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Lord Cameron will lead international efforts to drive the OHC’s mission of delivering 40 new therapies for rare diseases into clinical trials over the next ten years OXFORD, UK and CLEVELAND, Ohio, US, 15th October 2024 – The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, aimed at delivering cutting-edge rare disease breakthroughs, is delighted to welcome Former UK Prime Minister David Cameron as Chair of its Advisory Council. Under his leadership, the OHC aims to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years, while providing scalable solutions to address the rare disease challenge globally. Lord Cameron returns to the Advisory Council, having originally joined in October 2023 prior to his appointment as Foreign Secretary in the UK Government – a position he held between November 2023 and July 2024. As Chair of the OHC Advisory Council, Lord Cameron will lead the international efforts of the OHC in its philanthropic mission, build partnerships, expertise and networks, while also focusing on extending global awareness of the OHC and its objectives. Lord Cameron said: “Of the causes I have advocated, this one really is very personal. Setting up Genomics England and establishing a world-leading database with hundreds of thousands of genomes is one of my proudest achievements as Prime Minister. Yet, to be truly transformational, genomics requires the best of academia, life sciences, pharmaceutical companies, philanthropy and venture capital from around the world to come together. That is what the Oxford-Harrington Rare Disease Centre sets out to do, convening those key players and uniting around a bold mission: to deliver 40 new treatments for rare diseases in the next decade. “As a father I know all too well the impact of rare diseases. We miss our son Ivan every day – all these years on our loss is still so raw. But the steps we are taking now means that, in the not-too-distant future, families like ours will have hope. It is vital that we try to give it to them.” Lord Cameron is a passionate advocate of rare disease research and development as a parent of a child born with a rare and incurable disease. His family’s experience of caring for their son Ivan, who had a rare condition known as Ohtahara syndrome, made the issue of rare diseases deeply personal to him. While in Government, this personal connection inspired him to establish the 100,000 Genomes Project in 2012, working with leading scientists, healthcare advisors and agencies, including the UK National Health Service (NHS) and Medicines and Healthcare products Regulatory Agency (MHRA), all with the aim of accelerating progress in treating the millions of people affected by rare diseases. Genomics England has since helped position the UK as a leader in rare disease research, fostering collaboration and supporting the development of ground-breaking treatments. Matthew Wood, Director and Chief Scientific Officer of the OHC, added: “The OHC is aiming to make a global impact on the treatment of rare diseases by advancing pioneering science and driving the development of breakthrough therapies. Our Advisory Council plays a pivotal role not only in guiding our actions but also in bringing together the rare disease community with leading decision-makers to raise awareness of and address the key challenges being faced. We are proud to welcome Lord Cameron back as Chair of the Advisory Council, as he brings the passion and commitment from real world experience alongside a global profile. We are confident that his leadership, alongside the other members of our Council, can enable the progress needed to make a meaningful difference to patients and their families.” Jonathan S. Stamler, MD, President and co-Founder of Harrington Discovery Institute, said: “Rare diseases represent a global health issue that faces unique challenges. Individual disorders may affect very few patients, but collectively more than 400 million people worldwide are living with such a disease, about half of which are children. The OHC was created to make meaningful progress in changing the rare disease landscape for patients, their families and society. We will shortly announce the first class of Oxford-Harrington Scholars representing ground-breaking rare disease research programmes in the UK, US and Canada. We will need deep expertise and access at the highest levels if we are to have real impact and are privileged to be working with globally recognised leaders like Lord Cameron on our Advisory Council.” Lord Cameron joins Professor Sir John Bell (President, Ellison Institute of Technology Oxford, and former Regius Professor of Medicine at Oxford University from 2002 to 2024), Baroness Nicola Blackwood (Chair of Genomics England and of Oxford University Innovation), John F. Crowley (President and CEO of the US Biotechnology Innovation Association), Ronald G. Harrington (a renowned