Programme 2021

15th – 17th March 2021, All Timings in CET

SUBMIT A POSTER

7:40 AM - 8:40 AM

Registration & Refreshments

8:40 AM - 8:50 AM

Chair's Opening Remarks

8:50 AM - 9:35 AM - Keynote

Technology & Innovation

Connected Drug Delivery Devices and their Role in Enabling a Flexible Care Setting

Beate Bittner, Product Optimization Franchise Leader, Roche

Today, in times of increasing pressure on global healthcare systems and in light of the COVID-19 pandemic, the value of improved drug delivery to reduce dosing complexity is broadly recognized. Especially for biotherapeutics that need to be dosed parenterally, product optimizations that facilitate a flexible care setting in which patients can choose the place of drug administration are increasingly in the focus of manufacturers, regulators, payers, and healthcare providers. In this context, subcutaneous dosing alternatives to intravenous infusions and ready-to-use device technologies have been demonstrated to reduce administration related costs and resources.

A key requirement when it comes to parenteral administration outside of a controlled healthcare setting is that patients and lay caregivers understand the administration procedure and still adhere to the treatment regimen. So-called connected devices and accompanying health apps may play a pivotal role in ensuring drug administration outside of a clinic and in enabling early treatment decisions by ensuring an ongoing dialogue between the patient and healthcare provider.

The presentation will discuss the evolution of drug delivery technologies that facilitate administration of biotherapeutics. The opportunities and challenges of connected drug delivery devices in supporting a flexible care setting will be discussed.

9:40 AM - 10:15 AM - Case Studies

Small Molecules

Understanding and Designing Manufacturing Operations and Product Behavior with Coupled CFD-DEM Modeling

Andrea Benassi, CMC – Chemistry Manufacturing & Controls, Drug Product manufacturing & Innovation, New Technology & Innovation Scientist, Chiesi

In our activity of DPI process and product design we daily deal with complex, non-linear, multi-phase flows involving powders and gasses, e.g. the dose entrainment in the swirling air flow inside inhalers, the fine API particles deposition in the deep airways, the air/nitrogen borne particles colliding during API or excipient micronization in jet mills. Standard techniques of computational engineering, such as computational fluid dynamics (CFD), discrete element modeling (DEM) and their coupling, proved to be ideal tools to tackle the above-mentioned phenomena. Applications of the coupled CFD-DEM approach to both process and product design will be shown highlighting their potential and describing their current limitations.

Technology & Innovation

How to Utilize Transporters for Brain-Targeting

Kristiina M. Huttunen, Adjunct Professor (Docent) and Finnish Academy Research Fellow, University of Eastern Finland

During the drug development, it has been very challenging to create potent compounds with perfect pharmacokinetic properties. Therefore, for example, the majority of central nervous system (CNS) drugs fail in clinical trials as they are not delivered to their site of actions, and thus, they lack efficacy and cause toxic side effects. The blood-brain barrier (BBB) prevents the uptake of numerous drug molecules into the brain, however, the cell membranes and the delivery to specific brain cell types may also create a secondary barrier for CNS-drugs. In this presentation, I will show how L-type amino acid transporter 1 (LAT1) can be utilized for brain-targeting purposes and what kind of methodology is required to develop transporter-utilizing brain-targeted prodrugs. The presentation will also cover the main challenges that can be faced when working with this approach.

Device Development

Microneedles for Drug and Vaccine Delivery

Ryan F. Donnelly, Chair in Pharmaceutical Technology , Queen's University Belfast

This presentation will look at advanced microneedle delivery systems, their utility in a pandemic situation and their progression to the clinic

10:20 AM - 10:55 AM - Solution Spotlights

Small Molecules

Unique Nanoforming Technology for Poorly Soluble APIs by Nanoform

Satu Lakio, Pharmaceutical Development Director, Nanoform

Controlled Expansion of Supercritical Solutions (CESSᵀᴹ) technology produces nanonised ‘designed-for-purpose’ API particles. This enables poorly soluble molecules in the pharmaceutical pipeline to progress into clinical development by increasing their rate of dissolution and improving their bioavailability. Our unique nanoformingᵀᴹ technology also provides novel opportunities in diverse value enhancing drug delivery applications. The patented technology is based on supercritical CO2. The drug solution is expanded through a controlled process to produce pure drug nanoparticles. The process is more controlled than conventional supercritical technologies, and it produces smaller and more uniformly sized particles. This presentation will give an introduction Nanoforms technology and presents case studies to support that Small in powerful.

