Prosensa Therapeutics R&D in ultra-rare disease
|
|
- Joella Chapman
- 6 years ago
- Views:
Transcription
1 Prosensa Therapeutics R&D in ultra-rare disease European Business Development Conference Dusseldorf, September 24, 2013 Tina C Flatau VP Alliances and Project Management
2 Forward-Looking Statements This presentation may contain statements that constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of Forwardlooking statements are statements other than historical fact and may include statements that address future operating, financial or business performance or Prosensa s strategies or expectations. In some cases, you can identify these statements by forward-looking words such as may, might, will, should, expects, plans, anticipates, believes, estimates, predicts, projects, potential, outlook or continue, and other comparable terminology. Forward-looking statements are based on management s current expectations and beliefs and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. These risks and uncertainties include, but are not limited to, the timing and conduct of clinical trials of drisapersen and Prosensa s other product candidates, plans to pursue research and development of product candidates for DMD and other indications, the clinical utility of Prosensa s product candidates, the timing or likelihood of regulatory filings and approvals, Prosensa s intellectual property position, expectations regarding payments under Prosensa s collaborations and Prosensa s competitive position. These risks and uncertainties also include those described under the captions Risk Factors and Management s Discussion and Analysis of Financial Condition and Results of Operations in Prosensa s Registration Statement on Form F-1 and future filings with the Securities and Exchange Commission. Forward-looking statements speak only as of the date they are made, and Prosensa does not undertake any obligation to update them in light of new information, future developments or otherwise, except as may be required under applicable law. All forward-looking statements are qualified in their entirety by this cautionary statement. 2
3 Our Mission To develop innovative, RNA-based therapeutics to fill unmet medical needs for patients with rare genetic diseases
4 Our story Rare disease company founded in 2002 and grown out of the University of Leiden, the Netherlands Our lead compound is in Phase III for Duchenne Muscular Dystrophy We have a pipeline of genotype-specific treatments for Duchenne and other neuromuscular diseases Our technology is an RNA modulation platform, applicable to rare and common diseases Our biggest collaboration is with GSK for selected parts of our Duchenne pipeline 4
5 BioPartner Accelerator BioPartner Incubator Key collaboration University Leiden Key collaboration Dutch Authorities GMP inspection Employees: 80+ PNAtix Prosensa Therapeutics BV Dutch Authorities GMP licence Start PRO051 Clinical phase III Employees: 39 Start PRO044 Clinical phase I/II 2002 Employees: 7 Start PRO051 Clinical phase I/II Employees: 3 First human study
6 Duchenne Muscular Dystrophy Age walking problems wheel chair - skeletal deformity very limited use of arms ventilation at night ventilation 24 h death Clinical symptoms
7 Duchenne Muscular Dystrophy Cause: no dystrophin protein in muscles
8 Dystrophin
9 R&D Pipeline Indication Compound Discovery Pre-clinical Phase I/II Phase III Duchenne Myotonic Dystrophy Huntington s Disease PRO051 PRO044 PRO045 PRO053 PRO052 PRO055 PROSPECT PRO135 PRO289 drisapersen License GSK Option GSK Unencumbered Prosensa
10 the biotech dilemma to partner or not to partner
11 Biopharma and the importance of collaborations Emerging biotech companies Academic background and links Good IP basis Work virtually used to collaborations Innovative, energetic Need Good friends advisors, investors, backers A flow of $$ to keep working and grow Guidance A realistic growth/ game plan Good connections 11
12 Why collaborate with Big Pharma? Development is too costly for innovator companies Research Preclinical Clinical 12
13 13
14 Why collaborate with Big Pharma? Experience Guidance Learning Proven track record Get you there safely A guiding partner focused on getting you to where you want to be
15
16 Biotechnology..drug development with more twists and turns Umm I have never done it like THIS before
17 Pharma partners may be prepared to help us create the development pathway
18 and prefer a quiet collaborator
19 Rare disease development is difficult
20 Rare disease biotech to boldly go where no man has gone before
21 Expedited development and ultra-rare disease Speed Patient groups urge us to push ahead with progress choose speed over perfection DMD is ultra-rare, disabling and ultimately fatal We aim to generate evidence for expedited development of a whole series of similar treatments Challenges Ultra-rare diseases tend to not be studied well; no treatment so no diagnostic pathway; no imperative from payers to improve on an existing treatment Clinical end points yet to be validated in regulatory submissions; no proven surrogate EMA PIP applies even for rare childhood disease so you have to start out with an end to end plan FDA: Orphan drugs are held to the same statutory requirements for demonstrating effectiveness and safety
22 Patients
23 Patient Organizations Are partners on this journey of exploration They put pressure on politicians, regulators and payers They shape the development of regulatory policy They raise $$ to support research and development They help to educate us to understand life with the disease They help develop measures for outcomes and quality of life
24 the rare disease community
25 communication newborn screening creating disease awareness informing patients establishing registries
26 communication or promotion? newborn screening creating disease awareness informing patients establishing registries enlarging the market generating more revenues promotion to consumers lining up patients
27 patients as partners talking ABOUTpatients
28 balanced communication with patients information
29 Hello, We are a family from xxx. We have a son. His name is xxx and he is diagnosed with muscular dystrophy -deletion of exons 49 and 50. We have tested him in genetics labs in xxx, yyy and zzz. He was diagnosed when he was 11 years old. In August xxx will be 17. He is very determined boy, and he believes with stretching and exercising he will walk soon again. I am sending you all the test results we have, and if it is necessary we will make more or different tests. Please help our son. It is a dream of a mother and a father. We are ready to do anything that will help our son feel better.
