1ST EUROPEAN ATTR AMYLOIDOSIS MEETING FOR PATIENTS AND DOCTORS CHANGING THE LIVES OF PATIENTS WITH ATTR: Thursday 2 November :30 18:30 Adenauer

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1 2+3 NOV PAGE DE COUVERTURE VISUEL FLYER - SANS LOGOS - AVEC AJOUTS SUIVANTS: MAISON INTERNATIONALE - 17 BOULEVARD JOURDAN PARIS - FRANCE M A I S O N INTERNATIONALE PARIS 1 PROGRAM partnership international meeting exchange hope innovation MAISON INTERNATIONALE 17 BOULEVARD JOURDAN PARIS FRANCE administrative office: therapy

2 1ST EUROPEAN ATTR AMYLOIDOSIS MEETING FOR PATIENTS AND DOCTORS CHANGING THE LIVES OF PATIENTS WITH ATTR: Thursday 2 November :30 18:30 Adenauer Room Maison Internationale, Paris, Co-Organizers and Main topics p. 2 Welcome address p. 3 Committees & Boards p. 4 Map of the venue / Sponsors & Partners p. 5 Plenary - Thursday, Nov. 2nd PROGRAM FOR DOCTORS AND SPECIALISTS p. 6 POSTER SESSIONS p. 8 Thursday, Nov. 2nd PROGRAM FOR PATIENTS p. 13 Plenary - Friday, Nov. 3rd PROGRAM FOR PATIENTS AND DOCTORS p. 14 General information p. 16 Notes p. 17 nd Chair: David Adams () Agenda Time Session 17:30 17:35 Welcome and introduction 17:35 17:50 TTR: why is it important? 17:50 18:05 Long-term treatment of TTR-FAP: what does this mean for our patients? 18:05 18:20 Treating patients in the real world 18:20 18:30 Q&A and closing remarks PP-VYN-EUR-0274 Date of preparation: October 2017 David Adams () Neurologist Laura Obici (Italy) Internal Medicine Isabel Conceição (Portugal) Neurologist Lucía Galán (Spain) Neurologist Chaired by David Adams SUMMARY SUMMARY Reflecting on long-term safety and efficacy data 1

3 CO-ORGANIZERS & MAIN TOPICS 2 CO-ORGANIZERS WELCOME ADDRESS DOCTORS ATTR amyloidosis comprises a group of rare multisystem diseases including non-hereditary wild type ATTR amyloidosis (also known as senile cardiac amyloidosis, senile systemic amyloidosis), Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FAC). David ADAMS Philip HAWKINS Hartmut SCHMIDT Germany PATIENTS Jean-Christophe FIDALGO Koenraad VERHAGEN The Netherlands MAIN TOPICS NEW TOOLS FOR DIAGNOSIS EPIDEMIOLOGY, GENOTYPES, PHENOTYPES AND NATURAL HISTORY OF ALL TYPES UPDATES IN THE TREATMENT END ORGAN TARGETED THERAPIES Susanne BERGLUND & Ove REHNBERG Sweden FAP was first described in the 1950s in Portugal presenting as autosomal dominant disease, whereas wild type ATTR amyloidosis was recognized in the 1980s as a non-hereditary form of restrictive cardiomyopathy in older patients. ATTR amyloidoses are global diseases that are now being identified in most countries. They are progressive and life-threatening, and diagnosis is usually delayed. The First European meeting for ATTR amyloidosis for doctors and patients will inaugurate a new era in relations between patients, between doctors, and between doctors and patients from many European countries with the ultimate aim of improving diagnosis, treatment and care of this serious disease. Advocacy and Education experts from international institutions including EURORDIS (the Voice of Rare Disease Patients in Europe), ARC (Amyloidosis Research Centre), and the ISA (International Society of Amyloidosis) will be present. Since it is the first event of this nature, we propose three interconnected meetings over the course of two days. On Day One, Patients and Doctors will each have their own meeting; on Day Two, Patients and Doctors will share a common session. During the meeting for specialists, six keynote lectures and 59 abstracts will be presented, comprising 11 oral communications and 48 posters. Final data from two major phase 3 clinical trials for TTR-FAP will be presented. Eleven national Patients Organizations will participate. Attendees will include specialists predominantly from Europe but also from USA, South America and Asia. WELCOME ADDRESS 3

