Program 2018

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  • Small Molecules

  • Biologics

  • Technology & Innovation

  • Device Development

7:30 AM - 8:20 AM

Registration & Refreshments

Register, get yourself some refreshments and say hello to your fellow attendees

8:30 AM - 9:10 AM

Small Molecules

Transforming the Small Molecule to Medicine Product Design Process?

Brian Henry, Vice President Drug Product Design, Pfizer

 

 

  • Increasing small molecule complexity and accelerated regulatory filings timings are leading us to rethink our drug delivery and development strategy
  • Computational and predictive sciences will transform all aspects of accelerated drug product development
  • A focus on continuous manufacturing will revolutionise our approach to scale up and variable commercial batch sizes.
  • Developing a talent blueprint for our workforce will be key to our exciting future in drug development

9:15 AM - 9:50 AM - Case Studies

Small Molecules

Solvent-Induced Phase Separation in Amorphous Solid Dispersions

Gabriele Sadowski, Faculty Head, TU Dortmund University

  • Amorphous solid dispersions
  • Amorphous-amorphous phase separation
  • Solvent selection for ASD preparation
  • Thermodynamic phase behaviour

Biologics

Fixed Dose mAb Combinations: A recent case study highlighting CMC development challenges and strategies

Tarik Khan, Group Leader, Late-Stage Pharmaceutical and Processing Development, Roche

  • A recent surge in clinical trials using more than a single mAb has brought interest to CMC strategies to make patient administration less burdensome
  • Co-administration and co-formulation of multiple mAbs is now a hot topic for CMC development
  • Developing fixed dose combinations is not straightforward and requires balancing multiple factors
  • Analytical control strategies require significant more effort to resolve product quality attributes

Technology & Innovation

Implementation of QbD based Control Strategy during Drug Product Continuous Manufacturing

Frantz Elbaz, Formulator Project Leader and Pharm Development QBD Network Leader, Novartis

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  • Which QbD elements are used throughout the product development in CM
  • Strategy to identify Critical Process Parameters and Critical Material Attributes
  • Implementing the appropriate Control Strategy and keeping the process in a state of control

Device Development

Application of Decision Analytics to Device Selection and Development

Martin McLoughlin, Head of Device Development, Bristol-Myers Squibb

In the context of drug delivery device development a number of critical decisions have to be made. Getting these decisions right or wrong can make or break a product and the financial impact of these decisions may be in the $100 millions. Examples are, whether to invest in a device at all? When to introduce a device? Whether to make or buy? Which device to select? Formal decision analytic techniques provide a framework for rational decision making which can be applied to large and small decisions alike. The presentation introduces decision theoretic concepts, tools and methods and applies to some typical device development and selection questions.

9:50 AM - 10:25 AM - Case Studies

Small Molecules

Biopharmaceutics Risk Assessment During Formulation and Clinical Development

Filippos Kesisoglou, Senior Principal Scientist, Biopharmaceutics and Pharmaceutical Sciences, MSD

 

  • Role of in vitro, in silico and in vivo tools in formulation develop: Case Studies
  • Combination of preclinical data with modelling and simulation to inform clinical formulation selection
  • Driving Quality by Design via Biopharmaceutics Risk Assessment: Case Studies

Biologics

Introduction on “Next Generation Biologics

Carsten Olbrich, Product Quality Steward Eylea and Anetumab Ravstantine , Bayer

  • New therapeutic approaches
  • Treatment of severe disease on genetic roots
  • An introduction on innovative approaches, opportunities and challenges.

Technology & Innovation

Manufacturing the Future: a strategic approach to Product and Process development by Continuous Manufacturing

Giustino Di Pretoro, Associate Director, Johnson & Johnson

  • J&J deployment strategy in continuous manufacturing
  • CM in development – strategic approach
  • Minimization of API consumption
  • Case study of a J&J compound in development

Device Development

Left to Our Own Devices: Design Control and Risk Management Strategies for Combination Products

Richard Wedge, Compliance Lead , Pfizer Ltd

 

  • Design controls are a relatively new regulatory requirement for the development of drug/device combination products.
  • Manufacturers are now expected to establish and maintain Design History Files throughout the lifecycle of such products.
  • The requirements associated with medical device risk management activities are also expected to be followed as part of combination product development and lifecycle management.
  • Subtle differences exist between the approaches to medical device and drug product risk management; however, the two can be reconciled without either being compromised.

10:25 AM - 11:25 AM

iSolve & Break

10:58 AM - 10:58 AM - Case Studies

Technology & Innovation

Combining the Best of Two Worlds: Using High Throughput Physico-Chemical Testing & Biorelevance Assays in Biotherapeutics Development

Sabine Eichling, Senior Scientist II, NBE High-Throughput and Advanced Formulation Sciences, Abbvie

  • Formulation development for biotherapeutics
  • High throughput screening
  • Biorelevance after subcutaneous injection

11:30 AM - 12:05 PM - Case Studies

Small Molecules

Microenvironmental pH Modulation – An Effective Tool in Enhancing Dissolution and Oral Absorption of Solid Dosage Forms

Helen Hao Hou, Senior Scientist Small Molecule Pharmaceutical Sciences , Genentech

  • Pros and cons of using high-energy solids (salts, co-crystals, or amorphous solid dispersions) to generate supersaturation and enhance drug bioavailability
  • Dissolution from solid and diffusion layer model
  • Microenvironmental pH modulation to control dissolution behaviour of solid dosage forms
  • Case study in enhancing oral absorption of the salt of a weakly acidic BCS Class II compound

Biologics

A Thermodynamic Approach to Explore Excipient Mixtures and Novel Excipients in High Concentration Protein Formulations

Christoph Brandenbusch, Associate Professor, University of Dortmund

 

  • How to identify synergic effects in excipient selection?
  • Do we need novel excipients or can we determine the “sweet spot” for formulation development using classical excipients?
  • How many experimental data-points do we need?
  • How far can modelling approaches guide us?
  • Why consider thermodynamics in formulation development
  • Those results will be presented and discussed.