30 USA orphan designations and approvals to 2009 A.Pariser, FDA: Orphan drugs must demonstrate substantial evidence of effectiveness/clinical benefit (according to 21CFR ) Substantial evidence of benefit requires adequate and well controlled clinical trials There is no one right way to do things for rare diseases
31 Hurdles in Rare Disease Development Rare diseases are complex and heterogenous Costly and slow to find subjects for studies Difficult biology and end points Disease natural history not well-studied or understood Standards of care vary widely and affect outcomes In many territories there is no infrastructure to screen, record and locate subjects Lack of well-defined end point makes studies hard to design and regulatory and payer proofs hard to make Regulators are unlikely to have an expert reviewer for the rare disease in question Only the potentially large markets are prioritized by pharmas [E. Kakkis CureTheProcess Campaign]
32 sustainable pricing
33 European Orphan Medicinal Products Regulation Introduced in 2000 Aims to encourage development of orphan drugs Orphan drugs have been a very small part of the overall drugs budget. Now there is growing concern with: High prices of orphan drugs Inability to meet the standard measures of cost-effectiveness used by payers In Europe, proposals for better ways to assess in terms of valuefor money to the healthcare system
34 Paying for the Orphan Drug System: break or bend? Is it time for a new Evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet Journal of Rare Diseases 2012, 7:74.. Wills Hughes-Wilson (wills.hughes-wilson@sobi.com)... Ana Palma (apalma@shire.com). Ad Schuurman (ASchuurman@cvz.nl). Steven Simoens (steven.simoens@pharm.kuleuven.be).
35 Health Technology Assessments Rare Disease Proposed evaluation criteria: Rarity Investments Efficacy uncertainty Manufacturing complexity Follow-up measures being undertaken Disease severity Lack of alternative therapies Impact of treatment/ disease modification Unique target disease
36 Achievements small biotech
37 Speed vs reach + big pharma
38 Big Pharma vs Small Biotech development capabilities contacts with regulators payer relationships manufacturing know-how commercial infrastructure research capabilities patient proximity fast decision making entrepreneurial spirit sense of urgency Adapted for standardized R&D- Commercialization Specialized, adaptive and interconnected for Rare Disease R&D
39 Drisapersen Clinical Program Repeat dose escalation (5wks) PRO051-CLIN Subjects, mg/kg/week DMD Dose-escalation, placebo controlled (3:1) 18 Non-ambulant subjects -> 12mg/kg single dose PK/safety Dose regime comparison, placebo-controlled (2:2:1:1) DEMAND II/DMD Subjects; 6mg/kg, 24 wks efficacy/48 wks safety DEMAND III/DMD DEMAND V/DMD Pivotal study, placebo-controlled (2:1) 186 Subjects; 6mg/kg/week, 48 weeks Extension study ongoing (more than 3 years) 12 Subjects; 6mg/kg/week Dose comparison, placebo-controlled (2:2:1:1) 51 Subjects; 3 or 6mg/kg/week, 48 weeks DEMAND IV/DMD Extension study for DEMAND II and III the largest R&D effort ever in DMD Completed Enrolled DEMAND VI Natural History study 250 subjects (aged 3 18 years), 3 year follow-up More than 300 patients in fully enrolled or completed studies
40 Drisapersen Clinical Program the the the the the the the the most most most most most most most most global global global global global global global global effort effort effort effort effort effort effort effort ever ever ever ever ever ever ever ever in in in in in in in in DMD DMD DMD DMD DMD DMD DMD DMD More than 50 trial sites in 25 countries on 5 continents More than 50 trial sites in 25 countries on 5 continents
41 Thank you
Delivering on the promise: the clinical application of new diagnoses and treatments for RD K A T E B U S H B Y N E W C A S T L E U N I V E R S I T Y
Delivering on the promise: the clinical application of new diagnoses and treatments for RD K A T E B U S H B Y N E W C A S T L E U N I V E R S I T Y PERSPECTIVE/ DISCLAIMERS Doctor with >24 years experience
More informationDUCHENNE MUSCULAR DYSTROPHY CLINICAL DEVELOPMENT PROGRAM
DUCHENNE MUSCULAR DYSTROPHY CLINICAL DEVELOPMENT PROGRAM Dana R. Martin, PharmD Sarepta Therapeutics PPMD ANNUAL CONNECT CONFERENCE ORLANDO, FLORIDA JUNE 27, 2016 FORWARD LOOKING STATEMENTS This presentation,
More informationProsensa s continued development of exon-51 skipping with drisapersen after the failure of its phase-iii clinical trial.
