·There is an urgent need for earlier detection of DRD and novel treatments that work in earlier disease states before vision loss occurs. Ingelheim, Germany and Ann Arbor, MI, USA, November 12, 2024 – Boehringer Ingelheim and the Mary Tyler Moore Vision Initiative (MTM Vision) today jointly announce the start of a long-term collaboration as Boehringer becomes the first pharmaceutical company to join the MTM Vision Consortium. Housed at the University of Michigan, the MTM Vision Consortium aims to unite innovators in the pre-competitive space from universities, foundations, as well as pharmaceutical and biotech companies. MTM Vision leads this international collaboration, motivated by the urgency of millions of people worldwide who have lost or are at risk of losing their eyesight as a consequence of diabetes. The Consortium members share several goals: address the need for novel means to better diagnose and stage DRD; validate promising clinical trial endpoints and biomarkers; and accelerate the development of breakthrough therapies. These efforts will enable treatment earlier in disease before vision loss occurs, and contribute to the creation of new regulatory pathways for drug approval. “We are excited to join forces with MTM Vision in leading a new era of vision preservation and protection for people living with diabetes. This collaboration will help us enhance our understanding of the progression of diabetic retinopathy and the role of retinal non-perfusion, supporting the development of our portfolio in diabetic retinal diseases” said Dr. Ulrike Graefe-Mody, Global Head of Retinal Health at Boehringer Ingelheim. “It also gives us access to a unique biospecimen bank, accelerating our research and biomarker identification. In return, we are openly sharing our current efforts for evidence generation to further understand DRD and support MTM Vision’s work. Together, we are setting a course to fundamentally change our understanding of vision loss from diabetes and how it is treated.” “Here at our third Annual Global Symposium on curing vision loss from diabetes, which coincides with Diabetic Eye Disease Awareness Month, we are delighted to announce that Boehringer Ingelheim has joined us as a founding member of our MTM Vision Consortium” said Dr. S. Robert Levine, founder and CEO of MTM Vision. “This Symposium provides a platform for the convening of global leaders dedicated to accelerating the development of innovative therapies to prevent and cure vision loss from diabetes. Boehringer Ingelheim’s commitment to our pre-competitive Consortium marks a pivotal moment along our path to ensure better patient outcomes by advancing research and sharing critical data. This will significantly strengthen our efforts to catalyze the development of new methods to diagnose and treat diabetic retinal disease earlier, which may preserve vision for millions of people at risk.” For more information about the Mary Tyler Moore Vision Initiative visit MaryTylerMoore.org. For more information about Boehringer Ingelheim’s commitment to retinal health, please visit Boehringer-Ingelheim.com. Notes to Editors: About diabetic retinal diseaseDiabetic retinal disease (DRD) includes all diabetes-related changes in the retina, including harm to retinal blood vessels (diabetic retinopathy or DR), swelling in the retina (diabetic macular edema or DME), restriction of blood supply to the macula (diabetic macular ischemia or DMI), and damage to retinal nerve cells that help us see (diabetic retinal neuropathy or DRN). 4,5 About Boehringer IngelheimBoehringer Ingelheim is a biopharmaceutical company active in both human and animal health. As one of the industry’s top investors in research and development, the company focuses on developing innovative therapies that can improve and extend lives in areas of high unmet medical need. Independent since its foundation in 1885, Boehringer takes a long-term perspective, embedding sustainability along the entire value chain. More than 53,500 employees serve over 130 markets to build a healthier, more sustainable, and equitable tomorrow. Learn more at www.boehringeringelheim.com. About the Mary Tyler Moore Vision Initiative and the MTM Vision ConsortiumThe Mary Tyler Moore Vision Initiative (MTM Vision) is a one-of-a-kind non-profit dedicated to accelerating the development of new therapies to preserve and restore vision in people with diabetes. MTM Vision was founded by Dr. S. Robert Levine to honor his wife, Mary Tyler Moore’s leadership in diabetes research advocacy. With a focus on fostering groundbreaking research and global collaboration among leading academic institutions, research advocacy groups, pharmaceutical and medical device companies, government agencies, tech firms, philanthropists, and leaders in the entertainment industry, MTM Vision stands at the forefront of the fight against diabetic retinal disease (DRD). In partnership with the University of Michigan (U-M) and the Joslin Diabetes Center, an affiliate of Harvard Medical School, and housed at the U-M Caswell Diabetes Institute and the U-M Kellogg Eye Center, MTM Vision has established the MTM Vision Consortium, a pre-competitive research initiative accelerating drug development for DRD. The Consortium offers members access to key resources like the MTM Vision Ocular Biorepository and Resource Center and the MTM Vision Clinical Endpoints and Disease Biomarkers Data Resource to support development of breakthrough therapies and FDA approvals. Boehringer Ingelheim’s Intended Audiences NoticeThis press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business. References Wong TY, Sabanayagam C. Ophthalmologica. 