Hilde Revets, Senior Research Fellow, Ablynx
Presentation at EIPG – VAPI-UPIP Symposium “Biotech and Advanced Therapies: Challenges and Opportunities” at the Faculty of Medicine and Pharmacy, Campus Jette, Vrije Universiteit van Brussel, Brussels 2013.
Biological method for the preparation of nanoparticles(Sheersho)Sheersha Pramanik 🇮🇳
I have described about the biological processes(other than physical,chemical) for the preparation of Nanoparticles.
do like comment share if you like it.
Multiplex PCR is a technique whereby PCR is used to amplify several different DNA sequences simultaneously. It is a type of target enrichment approach. It was first described in 1988 as a method to detect deletion mutations in the dystrophin gene – the largest known human gene
Biological method for the preparation of nanoparticles(Sheersho)Sheersha Pramanik 🇮🇳
I have described about the biological processes(other than physical,chemical) for the preparation of Nanoparticles.
do like comment share if you like it.
Multiplex PCR is a technique whereby PCR is used to amplify several different DNA sequences simultaneously. It is a type of target enrichment approach. It was first described in 1988 as a method to detect deletion mutations in the dystrophin gene – the largest known human gene
An antibody (Ab), also known as an immunoglobulin (Ig), is a large, Y-shaped protein produced mainly by plasma cells that is used by the immune system to neutralize pathogens such as pathogenic bacteria and viruses.
Monoclonal antibodies are important reagents used in biomedical research, in diagnosis of diseases, and in treatment of such diseases as infections and cancer.
These antibodies are produced by cell lines or clones obtained from animals that have been immunized with the substance that is the subject of study.
Presentation from Andreas Hermann, Oeko-Institut, about specific project activity on the risk management measures for nanomaterials, on the "Strategic workshop on nanotechnology" in Brussels,
10th February 2015.
Until recently, the properties and compositions of the microbiota in the planet are still largely a black box. Next generation sequencing (NGS) has proven to be an invaluable tool for investigating diverse environmental and host-associated microbial communities, helping to generate enormous new data sets that can be mined for information on the composition and functional properties of vastly great numbers of microbial communities.
An antibody (Ab), also known as an immunoglobulin (Ig), is a large, Y-shaped protein produced mainly by plasma cells that is used by the immune system to neutralize pathogens such as pathogenic bacteria and viruses.
Monoclonal antibodies are important reagents used in biomedical research, in diagnosis of diseases, and in treatment of such diseases as infections and cancer.
These antibodies are produced by cell lines or clones obtained from animals that have been immunized with the substance that is the subject of study.
Presentation from Andreas Hermann, Oeko-Institut, about specific project activity on the risk management measures for nanomaterials, on the "Strategic workshop on nanotechnology" in Brussels,
10th February 2015.
Until recently, the properties and compositions of the microbiota in the planet are still largely a black box. Next generation sequencing (NGS) has proven to be an invaluable tool for investigating diverse environmental and host-associated microbial communities, helping to generate enormous new data sets that can be mined for information on the composition and functional properties of vastly great numbers of microbial communities.
Presentation from OIS@ASCRS 2016
Co-Moderators:
Gilbert H. Kliman, MD, Managing Director – InterWest Partners
Stephen Slade, MD
Company Presentations
Equinox | John Berdahl, MD, Founder & CEO
Presbyopia Therapies | Jim McCollum, Co-Founder
Stroma Medical | Doug Daniels, CEO
Eyenovia | Curt LaBelle, MD, Director
Wicab | Robert Beckman, President & COO
Pitches from the 10 finalist teams selected to compete for the £100,000 Grand Prize of the inaugural OneStart competition in 2013, hosted by the Oxbridge Biotech Roundtable and SR One.
Learn more about this year's competition: http://oxbridgebiotech.com/onestart
Life science companies have the responsibility to collect, assess and potentially report any adverse event (AE) information that field sales representatives or clinical research associates learn while in the field. The task of collecting that report from the health care provider and communicating it to the company's product vigilance team, however, is only a small part of the field sales representative's or clinical research associate's job requirements. Life science companies' field personnel require a simple, modern and effective solution to automate this task so they can focus their time on other important responsibilities.
At this one-hour webinar, we will show you how our mobile AE intake tool, PRIMO Mobile, empowers your field sales representatives, clinical research associates and product safety team members with a comprehensive and easy-to-use solution to manage the case data intake process on their tablets, smartphones or laptops.
