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Anetumab Ravtansine
June 2016 Company Update Safe Harbor This presentation includes forward-looking statements. Actual results could differ materially from those included in the forward-looking statements due to various risk factors and uncertainties including changes in business, economic competitive conditions, regulatory reforms, foreign exchange rate fluctuations and the availability of financing. These and other risks and uncertainties are detailed in the Company’s Annual Report. © MorphoSys AG, Company Update - June 2016 2 MorphoSys at a Glance MorphoSys is developing a pipeline of truly differentiated therapeutic antibodies built using proprietary technologies Munich, Germany-based biopharmaceutical company The industry’s largest antibody therapeutic pipeline assembled using proprietary technologies: 104 active therapeutic programs 26 antibodies in clinical trials Growing portfolio of attractive proprietary assets Strong balance sheet with recurring cash-flows supports growing investment in R&D Successful track-record of partnering world-wide Listed on the German TecDAX © MorphoSys AG, Company Update - June 2016 3 The MorphoSys Pipeline 26 Clinical Product Candidates, 104 Total Most advanced development stage Program Partner Target Disease Area Discovery Preclinic Phase 1 Phase 2 Phase 3 Bimagrumab (BYM338) Novartis ActRIIB Musculoskeletal diseases Guselkumab (CNTO1959) Janssen IL23p19 Psoriasis Gantenerumab Roche Amyloid-ß Alzheimer’s disease MOR208 - CD19 ALL, CLL, NHL MOR202 - CD38 Multiple myeloma MOR103/GSK3196165 GSK GM-CSF Inflammation -
9.0 Bn 23 000 200 +
42 | Novartis Annual Report 2017 Innovation The Novartis Institutes for BioMedical Research works in concert with our Global Drug Development group to bring innovative treatments to patients around the world. In 2017, we advanced our drug discovery and development efforts by encouraging greater collaboration and out-of-the-box thinking, exploring new approaches that could improve how we work, and investing in promising tools and technologies. We made progress in priority disease areas with high unmet medical needs. We also marked several key milestones, including US FDA approval – the first of its kind – for a type of personalized cell therapy that could change the course of cancer care. 9.0 bn 23 000 200 + Research and development Scientists, physicians and business Projects in clinical development spending in 2017, amounting to professionals working in research 18.3% of net sales (USD) and development worldwide Collaborative Efficient, effective Progress in important science drug development disease areas We are increasing collaboration We are using digital technology We highlight areas of our work where in research as we try to leverage and data analysis to make drug we are driving significant innovation, innovation from a variety of sources. development swifter and more or where we can potentially have an We are also exploring ways to effective. And we are taking steps important impact on patients and harness digital technology in drug to strengthen our pipeline. public health. discovery, as well as new therapeutic k page 46 k Immuno-oncology page 48 approaches such as cell therapies. k Multiple sclerosis page 50 k page 43 k Liver disease page 52 k Ophthalmology page 53 k Asthma page 55 k Malaria page 56 INNOVATION Novartis Annual Report 2017 | 43 Discovery For new treatments, the journey from laboratory to patient Promoting open innovation starts in the discovery phase where researchers try to Our efforts to increase the flow of ideas between identify potentially groundbreaking therapies. -
Stock Price Effects of Breakthrough Therapy Designation
NEWS & ANALYSIS SUPPLEMENTARY INFORMATION BIOBUSINESS BRIEFS In format as provided by the authors Stock price effects of breakthrough therapy designation David Hoffmann, Shane Van Dalsem and Frank S. David NATURE REVIEWS | D RU G D IS COVERY Supplementary Box 1 | Methods and data 1. Methods 1a. Sample definition and data collection Event data collection The website Friends of Cancer Research (http://www.focr.com/) lists all publicly available Breakthrough Therapy Designations (BTD), which were downloaded with a cut-off on 06/30/18. For partnered products, we treated each partner as if it had independently received the BTD, and thus generated an entry for each co-developing company. For each BTD, we identified the original press release and excluded companies that did not disclose the exact BTD announcement date. Further, we excluded companies that are not publicly traded on a US stock exchange, and those that had incomplete stock price data in the event period (as of 08/24/18). We also excluded BTDs that coincided with major corporate press releases (e.g., quarterly results or clinical trial news). In cases where the same company announced several BTDs on the same date, we retained only one for analysis. This yielded 146 BTDs (102 commercial, 44 pre-commercial). Subsequently, we identified three outliers and excluded them from the final CAAR analysis (see later section). 1 Stock price collection The historical closing stock prices (unadjusted) for the range of -111 days to +90 days around the disclosure of the BTD events were downloaded from https://finance.yahoo.com/. Historical stock prices from delisted companies were downloaded from https://amigobulls.com or https://barchart.com. -