Technology & Innovation

Facilitating meaningful differentiation of products by establishing a culture of innovation

Scott Ariagno, Head, Infusion and Reconstitution Systems / Director, Device Development and External Manufacturing, Shire

  • Case study on fostering a culture of innovation
  • Approach & Guiding Principles
  • Selected examples of how building on existing platforms allows to differentiate existing products and deliver value to the patient
  • Observations & take aways

Device Development

Risk Management and Formative Human Factor studies

Muriel Didier, Human Factors Team Head, Novartis

Auto injector is one of the delivery device that users self-injecting in home setting appreciate. It is easy to use, does not look too “medical/aggressive”. Many of them are currently marketed. There are differences in their handling but at the same time they can look similar. Using an auto-injector on a regular basis or only occasionally in emergency situation may make a difference in terms of use errors.

Is it the reason why the FDA has placed the auto injector on the list of devices that need to demonstrate safety and efficacy at least with HFE summative study and even sometimes with real use study?

What could be done to better support the user of auto injectors? Technical development: Differentiation (right drug, right dosage), handling (different mental models), intuitiveness (identification of needle end, click associated with end of injection). What can be improved? Connectivity, feedback identification, cap removal?

Regulation perspective: Harmonisation of the handling steps by developing some standards like it is done in the automotive field? Pro/Cons

12:10 PM - 12:45 PM - Solution Spotlights

Small Molecules

Solution Spotlight with Adare Pharmaceuticals How-to Use Oral Drug Delivery Technology Innovatively

Dr. Jin Wang-Lai, Senior Director - Pharmaceutical Development, Adare Pharma

 

Technology & Innovation

Solution Spotlight with Losan Pharma Efficient and predictive screening tools –facilitation of the development of nanoparticulate formulations for oral, parenteral and topical applications

Dr. Matthias Rischer, Director Drug Delivery & Innovation Projects , Losan Pharma

  • Efficient screening tool for Nanoformulations
  • Reduction of formulation time and efforts to gain POC
  • Use of Nanoformulations in parenteral, topical and oral applications
  • Examples

Device Development

Solution Spotlight with Datwyler Enabling device innovation through component customisation

Carina Van Eester, Platform leader PFS and cartridges, Datwyler Sealing Solutions

12:10 PM - 12:45 PM - Case Studies

Biologics

Solution Spotlight with Pfanstiehl Understanding Sucrose: Matching Sucrose Quality to Functional Need

Dr. Christian Lotz, Director, Business Development, Pfanstiehl

  • Not all compendial sucrose products should be considered to be equal.
  • Critical quality attributes that go beyond those found on the typical compendial specification and their potential impact on function and biological product quality will be discussed.
  • Highlighted will be the relationship of the necessity of control for low level endotoxin testing, understanding glucan load and the interactive relationship between them.
  • Recommendations will also be summarized for consideration by end users to truly understand the impact and level of control necessary for biologic fermentation, purification and formulation under a higher level of control and understanding.

12:45 PM - 1:45 PM

Networking Lunch

Wolfgang Beier, CSO, Leon-nanodrugs

 

 

 

Lunch and Learn Roundtable with Leon-nanodrugs GmbH
Innovative precipitation nanotechnology – bringing added value to product development and patients

 

  • Poor water solubility of majority of marketed therapeutics is a major challenge for sufficient bioavailability
  • Nanotechnology provides a solution to overcome the issue of poor solubility
  • Nano-formulated products can lead to added values for patients
    - GREATER FLEXIBILITY (no food effect, dosing) 
    - SAFER (dose reduction, less side effects, substitution of toxic/allergenic ingredients) 
    - BETTER ABSORPTION (faster onset of action) 
    - MORE CONVENIENT (improved route of application, dosage form, storage/stability)
  • MJR® Technology platform: a tool for the development and production of pharmaceutical drug products based on nanoparticle & nanoemulsion solutions
  • Examples of the use of MJR® Technology in product development

1:45 PM - 2:20 PM - Case Studies

Small Molecules

Differentiating Itraconazole Formulations Based on the Flux through Artificial Lipophilic Membrane

Bernard van Eerdenbrugh, Pre-clinical Formulation Expert, Chem. & Pharmaceut. Profiling Technical R&D, Novartis

 

 

 

  • Different itraconazole formulations were evaluated using a miniaturized dissolution-permeation apparatus
  • Flux values obtained correlated well with published in-vivo data in rodentsFor the solid dispersions, a decrease in flux was observed over time
  • The latter observation could be explained by crystallization, as supported by DSC and XRPD results

Biologics

Protein aggregation in multidose parenteral formulations: challenges during formulation development

Kathrin Spielberger, Lab Head Formulation Development, Sanofi

  • Stability of proteins in aqueous solutions
  • Key development challenges during formulation development
  • Siliconization processes of packaging material
  • Implementation of silicone-free packaging material: a case study

Device Development

Who moved my Device?” – a talk on working within the changing regulatory landscape around combination and medical devices.

Bjørg Hunter, Device Engineering Manager, GlaxoSmithKline

  • Challenges of developing combination products through a changing regulatory landscape.
  • Working across boundaries to achieve common goals.
  • Human Factors and usability studies.
  • Case study on the review of human factors protocols by the FDA.
  • Engaging with the industry and regulators.