Biologics

Protein or Not? Advanced High Throughput Aggregate Analysis with the Aura

Bernardo Cordovez, Chief Science Officer and Founder, Halo Labs

In protein-based formulations, distinguishing aggregated API from other particle types is important for understanding the root cause of instability. Until now, existing methods have been either unreliable or too cumbersome and difficult to use in many workflows. Here we introduce the Aura, a 96-well low-volume aggregate and particle imaging system that can rapidly size, count, and characterize particles and identify them as proteins, non-proteins, hydrophobic, or other types of molecules.

Technology & Innovation

Dissolvable Microarray Patch Systems – Microneedles That Are Able to Meet The Content Uniformity Requirements

Sebastian Braun, Director - Head of Formulation Development and Head of Manufacturing for Innovative Injection Systems, LTS LOHMANN Therapie-Systeme AG

Different microneedle systems are in discussion for industrial application. Only a few seem to be able to meet the regulatory standards of pharmaceutical drug products. One of these systems are dissolvable microneedles. Due to the unique manufacturing process and the simple way of incorporating the API in the needle polymer, this system tackles the challenges of content uniformity far better compared to the other microneedle systems. Process development data suggests that dissolvable microneedle systems show content uniformity comparable to standard dosage forms. Data that brings this technology one step closer to the market.

10:55 AM - 11:45 AM

iSolve & Networking Break

11:45 AM - 12:20 PM - Case Studies

Small Molecules

Powder Characterization for Small Molecule Formulation – understanding the Particle-Powder-Processability Interface

Carolin Riehl, Principal Scientist | Lab Head Drug Delivery & Innovation, Merck

Processability issues in development of oral solid dosage forms are often triggered by drug substance variability or scale-up effects. In this context, we need predictive and discriminating analytical methods to select both the right formulation and the right process. Powder rheological and as well as mechanical properties are not assessed at all or at least standardized methods are missing. Therefore, we need to characterize the drug substances, excipients and powder blends in an appropriate manner to better understand how we can use the data to optimize particle morphology and predict processability.

Biologics

Disruptive Technologies for Formulation, Manufacturing and/or Delivery: Enabling Advancements for Biologics and Vaccines Products into the Future

Jeffrey T. Blue, Executive Director, Vaccine Drug Product Development PR&D, MSD

  • Abstract TBC

Technology & Innovation

How to Anticipate Formulation and Stability with Modeling?

Olivier Brass, Senior scientist, lead of research unit, research department , Sanofi

  • Combine modelling approaches for formulation and stability description and prediction
  • Molecular understanding of excipient formulation through molecular dynamic
  • Accelerated stability as a good prediction way for long-term and thermal excursion prediction
  • Case studies

Device Development

A Unique Needle-Free Delivery System for Injectable, Precision Engineered, Thermally Stable, Unit Solid Dose Vaccines

David Grant, Head of Device Development , Enesi Pharma

  • Reviewing the top historic problems with needle free devices
  • Generating high pressures inside the devices and maintaining flexibility
  • Delivering biologics via needle-free devices

12:25 PM - 1:00 PM - Solution Spotlights

Small Molecules

Minitablets – a Patient Centric Dosage Form

Luigi Boltri, Ph.D., Senior. Director Pharmaceutical Sciences, Business Support and New Technologies, , Adare Pharma Solutions