In this session with Brad Gallien, you will discover:
- How to utilize your field representatives' standard tablet, smartphone or laptop to quickly and easily submit adverse events they uncover in the field.
- The power and flexibility of mobile solutions to support AE intake challenges
- A fast and powerful way to facilitate communication between the product vigilance department and field representatives
- A modern approach to manage the intake and review of potentially reportable adverse events and product complaints
SMi Group are thrilled to announce the launch of the Ophthalmic Drugs Conference, being held on the 28-29 November 2017 at the Copthorne Tara Hotel in London, UK. www.ophthalmicdrugs.com/slideshare
Specialized oncology reference laboratory providing the latest testing technologies, global/tech-only options, and interactive education to the pathology community
Offer the complete spectrum of diagnostic services through nationwide network of laboratories
Dedicated to providing superior service, faster turn-around times, and complete attention to the needs of our clients and their patients
Maurizio Battistini, EIPG Vice-President
EIPG Presentation at Pharmaceutical Supply Chains I, a training school organised by Cost Action CA15105: European Medicines Shortages Research Network – addressing supply problems to patients (Medicines Shortages), Lisbon 2017
Claude Farrugia, EIPG President
EIPG Presentation at European Clinical Trial Day, an international conference organised by AFI and Regione Lombardia, endorsed by EIPG, Milan 2017
Claude Farrugia, EIPG Vice-President
EIPG Presentation at VAPI-UPIP Seminar “Implementation of the new Delegated Act on Falsified Medicines”, Limelette 2016.
Prof Kristien De Paepe
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Dr Gavin Halbert
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Prof Clive Badman OBE
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Prof Angela Timoney
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Prof Alastair Florence
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Jean Pierre Paccioni, EIPG President
Presentation at EIPG – BIPA Symposium “Clinical Trials Research” at the Faculty of Pharmacy, Medical University of Sofia, Sofia 2014.
Jacques Morénas, Deputy Director, Inspection Division, ANSM
Presentation at EIPG – BIPA Symposium “Clinical Trials Research” at the Faculty of Pharmacy, Medical University of Sofia, Sofia 2014.
Borislav Borissov, Former Head of BDA
Presentation at EIPG – BIPA Symposium “Clinical Trials Research” at the Faculty of Pharmacy, Medical University of Sofia, Sofia 2014.
Georgina Gal, Regulatory Affairs Manager, AbbVie, Hungary
Presentation at EIPG – BIPA Symposium “Clinical Trials Research” at the Faculty of Pharmacy, Medical University of Sofia, Sofia 2014.
More from European Industrial Pharmacists Group (20)
Title: Sense of Taste
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
Causes: Nerve damage, neurological disorders, infections, poor oral hygiene, adverse drug effects, deficiencies, aging, tobacco use, altered neurotransmitter levels
Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
Prix Galien International 2024 Forum ProgramLevi Shapiro
June 20, 2024, Prix Galien International and Jerusalem Ethics Forum in ROME. Detailed agenda including panels:
- ADVANCES IN CARDIOLOGY: A NEW PARADIGM IS COMING
- WOMEN’S HEALTH: FERTILITY PRESERVATION
- WHAT’S NEW IN THE TREATMENT OF INFECTIOUS,
ONCOLOGICAL AND INFLAMMATORY SKIN DISEASES?
- ARTIFICIAL INTELLIGENCE AND ETHICS
- GENE THERAPY
- BEYOND BORDERS: GLOBAL INITIATIVES FOR DEMOCRATIZING LIFE SCIENCE TECHNOLOGIES AND PROMOTING ACCESS TO HEALTHCARE
- ETHICAL CHALLENGES IN LIFE SCIENCES
- Prix Galien International Awards Ceremony
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...i3 Health
i3 Health is pleased to make the speaker slides from this activity available for use as a non-accredited self-study or teaching resource.
This slide deck presented by Dr. Kami Maddocks, Professor-Clinical in the Division of Hematology and
Associate Division Director for Ambulatory Operations
The Ohio State University Comprehensive Cancer Center, will provide insight into new directions in targeted therapeutic approaches for older adults with mantle cell lymphoma.