2:25 PM - 3:00 PM - Solution Spotlights

Small Molecules

Solution Spotlight with Grace New Insights in Mesoporous Silica Drug Delivery: Scalable Processing and Solvent Free Techniques

Fred Monsuur, TCS and Business Development Manager Excipients, W.R. Grace

  • Silica-based Drug Delivery is a novel platform for solubility enhancement of poorly soluble drugs
  • Current work focused on practical strategies for loading API on mesoporous silica
  • Solvent free strategies were found to be very effective in loading the API on mesoporous silica
  • Scalable strategies have advanced the adoption of this novel technology platform

Biologics

Solution Spotlight with Unchained Labs Rip through formulation prep with a Junior, a Lunatic and an Uncle

Joe Barco, Sr. Director Marketing, Unchained Labs

  • Identifying formulations for biologics is a painful process
  • Automated buffer exchange coupled with volume control can streamline the buffer exchange process
  • Lower volume analytic measurements provides a means to get to key formulations faster and more effectively
  • Combining automated buffer exchange with low sample volume analytics increases throughput and reduces sample burdens for discovery

Device Development

Solution Spotlight with Enable Injections Enable Injections: A Drug Delivery Technology to Enable your Portfolio

Mike Hooven, President and CEO, Enable Injections, Inc.

  • Technology – overview of the device technology and the benefits it provides to patients, companies, and overall healthcare
  • Business Structure – a mix of pharma and device executives
  • Manufacturing – overview of Clinical and Commercial Manufacturing, highlighting our partnership with Flex
  • Partnering Process – discuss pipeline from feasibility to commercialization

2:25 PM - 3:00 PM

Solution Spotlight with LTS Lohman

3:05 PM - 4:00 PM

iSolve & Networking Refreshments

4:05 PM - 4:35 PM - Case Studies

Small Molecules

The unmet need of biodegradable parenteral controlled release DDS: concept, performance and perspectives

Prof. Dr. Karsten Mäder, Head of Pharmaceutical Technology, Martin Luther University Halle-Wittenberg

  • Parenteral CR is dominated by PLA and PLGA based DDS
  • Many clinically used DDS have undesired release profiles (very high burst release or lag times, complex release profiles)
  • Problems are associated with high acidity of PLA / PLGA monomers
  • Alternatives are needed to increase selection portfolio
  • Promising alternatives will be presented (lipid and polymer based)

Biologics

High-concentration protein formulations: How high is high? Some reflections on feasibility

Patrick Garidel, Associate Director Protein Science, Boehringer Ingelheim

 

  • Between clinical desires and physico-chemical reality
  • The ongoing challenges …
  • Current topics related to HCLF biologics
  • Same related case studies

Technology & Innovation

Novel Developments in the Formulation of Amorphous Solid Dispersions

Thomas Rades, Chair Pharmaceutical Sciences, University of Copenhagen

 

  • Amorphous solid dispersions
  • Drug solubility in polymers
  • Alternatives to polymers
  • Spring and parachute effect
  • In vitro in vivo correlation

Device Development

Risk Management and Formative Human Factor

Muriel Didier, Human Factors Team Head, Novartis

Auto injector is one of the delivery device that users self-injecting in home setting appreciate. It is easy to use, does not look too “medical/aggressive”. Many of them are currently marketed. There are differences in their handling but at the same time they can look similar. Using an auto-injector on a regular basis or only occasionally in emergency situation may make a difference in terms of use errors.

Is it the reason why the FDA has placed the auto injector on the list of devices that need to demonstrate safety and efficacy at least with HFE summative study and even sometimes with real use study?

What could be done to better support the user of auto injectors?

Technical development: Differentiation (right drug, right dosage), handling (different mental models), intuitiveness (identification of needle end, click associated with end of injection). What can be improved? Connectivity, feedback identification, cap removal?

Regulation perspective: Harmonisation of the handling steps by developing some standards like it is done in the automotive field? Pro/Cons

4:40 PM - 5:15 PM - Case Studies

Small Molecules

Absorption Modelling in Regulatory Context – from IVIVC to PBPK

Bertil Abrahamsson, Senior Principal Scientist, Astrazeneca

 

  • In vitro/in vivo correlations (IVIVC)
  • In vitro/in vivo relationships (e.g. safe-space) within context of Quality by Design
  • Physiologically based pharmacokinetic (PBPK) based absorption modelling

Biologics

Protein-based nanoparticles for drug delivery purposes

Juan Irache, Professor of Pharmaceutical Technology, University of Navarra

  • Protein based-nanoparticles: advantages and drawbacks
  • Nanoparticles from food proteins: oral delivery of bioactives
  • Nanoparticles from food proteins: oral delivery of biomacromolecules
  • Albumin-based nanoparticles: intravenous delivery of monoclonal antibodies

Device Development

Bridging between device types during clinical development

 

  • Role of devices during development programs
  • Reasons for bridging during development
  • Options and risks for bridging between devices
  • Evaluation of the minipig as a model to de-risk clinical bridging

8:05 AM - 8:35 AM

Registration & Refreshments

Register, get yourself some refreshments and say hello to your fellow attendees

8:40 AM - 9:15 AM - Keynote

Technology & Innovation

Jet milling Industrialization of sticky Active Pharmaceutical Ingredient using QbD approach

Mostafa Nakach, Head of Pharmaceutical engineering-Paris, Sanofi

 