4 STEERING COMMITTEE MAP DOCTORS PATIENTS David ADAMS Jean-Christophe FIDALGO Philip HAWKINS Koenraad VERHAGEN Hartmut SCHMIDT Susanne BERGLUND French Reference Center for FAP - NNERF, University College London - HONNORAT ROOM PARTNERS PLATINIUM Association Française contre l Amylose, Stichting Amyloïdose, The Netherlands 4 DOCTORS LOCAL COMMITTEE Shahram ATTARIAN Dominique LEGULUDEC Didier SAMUEL Jocelyn INAMO Laurent MAGY Michel SLAMA CHU Marseille, CHU Fort de, NNERF, Martinique CHU Limoges, NNERF, MEDICAL ADVISORY BOARD CHU P. Brousse, NNERF, Theodores KYRIAKIDES Ivailo TOURNEV Isabel CONCEICAO Sadeh MENACHE Peter VAN DEN BERGH Pablo GARCIA PAVIA Yesim PARMAN Julian GILLMORE Davide PAREYSON Alexander VRANCKEN Arnt KRISTEN Claudio RAPPEZZI Janez ZIDAR Thierry KUNTZER Mary REILLY Cyprus Portugal Israël Spain Turkey Germany Switzerland Bulgaria Belgium Italy Italy GOLD POSTERS NNERF, Juan BUADES Spain ORGANIZATIONS 5 OTHER PARTNERS ENTRANCE Giuseppe VITA Italy The Netherlands PROTHENA Slovenia ALNYLAM EXHIBITION ENTRANCE PATIENTS ADVISORY BOARD Bouke HAZENBERG EIDOS Ole SUHR INSTITUTIONAL PARTNERS The Netherlands Sweden PATIENTS ORGANIZATIONS Amyloidosis Israël Israël Asociación Española de la Enfermedad de Andrade Amyloidosis Research Consortium Spain Associação Brasileira de Paramiloidose Associação Portuguesa de Paramiloidose Italy USA Brazil Portugal Association française contre l Amylose Associazione Italiana Amiloidosi Familiare Central Eastern Europe Amyloidosis Research Cooperative Groupe Russia MAP & PARTNERS COMMITEES & BOARDS Klinik für Tranplantations medizin Muenster - Germany & Ove REHNBERG Famy-Norrbotten, Sweden IMPETO FOOD BUFFET FAMY-Norrbotten PFIZER Sweden Myeloma Patients Europe Belgium Patientenverband FAP Germany Stichting Amyloïdose Nederland The Netherlands ENTRANCE