STATEMENT OF NEED
Mantle cell lymphoma (MCL) is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) accounting for 5% to 7% of all lymphomas. Its prognosis ranges from indolent disease that does not require treatment for years to very aggressive disease, which is associated with poor survival (Silkenstedt et al, 2021). Typically, MCL is diagnosed at advanced stage and in older patients who cannot tolerate intensive therapy (NCCN, 2022). Although recent advances have slightly increased remission rates, recurrence and relapse remain very common, leading to a median overall survival between 3 and 6 years (LLS, 2021). Though there are several effective options, progress is still needed towards establishing an accepted frontline approach for MCL (Castellino et al, 2022). Treatment selection and management of MCL are complicated by the heterogeneity of prognosis, advanced age and comorbidities of patients, and lack of an established standard approach for treatment, making it vital that clinicians be familiar with the latest research and advances in this area. In this activity chaired by Michael Wang, MD, Professor in the Department of Lymphoma & Myeloma at MD Anderson Cancer Center, expert faculty will discuss prognostic factors informing treatment, the promising results of recent trials in new therapeutic approaches, and the implications of treatment resistance in therapeutic selection for MCL.
Target Audience
Hematology/oncology fellows, attending faculty, and other health care professionals involved in the treatment of patients with mantle cell lymphoma (MCL).
Learning Objectives
1.) Identify clinical and biological prognostic factors that can guide treatment decision making for older adults with MCL
2.) Evaluate emerging data on targeted therapeutic approaches for treatment-naive and relapsed/refractory MCL and their applicability to older adults
3.) Assess mechanisms of resistance to targeted therapies for MCL and their implications for treatment selection
micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
Tom Selleck Health: A Comprehensive Look at the Iconic Actor’s Wellness Journeygreendigital
Tom Selleck, an enduring figure in Hollywood. has captivated audiences for decades with his rugged charm, iconic moustache. and memorable roles in television and film. From his breakout role as Thomas Magnum in Magnum P.I. to his current portrayal of Frank Reagan in Blue Bloods. Selleck's career has spanned over 50 years. But beyond his professional achievements. fans have often been curious about Tom Selleck Health. especially as he has aged in the public eye.
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Introduction
Many have been interested in Tom Selleck health. not only because of his enduring presence on screen but also because of the challenges. and lifestyle choices he has faced and made over the years. This article delves into the various aspects of Tom Selleck health. exploring his fitness regimen, diet, mental health. and the challenges he has encountered as he ages. We'll look at how he maintains his well-being. the health issues he has faced, and his approach to ageing .
Early Life and Career
Childhood and Athletic Beginnings
Tom Selleck was born on January 29, 1945, in Detroit, Michigan, and grew up in Sherman Oaks, California. From an early age, he was involved in sports, particularly basketball. which played a significant role in his physical development. His athletic pursuits continued into college. where he attended the University of Southern California (USC) on a basketball scholarship. This early involvement in sports laid a strong foundation for his physical health and disciplined lifestyle.
Transition to Acting
Selleck's transition from an athlete to an actor came with its physical demands. His first significant role in "Magnum P.I." required him to perform various stunts and maintain a fit appearance. This role, which he played from 1980 to 1988. necessitated a rigorous fitness routine to meet the show's demands. setting the stage for his long-term commitment to health and wellness.
Fitness Regimen
Workout Routine
Tom Selleck health and fitness regimen has evolved. adapting to his changing roles and age. During his "Magnum, P.I." days. Selleck's workouts were intense and focused on building and maintaining muscle mass. His routine included weightlifting, cardiovascular exercises. and specific training for the stunts he performed on the show.
Selleck adjusted his fitness routine as he aged to suit his body's needs. Today, his workouts focus on maintaining flexibility, strength, and cardiovascular health. He incorporates low-impact exercises such as swimming, walking, and light weightlifting. This balanced approach helps him stay fit without putting undue strain on his joints and muscles.
Importance of Flexibility and Mobility
In recent years, Selleck has emphasized the importance of flexibility and mobility in his fitness regimen. Understanding the natural decline in muscle mass and joint flexibility with age. he includes stretching and yoga in his routine. These practices help prevent injuries, improve posture, and maintain mobilit
Explore natural remedies for syphilis treatment in Singapore. Discover alternative therapies, herbal remedies, and lifestyle changes that may complement conventional treatments. Learn about holistic approaches to managing syphilis symptoms and supporting overall health.
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdfAnujkumaranit
Artificial intelligence (AI) refers to the simulation of human intelligence processes by machines, especially computer systems. It encompasses tasks such as learning, reasoning, problem-solving, perception, and language understanding. AI technologies are revolutionizing various fields, from healthcare to finance, by enabling machines to perform tasks that typically require human intelligence.