  • There is a significant unmet need to formulate medicines that are easy to administer to children to optimize acceptance, encourage adherence and improve health outcomes.
  • Minitablets can be one of the solutions also for elderly patients and those suffering dysphagia. Flexibility for dose adjustments is another key advantage to be underlined.
  • Thanks to recent guidelines, scientific findings and a growing understanding of children swallowing capability, Minitablets are more and more considered a popular patient centric solution, especially when administered as a sprinkle.
  • On the other side Regulatory Authorities raised their concerns about the practice of sprinkling and mixing drugs with liquids and soft food, potentially impacting the bio-pharmaceutical properties of the drug product.
  • An alternative and compliant dosing vehicle should be therefore identified in order to maintain the possibility to administer Minitablet, but more in general small multiparticulates, as a sprinkle, when swallowing difficulties can have an impact on therapy adherence.

Biologics

Parenteral Delivery of Therapeutic Proteins and Peptides using Biodegradable Silica Matrix

Frederic Dargelas, Director, Head of Business Development and Alliance Management, DelSiTech

Parenteral delivery of therapeutic proteins and peptides using biodegradable silica.

DelSiTech Silica Matrix is an advanced delivery technology for parenteral and local administration of injectable depot, implant and eye drop dosage forms. The proprietary technology is based on Silica (silicon dioxide, SiO2) Matrix into which the active ingredient is embedded. The matrix can be designed to biodegrade at the required rate to assure a tightly controlled release of the active substance over extended periods of time, providing a unique therapeutic effect in situ and causing low systemic and local adverse events compared to alternative delivery systems.

The dissolution of the matrix takes place mainly through an erosion mechanism. The biodegradation process does not change the pH within silica gel nor in the surrounding tissue in contrast to most commonly used alternative drug delivery matrices. The Silica Matrix is inert and can stabilize the APIs, from small molecules to large proteins.

In this presentation we will review the case of parenteral delivery of therapeutic proteins and peptides using biodegradable silica, and how non-porous silica matrix can provide long-acting protein

Technology & Innovation

Using Computer Modelling to Inform Formulation Development for Small Molecule Drugs and Biologics

John C. Shelley, Fellow, Schrödinger

  • API and excipient property prediction for small molecule drug formulations
  • Molecular modelling provides a basic understanding of the structure and behaviour of drugs as formulated and inform decision making in drug formulation that compliments experimental data
  • Characterization of drug-drug and drug-excipient including drug-polymer association in small molecule and biologics formulations
  • Predict viscosities and provide structural insights into concentrated globular protein and antibody solutions including excipient effects

Complex and evolving structures, often in fluid states, play a crucial role in the pharmaceutical, industry. Selecting and combining the right ingredients in the right manner to obtain optimal properties are essential for success given the inherent challenges and a competitive market. With advances in modern simulation techniques and computer hardware, molecular modelling is starting to provide timely and invaluable information that is complementary to experimental characterization.  We present a cross-section of capabilities within Schrödinger’s Material Science Suite for modelling either small-molecule drugs or biologics during the development process.   For small-molecule drugs workflows are available for characterizing degradation risk and crystal morphology as well as calculating elastic constants (bulk modulus, shear modulus, etc.), powder diffraction pattern, glass transition temperatures (Tg), diffusion constants, melting points, water adsorption, and solubility.   For biologics our toolset permits the prediction of aggregation propensity, titration curves and isoelectric points, among other things.    For both small-molecule and biologics formulations powerful simulation tools using atomistic, coarse-grained models to permit the characterization of molecular interactions and nanoscale structuring, sometimes within otherwise disordered bulk systems (e.g., self-assembly of polymer-based structures, dissolving amorphous solid dispersions, liposomes and protein-excipient interactions). We will present some prototypical studies for both small molecule drugs and biologics.