5 11:45 PM PLENARY - THURSDAY, NOV. 2nd FOR DOCTORS AND SPECIALISTS FOR DOCTORS AND SPECIALISTS 08:00 AM WELCOME 08:30 AM INTRODUCTION 08:40 AM Chairmen: P. Van den Bergh - Belgium and M. Théaudin - Switzerland LECTURE 1 08:35 AM UPDATE IN EPIDEMIOLOGY AND PHENOTYPES IN TTR-FAP Y. Parman - Turkey 09:00 AM M. Reilly - 09:30 AM ORAL SESSION - Chairmen: D. Pareyson - Italy and P. Garcia Pavia - Spain EPIDEMIOLOGY GENOTYPES, PHENOTYPES AND NATURAL HISTORY OF ALL TYPES OF ATTR O1 O3 O4 Transthyretin A new staging system amyloidosis for cardiac ATTR cardiomyopathy: amyloidosis Genotype, phenotype, quality of life and outcome in 997 patients in the United Kingdom Transthyretin Familial Amyloid Polyneuropathy (ATTR V30M) in Cyprus: An updated epidemiological, clinical and genetic study. TTR FAP in Central and Eastern Europe T. Lane S. Andreou I. Tournev 6 O2 J. Gillmore 10:30 AM Coffee break and POSTERS SESSION 11:00 AM Chairman: C. Rapezzi - Italy LECTURE 3 Cyprus O5 O6 O7 Mass Spectrometry and Immunohistochemistry are complementary techniques for typing of cardiac amyloid T1, extracellular volume and rest myocardial blood flow mapping: a multiparametric mapping approach in ATTR cardiac amyloidosis Rethinking heart failure in TTR amyloidosis T. Rezk A. Martinez-Naharro R. Falk USA P 12:30 PM Lunch and POSTERS SESSION 01:30 PM ALNYLAM S SYMPOSIUM - Chairman: O. Suhr - Sweden 02:30 PM Chairmen: P. Hawkins - and I. Conceicao - Portugal LECTURE 5 MULTIPLE SYMPTOMS, ONE DISEASE MONITORING HATTR AMYLOIDOSIS FROM HEAD TO TOE UNMET NEED FOR ATTR AMYLOIDOSIS THERAPY? O. Suhr - Sweden 02:50 PM LECTURE 6 HOW TO MONITOR TTR-FAP UNDER TREATMENT? G. Vita - Italy 03:20 PM Bulgaria P IMAGING BY MRI IN TTR AMYLOIDOSIS M. Fontana - 11:20 AM ON NEW TOOLS FOR DIAGNOSIS INCLUDING SCINTIGRAPHIC IMAGING AND CARDIAC MRI LECTURE 2 WHERE ARE WE NOW AND WHERE ARE WE GOING IN FAP? ORAL SESSION - Chairmen: J. Gillmore - and A. Kristen - Germany ORAL SESSION - Chairmen: M. Reilly - and M. Slama - O8 O9 O10 O11 Safety and efficacy of Inotersen in patients with hereditary transthyretin amyloidosis with polyneuropathy (hattr-pn) Patisiran, an investigational RNAi therapeutic for patients with hereditary transthyretinmediated (hattr) amyloidosis with polyneuropathy: Results from the Phase 3 APOLLO study Death cause analysis and temporal trends in survival after liver transplantation for transthyretin familial amyloid polyneuropathy Proposal for a minimal neuropathy assessment protocol for ATTR amyloidosis in routine clinical practise M. Benson D. Adams M. Slama M. Reilly LECTURE 4 MULTIMODAL IMAGING OF CARDIAC AMYLOIDOSIS IN FAP M. Slama - PLENARY - THURSDAY, NOV. 2nd FOR DOCTORS AND SPECIALISTS PLENARY - THURSDAY, NOV. 2nd USA P 04:30 PM Coffee break and POSTERS SESSION 05:30 PM SYMPOSIUM PFIZER - Chairman: D. Adams - 06:30 PM CONCLUSION CHANGING THE LIVES OF PATIENTS WITH ATTR: REFLECTING ON LONG-TERM SAFETY AND EFFICACY DATA 7