Ozempic: Preoperative Management of Patients on GLP-1 Receptor Agonists Saeid Safari
Preoperative Management of Patients on GLP-1 Receptor Agonists like Ozempic and Semiglutide
ASA GUIDELINE
NYSORA Guideline
2 Case Reports of Gastric Ultrasound
Maxilla, Mandible & Hyoid Bone & Clinical Correlations by Dr. RIG.pptx
Nanobodies: Journey from Research to Commercial
1. Nanobodies® -
Inspired by nature
UPIP-VAPI
VUB Campus Jette April 2013
Hilde Revets
Senior Research Fellow
Nanobodies®: journey from
research to commercial
2. www.ablynx.com
Forward looking statements
Certain statements, beliefs and opinions in this presentation are forward-looking, which
reflect the Company’s or, as appropriate, the Company’s directors’ current expectations
and projections about future events. By their nature, forward-looking statements involve a
number of risks, uncertainties and assumptions that could cause actual results or events
to differ materially from those expressed or implied by the forward-looking statements.
These risks, uncertainties and assumptions could adversely affect the outcome and
financial effects of the plans and events described herein. A multitude of factors including,
but not limited to, changes in demand, competition and technology, can cause actual
events, performance or results to differ significantly from any anticipated development.
Forward looking statements contained in this presentation regarding past trends or
activities should not be taken as a representation that such trends or activities will
continue in the future. As a result, the Company expressly disclaims any obligation or
undertaking to release any update or revisions to any forward-looking statements in this
presentation as a result of any change in expectations or any change in events, conditions,
assumptions or circumstances on which these forward-looking statements are based.
Neither the Company nor its advisers or representatives nor any of its of their parent or
subsidiary undertakings or any such person’s officers or employees guarantees that the
assumptions underlying such forward-looking statements are free from errors nor does
either accept any responsibility for the future accuracy of the forward-looking statements
contained in this presentation or the actual occurrence of the forecasted developments.
You should not place undue reliance on forward-looking statements, which speak only as
of the date of this presentation.
2
3. www.ablynx.com
Outline
From research to commercialization
• The story of Ablynx
The Nanobody technology
Product pipeline and examples of clinical assets
• anti-IL-6R to treat RA – strong efficacy and safety results in Phase II
• anti-vWF (caplacizumab) to treat TTP
• anti-RSV
3
4. www.ablynx.com 4
Commercialization via Start-up/Spin-Off Company
What do you need to create a Start-up/Spin-Off Company?
Creating a Spin-Off Company: steps and issues involved
An invention arises from university research
A platform technology is built up
If the technology (invention) is a platform on which could be built
multiple commercial products, it can form basis for a new company
• New business allows a researcher to be personally involved in the
translation of its discoveries into products & services and see the
correlation between hard work and financial reward
5. www.ablynx.com 5
The Business Opportunity Document
• A key marketing document that describes the business opportunity
Development of Business Plan
Protection and exploitation of Intellectual Property
• Multi-layered approach (platform, drugs, formulation,…)
• Life cycle management
Finding investors
Finding infrastructure
Negotiation and legal support
Creating a Spin-Off Company: steps and issues involved
6. www.ablynx.com 6
In the beginning….
Early ’90: discovery of camelid heavy-
chain only antibodies at ALBI (VUB)
Further characterization and development
of the VHH platform technology
In 1996: ALBI joins VIB
Intensive collaboration between VIB
headquarters and ALBI (VIB6) to validate
the technology for potential spin-off
Generation of IP
Development of Business Plan
Patent Portfolio (University/VIB)
In 2001: ABLYNX established
In 2002: ABLYNX incorporated (completed
first financing round)
Nanobody technology R. Hamers
7. www.ablynx.com 7
Rapid evolution from platform to product based company
End 2001 End 2002 End 2007 Today
Foundation
Discovery platform
• No partners
• €5M seed financing
• No products
• 10 staff
• Platform building
Discovery and early
development
• 3 partners
• €70M private equity
• €85M IPO (NYSE)
• 11 R&D projects
• 1 Nanobody in clinic
• 144 staff
• Platform upscaling
Discovery and later
development
• 4 partners
• > €200M equity funding
• €160M in cash from partners
• ~ 25 R&D projects
• >700 people treated
• 7 Nanobody products in clinic
• 2 clinical POC (RA)
• < 250 staff
• Commercial production
VALUE
CREATION
8. www.ablynx.com
Nanobodies – demonstrated track record
>750 patients and subjects
have received Nanobodies
Nanobodies have been
tested in 18 countries, 4
continents
Clinical grade material
produced up to 2,500L scale
Two clinical POCs in RA
8
1st inhaled Nanobody
successfully completes
Phase I safety study
9. www.ablynx.com 9
Three-pronged approach to balancing risk and reward
* No investment in clinical trials made to date by Ablynx
Balancing risk and reward
€160M in non-dilutive cash from collaborators received to date
1.