  • Firstly, an Initial risk assessment based on quality target product profile and prior knowledge was performed to anticipate the risks linked to process development and its scale-up to industrial scale. An action plan was proposed to mitigate the identified risks.
  • Secondly, basic data that are required for process understanding were acquired at lab scale
  • Thirdly, process development was carried out at pilot scale to define the design space were the product will be produced with the appropriate attributes. The process was designed to meet two key performance indicators: high Process yield and compliance to environment hygiene and safety regulations.
  • Finally, process fine tuning was performed at intended scale for industrialization

9:20 AM - 9:50 AM - Case Studies

Small Molecules

Leveraging prediction tools to accelerate product development

Jatin Patel, Head – Oral Product Design & Development, Bristol-Myers Squibb

  • Product development can be significantly accelerated by having a well-defined Quality Target Product Profile (QTPP)
  • Evaluating the risk of achieving the QTPP can be expedited by the use of various prediction tools
  • Prediction tools include those that establish the composition of the product, the stability of the product as well as those that establish the drug delivery requirements
  • An integrated assessment using these prediction tools facilitates the decision making process and positive outcomes for the patient
  • Several process analytical tools can be used to further verify product & process robustness

Biologics

The Power of Predictive Stability Modelling in Drug Product Development

Ellen Verheyen, Senior scientist – Stability Expert, Janssen

  • Introduction to Risk-Based Predictive Stability Modelling to enable fast and better product understanding
  • Case study on the use of stability modelling for shelf life setting and packaging selection
  • Case study on how stability modelling can drive the decision to adapt the formulation strategy

Technology & Innovation

Fill and Finish processing of Biotech products

Jean-René Authelin, Global director of Pharmaceutical Engineering , Sanofi

  • Protein aggregation is a major risk for patients
  • The root cause of aggregation is still highly debated: shear or interfacial effects
  • Whatever the root cause, some precautions and de-risking actions have to be taken for the scale up of the Fill & Finish process. Practical examples will be provided.

Device Development

Panel Discussion: Challenges and opportunities in combination product design, development and use

Bastiaan de Leeuw, Head of Business Development – Drug Delivery, Cambridge Design Partnership

Muriel Didier, Human Factors Team Head, Novartis

  • Critical information needs (from e.g. user, formulation, new device technology) that would enable us to improve design, accelerate development and encourage use
  • The need for and impact of technological advances
  • Bringing molecules, formulations and devices together - the collaboration between suppliers and users

9:50 AM - 10:50 AM

iSolve & Networking Break

10:50 AM - 11:25 AM - Case Studies

Small Molecules

Biopharmaceutics as Catalyst for Quality by Design

Jerome Mantanus, Head of Formulation and Process Characterisation, UCB

 

  • Definition of Biopharmaceutics & relationship with QbD
  • Biopharmaceutical tools (Classifications, IVIVC / IVIVR, PBPK modelling).
  • Overall impact of Biopharmaceutics:
  • CQAs understanding.
  • Prediction in anticipation of clinical studies.
  • Clinically Relevant Control.
  • Virtual evaluation of Life Cycle Management scenarios.
  • Case studies / examples of applications

Biologics

Formulation Design of Smaller Proteins and Insulin for Multiple Injection Dosing and/or Infusion

Svend Ludvigsen, Senior Scientific Director, Novo Nordisk

 

  • Formulation design of insulins, examples
  • Formulation of smaller proteins for subcutaneous injection: balance stability and pharmacokinetic properties
  • Accelerating insulin absorption to the blood stream
  • Rapid acting Insulins for injection are they fitted for pump infusion?

Technology & Innovation

Predictive strategies for physical and biopharmaceutical behaviour in early phase development: from crystallization to taste

Professor Duncan Craig, Director, Chair in Drug Delivery, UCL School of Pharmacy

 

  • Methods for assessing the miscibility of drugs in polymers for solid disperse and other formulations. This will include using quasi-isothermal thermal methods for studying drug solubility in polymers.
  • Methods for understanding the spatial distribution, and changes in spatial distribution, of drugs in polymeric carriers. This will include methods such as nanotherrmal and photothermal analysis as well as transition temperature microscopy.
  • Methods for assessing taste at early stages of development, including both biosensor approaches and animal models.

Device Development

Trends and challenges in inhaled drug delivery transforming respiratory drug portfolios

Gunilla Petersson, Science & Innovation Director, Astrazeneca Sweden

  • Future demands from a drug portfolio perspective
  • Redesign of standard device platforms to match future needs
  • Examples on device inventions addressing new needs
  • Patient centric device improvements for compliance and adherence

11:30 AM - 12:05 PM - Solution Spotlights

Small Molecules

Solution Spotlight with Hovione Amorphous Solid dispersions. What’s next?

Márcio Temtem, Associate Director for Drug Product Development, Hovione

  • Review of the market portfolio
  • Saving API during Formulation Screening (intermediate and drug product)
  • Addressing scale up challenges
  • Alternative manufacturing process

Biologics

Solution Spotlight with Albumedix Formulation of Hard-to-Stabilize Biopharmaceuticals

Eleonora Cerasoli, Senior Research Scientist, Albumedix

  • Recombumin® for the formulation of next generation therapeutics: While in many cases the use of sugars, amino acids and detergents are sufficient there are many more challenging biologics where traditional approaches still fail or are suboptimal.
  • Recombumin® the importance of quality and reliable manufacture: cGMP quality meeting USP-NF standards. Commercially validated in marketed and late-stage clinical drug and vaccine candidates. Expert technical and regulatory support.
  • Recombumin® the importance of a controlled process: The lack of endogenous ligands guarantees batch-to-batch consistency and consequently performance reproducibility. Depending on the mechanism of albumin stabilisation we would expect a different influence of excipients, buffer components and, in case of serum derived HSA by endogenous ligand present.
  • Example: cells cryopreservation: the viability of cells was kept above the set release criteria, even after 72 hours and AlbIX maintained viability of the cells after thawing significantly better compared to HSA.