Tables-Of-Phase-3-Mabs.Pdf
Table 3. Monoclonal antibodies in Phase 2/3 or 3 clinical studies for cancer indications Most advanced Primary sponsoring company INN or code name Molecular format Target(s) phase Phase 3 indications Therapeutic area Janssen Research & Development, LLC JNJ-56022473 Humanized mAb CD123 Phase 2/3 Acute myeloid leukemia Cancer Murine IgG1, Actinium Pharmaceuticals Iomab-B radiolabeled CD45 Phase 3 Acute myeloid leukemia Cancer Humanized IgG1, Seattle Genetics Vadastuximab talirine ADC CD33 Phase 3 Acute myeloid leukemia Cancer TG Therapeutics Ublituximab Chimeric IgG1 CD20 Phase 3 Chronic lymphocytic leukemia Cancer Xencor XMAB-5574, MOR208 Humanized IgG1 CD19 Phase 2/3 Diffuse large B-cell lymphoma Cancer Moxetumomab Murine IgG1 dsFv, AstraZeneca/MedImmune LLC pasudotox immunotoxin CD22 Phase 3 Hairy cell leukemia Cancer Humanized scFv, Viventia Bio Oportuzumab monatox immunotoxin EpCAM Phase 3 Bladder cancer Cancer scFv-targeted liposome containing Merrimack Pharmaceuticals MM-302 doxorubicin HER2 Phase 2/3 Breast cancer Cancer MacroGenics Margetuximab Chimeric IgG1 HER2 Phase 3 Breast cancer Cancer Gastric cancer or gastroesophageal junction Gilead Sciences GS-5745 Humanized IgG4 MMP9 Phase 3 adenocarcinoma; ulcerative colitis (Phase 2/3) Cancer; Immune-mediated disorders Depatuxizumab Humanized IgG1, AbbVie mafodotin ADC EGFR Phase 2/3 Glioblastoma Cancer AstraZeneca/MedImmune LLC Tremelimumab Human IgG2 CTLA4 Phase 3 NSCLC, head & neck cancer, bladder cancer Cancer NSCLC, head & neck cancer, bladder cancer, breast AstraZeneca/MedImmune -
A Vicious Cycle of Osteosarcopenia in Inflammatory Bowel Diseases
nutrients Review A Vicious Cycle of Osteosarcopenia in Inflammatory Bowel Diseases—Aetiology, Clinical Implications and Therapeutic Perspectives Dorota Skrzypczak *, Alicja Ewa Ratajczak , Aleksandra Szymczak-Tomczak, Agnieszka Dobrowolska , Piotr Eder and Iwona Krela-Ka´zmierczak* Department of Gastroenterology, Dietetics and Internal Diseases, Poznan University of Medical Sciences, 49 Przybyszewskiego Street, 60-355 Poznan, Poland; [email protected] (A.E.R.); [email protected] (A.S.-T.); [email protected] (A.D.); [email protected] (P.E.) * Correspondence: [email protected] (D.S.); [email protected] (I.K.-K.) Abstract: Sarcopenia is a disorder characterized by a loss of muscle mass which leads to the reduction of muscle strength and a decrease in the quality and quantity of muscle. It was previously thought that sarcopenia was specific to ageing. However, sarcopenia may affect patients suffering from chronic diseases throughout their entire lives. A decreased mass of muscle and bone is common among patients with inflammatory bowel disease (IBD). Since sarcopenia and osteoporosis are closely linked, they should be diagnosed as mutual consequences of IBD. Additionally, multidirectional treatment of sarcopenia and osteoporosis including nutrition, physical activity, and pharmacotherapy should include both disorders, referred to as osteosarcopenia. Keywords: sarcopenia; IBD; Crohn’s disease; ulcerative colitis; malnutrition; low muscle mass; low muscle strength; nutritional therapy Citation: Skrzypczak, D.; Ratajczak, A.E.; Szymczak-Tomczak, A.; Dobrowolska, A.; Eder, P.; Krela-Ka´zmierczak, I. A Vicious Cycle 1. Introduction of Osteosarcopenia in Inflammatory Bowel Diseases—Aetiology, Clinical Sarcopenia is a syndrome associated with a reduction of skeletal muscle mass, leading Implications and Therapeutic to decreased muscle strength [1]. -
Monoclonal Antibodies As Neurological Therapeutics