Fully Funded + Milestones
and Royalties
Boehringer Ingelheim, Novartis
and Merck & Co
•11 active programmes
• €113 million in cash received
since 2005
• BI is current shareholder (4.9%)
2.
Co-discovery/Co-
development
Merck Serono – Ablynx
• 5 active programmes in
inflammation, immunology and
oncology
• First Phase I expected in
2013
• €47 million in cash received
since 2008
3.
Wholly-owned clinical
assets
Ablynx
• TNFa (ozoralizumab) – Ph II*
• vWF (caplacizumab) – Ph II
• IL-6R (ALX-0061) – Ph II
• RANKL (ALX-0141) – Ph I
• RSV (ALX-0171) – Ph I
10. www.ablynx.com
Outline
From research to commercialization
• The story of Ablynx
The Nanobody technology
Product pipeline and examples of clinical assets
• anti-IL-6R to treat RA – strong efficacy and safety results in Phase II
• anti-vWF (caplacizumab) to treat TTP
• anti-RSV
10
11. www.ablynx.com
Ablynx’s Nanobodies – proven single variable domain approach
11
Camelidae family has both forms
Conventional antibody
• Heavy and light chains
• Both chains required for antigen
binding and stability
• Large size and relatively low
formatting flexibility
• Administered through injection
Heavy-chain antibody
• Only heavy chains
• Full antigen binding capacity
and very stable
VH
VL
CL
CH1
CH3
CH2
VHH
Ablynx’s Nanobody®
•Small (1/10 size of a mAb)
•Flexible formatting
•Highly potent, robust and stable
•Broad target applicability
•Multiple administration routes
•Ease of manufacture
•Speed of discovery
VHH
CH3
CH2
12. www.ablynx.com
Nanobody discovery process – the power of evolution
Conventional
antibodies
Select Nanobodies
of interest
Immunize llama
with antigen
Draw blood 6–12
weeks later
Clinical trials
VHH
Ablynx’s
Nanobody®
VHH
CH3
CH2
Manufacture in
micro-organisms
Format Nanobody to achieve
desired properties
plus half-life
extension
(HLE)
12
14. www.ablynx.com
Outline
From research to commercialization
• The story of Ablynx
The Nanobody technology
Product pipeline and examples of clinical assets
• anti-IL-6R to treat RA – strong efficacy and safety results in Phase II
• anti-vWF (caplacizumab) to treat TTP
• anti-RSV
14
15. www.ablynx.com Blank boxes: non-disclosed targets 15
Pipeline – internal and funded programmes
Fullyowned
Therapeutic area Product name Target
Inflammation/
Immunology/
Infection
Haematology
Oncology
Pulmonary
Discovery
ALX-0061
NA
Pre-clinical Phase I Phase II Phase III Filing
IL-6R
caplacizumab vWF
ALX-0171
Various
Inflammation/
Immunology
Neurology
Oncology
Various
ozoralizumab TNFα
ALX-0141 RANKL
Oncology
Pulmonary
ALX-0761
NA
ALX-0751
NA
NA
NA
NA
NA
Validated targets (clinic)
1st in class
RSV
50%Co-Co
NA
NA
Various
Various
Various
Various
IgE
Fullypartnered
NA
16. www.ablynx.com
ALX-0061 – designed to be potentially best-in-class
Features Potential Benefits
Small (26kD) • penetrates faster and more effectively into
tissues
Targets human serum albumin (HSA) • prolongs half-life
• improved trafficking to inflamed tissue
Monovalent binding • avoids target cross-linking
Preferential binding of soluble vs.