A new research report by Dr. Guenter Scheuerbrandt Prosensa s continued development of exon-51 skipping with drisapersen after the failure of its phase-iii clinical trial. In January 2013 you received
More informationPfizer Program in DMD. Beth Belluscio, MD-Ph.D. Pfizer Rare Disease September 9, 2017
Pfizer Program in DMD Beth Belluscio, MD-Ph.D. Pfizer Rare Disease September 9, 2017 Myostatin Inhibitor for the Potential Treatment of Duchenne Muscular Dystrophy Disclaimer This presentation includes
More informationPTC Therapeutics: 20 years of commitment to bringing new treatments to patients with rare disorders. March 2018
PTC Therapeutics: 20 years of commitment to bringing new treatments to patients with rare disorders March 2018 Mar-18 Page 1 Forward looking statements within the meaning of The Private Securities Litigation
More informationSarepta Therapeutics, Inc. (SRPT-NASDAQ)
January 27, 2015 Sarepta Therapeutics, Inc. (SRPT-NASDAQ) Current Recommendation SUMMARY DATA NEUTRAL Prior Recommendation Outperform Date of Last Change 08/12/2013 Current Price (01/26/15) $12.44 Target
More informationSwissmedic Accepts Santhera's Filing of SNT-MC17 in Friedreich's Ataxia
Published: 07:00 04.10.2007 GMT+2 /HUGIN /Source: Santhera Pharmaceuticals Holding AG /SWX: SANN /ISIN: CH0027148649 Swissmedic Accepts Santhera's Filing of SNT-MC17 in Friedreich's Ataxia Liestal and
More informationataluren overview A New Approach to Genetic Disorders
ataluren overview A New Approach to Genetic Disorders Ataluren Scientific Background What is ataluren? Ataluren(formerly PTC124) is the first investigational therapy with the potential to enable the formation
More informationExon Skipping. Wendy Erler Patient Advocacy Wave Life Sciences
Exon Skipping Wendy Erler Patient Advocacy Wave Life Sciences Forward Looking Statements This document contains forward-looking statements. All statements other than statements of historical facts contained
More informationSecond Quarter 2016 Financial Results. August 4, 2016
Second Quarter 2016 Financial Results August 4, 2016 Cautionary Note Regarding Forward-Looking Statements This presentation and various remarks we make during this presentation contain forward-looking
More informationConsortia-Based Strategies in Neurodegenerative Diseases: Critical Path Institute s Track Record in Collaborative Efforts
Consortia-Based Strategies in Neurodegenerative Diseases: Critical Path Institute s Track Record in Collaborative Efforts Martha A. Brumfield, PhD President & CEO Agenda C-Path Model for Collaboration
More informationuniqure Completes Strategic Review to Refocus its Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value
uniqure Completes Strategic Review to Refocus its Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value ~ Company Structure Simplified; Manufacturing to be Consolidated into Lexington,
More informationHD Regulatory Science Consortium (HD-RSC) A consortium aimed at accelerating treatments for Huntington s disease
HD Regulatory Science Consortium (HD-RSC) A consortium aimed at accelerating treatments for Huntington s disease Critical Path Institute / CHDI Foundation Huntington s Disease Regulatory Science Consortium
More informationPartnering with Rare Disease Patient Groups. Namrata Taak R&D External Communications, Rare Diseases
Partnering with Rare Disease Patient Groups Namrata Taak R&D External Communications, Rare Diseases What we do Pharmaceuticals We develop and make medicines to treat a range of conditions including respiratory
More informationAntisense Therapeutics Ltd ASX:ANP January 2017
Antisense Therapeutics Ltd ASX:ANP January 2017 Forward Looking Statements This presentation contains forward-looking statements regarding the Company s business and the therapeutic and commercial potential
More informationCorporate Presentation. April 2016
Corporate Presentation April 2016 1 Forward Looking Safe Harbor Statement This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.
More informationRegulatory Challenges and Threats in Orphan Drug Development Are Ultra Orphans Easy?