Device Development

Solution Spotlight with Cambridge Design Partnership

Tom Lawrie-Fussey, Digital Services Specialist, Cambridge Design Partnership

 

The patient experience by CDP: enabling informed design development decisions by using our latest insight tools and techniques to understand actual user behaviour

Abstract: Established methods of user research are invaluable, and yet sometimes fall short of truly de-risking product launches. We have developed a new methodology that helps us to uncover quantified opportunities to innovate, focusing investment decisions on where these will likely have the greatest impact. This approach allows us to link the patient experience to business benefits, by deploying digital tools such as miniature sensors and covert logging platforms, with our considerable data analysis expertise, to uncover quantifiable truth data. CDP is constantly enhancing how we help our clients, from initial ideation and co-creation, through rapid prototyping device development and build, to high-volume manufacturing support.

11:30 AM - 12:05 PM - Case Studies

Technology & Innovation

Solution Spotlight with DFE Pharma Introduction of a new highly compactable, water soluble, lactose based functional excipient (SuperTab® 40LL).

Dr Bastiaan Dickhoff, Product Application Specialist OSD , DFE Pharma

12:10 PM - 12:45 PM - Case Studies

Small Molecules

Oral delivery of hydrophobic drugs using mixed micelles

Rene Van Nostrum, Associate Professor, Utrecht University

  • Hydrophobic drugs having low bioavailability can be formulated in bile-salt containing mixed micelles
  • Those mixed micelles can be stabilized in gastric conditions by a few different ways, including PEGylation
  • PEGylated mixed micelles show good mucus penetration and transport through endothelial cells
  • PEGylated mixed micelles are designed to provide proper bioavailability even under cholestatic conditions

Biologics

Oral Peptide Technologies Progressing in the Clinic: Addressing Challenges to Bring New Products to the Market

Joel Richard, Senior Vice President, Peptides, Ipsen

  • Importance of non-invasive routes of administration for peptides
  • Focus on oral peptide delivery: marketed products and expectations from the market
  • Main challenges for oral peptide delivery: stability and permeability through the intestinal epithelium
  • Key formulation components in oral peptide technologies progressed in the clinic
  • Lessons learnt from oral peptide clinical studies
  • Anticipating challenges to switch from injection to the oral route
  • Innovative approaches to address the remaining hurdle of low oral peptide absorption

Technology & Innovation

Absorption Modelling in Regulatory Context – from IVIVC to PBPK

Bertil Abrahamsson, Senior Principal Scientist, Astrazeneca

  • In vitro/In vivo correlations (IVIVC)
  • In vitro/in vivo relationships (e.g. safe-space) within context of Quality by Design
  • Physiologically based pharmacokinetic (PBPK) based absorption modelling

Device Development

Tissue Specific Delivery Using Caveolae Technology Improves Drug Efficacy

Ruchi Gupta, Scientist I, MedImmune

12:45 PM - 1:45 PM

Networking Lunch

Lunch and Learn Roundtable with ShinEtsu 

Pediatric and geriatric formulations: common challenge

Patient compliance has becoming a hot topic for pharmaceutical companies in the past decade especially for children and the elderly population. Beside the available EMA guidelines and reflection papers, research has been done to compare both patient groups. During this table round, we will review common challenges and provide solutions based on cellulose derivatives for administrating medicines to children and elderly people in terms of:

  • Selection of excipient for solid dosage forms, regulatory aspect
  • Palatability, Acceptability, Swallowability
  • Analytical challenges

1:45 PM - 2:20 PM - Case Studies

Small Molecules

Early “Developability” Assessment, Key to Success in Development Phases

Philippe Lienard, CMC Discovery Coordinator, Sanofi

 

  • How to scientifically select a new molecular entities (NMEs) in a research leads portfolio
  • How to characterize physico-chemical properties as function of the expected Target Product Profile (TPP)
  • Rationally select polymorphism before development
  • How and when do you need to utilize enabling technologies to streamline drug development of molecule presenting limitations/weaknesses.

Biologics

Mucoadhesive polymers: product developments and future trends

Andreas Bernkop-Schnürch, Professor, University of Innsbruck

  • Prolonged mucosal residence time
  • Combination of mucoadhesive and in situ gelling polymers
  • IVIV correlation of different in vitro test methods
  • Product developments

Technology & Innovation

Microneedles: From Basic Research to a Drug Product

Yash Kapoor, Associate Principal Scientist, MSD

 

  • Advantages of microneedles as a minimally invasive delivery system
  • Improving health and compliance worldwide as needle-phobia declines
  • Intelligently addressing the cold-chain burden

Device Development

Dry Powder Inhaler (DPI) products: From small to industrial scale at Chiesi

Isabel Sequeira Lopes, Senior Formulation Scientist/Project Coordinator, Chiesi

The goal of this presentation is to guide the audience in the some of the processes associated to the development of a Dry Powder Inhaler (DPI) product, at Chiesi, from pre-clinical up to full scale industrialization. Additionally, the objective will be to present some of the methodologies being currently used to aid a comprehensive product and process understanding.