Device Development

Enabling a Flexible Autoinjector Platform for the Delivery of High Viscosity or High-Volume Parenteral Formulations

Alex Vasiev, Manager of Device Development , Oval Medical Technologies

Injectable drugs are expected to be the largest growth segment within drug delivery in the coming decade. Most of this growth is driven by the continued development of new biologic drugs and biosimilars. Biologics tend to have high dosage mass, higher viscosity and aggregate at higher concentrations. Balancing formulation stability, manufacturability, pharmacokinetics and injectability can be a great challenge.

As the industry moves towards self-administration to reduce healthcare costs and improve convenience for patients, there is a compelling need for simple-to-use, low-cost disposable devices for domestic use. Considerations for the secondary packaging often comes very late in the development process, diminishing the number of viable options.

Oval has developed an autoinjector technology to offer flexibility in early-stage development by allowing for the delivery of either high viscosity or high-volume biologic formulations. The system uses Oval’s proprietary cyclic olefin technology and is lubricant free. Optimisation for extremely high pressures means the system is able to achieve performance not possible with traditional glass primary packaging.

1:00 PM - 2:00 PM

Networking Lunch

2:00 PM - 2:35 PM - Case Studies

Small Molecules

New Insights into Developing Enabling Formulations for Problem Drugs

Anette Müllertz, Professor, Head of Center, University of Copenhagen

  • New approached to formulate poorly soluble drugs
  • Lipid based drug delivery systems
  • Amorphous drugs
  • In vivo predictive in vitro models

Technology & Innovation

Lyophilization Cycle Transfer and Scale-Up Case Study: Impact of Freeze Dryer Configuration

Stefan Schneid, Laboratory Head Formulation Development Parenterals, Bayer

  • Introduction and critical factors for process transfer
  • Influence of freeze dryer configuration and vial loading
  • Case studies for project transfers
  • Recommendations for risk mitigation

2:40 PM - 3:15 PM - Case Studies

Small Molecules

Drug Delivery Across Biological Barriers in the Context of Infectious Diseases

Claus-Michael Lehr, Head, Dept. of Drug Delivery (DDEL), Helmholtz-Institute for Pharmaceutical Research Saarland (HIPS)

Infectious diseases are on the raise and increasing challenge to human health with mortality rates predicted to soon exceed those of cancer and other diseases. The lecture will provide an introduction into this still emerging field of drug delivery research with focus on novel nanomedicines as innovative human cell- and tissue models as alternative to animal testing 

Bacterio-mimetic nanocarriers for combatting intra-cellular infections 
Predicting & optimizing intra-bacterial delivery of anti-infectives across the gram-negative bacterial cell envelope  
Non-invasive delivery of antigens and drugs via skin hair follicles  
Combatting infections of the lung by biofilm-forming bacteria

Biologics

Challenges in the Pharmaceutical Development of Very Low Concentration Dosage Forms for Highly Potent Immunotherapy Molecules

Naila El Kechai, Formulation and Drug Product Team Leader, Sanofi

  • Formulation to maintain safe, stable, low concentration dosing
  • Overcoming adsorption to ensure concentration consistency during DP manufacturing process
  • Ensuring safety and dose accuracy during preparation and clinical administration

Device Development

Impact of article 117 on the Development of New Combination Products

Dr. Fatima Bennai-Sanfourche, Director Medical device Safety, Medical Device Safety Officer, Bayer

3:15 PM - 4:05 PM

iSolve & Networking Break

4:05 PM - 4:15 PM

Poster Presentation - Advanced Non-Viral Gene Delivery Vehicles

Jian Bao, CEO, ZY Therapeutics

Poster Presentation - Prediction Model of Drug Release from Long-Acting Implantable Dosage Forms Obtained by 3D Printing

Giuseppe Manini, PhD student, ULB

Poster Presentation - Characterisation and Delivery of Ultra-Viscous and Non-Newtonian Formulations

James Hance, Senior Device Development Engineer, Oval Medical Technologies Ltd

Poster Presentation - Novel Nanomedicines with the DELOS Platform: Nanoformulation of an Enzyme in Peptide Targeted Nanovesicles to Treat Fabry Disease

Alba Córdoba Insensé, Technology Transfer & BD Director, Nanomol Technologies S.L.