entrepreneur and philanthropist, and co-Founder of the Harrington Discovery Institute), and Jonathan S. Stamler, MD (President and co-Founder of the Harrington Discovery Institute), on the OHC Advisory Council. *** Notes to Editors About Rare DiseasesMore than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects 10 percent of the US population. Similarly, Europe has approximately 30 million people who suffer from a rare disease. The majority of rare diseases are genetic in origin, which means they are present throughout a person’s life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option. About Oxford-Harrington Rare Disease CentreThe Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, in Cleveland, Ohio, US. The OHC combines world-leading strengths in research and therapeutics development from across these organizations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare diseases across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | LinkedIn | X About the University of OxfordThe University of Oxford is rated the best in the world for medicine and life sciences, and it is home to the UK’s top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. The University of Oxford’s Medical Sciences Division, which includes the Department of Paediatrics where the OHC is based in Oxford, is one of the largest biomedical research centres in Europe. For more information, visit: www.ox.ac.uk About Harrington Discovery InstituteHarrington Discovery Institute at University Hospitals in Cleveland, Ohio aims to advance academic discoveries into medicines to address unmet need. It was created in 2012 with a $50 million founding gift from the Ronald G. Harrington family and has supported more than 200 scientists throughout the U.S., U.K. and Canada. Harrington Discovery Institute uses an innovation model that surrounds scientists with drug development and business expertise and employs both philanthropic funding and catalytic investment capital to advance projects into the clinic and through commercialization. For more information, visit: HarringtonDiscovery.org| LinkedIn | X Media contacts: UK/ EU – MEDiSTRAVA Sylvie Berrebi / Mark SwallowOHC@medistrava.com US – For University HospitalsAnsley Kelmansley.kelm@UHhospitals.org

One-year outcomes from open-label extension to BouNDless study reaffirm previously published data regarding long-term efficacy of ND0612 Additional new data evaluating quality of life, daily motor experiences and motor fluctuations favor ND0612 treatment vs. oral immediate-release levodopa/carbidopa JERSEY CITY, N.J., Sept. 30, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of three posters discussing new data from the pivotal Phase 3 BouNDless trial (NCT04006210) and its ongoing open-label extension (OLE) evaluating investigational ND0612 in people with Parkinson's disease (PD) experiencing motor fluctuations, as part of the 2024 International Congress of Parkinson's Disease and Movement Disorders (MDS). ND0612 is being studied as a 24-hour, continuous, subcutaneous (SC) infusion of liquid levodopa/carbidopa (LD/CD). The data highlight one-year outcomes from the BouNDless-OLE phase that are consistent with previously-reported 12-week data from the study evaluating the long-term efficacy of ND0612. Additional presentations discussed findings from patient-reported outcomes favoring ND0612 vs. oral immediate-release (IR) LD/CD across various measures of quality of life (QoL) and the effect of treatment on motor signs and motor experiences of daily living (m-EDL). "The longer-term data presented at MDS provide insights into the potential of ND0612 to not only improve symptoms, but also maintain its efficacy, which may provide patients with a meaningful reduction in symptom burden," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "These findings reinforce our commitment to studying ND0612, if approved, as a potential new treatment option for people with Parkinson's disease experiencing motor fluctuations, as well as ensuring the latest scientific knowledge is continuously shared with the community." Key highlights presented at the meeting include: Abstract #653: Long-Term Efficacy of 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612) for Motor Fluctuations in Parkinson's Disease Patients who completed the double-blind double-dummy (DBDD) phase of the BouNDless study were eligible to enter the OLE phase. At six months, patients treated with ND0612 experienced an average reduction of 2.2 hours in daily OFF time and an increase of 2.4 hours in daily ON time without troublesome dyskinesia. These results were sustained at 12 months, showing an average reduction of 2.02 hours in OFF time and an increase of 2.11 hours in ON time without troublesome dyskinesia. In addition, patients with one or more hours of ON time with troublesome dyskinesia at ND0612 initiation (n=45) experienced an average reduction of troublesome dyskinesia by 1.5 hours at 12 months, further demonstrating sustained efficacy. Abstract #648: Quality of Life with 24-hour Subcutaneous Levodopa/Carbidopa Infusion (ND0612): PDQ-39 Results from a Phase 3 Randomized, Active-Controlled Study Following two sequential open-label periods to optimize oral IR-LD/CD and ND0612, study participants were randomized to a 12-week DBDD treatment period with either ND0612 or IR-LD/CD regimens. Change in QoL was assessed at the end of the DBDD period across eight domains of daily living using the PD Questionaire-39 (PDQ-39).