6 POSTER SESSION - THURSDAY Chairmen: J. INAMO - Martinique GENOTYPE, PHENOTYPE P14 The frequency of wild-type transthyretin amyloidosis in Russia according to the results of an autopsy study A. Poliakova - Russia P15 Echocardiographic characteristics of patients with localised wild-type transthyretin amyloidosis B. Pawarova - Russia P16 Machine Learning Predicts Mortality Better than Biomarker Staging in WildType Transthyretin Cardiac Amyloidosis A. Singh - USA P17 Characterization of wild-type transthyretin amyloidosis among women: preliminary results from an international multicenter study C. Quarta - 8 P1 Genetic diagnosis in ATTR amyloidosis; a single UK Centre 26 year experience. D. Rowczenio - P2 Clinical genetic correlations in Bulgarian TTR FAP cohort ten years observations S. Sarafov - Bulgaria P3 Genotypic and phenotypic presentation of Val30Met mutation in a Turkish cohort A. Çakar - Turkey P4 Transthyretin amyloidosis in Slovenia J. Zidar - Slovenia P6 Late-onset transthyretion familial amyloid polyneuropathy: characterization of Brazilian subjects from the THAOS registry M. Waddington Cruz - Brazil P7 Amyloidosis Research Consortium cardiac amyloidosis survey: Results from patients with ATTR amyloidosis and their caregivers I. Lousada - USA Chairmen: G. VITA - Italy PHENOTYPIC VARIABILITY P8 Phenotypic variability of TTR Val122Ile mutation: a Caucasian patient with axonal neuropathy and normal heart after two years follow up C. Stancanelli - Italy P9 The c G>C mutation in the transthyretin gene is Amyloidogenic S. Khella - USA P10 Rare cases of onset with cardiac involvement in a large cohort of patients with Val30Met Transthyretin amyloidosis from Majorca focus T. Rhippol-Vera - Spain P11 A Val30Met sporadic familial amyloid polyneuropathy case with atypical presentation: Upper limb onset of symptoms E. Sahin - Turkey P12 Proteinuric Renal Phenotype in the Mallorca Cohort of Hereditary Transthyretin Amyloidosis Patients A. Ferrer-Nadal - Spain P13 Ocular Manifestations in S77T transthyretin-related familial amyloid polyneuropathy R. Bunod - WILD TYPE TTR AMYLOIDOSIS Chairmen: B. HAZENBERG - The Netherlands CLINICAL BIOLOGY P18 Serum Transthyretin Levels Are Significantly Lower in Val122Ile Cardiomyopathy Compared With Wild-Type Transthyretin Cardiomyopathy A. Singh - USA P19 Investigation of the protein quality control in patients with ATTR amyloidosis C. Niemetz - Germany P20 Stem cell-based hepatocyte model for assessment of tranthyretin tetramer stabilization A. Zibert - Germany P21 Mutual Dependence of TTR and SERPINA1 in ATTR Amyloidosis C. Niemetz - Germany P22 Immortalization of primary cells derived from ATTR patients P. Ballmaier - Germany P23 Mechano-enzymatic mechanism of transthyretin amyloidogenesis: long distance effects of optimally effective inhibitors PP. Mangione - P24 Serum protein electrophoresis (SPE): a review in Hereditary Transthyretin Amyloidosis J. Buades - Spain POSTER SESSION POSTER SESSION THEME 1 - Chairmen: T. KYRIAKIDES - Cyprus 9