2:25 PM - 3:00 PM - Solution Spotlights

Biologics

Solution Spotlight Revox Sterilization Solutions Sterilization Solutions for Todays Drug/Device Combinations

Herve Barre, Chef de projet Sterilisation WW, Industrial Engineer , BD Medical Pharmaceutical Systems

James A. McCoy, Staff Scientist, Head of Sterilization R&D/Innovation Center, BD Medical Pharmaceutical Systems

  • Explain the challenges and processes today - regulatory/quality/operational and trends that indicate the need for more elegant solutions.
  • Incorporating electronic devices
  • Present research/testing findings
  • Present solutions offered through VPA (or other novel approaches)
  • Next steps
  • Future of the industry and how VPA will make an impact

Technology & Innovation

Solution Spotlight with Lonza

Prof. Dr. Sven Stegemann, Director Pharmaceutical Business Development, Lonza

Moving multi particulates from pediatric formulation to patient centric drug product design

  • Patient centric drug product design – definition, regulation and outcomes evidence.
  • Review of multiparticulate technology platforms
  • Case study

Device Development

Solution Spotlight with Portal Instruments Transforming the Patient Experience with a Novel Needle Free Drug Delivery Device

Patrick Anquetil, CEO, Portal Instruments

 

Delivering high viscosity, high concentration biologics is a challenge for the biopharma industry. Injections are slow, painful, and patients dislike needles, leading to sub-par adherence rates for these treatments. Portal is transforming this patient experience with a needle-free device that is computer-controlled, easy to use, and fits into the patient’s life. The device platform is viscosity agnostic and can be used seamlessly across a vast portfolio of injectables. The Portal cartridge was developed in collaboration with Gerresheimer of Germany and is easily pre-fillable with standard fill/finish lines compatible with the nest and tub format.

  • In injectable therapies, the drug delivery device embodies the experience that a patient has with a treatment.
  • There is a competitive advantage for pharma companies who understand this paradigm and are able to market a drug / device combination product that is intuitive, easy to use and reliable
  • Portal Instruments has developed a needle free drug delivery device that transforms the patient experience as no sharps are used to perform an injection.
  • The Portal device is computer controlled and automatically detects and adjusts for the drug volume and viscosity. Drug developers can use this feature to widen the range of design parameters available to them.

3:40 PM - 4:15 PM - Case Studies

Small Molecules

User Friendly Medicines

Clive Wilson, J.P. Professor of Pharmaceutics, University of Strathclyde

Medicines need to be designed to reflect the lifestyle and physiological status of patients at the extremes of age, we have to consider palatability and appropriateness of medicines

Medicines are expensive items. If they are not taken as intended, issues with regard to effectiveness will arise and unnecessary costs will be expended by the health provider

Patients may have hidden pathophysiological changes that cause issues with exposure. At a certain stage, additional support including home help may be necessary.

Controlled release dosage forms and other novel constructs may reduce the need for expensive nursing care

Biologics

Thermal Stabilization of Freezing Sensitive Adjuvanted Vaccine Formulations Through Spray Drying

Olivier Brass, Formulation Scientist, Sanofi Pasteur

 

  • Maintaining thermal stability and immunogenicity during manufacturing and cold chain storage of vaccines.
  • Spray-dry as an emerging technology for biologics.
  • Reviewing the POC study on stable dried adjuvant-containing vaccine formulations produced through spray dry

Technology & Innovation

Novel approaches for efficient topical delivery of small - and biomacromolecules

Prof. Dr. Sarah Hedtrich, Professor, Freie Universitat Berlin

  • Topical delivery of therapeutic proteins
  • Polyglycerol-based nanocarriers
  • Stimuli-triggered drug release
  • New approaches for severe genetic skin diseases

Device Development

Predictive tools for rational development of dry powder inhalation (DPI) products and process

Dr. Amrit Paudel, Assistant Professor Institute of Process and Particle Engineering, Graz University of Technology

  • Designing and manufacturing micro particles with delivery method in mind
  • Device-capsule-formulation interaction
  • Recent advancement in the use of physiologically based pharmacokinetic modelling (PBPK)

4:20 PM - 4:55 PM - Case Studies

Small Molecules

Solution Spotlight with Patheon Scalability, a Topic to Consider in Early Development?

Kaspar van den Dries, Senior Director and Principle Scientist, Solid Dose Development, Patheon, Part of Thermo Fisher Scientific

There are many challenges to progress your new molecule through the various stages of clinical development: Will the molecule have the appropriate pharmacokinetic, safety and efficacy profiles? How do I set up the clinical studies to obtain proof of concept?

Typically, concerns around formulation options receive little attention, let alone any considerations around scale-up. Nevertheless, the path you start in early development will typically determine the route into late stage development and commercialization and the challenges you might face.

Join Kaspar van den Dries as he discusses how developing a solid understanding of the API, formulation, and process during early stage development can ensure your molecule is prepared for scaling to late stage development and commercialization.

Biologics

Solution Spotlight biopharma Group Freeze Drying Protein Formulations: A Journey from Challenge to Opportunity

Dr. Roberto Castangia, Global Sales Manager, biopharma group

 

 

Device Development

Solution Spotlight with Medical Device Usability Ltd Auto injectors – an Insight into User Needs

Richard Featherstone, Managing Director, Medical Device Usability Ltd

4:20 PM - 4:55 PM - Solution Spotlights

Technology & Innovation

Solution Spotlight with ADM SIO Tackling the Challenge of Natural Variability Related to Plant-Based Pharmaceutical Ingredients

Stéphanie Bureau, R&D Director, ADM-SIO

 

  • Importance of a strong knowledge of raw material variability origins when working with natural based products
  • Major importance of raw material sourcing and close collaboration with suppliers to guarantee a high level quality and good understanding of pharma specific needs
  • In-depth understanding of critical product parameters and their link to process parameters to design robust process and ensure constant quality
  • Strong process knowledge to know how to adapt process conditions to raw material variability