2020;243(1):9–20.Teo ZL, et al. Ophthalmology. 2021;128(11):1580-91.Causes of blindness and vision impairment in 2020 and trends over 30 years, and prevalence of avoidable blindness in relation to VISION 2020: the Right to Sight: an analysis for the Global Burden of Disease Study. Lancet Glob Health 2021 Feb;9(2):e144-e16.Michael D. Abramoff MD, et al. Investigative Ophthalmology & Visual Science. 2018;59:519–527. Jennifer K. Sun, MD, MPH, et al. Ophthalmology. 2020;128:490-3, 2021. Media Contacts Boehringer Ingelheim: Linda Ruckel Boehringer Ingelheim Corporate Center GmbH Innovation Unit/Bio Comms, Corp. Affairs Media + PR press@boehringer-ingelheim.com Mary Tyler Moore Vision Initiative: Laura BachrachThe Mary Tyler Moore Vision Initiative, Corporate Communicationslb@laurabachrach.com

Accelerate the Cure for Vision Loss and Blindness from Diabetes; 530 Million People Worldwide at RiskThe Mary Tyler Moore Vision Initiative has produced their first PSA campaign to galvanize the public to support the advancement of medical research to preserve and restore vision in people with diabetesNarrated by Kevin Kline, this PSA includes remarks by Julia Louis-Dreyfus, Oprah Winfrey and Reese Witherspoon and is directed by Academy Award-nominated filmmaker James Keach LOS ANGELES, July 16, 2024 /PRNewswire/ -- Today, the Mary Tyler Moore Vision Initiative (MTM Vision) has launched their first Public Service Announcement (PSA) campaign. Created to honor the iconic actress Mary Tyler Moore, MTM Vision seeks to make Mary's dream of a world without vision loss and blindness from diabetes come true. MTM Vision's PSA educates the public about the critical need for medical research to find ways to better treat and cure diabetic retinal disease (DRD) and related vision loss and blindness from diabetes. Today, more than 530 million people worldwide with diabetes are at risk for DRD. Mary Tyler Moore Vision Initiative PSA: The Sight You Save May Be That of Someone You Love

On May 1, 2020, the Food and Drug Administration approved daratumumab and hyaluronidase-fihj (DARZALEX FASPRO, Janssen Biotech, Inc.) for adult patients with newly diagnosed or relapsed/refractory multiple myeloma. This new product allows for subcutaneous dosing of daratumumab. Daratumumab and hyaluronidase-fihj is approved for the following indications that intravenous daratumumab had previously received: in combination with bortezomib, melphalan and prednisone  in newly diagnosed patients who are ineligible for autologous stem cell transplant, in combination with lenalidomide and dexamethasone  in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy, as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent. Efficacy of daratumumab and hyaluronidase-fihji (monotherapy) was evaluated in COLUMBA (NCT03277105), an open-label non-inferiority trial randomizing 263 patients to daratumumab and hyaluronidase-fihj and 259 to intravenous daratumumab (daratumumab IV). The trial’s co-primary endpoints were overall response rate (ORR) and pharmacokinetic (PK) endpoint of the maximum Ctrough on cycle 3, day 1 pre-dose. Daratumumab and hyaluronidase-fihj was non-inferior to daratumumab IV in evaluating these two endpoints. The ORR was 41.1% for daratumumab and hyaluronidase-fihj and 37.1% for daratumumab IV with a risk ratio of 1.11 (95% CI: 0.89, 1.37). The geometric mean ratio comparing daratumumab and hyaluronidase-fihj to daratumumab IV for maximum Ctrough was 108% (90% CI: 96,122). Efficacy of daratumumab and hyaluronidase-fihj in combination with VMP (D-VMP) was evaluated in a single-arm cohort of PLEIADES (NCT03412565), a multi-cohort, open‑label trial. Eligible patients were required to have newly diagnosed multiple myeloma and were ineligible for transplant. The major efficacy outcome measure, ORR,  was 88.1% (95% CI: 77.8, 94.7). Efficacy of daratumumab and hyaluronidase-fihj in combination with Rd (D-Rd) was evaluated in a single-arm cohort of this trial. Eligible patients had received at least one prior line of therapy. ORR was 90.8% (95% CI: 81.0, 96.5).   The most common adverse reaction (≥20%) with daratumumab and hyaluronidase-fihj monotherapy is upper respiratory tract infection. The most common adverse reactions (≥20%) with D-VMP are upper respiratory tract infection, constipation, nausea, fatigue, pyrexia, peripheral sensory neuropathy, diarrhea, cough, insomnia, vomiting, and back pain. The most common adverse reactions (≥20%) with D-Rd are fatigue, diarrhea, upper respiratory tract infection, muscle spasms, constipation, pyrexia, pneumonia and dyspnea.   The most common hematology laboratory abnormalities (≥40%) with daratumumab and hyaluronidase-fihj are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin. The recommended dose of daratumumab and hyaluronidase-fihj is 1,800 mg daratumumab and 30,000 units hyaluronidase administered subcutaneously into the abdomen over approximately 3 to 5 minutes according to recommended schedule. View full prescribing information for DARZALEX FASPRO. This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. For information on the COVID-19 pandemic, see the following resources: FDA: Coronavirus Disease 2019 (COVID-19) NCI: Coronavirus: What People With Cancer Should Know CDC: Coronavirus (COVID-19) Follow the Oncology Center of Excellence on Twitter @FDAOncology.