Regulatory Challenges and Threats in Orphan Drug Development Are Ultra Orphans Easy? EPLS September 24, 2014 Ulrich Granzer Granzer Regulatory Consulting & Services The Challenge: What needs to be done
More informationCRO partner in Rx/CDx Co-Development
CRO partner in Rx/CDx Co-Development DEDICATED DIALOGUE A sponsored roundtable discussion published in Pharmaceutical Executive Two Covance executives discuss a CRO s role in supporting Companion Diagnostics
More informationSecond Quarter 2017 Financial Results. August 8, 2017
Second Quarter 2017 Financial Results August 8, 2017 Agios Conference Call Participants Prepared Remarks Introduction RENEE LECK, Sr. Manager, Investor Relations Business Highlights & 2017 Key Milestones
More informationIdorsia Company Profile
Idorsia Headquartered in Switzerland - a biotech-hub of Europe - Idorsia is specialized in the discovery and development of small molecules, to transform the horizon of therapeutic options. Idorsia has
More informationWelcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications
Welcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications 1 Alnylam Forward Looking Statements This presentation contains forward-looking statements, within the meaning
More informationClinical Trial Methods Course 2017 Trials in Rare Diseases. Erika Augustine, MD, MS University of Rochester Medical Center August 10, 2017
Clinical Trial Methods Course 2017 Trials in Rare Diseases Erika Augustine, MD, MS University of Rochester Medical Center August 10, 2017 Overview Challenges in studying rare diseases Strategies for trial
More informationAgios Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Charter)
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of report (Date of earliest event
More informationDisease Specific Registries vs Product Registries
1 Disease Specific Registries vs Product Registries Professor Hanns Lochmüller Newcastle University 2 What is TREAT-NMD? A network of excellence funded by the European Union (but with global collaborations)
More informationClinical trials for skipping exon 51 as a therapy for Duchenne muscular dystrophy.
Two Interviews. Clinical trials for skipping exon 51 as a therapy for Duchenne muscular dystrophy. Interview with Professor Kate Bushby TREAT-NMD Coordinator, Newcastle upon Tyne, UK This interview was
More informationSpark Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter)
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event
More informationInaugural Fraunhofer Delaware Technology Summit
Inaugural Fraunhofer Delaware Technology Summit Energy and Life Sciences Solu
More informationORPHANET s Services for Researchers. Valérie THIBAUDEAU Database Manager Orphanet
ORPHANET s Services for Researchers Valérie THIBAUDEAU Database Manager Orphanet Services for Researchers Needs Access classifications, genes Identify experts Collaborate Quickly recruit volunteers Establish
More informationSpeed your time to market with FDA s expedited programs
Regulatory Sciences Expediting drug approval Speed your time to market with FDA s expedited programs The faster way to marketing submission and drug approval for serious conditions and rare diseases In
More informationDrug Development and Clinical Trials Pat Furlong Parent Project Muscular Dystrophy
Drug Development and Clinical Trials Pat Furlong Parent Project Muscular Dystrophy ParentProjectMD.org -LATE 1990 s Where Are We? - Total NIH funding for MD is $17M -No new drugs in development -Average
More informationMedical Policy An independent licensee of the Blue Cross Blue Shield Association
Eteplirsen (Exondys 51) for DMD Page 1 of 8 Medical Policy An independent licensee of the Blue Cross Blue Shield Association Title: Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy Professional
More informationABPI response to European Commission consultation on advanced therapy medicinal products
ABPI response to European Commission consultation on advanced therapy medicinal products 28 March 2013 ABPI response to European Commission consultation on the regulation of advanced therapy medicinal
More informationMuscular Dystrophy Australia. Research RoadShow
Muscular Dystrophy Australia Research RoadShow Research at NMDRC Skeletal muscle Development and Hypertrophy Dystrophic Pathology Skeletal Muscle Regeneration Stem cells and Cell Transplantation Identification
More informationRWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact.