5:45 PM - 5:55 PM

8:00 AM - 8:20 AM

Registration & Refreshments

Register, get yourself some refreshments and say hello to your fellow attendees

8:30 AM - 9:05 AM - Keynote

Technology & Innovation

9:10 AM - 9:45 AM - Case Studies

Small Molecules

Predictive Stability in Pharmaceutical Development

Helen Williams, Associate Principal Scientist, AstraZeneca

 

  • Applications of predictive stability tools in pharmaceutical development, focusing on Accelerated Stability Assessment Program (ASAP) studies and packaging predictions.
  • Case studies demonstrating the use of predicted stability data in regulatory submissions from ASAP studies in early clinical submissions through to packaging predictions in a marketing application.
  • Results of a recently published survey of IQ member companies on the use of predictive stability across the industry
  • Update from the IQ Risk-Based Predictive Stability Working Group

Biologics

New Approaches to Advance Therapeutic Drug Delivery across the Blood-Brain Barrier to Brain and Brain Tumours TTUHSC

Stephen Buckley, Head of Department, Absorption and Biological MS, Novo Nordisk

  • The blood-brain barrier restrict the brain delivery of most currently active anticancer agents used in the treatment of brain tumours.
  • The magnitude of this restriction is extremely large (50-200 fold) for some of the most commonly used agents cytotoxic agents (paclitaxel, doxorubicin, vinorelbine,) and newer molecularly-target agents.
  • In only a very small subset (<5%) of brain metastases is the barrier sufficiently compromised to allow marked drug accumulation.
  • This restriction was found in matching human brain metastases.
  • This drug delivery compromise can be overcome in brain metastases using several approaches, including drug agents which show poor affinity for barrier active efflux transport, enhanced barrier passive permeability, elevated active efflux, or molecularly targeting mechanism, such as those in using LRP, transferrin, insulin receptor, or other mechanisms.

Technology & Innovation

Recent Advances in the Development of Ocular Drug Delivery Systems

Gregoire Schwach, Head of Drug Delivery, Pharmaceutical Development and Supplies Biologics, EU F. Hoffmann-La Roche

  • The eye: a complex organ to target and a big unmet need for even longer acting therapies
  • Drug delivery approach (e.g. long acting, sustained release) has impact on major product development areas like processes, formulation, analytics, technologies
  • Benefits of investigational predictive ocular models during early development
  • Keep it simple to increase probability of (technical) success, many other things can go wrong!

9:50 AM - 10:25 AM - Solution Spotlights

Small Molecules

Solution Spotlight with Quotient Sciences Accelerating development of spray dried dispersions for early phase clinical trials and beyond

Nikki Whitfield, Vice President CDMO Services, Quotient Sciences

  • Benefits of spray-drying to address poor drug solubility
  • Fit-for-purpose and fit-for-phase systems in early clinical research
  • Effectively transitioning to solid oral dosage forms
  • Adaptive manufacturing “tailored” to the clinical study and patient recruitment
  • Overcoming challenges with highly potent molecules

Biologics

Solution Spotlight with Roquette Exploration of Anti-Aggregation and Protein Stabilization Properties of Novel Carbohydrates’

Sofiya Yashchuk, Technical Applications Specialist, Roquette

 

Protein aggregation is a major challenge encountered during manufacturing, storage and transportation of biopharmaceuticals. Aggregation of proteins is often irreversible, leading to aggregates that are inactive and potentially immunogenic; resulting in reduced efficacy and possibly toxicity of the biologic drug. Thermal stability of proteins can be improved by formulating with carbohydrates (commonly: sucrose, trehalose, and sorbitol), which prevent protein unfolding and structural loss. In our study, we investigated the stabilizing e-ffects of two ß-cyclodextrin derivatives (KLEPTOSE® HPB hydroxypropyl-ß-cyclodextrin with MS=0.65, and KLEPTOSE® HP hydroxypropyl-ß-cyclodextrin with MS=0.9), and the polyol Maltitol (a hydrogenated disaccharide), using high-throughput formulation screening (iFormulate™) and nanoDSF (Di¬erential Scanning Fluorimetry). The iFormulate™ DOE plate design provided a rapid evaluation of four critical formulation variables: pH, ionic strength, buffer concentration, and stabilizer concentration. The influence on thermal stability of two proteins, the human growth hormone (hGH) and an IgG-type therapeutic antibody (Infliximab), was evaluated under the DOE approach. Simultaneous evaluation of Tm (melting temperature) and relative degree of aggregation of proteins, with increasing concentrations of Maltitol, KLEPTOSE® HPB and HP, was performed. Our study results indicate that all three carbohydrates can be effective aggregation modulators, offering a wide range of application possibilities to address process and formulation challenges.

Technology & Innovation

Solution Spotlight with Merck KGaA

Dr. Finn Bauer, Head of Solid Formulations R&D, Merck KGaA

  • Hot melt extrusion with polyvinyl alcohol (PVA)
  • Characterization of single and multi-phasic amorphous systems
  • Evaluation of solubility and permeability enhancement
  • Discussion of stability considerations

10:25 AM - 10:45 AM

Networking Break & Refreshments

10:45 AM - 11:20 AM - Case Studies

Small Molecules

Ensuring Age-Appropriate Medicines for Children

Hannah Batchelor, Senior Lecturer in Pharmaceutics, Formulation and Drug Delivery, University of Birmingham

  • What does acceptable medicine mean to children?
  • How is acceptability measured?
  • What medicines do children accept?
  • How can we ensure that all future medicines are acceptable to children?