pharmaceuticals Review Monoclonal Antibodies as Neurological Therapeutics Panagiotis Gklinos 1 , Miranta Papadopoulou 2, Vid Stanulovic 3, Dimos D. Mitsikostas 4 and Dimitrios Papadopoulos 5,6,* 1 Department of Neurology, KAT General Hospital of Attica, 14561 Athens, Greece; [email protected] 2 Center for Clinical, Experimental Surgery & Translational Research, Biomedical Research Foundation of the Academy of Athens (BRFAA), 11527 Athens, Greece; [email protected] 3 Global Pharmacovigilance, R&D Sanofi, 91385 Chilly-Mazarin, France; vid.stanulovic@sanofi.com 4 1st Neurology Department, Aeginition Hospital, National and Kapodistrian University of Athens, 11521 Athens, Greece; [email protected] 5 Laboratory of Molecular Genetics, Hellenic Pasteur Institute, 129 Vasilissis Sophias Avenue, 11521 Athens, Greece 6 Salpetriere Neuropsychiatric Clinic, 149 Papandreou Street, Metamorphosi, 14452 Athens, Greece * Correspondence: [email protected] Abstract: Over the last 30 years the role of monoclonal antibodies in therapeutics has increased enormously, revolutionizing treatment in most medical specialties, including neurology. Monoclonal antibodies are key therapeutic agents for several neurological conditions with diverse pathophysio- logical mechanisms, including multiple sclerosis, migraines and neuromuscular disease. In addition, a great number of monoclonal antibodies against several targets are being investigated for many more neurological diseases, which reflects our advances in understanding the pathogenesis of these -
AHRQ Healthcare Horizon Scanning System – Status Update
AHRQ Healthcare Horizon Scanning System – Status Update Horizon Scanning Status Update: January 2014 Prepared for: Agency for Healthcare Research and Quality U.S. Department of Health and Human Services 540 Gaither Road Rockville, MD 20850 www.ahrq.gov Contract No. HHSA290201000006C Prepared by: ECRI Institute 5200 Butler Pike Plymouth Meeting, PA 19462 January 2014 Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290201000006C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. A novel intervention may not appear in this report simply because the System has not yet detected it. The list of novel interventions in the Horizon Scanning Status Update Report will change over time as new information is collected. This should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual target technology reports are developed for those that appear to be closer to diffusion into practice in the United States. A representative from AHRQ served as a Contracting Officer’s Technical Representative and provided input during the implementation of the horizon scanning system. AHRQ did not directly participate in the horizon scanning, assessing the leads or topics, or provide opinions regarding potential impact of interventions. -
Inflammatory Myopathies: Update on Diagnosis, Pathogenesis and Therapies, and COVID-19-Related Implications
Thomas Jefferson University Jefferson Digital Commons Department of Neurology Faculty Papers Department of Neurology 12-1-2020 Inflammatory myopathies: update on diagnosis, pathogenesis and therapies, and COVID-19-related implications. Marinos C. Dalakas Follow this and additional works at: https://jdc.jefferson.edu/neurologyfp Part of the Neurology Commons Let us know how access to this document benefits ouy This Article is brought to you for free and open access by the Jefferson Digital Commons. The Jefferson Digital Commons is a service of Thomas Jefferson University's Center for Teaching and Learning (CTL). The Commons is a showcase for Jefferson books and journals, peer-reviewed scholarly publications, unique historical collections from the University archives, and teaching tools. The Jefferson Digital Commons allows researchers and interested readers anywhere in the world to learn about and keep up to date with Jefferson scholarship. This article has been accepted for inclusion in Department of Neurology Faculty Papers by an authorized administrator of the Jefferson Digital Commons. For more information, please contact: [email protected]. ACTA MYOLOGICA 2020; XXXIX: p. 289-301 doi:10.36185/2532-1900-032 Inflammatory myopathies: update on diagnosis, pathogenesis and therapies, and COVID-19-related implications Marinos C. Dalakas Department of Neurology, Thomas Jefferson University, Philadelphia, PA USA and the Neuroimmunology Unit, National and Kapodistrian University University of Athens Medical School, Athens, Greece The inflammatory myopathies constitute a heterogeneous group of acquired my- opathies that have in common the presence of endomysial inflammation. Based on steadily evolved clinical, histological and immunopathological features and some autoantibody associations, these disorders can now be classified in five character- istic subsets: Dermatomyositis (DM) Polymyositis (PM), Necrotizing Autoimmune Myositis (NAM), Anti-synthetase syndrome-overlap myositis (Anti-SS-OM), and Inclusion-Body-Myositis (IBM). -
Lääkeaineiden Yleisnimet (INN-Nimet) 21.6.2021
Lääkealan turvallisuus- ja kehittämiskeskus Säkerhets- och utvecklingscentret för läkemedelsområdet Finnish Medicines Agency Lääkeaineiden yleisnimet (INN-nimet) 21.6. -
(INN) for Biological and Biotechnological Substances