Real Real World World Data Data Across Across the the Product Product Lifecycle: Lifecycle: RWE from pre-clinical to launch RWE from pre-clinical to launch Standard of care Unmet needs Disease burden Budget
More informationExpanded Access and the Individual Patient IND
Expanded Access and the Individual Patient IND Research Wednesdays April 26, 2017 Erika Segear Johnson, PhD, RAC Associate Director of Regulatory Affairs Office of Regulatory Affairs and Quality Office
More informationPhase 1 SMA Type 2 Trial Initiation and Study Design. December 2017
Phase 1 SMA Type 2 Trial Initiation and Study Design December 2017 Disclaimers This presentation contains forward-looking statements, including statements about: the timing, progress and results of preclinical
More informationPresentation to the Committee on Accelerating Rare Disease Research and Orphan Product Development
Presentation to the Committee on Accelerating Rare Disease Research and Orphan Product Development 23 November 2009 Sharon F. Terry, MA President & CEO, Genetic Alliance Executive Director, PXE International
More informationDecision Tree for selection of model agreements for collaborative commercial clinical research
Decision Tree for selection of model agreements for collaborative commercial clinical research 1 Research interactions involving industry, universities and the NHS Industry, universities and the NHS cooperate
More informationREGENXBIO Inc. Ticker: RGNX
REGENXBIO Inc. Ticker: RGNX I know what you are thinking. You are about to read another biotech report with more acronyms than words you know the definitions. I promise you this report is written in layman
More informationPAREXEL GENOMIC MEDICINE SERVICES. Applying genomics to enhance your drug development journey
PAREXEL GENOMIC MEDICINE SERVICES Applying genomics to enhance your drug development journey YOUR JOURNEY. OUR MISSION. Genomic expertise to simplify the route to product approval and maximize patient
More informationUnderstanding the impact of publications in specialist areas: focus on orphan drugs
2 0 1 5 E U R O P E A N M E E T I N G O F I S M P P Understanding the impact of publications in specialist areas: focus on orphan drugs 2015 EUROPEAN MEETING OF ISMPP 1 2 0 1 5 E U R O P E A N M E E T
More informationNational Foundation for Women Legislators Annual Meeting September 12, 2015 Tara Ryan Vice President, State Government Advocacy
From Hope to Cures: The Value of Biopharmaceutical Innovation National Foundation for Women Legislators Annual Meeting September 12, 2015 Tara Ryan Vice President, State Government Advocacy HIV/AIDS: Then
More informationKRISANI BIO SCIENCES PVT. LTD.
KRISANI BIO SCIENCES PVT. LTD. Flat B2, 2 nd Floor, Suryateja Apartments, Hindi Nagar, Punjagutta, Hyderabad, Telangana 500078. Email: kkr@krisanibio.com. URL: www.krisanibio.com Contact Number: +91 98494
More informationALLERGAN ENTERS STRATEGIC R&D ALLIANCE WITH EDITAS MEDICINE TO DISCOVER AND DEVELOP CRISPR GENE EDITING PROGRAMS FOR OCULAR DISEASES
ALLERGAN ENTERS STRATEGIC R&D ALLIANCE WITH EDITAS MEDICINE TO DISCOVER AND DEVELOP CRISPR GENE EDITING PROGRAMS FOR OCULAR DISEASES Novel Development Programs for Potential Treatments of Serious Ocular
More informationCENTRAL NERVOUS SYSTEM (CNS) BIOMARKERS: TECHNOLOGIES AND GLOBAL MARKETS
CENTRAL NERVOUS SYSTEM (CNS) : TECHNOLOGIES AND GLOBAL MARKETS BIO074C March 2016 Jackson Highsmith Project Analyst ISBN: 1-62296-246-X BCC Research 49 Walnut Park, Building 2 Wellesley, MA 02481 USA 866-285-7215
More informationADAPTIVE PHASE II STUDY OF BAN2401 IN EARLY ALZHEIMER S DISEASE CONTINUES TOWARD 18-MONTH ENDPOINT
FOR IMMEDIATE RELEASE December 21, 2017 Eisai Co., Ltd. Biogen Inc. ADAPTIVE PHASE II STUDY OF BAN2401 IN EARLY ALZHEIMER S DISEASE CONTINUES TOWARD 18-MONTH ENDPOINT CRITERIA FOR SUCCESS AT 12-MONTH ANALYSIS
More informationINVEST IN TOPADUR! HEALTH IS THE MOST IMPORTANT THING IN LIFE!
INVEST IN TOPADUR! HEALTH IS THE MOST IMPORTANT THING IN LIFE! TOPADUR PHARMA AG a promising Swiss start-up investment Global Medical Need: Diabetes mellitus is one of the major health concerns worldwide.
More informationPATIENT GROUPS & CLINICAL TRIALS CASE STUDY
February 1, 2017 PATIENT GROUPS & CLINICAL TRIALS CASE STUDY Jeffrey W. Sherman, M.D., FACP Past President Inaugural Fellow Chief Medical Officer Executive Vice President Disclaimer The views and opinions
More informationMaximizing Market Access: THE 5 MOST CRITICAL QUESTIONS TO ASK WHEN LAUNCHING A SPECIALTY DRUGS
Maximizing Market Access: THE 5 MOST CRITICAL QUESTIONS TO ASK WHEN LAUNCHING A SPECIALTY DRUGS by Jan Nielsen, division president, Access & Patient Support Are You Asking the Right Questions? We ve all
More informationFDA Drug Approval Process Vicki Seyfert-Margolis, Ph.D.