7 THEME 2 - Chairmen: M. SLAMA - and I. TOURNEV - Bulgaria POSTER SESSION NEW TOOLS 10 P25 Diagnostic accuracy of 99mTc-DPD scintigraphy for detecting ATTR cardiac amyloid deposits D. Hutt - P26 Spectrum and significance of CMR findings in cardiac transthyretin amyloidosis A. Martinez-Naharro - P27 Clinical utility of T1 mapping in cardiac ATTR amyloidosis diagnostic performance and prognostic capability A. Martinez-Naharro - P28 The Effect of Tracer Kinetics on Heart to Contralateral Ratio in 99mTc-DPD Scintigraphy J. Page - P29 The relationship between 99mTc-DPD uptake and amyloid fibril composition in hereditary cardiac TTR amyloidosis: is the Glu92Lys variant an exception to the rule? L. Obici - Italy P30 Comparison of immunohistochemistry and proteomic analysis for identification and typing of amyloid in different histological tissues JA. Gilbertson - P31 Screening for transthyretin amyloidosis by using abdominal fat ultrasonography Y. Misumi - Japan P32 A novel serum microrna signature to screen ATTR M. Aguennouz - Italy P33 Strange location for diagnosis of ATTR amyloidosis M. Raya-Cruz - Spain P34 Peripheral nerve ultrasonography in patients with transthyretin amyloidosis J. Zidar - Slovenia P37 P39 Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in transthyretin familial amyloid polyneuropathy L. Amass - USA P40 Reasons for Discontinuation of TTR stabilizers and/or TTR fibril disrupter: An Analysis of Baseline Demographics of Patients with Hereditary ATTR (hattr) Amyloidosis in the Phase 3 APOLLO study H. Schmidt - Germany Chairmen: Y. Parman - Turkey J Gillmore - Characteristics of patients with hereditary transthyretin-mediated amyloidosis on the US liver transplant waiting list H. Schmidt - Germany P42 Cardiac involvement after liver transplantation in patients with Val30Met Transthyretin amyloidosis from Majorca focus T. Ripoll-Vera - Spain P43 Cardiomyopathy and peripheral polyneuropathy progression in patients with hereditary transthyretin-related amyloidosis associated with Glu8 9 Gln mutation treated with Tafamidis M. Gospodinova - Bulgaria P44 Long-term treatment of ATTR with tafamidis: the Sicilian experience L. Gentile - Italy P45 Long-term, open-label clinical experience with patisiran, an investigational RNAi therapeutic for patients with hereditary transthyretin-mediated (hattr) amyloidosis with polyneuropathy JL. Berk - USA P46 Rationale and Design of the Phase 3 Transthyretin Amyloid Cardiomyopathy Tafamidis Clinical Trial (ATTR-ACT) M. Waddington Cruz - Brazil Treatment of ATTR cardiomyopathy with a TTR specific antisense oligonucleotide N. Dasgupta - USA P47 Tafamidis delays disease progression in TTR familial amyloid polyneuropathy: supportive analyses from a pivotal trial D. Keohane - USA Decreased S100A8/A9 in V30M related familial amyloid polyneuropathy: a possible pathway in misregulation of Schwann cells J. Moreira - Portugal P48 Real-life ILUVIEN (Fluocinolone Acetonide) case sudy: rapid drying of the macula edema and improved vision within 1 Year after therapy initiation in ATTRV30M retinal angiopathy J. Beirao - Portugal UPDATES IN THE TREATMENT P36 Long-term safety and efficacy of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy: results up to 6 years F. Barroso - Argentina P41 THEME 3 - Chairmen: J. BUADES - Spain P35 P38 Tafamidis delays disease progression comparably across Val30Met and nonval30met genotypes in transthyretin familial amyloid polyneuropathy B. Gundapaneni - USA POSTER SESSION 11

8 THURSDAY, NOV. 2nd 08:45 AM WELCOME 08:50 AM PATIENT DELEGATES PRESENT THEMSELVES 09:15 AM CHAIRMAN: S. BERGLUND - Sweden MEDICINES COMBATING HEREDITARY ATTR POLYNEUROPATHY A. Vrancken - The Netherlands POSTER SESSION 09:45 AM DIFFERENCES IN ACCESS TO MEDICINES BETWEEN COUNTRIES C. Langenstroer & A. Albers - Germany 10:15 AM Coffee break 10:45 AM CHAIRMAN: K. VERHAGEN - The Netherlands TREATMENT OF WILD TYPE ATTR J. Gillmore - 11:15 AM BEST PRACTICES OF POS. LEARNING FROM EACH OTHER PO delegates Progressive diseases require 12 transformational science. 12:15 PM Lunch break 01:45 PM CHAIRMAN: G. D ALESSIO - Italy ISA MEETS THE PATIENTS: UNMET NEEDS AND CONCERTED ACTIONS PLENARY - THURSDAY, NOV. 2nd FOR PATIENTS ORGANIZATIONS FOR PATIENTS ORGANIZATIONS G. Merlini, Vice-president ISA - Italy 02:15 PM HOW PATIENTS OF A RARE DISEASE CAN ORGANISE THEMSELVES? THE CASE OF MYELOMA PATIENTS EUROPE H. Scheurer, President Myeloma Europe - Belgium 02:45 PM Ionis, the leader in antisense technology, is committed to helping patients who suffer from serious diseases. WHAT A PATIENT DRIVEN, MULTI-STAKEHOLDER ORGANISATION CAN DO AND ACHIEVE? THE CASE OF THE ARC (AMYLOIDOSIS RESEARCH CONSORTIUM) I. Lousada, ARC President/CEO - USA 03:15 PM Coffee Break 03:45 PM CHAIRMAN: J-C FIDALGO - Revolutionizing Medicine. Saving Lives. NETWORKING AND ORGANIZING OURSELVES AT EUROPEAN LEVEL AND BEYOND G. d Alessio - Italy G. Fischer - Austria F. Figueiredo de Almeida - Brazil 05:15 PM End of Day One 13