[1] At the end of 12 weeks, patients in the continuous 24-hour ND0612 treatment group reported improved QoL (-2.1 [-3.7, -0.6]) compared to no change in the IR-LD/CD group (+0.6 [-0.9, 2.1]). The PDQ-39 domain analyses favored ND0612 treatment in most domains; mean changes were mobility, bodily discomfort, cognition, activities of daily living, stigma, social support, communication and emotional well-being. Abstract #685: Efficacy of Continuous Subcutaneous Levodopa/Carbidopa Infusion (ND0612) on Motor Signs of PD and Experiences of Daily Living During the BouNDless trial, at both eight and 12 weeks, treatment with ND0612 showed superior improvements in m-EDL vs. oral IR-LD/CD, as measured by the Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II and Part III scores. Additionally, a post-hoc analysis of treatment differences for a number of motor signs showed treatment with ND0612 was superior to oral IR-LD/CD, with improvements observed in postural instability-gait disorder (–0.26 vs. 0.02, p=0.0012), speech and oral health (–0.11 vs. 0.05, p=0.0140), tremor (–0.15 vs. –0.05, p=0.0992) and self-care (–0.08 vs. 0.09, p=0.0528). No relevant differences were observed for rigidity and bradykinesia. In the BouNDless trial, ND0612 was generally well tolerated. In patients treated with ND0612, the most common systemic treatment emergent adverse events (TEAEs) reported during the open-label run-in period of the study included dyskinesia (9.3% of subjects), ON and OFF phenomenon and fall (each reported in 5.3% of subjects). During the maintenance period of the clinical trial, 2% of patients in both the ND0612 and oral IR-LD/CD-only groups reported dyskinesia. Additionally, only 6% of study participants treated with ND0612 discontinued the trial during the maintenance period due to any reason – including 5% due to AEs – compared to discontinuation rates of 6% and 3%, respectively, of study participants in the oral IR-LD/CD groups.[2] About ND0612 ND0612 is an investigational drug-device combination therapy – a 24-hours/day, continuous subcutaneous (SC) infusion of liquid levodopa/carbidopa (LD/CD) for the treatment of motor fluctuations in people with Parkinson's disease (PD). Development of investigational ND0612 is being led by NeuroDerm, Ltd., a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on X (formerly Twitter), Facebook and LinkedIn. About Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future". MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction and additionally working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to . About NeuroDerm, Ltd. NeuroDerm, Ltd. is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), based in Israel, inspired to reduce disease burden and improve the quality of life of patients and their families through innovative drug-device combination therapies and technologies. NeuroDerm is an integrated pharmaceutical and medical technology company developing central nervous system (CNS) product candidates. For additional information, please visit NeuroDerm's website at or follow the Company on LinkedIn. Media inquiries: [email protected] 1 Parkinson's Disease questionnaire. Oxford University Innovation. Published August 31, 2023. 2 Espay A, Stocchi F, Pahwa R, et al (2024). Safety and efficacy of continuous subcutaneous levodopa-carbidopa infusion (ND0612) for Parkinson's disease with motor fluctuations (BouNDless): a phase 3, randomized, double-blind, double-dummy, multicentre trial. Lancet Neurol. (24)00052-8. SOURCE Mitsubishi Tanabe Pharma America WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

OXFORD, UK and CLEVELAND, Ohio, US, 11th September 2024 – The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, aimed at driving cutting-edge rare disease breakthroughs, announces the appointment of Baroness Nicola Blackwood to the OHC Advisory Council. In her position on OHC’s Advisory Council, Baroness Blackwood will contribute to advancing OHC’s mission to drive life-changing discoveries in rare disease, with a particular focus on addressing critical unmet needs in the UK. Baroness Blackwood is Chair of both Genomics England and Oxford University Innovation. She is also a Board Member of the biotechnology company BioNTech and investment fund RTW Biotech Opportunities. She brings extensive experience in healthcare policy and innovation, including her involvement in the UK’s impactful 100,000 Genomes