Speaker Comparing The Effectiveness Of New Drugs: Should The FDA Be Asking 'Does It Work' Or 'Does It Work Better'? Vicki L. Seyfert-Margolis, PhD Senior Advisor, Science Innovation and Policy U.S. Food
More informationNPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide
NPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide Jefferies 2014 Global Healthcare Conference June 2, 2014 Francois Nader, MD President
More informationImplementing the 21 st Century Cures Act: Supporting Orphan Drug Development
Implementing the 21 st Century Cures Act: Supporting Orphan Drug Development Rare Disease Congressional Caucus Briefing March 2, 2017 Frank Sasinowski, M.S., M.P.H., J.D. Director, Hyman, Phelps & McNamara,
More informationBuilding on our specialty expertise. Shire acquires Transkaryotic Therapies Inc.
Building on our specialty expertise Shire acquires Transkaryotic Therapies Inc. "SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995 Statements included herein that are not
More informationLA RICERCA TELETHON PER LE MALATTIE RARE TELETHON RESEARCH ON RARE DISEASES
LA RICERCA TELETHON PER LE MALATTIE RARE TELETHON RESEARCH ON RARE DISEASES Francesca Pasinelli Direttore Generale 3 Congresso Nazionale SIFaCT Roma, 9 ottobre 2015 About Telethon Fondazione Telethon is
More informationComparability Studies for Autologous Cell Therapy Products. Chris Shen July, 2017
Comparability Studies for Autologous Cell Therapy Products Chris Shen July, 2017 Forward Looking Statements/Safe Harbor To the extent statements contained in this presentation are not descriptions of historical
More informationA BioPontis Alliance Report. Integrating Rare Disease Patients into Pre-Clinical Therapy Development; Finding our Way with Patient Input
A BioPontis Alliance Report Integrating Rare Disease Patients into Pre-Clinical Therapy Development; Finding our Way with Patient Input December 2016 Integrating Rare Disease Patients into Pre-Clinical
More informationRe: Docket No. FDA-2014-D-1461: Rare Pediatric Disease Priority Review Vouchers
February 13, 2015 Division of Dockets Management (HFA-305) Food and Drug Administration 5630 Fishers Lane, Rm. 1061 Rockville, MD 20852 Re: Docket No. FDA-2014-D-1461: Rare Pediatric Disease Priority Review
More informationRecombinant Biglycan for the Treatment! of Duchenne Muscular Dystrophy!!! PPMD Annual Conference, Jun 2013! By Joel B. Braunstein, MD!
Recombinant Biglycan for the Treatment! of Duchenne Muscular Dystrophy!!! PPMD Annual Conference, Jun 2013! By Joel B. Braunstein, MD! Tivorsan Overview Commercial operations began in 2010 around biglycan
More informationRare Diseases: Challenges and Opportunities NIH Perspective
Rare Diseases: Challenges and Opportunities NIH Perspective Stephen C. Groft, Pharm.D. Office of Rare Diseases Research (ORDR) National Center for Advancing Translational Science (NCATS) National Institutes
More informationUNITED STATES DISTRICT COURT FOR THE DISTRICT OF MASSACHUSETTS CLASS ACTION COMPLAINT
Case 1:14-cv-10201-MBB Document 1 Filed 01/27/14 Page 1 of 34 UNITED STATES DISTRICT COURT FOR THE DISTRICT OF MASSACHUSETTS MARK A. CORBAN, Individually and on Behalf of All Others Similarly Situated,
More informationIntec. Pharma Unfolding drug delivery solutions. Investor Presentation. Nasdaq: NTEC. June 2017
Intec Pharma Unfolding drug delivery solutions Investor Presentation Nasdaq: NTEC June 2017 Forward Looking Statements This presentation by Intec Pharma Ltd. (referred to as we or our ) contains forward-looking
More informationInnovative Pharmaceutical Solutions for Discovery Chemistry, Biology and cgmp Manufacturing
Innovative Pharmaceutical Solutions for Discovery Chemistry, Biology and cgmp Manufacturing Overview Serving biotech and pharma community since 1998 Proven track record of advancing small molecules from
More informationAdvanced Therapies in Europe
Advanced Therapies in Europe 1 ATMPs in Europe (2009-2017) ~ 500 clinical trials using ATMPs in EU ~ 270 ATMP classifications 18 MAAs reviewed ~ 250 scientific advice requests 9 ATMPs approved 2 3 withdrawn
More informationOffice for Human Subject Protection. University of Rochester
POLICY 1. Purpose Outline the responsibilities and regulatory requirements when conducting human subject research that involves the use of drugs, agents, biological products, or nutritional products (e.g.,
More informationDelivering on the Promise of RNA- Based Therapeu;cs
Delivering on the Promise of RNA- Based Therapeu;cs Parent Project Muscular Dystrophy Webinar June 20, 2011 Forward- Looking Statements This presenta,on includes forward- looking statements, including