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

Small Molecules

Biologics

Challenges and Opportunities in Cell Therapy Formulation and Delivery

Bharathi Vellalore, Scientist, Drug Product Development Discovery, Product Development and Supply, Janssen

  • Formulation of autologous and allogeneic cell therapy products
  • Cold supply chain: Freezing, storage, transport, thawing and administration
  • Novel drug delivery approaches for solid tumor CAR-T therapy

Technology & Innovation

Device Development

How to use Smart Devices Intelligently to Deliver Outcomes

Jace Blackburn, Smart Device Engineer, Genentech

  • Introduction to Smart DevicesTypes of Smart Devices & data they generate
    • Current Smart Device applications on the market
  • How to evaluate the fit of Smart Devices with a brand team’s digital strategyStrategies to dig into with your marketing colleagues
    • Strategies that should raise red flags
  • How to execute a make vs buy decision for companion applications to support your digital strategy
  • Delivering on the prioritized outcomes through the implementation of behavior design
    • Intro on behavior design
    • Analyzing reasons for medication non-adherence or non-persistence with a behavior design lens

5:00 PM - 5:45 PM - Panel Discussion

Device Development

The Role of Microneedle Delivery Systems in the COVID age?

Ryan F. Donnelly, Chair in Pharmaceutical Technology , Queen's University Belfast

Twinkle Christian, Process Development Scientist, Drug Product Technologies Group, Amgen

David Hipkiss, Chief Executive Officer, Enesi Pharma

Sudhakar Garad, Global Head of Chemical and Pharmaceutical Profiling, Novartis Institutes for BioMedical Research

Anne Moore, Professor , University College Cork

Jeffrey T. Blue, Executive Director, Vaccine Drug Product Development PR&D, MSD

This would consider how microneedles can allow at-home delivery of vaccines and medicines ordinarily requiring injection by a healthcare professional, thus taking pressure off over-burdened health systems on a long-term basis and talk about opportunities and challenges.

5:35 PM - 5:40 PM

Chair’s Closing Remarks

8:00 AM - 8:45 AM

Registration & Refreshments

8:45 AM - 8:50 AM

Chair's Opening Remarks

8:50 AM - 9:25 AM - Keynote

Biologics

The Boon and Bane of Polysorbate

Patrick Garidel, Head of Process, Purification and Pharma Development, Boehringer Ingelheim

Polysorbate 20 and 80 , also known as Tween, are the most used excipients for the stabilisation of biologics, due to specific beneficial properties. About 90% of commercialised biologics contain polysorbate. A number of vaccines too.

In the past, however, observations were published showing, that polysorbate may degrade and may be involved in the formation of particles. According to publications, this issue seems to be observed very broadly. The current talk summarises the current knowledge about polysorbate degradation pathways, analytics, quality, application etc.

9:30 AM - 10:05 AM - Case Studies

Small Molecules

Humidity Induced Collapse in Freeze Dried Cakes

Daryl Williams, Professor of Particle Science , Imperial College

Maintaining low moisture content is seen as crucial to sustaining long term stability in freeze dried (FD) cakes as higher moisture could lead to cake collapse, degradation and loss in biological potency. Using a combination of gravimetric data and video images captured from a Dynamic Vapour Sorption instrument the onset humidity Collapse Point (RHcp), the humidity Onset Crystallisation (RHc) and Onset Glass Transition (RHg) points for a series of freeze dried cakes at 10, 25 and 40°C have been determined. The moisture sorption behavior with respect to cake collapse and other morphological phase transitions are reported for a two freeze drying excipients and one product formulation; sucrose, trehalose (both 5% w/w) and an influenza antigen (A/Wisconsin/15/2009 H3N2 NYMCX-183, formulated with 1.1% w/w sucrose).  Stability maps for all three formulations tested were reported as a function of %RH and temperature using the methods described in this work, thus direct visualization of collapse behavior for any FD cake can now be standardized and routinely determined, facilitating the formulation of FD products with improved stability and storage performance.