Project, which continues to shape the lives of those with rare diseases. She has also led on NHS innovation, UK rare disease and regulation, and global health security through her previous roles as Minister in the UK Government’s Department for Health and Social Care. Her extensive professional experience combined with her own personal journey since being diagnosed with Ehlers-Danlos Syndrome will bring a unique perspective to OHC’s Advisory Council. Professor Sir John Bell, President of the Ellison Institute of Technology (EIT) Oxford, member of the OHC Steering Committee and Chair of the OHC Advisory Council, said: “We are delighted to welcome Nicola to the OHC Advisory Council. Nicola’s extensive experience in healthcare policy and her dedication to advancing medical innovation makes her the perfect fit for our mission. We look forward to working with her to transform cutting-edge science into life-changing therapies for those in need.” Baroness Nicola Blackwood commented: “Having dedicated my career to improving healthcare outcomes for patients with rare diseases, I am honoured to be joining OHC’s Advisory Council. From both a professional and personal perspective, I have witnessed how much diagnosis and treatment of a rare disease can change someone’s life. I am delighted to be joining OHC at this pivotal stage as they continue to progress towards driving meaningful change by developing innovative solutions for patients in need.” Matthew Wood, Director and Chief Scientific Officer of the OHC, and Professor of Neuroscience in Oxford’s Department of Paediatrics, added: “Nicola’s dedication to UK life sciences and the rare disease community is truly inspiring, and her advocacy efforts have significantly advanced healthcare in this area. Her contribution to shaping healthcare policy and her unwavering commitment to patients makes her a vital asset in achieving our objectives. We are all thrilled that Nicola has decided to join us at the OHC, and I look forward to collaborating with her to reach our goal of delivering 40 rare disease drugs in the next ten years.” The OHC Advisory Council includes the following founding members: Lord David Cameron, John Crowley, Professor Sir John Bell, Jonathan Stamler, MD, and Ronald G. Harrington. *** Notes to Editors About Baroness Nicola BlackwoodNicola is a leader in science and entrepreneurship. She is a member of the House of Lords, and Chair of Genomics England and Oxford University Innovation. She is also a Board member of the biotechnology company, BioNTech and investment fund RTW Biotech Opportunities. Nicola served as a Minister in the UK Government Department for Health and Social Care under two Prime Ministers. As Minister for Innovation, she led on Life Sciences, NHS Data and Digital Transformation, and Global Health Security. She was the first female Member of Parliament (MP) for Oxford and was elected by MPs of all parties to Chair the House of Commons Science and Tech Committee. She remains one of the youngest committee chairs in British history and the only woman to have chaired the Commons Science & Tech Committee. About Rare DiseasesMore than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects one in 10 Americans, or 10 percent of the US population. Similarly, Europe has approximately 30 million people who suffer from a rare disease. The majority of all rare diseases are genetic in origin, which means they are present throughout a person’s life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option. About Oxford-Harrington Rare Disease CentreThe Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, in Cleveland, Ohio, US. The OHC combines world-leading strengths in research and therapeutics development from across these organizations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare diseases across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | X About the University of OxfordThe University of Oxford is rated the best in the world for medicine and life sciences, and it is home to the UK’s top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. The University of Oxford’s Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. For more information, visit: www.ox.ac.uk About Harrington Discovery InstituteThe Harrington Discovery Institute at University Hospitals in Cleveland, Ohio – part of The Harrington Project for Discovery & Development – is a translational accelerator that advances promising discoveries from academic labs into the clinic for the benefit of patients and society. The institute was created in 2012 with a $50 million founding gift from the Harrington family and is a prime example of the commitment they share with University Hospitals to a Vision for a ‘Better World’. For more information, visit: HarringtonDiscovery.org| LinkedIn | X Media contacts: UK/ EU – MEDiSTRAVA Sylvie Berrebi / Mark SwallowOHC@medistrava.com US – For University HospitalsAnsley Kelmansley.kelm@UHhospitals.org