9 FOR PATIENTS AND DOCTORS PLENARY - FRIDAY, NOV. 3rd FOR PATIENTS AND DOCTORS 08:30 AM 14 PLENARY - FRIDAY, NOV. 3rd AFTERNOON FOR PATIENTS AND DOCTORS READ OUT DOCTORS DAY 02:00 PM READ OUT PATIENTS DAY 02:30 PM P. Hawkins - 09:00 AM P. Westermark - Sweden K. Verhagen - The Netherlands 09:30 AM ESTIMATING THE GLOBAL PREVALENCE OF TTR-FAP H. Schmidt - Germany 10:00 AM OPEN PANEL DISCUSSION ON ACCESS TO THERAPY Chairman: G. Merlini - Italy 10:30 AM Coffee break 11:00 AM 11:30 AM UPDATE ON SIRNA TRIALS (PATISIRAN, ALNYLAM) A HOLISTIC APPROACH TOWARDS CURE AND CARE OF ATTR PATIENTS I. Conceição - Portugal 03:00 PM THE CASE OF EDUCATION THERAPEUTIQUE C. Cauquil - 03:15 PM PLACE OF NURSES IN REFERENCE CENTERS Janita Bulthuis - The Netherlands Marine de Lambert - Daniele Gallo - Italy Thirusha Lane - Victorira Eugenia Llabres Esquilas - Spain D. Adams - 03:30 PM Coffee break UPDATE ON ANTISENSE TRIALS (INOTERSEN, IONIS) 04:00 PM FINAL PANEL DISCUSSION ON MOVING FORWARD Chairman: J-C. Fidalgo - M. Benson - USA 11:55 AM OPEN PANEL DISCUSSION TREATING ATTR AMYLOIDOSIS Chairman: H. Schmidt - Germany 12:25 PM SCREENING STRATEGIES IN IDENTIFYING ATTR PATIENTS LESSONS LEARNT FROM EURORDIS M. Bolz-Johnson - Germany 01:00 PM Lunch break 05:00 PM End of Day Two. Take home messages PLENARY - FRIDAY, NOV. 3rd FOR PATIENTS AND DOCTORS PLENARY - FRIDAY, NOV. 3rd MORNING 15

10 GENERAL INFORMATION ACCESS NOTES 16 Cité Universitaire Porte d Orléans Cité Universitaire This stop is accessible from both Airports Charles de Gaulle and Orly 21 Cité Universitaire 88 Jourdan-Montsouris 67 Stade Charléty SOCIAL EVENT WEDNESDAY NOV. 1st Meet and Greet Wine & Cheese cocktail from 6.00 PM to 8.00 PM at La Maison Internationale - Salon Gulbenkian THURSDAY NOV. 2nd Join us for the congress event to be held at the Musée d Orsay. Discover one of the most beautiful monuments of Paris. Enjoy a visit of the Musée d Orsay from from 6:30 PM or 7:30 PM followed by a dinner at the restaurant of the Musée d Orsay at 8:30 PM. REGISTRATION FEES Registration fees include: attendance to all sessions, posters session, lunches and coffee breaks. NOTES GENERAL INFORMATION MAISON INTERNATIONALE - CITÉ UNIVERSITAIRE 17 boulevard Jourdan Paris 17

11 To those who say impossible, impractical, unrealistic, we say: CHALLENGE ACCEPTED We are relentless in our pursuit of new treatments. Because patients shouldn t have to wait for hope. alnylam.com 2017 Alnylam Pharmaceuticals, Inc.

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