Technology & Innovation

Liquid Formulations for a Combination of Low and High Dose Drugs

Jayne Lawrence, Director, NoWCADD, University of Manchester

  • Conflicting demands of formulating low and high dose drugs
  • Nanosuspensions good for high dose drugs
  • Micro- and nanoemulsions good for low dose drugs

Technology & Innovation

BBB Pathophysiology-Independent Drug Delivery in Traumatic Brain Injury

Nitin Joshi, Instructor of Anesthesia, Center for Nanomedicine, Brigham and Women’s Hospital, Harvard Medical School

Over past few decades, researchers have identified several promising therapeutic agents that can mitigate the long-term sequelae of traumatic brain injury (TBI). However, their clinical translation has been limited due to poor permeability across the blood-brain barrier (BBB). One approach to overcoming this challenge involves treatment administration while the BBB is transiently breached after injury. However, it offers a limited window for therapeutic intervention and is applicable to only a subset of injuries with substantially breached BBB. We have developed a nanoparticle platform for BBB pathophysiology–independent delivery of siRNA in TBI. We achieved this by combined modulation of surface chemistry and coating density on nanoparticles, which maximized their active transport across BBB. Engineered nanoparticles injected within or outside the window of breached BBB in TBI mice showed threefold higher brain accumulation compared to nonengineered PEGylated nanoparticles and 50% gene silencing. Together, our data suggest that this nanoparticle platform is a promising next-generation drug delivery approach for the treatment of TBI.

Technology & Innovation

Dry powder pulmonary vaccine delivery: Inducing mucosal immunity at the site of pathogen entry

Camilla Foged, Professor Vaccine Design and Delivery Group, University of Copenhagen

10:10 AM - 10:45 AM - Solution Spotlights

Technology & Innovation

Planar Dosage Forms Advantages Over Classical Dosage Forms

Dr. Timo Taghizadeh, Head of Business Development & Marketing, tesa Labtec GmbH

tesa Labtec is working more than three decades in the field of planar dosage forms. Our technologies are transdermal and topical patches, orodispersible thin films and topical and mucoadhesive oral films. We are your full end B2B CDMO supporting to transfer your Product ideas to Market.

Planar dosage forms show many advantages over traditional administration forms.

Biologics

Trehalose and Sucrose: Essential Components of Platform Biopharma Formulations and Covid 19 Applications

Christian Lotz, General Manager EMEA, Pfanstiehl GmbH

  • Commercial Biotherapeutics Stabilized with Trehalose and Sucrose
  • Key Issues in Biopharma Formulation Development
  • Essential components of a “Platform Biopharma Formulation”
  • Examples for utilizations and functionalities of Sucrose and Trehalose in Covid 19 related formulations/vaccines and techniques (m-RNA, Viral vectors, mAbs etc.)
  • Understanding important physicochemical properties of Trehalose and Sucrose
    • Instability of sucrose at low pH – free glucose, protein glycation
    • Phase transition and crystallization of trehalose
    • Importance of Control of Glucose levels in Sucrose as well as Trehalose

  • Purity, Quality, Consistency in Pfanstiehl’s Trehalose and Sucrose
    • β-glucans in sucrose – interference with endotoxins
    • Trace metal specifications for Sucrose and Trehalose
    • Upcoming compendial changes
  • Advantages of Trehalose over Sucrose
  • Pfanstiehl’s Biopharma Stabilization Portfolio incld. newly launched L-Arginine  and L-Histidine base and HCl
  • Conclusions

Technology & Innovation

Spray Drying Formulations with HPMCAS (Hypromellose Acetate Succinate)

Shogo Warashina, Technical Sales Manager, SE Tylose GmbH & Co.KG

HPMCAS (Shin-Etsu AQOAT®) is the most popular excipient for amorphous solid dispersion formulation due to its unique properties of improving solubility, maintaining supersaturation, and good stability of the drug product.