INN Working Document 05.179 Update 2013 International Nonproprietary Names (INN) for biological and biotechnological substances (a review) INN Working Document 05.179 Distr.: GENERAL ENGLISH ONLY 2013 International Nonproprietary Names (INN) for biological and biotechnological substances (a review) International Nonproprietary Names (INN) Programme Technologies Standards and Norms (TSN) Regulation of Medicines and other Health Technologies (RHT) Essential Medicines and Health Products (EMP) International Nonproprietary Names (INN) for biological and biotechnological substances (a review) © World Health Organization 2013 All rights reserved. Publications of the World Health Organization are available on the WHO web site (www.who.int ) or can be purchased from WHO Press, World Health Organization, 20 Avenue Appia, 1211 Geneva 27, Switzerland (tel.: +41 22 791 3264; fax: +41 22 791 4857; e-mail: [email protected] ). Requests for permission to reproduce or translate WHO publications – whether for sale or for non-commercial distribution – should be addressed to WHO Press through the WHO web site (http://www.who.int/about/licensing/copyright_form/en/index.html ). The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. Dotted lines on maps represent approximate border lines for which there may not yet be full agreement. The mention of specific companies or of certain manufacturers’ products does not imply that they are endorsed or recommended by the World Health Organization in preference to others of a similar nature that are not mentioned. -
Thesis Versie 38 Without Cover.Indd 1 9-3-2020 15:10:38 © 2020 Mohsin El Amrani Cover Image: Adobe Stock Lay-Out and Design By: M
Quanti fi cati on of therapeuti c anti LC-MS/MSproteins with bodies and endogenous c on of therapeuti ficati Quanti Amrani Mohsin El Quanti fi cati on of therapeuti c anti LC-MS/MS proteins with bodies and endogenous c on of therapeuti ficati Quanti Quanti fi cati on of therapeuti c Quanti fi cati on of therapeuti c UITNODIGINGUITNODIGING anti bodies and endogenous proteins voor het bijwoonen van anti bodies and endogenous proteins voorde hetopenbare bijwoonen verdediging van with LC-MS/MS de openbarevan het verdediging proefschrift with LC-MS/MS van het proefschrift Quanti fi cati on of Quanti fi cati on of Mohsin El Amrani therapeuti c anti bodies Mohsin El Amrani therapeutiand endogenous c anti bodies and proteinsendogenous with proteinsLC-MS/MS with LC-MS/MS op donderdag 28 mei 2020 op donderdagom 14:3028 Mei uur 2020 om 14:30 uur Academiegebouw AcademiegebouwUniversiteit Utrecht UniversiteitDomplein 29Utrecht te Utrecht Domplein 29 te Utrecht Aansluitend recepti e ter Aansluitendplaatse recepti e ter plaatse Paranimfen Paranimfen Faiza El Amrani ([email protected])Faiza El Amrani ([email protected]) Gerard Graat Gerard Graat ([email protected]) Mohsin El Amrani ([email protected]) Mohsin El Amrani Mohsin El Amrani [email protected] [email protected] 2020 2020 Cover.indd 1 4-3-2020 15:53:28 Cover.indd 1 9-3-2020 12:54:39 Quantification of therapeutic antibodies and endogenous proteins with LC-MS/MS Thesis Versie 38 without cover.indd 1 9-3-2020 15:10:38 © 2020 Mohsin El Amrani Cover image: Adobe Stock Lay-out and design by: M. -
Rare Diseases, When Taken Together, Are Are Together, When Taken Diseases, Rare to According at All
2013 MEDICINES IN DEVELOPMENT REPORT Rare Diseases A Report on Orphan Drugs in the Pipeline PRESENTED BY AMERICA’s biopharmACEUTICAL RESEARCH COMPANIES More Than 450 Medicines in Development for Rare Diseases Rare diseases, when taken together, are A major area of this research targets Orphan Drugs in Development* not that rare at all. In fact, according to rare cancers, accounting for more than Application the National Institutes of Health (NIH), one-third of all rare disease medicines in Submitted 30 million Americans have one of the development. Other top research areas Phase III nearly 7,000 diseases that are officially include genetic disorders, neurologi- Phase II deemed “rare” because alone they each cal conditions, infectious diseases and Phase I affect fewer than 200,000 people in autoimmune disorders. the United States. Sometimes, only Despite some recent victories, research a few hundred patients are known to 105 into treatments for rare diseases is a have a particular rare disease. daunting quest. This ongoing innovation Simply receiving a diagnosis of a rare and the hundreds of new medicines in disease often becomes a frustrating development now offer hope that physi- 85 quest, since many doctors may have nev- cians will have new treatment options er before heard of or seen the disease. for patients confronting a rare disease. This is, however, a time of great progress and hope. Biopharmaceutical 65 Contents research is entering an exciting new era Innovative Orphan Drugs with a growing understanding of the in the Pipeline ......................................... 2 human genome. Scientific advances have given researchers new tools to Orphan Drug Approvals ...........................4 explore rare diseases, which are often Challenges in Clinical Trials ......................6 more complex than common diseases.