More informationWhitepaper. Small Molecule Vs Cell Therapy Clinical Trial Supply Chain. Martin Lamb, Executive Vice President for Sales and Marketing
Small Molecule Vs Cell Therapy Clinical Trial Supply Chain Page 1 Whitepaper Small Molecule Vs Cell Therapy Clinical Trial Supply Chain Martin Lamb, Executive Vice President for Sales and Marketing Small
More informationUNITED STATES DISTRICT COURT DISTRICT OF MASSACHUSETTS
GLANCY BINKOW & GOLDBERG LLP Lionel Z. Glancy Michael Goldberg Robert V. Prongay Casey E. Sadler 1925 Century Park East, Suite 2100 Los Angeles, California 90067 Telephone: (310) 201-9150 Facsimile: (310)
More informationRemco de Vrueh. Driving partnerships from idea to success
Remco de Vrueh Driving partnerships from idea to success Rare Disease Symposium: the rare disease patient in 2030 Outline 2 Current status Orphan drug development Public-Private Partnerships (PPPs) Lygature
More informationDrug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research
Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research Drug Development Process by which new chemical entities
More informationBuilding Biotech Technology Transfer Opportunities: Sponsor and Developer Strategies for Success
Building Biotech Technology Transfer Opportunities: Sponsor and Developer Strategies for Success Drug developers have long been under pressure to introduce new products in an environment of escalating
More informationDefining Clinical Benefit in Clinical Trials: FDA Perspective
Defining Clinical Benefit in Clinical Trials: FDA Perspective Jessica J. Lee, MD, MMSc Medical Team Leader Division of Gastroenterology and Inborn Errors Products Center for Drug Evaluation and Research
More informationUtilizing Innovative Statistical Methods. Discussion Guide
Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings Discussion Guide Background Rare diseases are a complex and diverse set of conditions which, when taken together, affect
More informationInternational Transfers of Personal Data at sanofi-aventis R & D
International Transfers of Personal Data at sanofi-aventis R & D Pierre-Yves Lastic, PhD Senior Director, Standards Management & Data Privacy Sanofi-aventis R&D CONFERENCE ON INTERNATIONAL TRANSFERS OF
More informationConference call transcript for the March 2017 Quarterly Report
ASX ANNOUNCEMENT Conference call transcript for the March 2017 Quarterly Report Sydney Australia, 31 May 2017: Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today lodged its transcript
More informationDecember 4, Dockets Management Branch (HFA-305) Food and Drug Administration 5630 Fishers Lane, Rm Rockville, MD 20852
December 4, 2014 Dockets Management Branch (HFA-305) Food and Drug Administration 5630 Fishers Lane, Rm. 1061 Rockville, MD 20852 Re: Docket No. FDA 2014 N 1698: Food and Drug Administration Activities
More informationOrphan Drug Commercial Models: How Pharma Can Optimize the Value and Impact of Orphan Therapies
Volume XX, Issue 27 Orphan Drug Commercial Models: How Pharma Can Optimize the Value and Impact of Orphan Therapies Orphan drugs are expected to play an increasingly prominent role in the biopharmaceutical
More informationAvailability of Masked and De-identified Non-Summary Safety and Efficacy Data; Request for
This document is scheduled to be published in the Federal Register on 06/04/2013 and available online at http://federalregister.gov/a/2013-13083, and on FDsys.gov 4160-01-P DEPARTMENT OF HEALTH AND HUMAN
More informationSamatasvir (IDX719), a Potent Pan-Genotypic NS5A Inhibitor, for the Treatment of Hepatitis C Virus (HCV) Infection
Samatasvir (IDX719), a Potent Pan-Genotypic NS5A Inhibitor, for the Treatment of Hepatitis C Virus (HCV) Infection Douglas Mayers, MD December 11, 2013 1 Idenix: Advancing All-Oral, Pan-Genotypic Combination
More informationVenture Philanthropy Models: The Leukemia & Lymphoma Society's Therapy Acceleration Program. A FasterCures Webinar June 19, 2013
Venture Philanthropy Models: The Leukemia & Lymphoma Society's Therapy Acceleration Program A FasterCures Webinar June 19, 2013 What is FasterCures? Founded in 2003, our mission is to save lives by saving
More informationRoche Committed to Innovation. Sal. Oppenheim European Healthcare Investors Conference Frankfurt, September 2, 2008
Roche Committed to Innovation Sal. Oppenheim European Healthcare Investors Conference Frankfurt, September 2, 2008 This presentation contains certain forward-looking statements. These forward-looking statements
More informationA full-service CRO with integrated early-stage capabilities