Technology & Innovation

Innovative Ocular Drug Delivery Systems

Thakur Raj Singh, Senior Lecturer in Pharmaceutics, Queen's University Belfast

  • Visual impairment and blindness are potentially the most devastating health problem worldwide.
  • Drug delivery to the eye is challenging due to the extremely delicate nature, relative inaccessibility, and barrier properties of ocular tissues
  • Topical and systemic routes of ocular drug delivery result in low or sub-therapeutic drug levels; drug delivery implants need surgical implantation.
  • Injections into the eye using conventional hypodermic can provide direct access to the target tissues. However, this method is highly invasive and causes considerable discomfort, pain and associated with a number of side effects.
  • We are working on innovative devices to allow minimally-invasive means of drug delivery to the eye. And, on formulations to allow us to maintain constant drug levels for long-term thereby avoid issues associated with frequent administration.

11:20 AM - 11:55 AM - Case Studies

Small Molecules

Suspendilicious: unveiling the marvels of lipid suspensions

Kristof Kimpe, Principal Scientist, Janssen Pharmaceutica (a division of Johnson & Johnson)

  • Pre-clinical oral dosing of suspensions of solid dispersions
  • Pre-clinical oral dosing of suspensions of salts of very weak bases

 

Furthermore, the investigation of factors which influence the mass transfer kinetics from lipid suspensions to gastro-intestinal fluids will be discussed along with strategies to modulate the mass transfer kinetics.

Biologics

Development of Antibody drug conjugate drug products

Daniel Schweizer, DP Project Lead TD NBE’s, Novartis

 

The CMC development of ADCs can be regarded as an extension of current MAb development strategies, and standard MAbs are further processed in order to attach cytotoxic payloads. This extension impacts resources and time required for process development (conjugation, formulation, and analytics), the complexity of these processes as well as the final product. The presentation shall provide insights into drug product development of ADCs at Novartis including:

Technology & Innovation

Nucleic Acid Based Therapeutics for Cancer: Targeted Delivery Systems and In Vivo Bioimaging

Manfred Ogris, Professor Pharmaceutical Sciences, Head of the Laboratory for Macromolecular Cancer Therapeutics, Division of Clinical Pharmacy and Diagnostics Vienna University

  • Non-viral gene delivery molecular conjugates
  • Disseminated cancer models
  • Multimodal imaging and optical tomography

11:55 AM - 12:30 PM - Solution Spotlights

Small Molecules

Solution Spotlight with PMC Isochem: The Multiple Functions of Vitamin E TPGS; a Solution for Your Drug Delivery Innovation?

Yves Robin, R&D Director, PMC Isochem (PMC Group)

Vitamin E TPGS overview:

  • Product characteristics
  • Approved & commercial formulations
  • Properties in drug delivery
  • Functions in drug delivery
  • Analog

Biologics

Solution Spotlight with Foster Innovation Twin-Screw Melt Granulation of Thermally Labile Drug: Effect of Processing Conditions

Feng Zhang, Assistant Professor, University of Texas at Austin

 

  • Processing temperature and reduction of GABA particle size during granulation are significant factors that can affect the chemical stability of GABA.
  • Small particle size of HPC can cause higher degradation due to higher surface area in contact with GABA particles.
  • Processing parameters (screw speed and feed rate) should be optimized to achieve the balance between improving compaction properties but still maintaining chemical stability of GABA.

Technology & Innovation

Solution Spotlight with Nanoform Nanoparticle Production with Supercritical CO2

Professor Edward Hæggström, Chief Executive Officer, Nanoform

  • We produce Pure Homogenous Nanoparticles under 20 nm
  • Improving bioavailability
  • Nanonization services aimed at present for pre-clinical investigations
  • Gearing up for process optimization, clinical trials and GMP manufacturing

12:30 PM - 1:30 PM

Networking Lunch

1:40 PM - 2:15 PM - Case Studies

Small Molecules

Quantitative Biopharmaceutics: Use of Modelling and Simulation to Inform API and Formulation Decisions

Filippos Kesisoglou, Senior Principal Scientist, Biopharmaceutics and Pharmaceutical Sciences, MSD

  • Case studies of use of absorption modelling for early phase API/formulation selection
  • Case studies of use of modelling for bioequivalence simulations
  • Addressing emerging gaps – case studies on simulation of amorphous solid dispersions and nanoparticles

Biologics

Bioinspired drug delivery systems

Lorenz Meinel, Chair for Drug Formulation and Delivery, Wurzburg University

  • Nature’s strategies for creating “drug depots”
  • Controlling attachment sites in biologics
  • Emulating natural strategies in drug delivery applications
  • Emulating natural strategies for easy to use diagnostics

Technology & Innovation

Cryopreservation of ex vivo Cell and Gene Therapies

Richard Zajicek, Head of Formulation Development, Cell and Gene Therapy Group, GSK

  • ex vivo stem cell therapies for ultra-rare diseases.
  • Current formulation has a limited shelf life restricting patient accessibility and release strategy.
  • A change to a cryoformulation will increase the shelf life to months/years
  • Careful assessment of the CQAs is vital along with comprehensive comparability

2:15 PM - 2:50 PM - Keynote

Small Molecules

Applying Biopharmaceutics in Global Public Health – Deworming Children More Safely

Christophe Tistaert, Senior Scientist, Janssen Belgium

  • Neglected tropical diseases
  • Mechanistic absorption modelling of locally acting compoundss
  • Pediatric formulation development
  • Food effect predictions