membrane bound IL-6R
• superior benefit/risk profile
Strong affinity to soluble IL-6R • fast target engagement resulting in fast onset
of action
Low immunogenic potential • improved safety profile
Tailored PK • extended therapeutic window
• convenient dosing and scheduling
16
17. www.ablynx.com
ALX-0061 – Phase II study design (MAD)
Randomisation(N=37)
Week 0 - 12 Week 12 - 24
ALX-0061 6mg/kg Q8W
ALX-0061 3mg/kg Q4W
ALX-0061 1mg/kg Q4W ALX-0061 1mg/kg Q4W
ALX-0061 3mg/kg Q4W
ALX-0061 3mg/kg Q4W
ALX-0061 3mg/kg Q4W
Placebo
ALX-0061 6mg/kg Q8W
ALX-0061 6mg/kg Q4W
ALX-0061 6mg/kg Q4W
Placebo
Dose modification based on EULAR
response at week10
N=6 N=3
N=3
N=9
N=10
N=9
N=8
N=1
N=8
N=2
N=8
N=1
unmodified
placebo
modified
unmodified
modified
unmodified
modified
switched
24/28 patients completed the study at their ALX-0061 starting dose
17
19. www.ablynx.com
ALX-0061 – strong induction of DAS28 remission
• All DAS28 components contributed substantially to the score
• 20/24 patients achieved low disease activity or remission
19
20. www.ablynx.com
Caplacizumab (anti-vWF) – designed to address an unmet
medical need in TTP
Unique Nanobody Format
Small
not an antibody
no Fc
rapid distribution and
onset of action
rapid clearance
limits toxicity risk
Specific
high potency
towards target
avoid “off-target”
effects
Robust
high stability
good
manufacturability
iv and sc formulation
liquid, lyophilised
Modular
bivalent interaction
with target
increased avidity
leads to higher
potency
Orphan Drug designation in US and EU
Patent term (excluding extensions) will run until 2026
Potential pivotal Phase II study on-going with the aim to complete recruitment in 2013
20
21. www.ablynx.com
ULvWF and anti-vWF Nanobody
ULvWF
Ex vivo platelet string formation
Anti-vWF Nanobody inhibits platelet
string formation caused by UL-vWF in
plasma of TTP patients
ULvWF
multimers
Platelet String
FormationEndothelium
ADAMTS13
anti-vWF Nanobody
Microthrombi form which block
the small blood vessels in
thrombotic thrombocytopenic
purpura (TTP)
Target for the Nanobody is
in the bloodstream, i.v. and
s.c. formulations ensure
desired exposure
21
Caplacizumab – blocks the platelet and ULvWF interaction
22. www.ablynx.com
Acquired TTP – an unmet medical need
Healthy active adult
Sudden onset:
severe fatigue,
headache, bizarre
behaviour, vertigo,
seizures, coma,
various other symptoms
Daily plasma exchanges in
hospital until recovery of
platelets count
Diagnosis
of TTP
Day 1 Day 2 Day 3 Day 4 Day 5 Day 8 ...Day 6 Day 7
+ caplacizumab
22
Potentially:
fewer days of PEX
reduction in relapse/exacerbations
improved longer term outcome
23. www.ablynx.com
Respiratory syncytial viral (RSV) infections – unmet need
Duration: 1-2 weeks
Evolves to
distressing
symptoms
8-20%
hospitalised
Symptomatic treatment
including inhaled
corticosteroid & bronchodilator
23
*medical cost year after infection
**risk asthma
* Shi et al., J Med Econ, 2011; **Sigurs et al., Thorax, 2010; Krishnamoorthy et al., Nature Medicine 2012
24. www.ablynx.com
ALX-0171 – anti-RSV Nanobody designed for delivery to
site of infection
24
Unique Nanobody Format
2,000 fold increase in potency compared with monovalent structure
Specific
high potency towards the
virus
avoid “off-target” effects
well tolerated in Phase I
study
Robust
high stability
efficient nebulisation
without loss in potency
potentially reduces viral
replication in the lungs
Convenient
inhalation
opportunity for once or
twice daily dosing
dosing time < 3 minutes
Patent term (including extensions) will run until 2035
25. www.ablynx.com
ALX-0171 – potential for transformational treatment of RSV
25
Safe
Biologic targeting the virus
Well tolerated in Phase I
In constrast to some vaccines, not associated with
enhanced RSV disease
Potent
Potentially most potent drug in clinical development
Broad coverage of viral clinical strains
Fast
Virus in the respiratory tract targeted
immediately through nebulisation
Inhaled
Opportunity for once daily dosing (< 3 minutes)
Airway model shows efficient delivery to infant lung
ALN-RSV01: Alnylam (PhII b completed to treat progressive bronchiolitis obliterans syndrome; primary endpoint not met); MDT637: Microdose (PhI completed to treat RSV)
26. Nanobodies® -
Inspired by nature
UPIP-VAPI
VUB Campus Jette April 2013
Hilde Revets
Senior Research Fellow
Nanobodies®: journey from
research to commercial