CHARLES RIVER TO ACQUIRE ARGENTA and BIOFOCUS A full-service CRO with integrated early-stage capabilities James C. Foster Chairman, President & CEO Thomas F. Ackerman Executive Vice President & CFO March
More informationJoint Horizon Scanning for pharmaceuticals
Joint Horizon Scanning for pharmaceuticals - KCE report and use of EMA data Irina Cleemput (KCE Belgium) Aldo Golja (Dutch ministry of Health) 1 BeNeLuxA Introduction Brood hal, Brussels BeNeLuxA what
More informationRimeporide in Duchenne Muscular Dystrophy January Photo credits : Ceridwen Hughes,
s Rimeporide in Duchenne Muscular Dystrophy January 2017 Photo credits : Ceridwen Hughes, http://www.samebutdifferentcic.org.uk/ Orphan Drug Market The orphan disease market is heterogeneous and complex
More informationBuilding Biotech Technology Transfer Opportunities
HEALTHCARE Building Biotech Technology Transfer Opportunities Sponsor and developer strategies for success By Alison Sahoo Alison Sahoo Alison Sahoo is a pharmaceutical industry analyst with more than
More informationThe Science of Small Clinical Trials
The Science of Small Clinical Trials Introduction to Small Clinical Trials an Industry Perspective Simon Day, PhD Confucius (allegedly) When it is obvious that the goals cannot be reached, don t adjust
More informationTRANSFORMING GLOBAL GENETIC DATA INTO MEDICAL DECISIONS
TRANSFORMING GLOBAL GENETIC DATA INTO MEDICAL DECISIONS THE DOORS ARE OPEN: FEEL FREE TO COME IN CENTOGENE UNLOCKS THE POWER OF GENETIC INSIGHTS TO IMPROVE THE QUALITY OF LIFE OF PATIENTS WITH GENETIC
More informationAPITOPE ANNOUNCES ITS INTENTION TO LAUNCH AN INITIAL PUBLIC OFFERING AND LISTING ON EURONEXT BRUSSELS
APITOPE ANNOUNCES ITS INTENTION TO LAUNCH AN INITIAL PUBLIC OFFERING AND LISTING ON EURONEXT BRUSSELS HASSELT, Belgium, and CHEPSTOW, UK, 31 October 2017 - Apitope (the Company or Apitope ), a clinical
More informationStrategic Plan. Uniting to care & cure
2017-2019 Strategic Plan Uniting to care & cure Table of Contents Message from the President & CEO Page 2 Overview Page 3 Mission Page 4 Core Values Page 5 2017-2019 Objectives & Strategies Page 6 Mission
More informationRegulatory Pathways. Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB
Regulatory Pathways Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB johnlaschinger@fda.hhs.gov 1 Disclosures and Disclaimer John C. Laschinger, M.D. I am a full
More informationAdaptive Design for Medical Device Development
Adaptive Design for Medical Device Development A guide to accelerate clinical development and enhance portfolio value Executive Summary In May 2015, the FDA released a draft guidance document regarding
More informationAdvancing New Treatments for DMD and C. difficile Infection
Advancing New Treatments for DMD and C. difficile Infection 34 th Annual Canaccord Genuity Growth Conference August 13, 2014 Legal Disclaimer FORWARD-LOOKING STATEMENTS This Document contains forward-looking
More informationGood morning and thank you for joining us for our quarterly update.
Q1 2018 Earnings Conference Call May 7, 2018 11:00 AM ET Introduction and Forward Looking Statements in APPENDIX I Garo Armen Good morning and thank you for joining us for our quarterly update. We have
More informationCORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016
CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016 Positive remimazolam data in pivotal U.S. Phase III study for procedural sedation during colonoscopy
More informationWHILE A SMALL NUMBER OF PEOPLE ARE SUFFERING FROM A SINGLE RARE DISEASE, 350 MILLION PEOPLE WORLDWIDE ARE SUFFERING FROM RARE DISEASES.
June 2017 WHILE A SMALL NUMBER OF PEOPLE ARE SUFFERING FROM A SINGLE RARE DISEASE, 350 MILLION PEOPLE WORLDWIDE ARE SUFFERING FROM RARE DISEASES. 3rd most populous country 50% of affected are children
More informationGene Replacement Therapy
Gene Replacement Therapy A Boy With the Will to Live From the moment of Joshua Frase s birth, doctors were saying that the odds were he wouldn t live through the day. How could he? None of the striated
More informationJanuary (San Francisco, CA) January 8, 2018
January 2017 J.P. Morgan 36 th Annual Management Healthcare Presentation Conference (San Francisco, CA) January 8, 2018 DISCLAIMER Certain information contained in this presentation relates to or is based
More informationISPOR 19 th International Meeting May 31 st June 4 th 2014, Montréal International Society for Pharmacoeconomics and Outcomes Research
ISPOR 19 th International Meeting May 31 st June 4 th 2014, Montréal International Society for Pharmacoeconomics and Outcomes Research Pascale Boyer Barresi, CFA Business Analysis, BD&L September 2014
More information