In this presentation, we investigate the Spray Drying Formulations with HPMCAS both in spray drying process and downstream process.

  • Spray Drying Formulations with HPMCAS (Hypromellose Acetate Succinate) as Carrier Polymer: Impact of process parameters to powder properties
  • Downstream processing of HPMCAS spray dried dispersion. Head to head comparison of L-HPC (low-substituted hydroxypropyl cellulose) as binder and disintegrant and MCC/croscarmellose blends

10:45 AM - 11:35 AM

iSolve & Networking Break

10:50 AM - 11:00 AM

Poster presentation - Development and Verification of Analytical Techniques for a Blinded Comparator Drug Productv

Juliet Symonds, Investigator, GSK

Poster Presentation - Adeno-Associated Virus Mediated Gene Delivery: Novel Analytical Tools

Dr. Johanna Simon, Scientific Expert Analytical and Preparative Liquid Chromatography, Merck

Poster Presentation - Study of the long‐term stability of the compactibility of spray‐dried mannitol powders containing α and β crystalline polymorphs

Philippe Leferve, Head of Global CTS Labs, Roquette

Poster Presentation -

11:35 AM - 12:10 PM - Case Studies

Small Molecules

In-Silico Excipient Screening for Pharmaceutical Formulations

Gabriele Sadowski, Professor for Thermodynamics, TU Dortmund

  • Reliable excipient selection based on thermodynamic modeling
  • Predicting long-term stability and influence of humidity  
  • Amorphous solid dispersions
  • Lipid-based formulations
  • Self-amorphous systems

Technology & Innovation

Formulation Development Strategies for Monoclonal and Bispecific Antibodies

Sachin Dubey, Senior Associate Director, Drug product and Analytical Development, Ichnos Sciences SA

Last couple of decades have witnessed significant growth of biopharmaceuticals; they are now playing pivotal role in the management of several diseases. Currently, they constitute around 30% of all the novel drugs approved each year; majority of biopharmaceuticals are proteins and monoclonal antibodies. Formulation development is an integral part of product development and is often used as a differentiating attribute. Multiple drug product presentations are employed during clinical development and commercial launch. Strategy of their development and timing of their launch are critical and defines product development efficiency. Increased importance of bispecifics/trispecifics have further made the formulation and drug product development   very important from strategic point of view.

12:15 PM - 12:50 PM - Solution Spotlights

Small Molecules

Improving Delivery of Poorly Soluble Compounds with Science-led Integrated Programs

Sarah Stevens, Vice President, Drug Development Sciences , Quotient Sciences

Improving solubility remains one of the greatest barriers to successful Drug Delivery. Pharmaceutical formulation scientists have an increasing range of technologies and novel platform approaches available to support successful solubility enhancement. Such technologies cannot be successful in isolation. Moving a molecule quickly towards application as a medicine requires a strategy underpinned by a fundamental understanding of the compound itself, and critically a formulation strategy that does not rely solely on potentially misleading in-vitro data. The ability to screen a range of technologies and dosage forms using physicochemical screening, biopharmaceutics to understand developability and ‘real time’ human PK data positions the drug development pathway for success. This presentation explores solubility challenges and discusses the benefits of integrating programs in order to deliver success.

Biologics

Hydroxypropyl-β-cyclodextrin as a Versatile Excipient in Biologics Processes – Application in Ultrafiltration/Diafiltration

Shiqi Hong, Senior biopharma scientist , Roquette

  • Biologics undergo various stresses during manufacturing, transport and storage.
  • HPβCD has been successfully used to mitigate aggregation in biologic formulations.
  • HPβCD is surface active and can potentially protect protein from interfacial stress during ultrafiltration/diafiltration (UF/DF) process.
  • Case study: Application of HPβCD in protein formulation during UF/DF process 
    • Concentration of HPβCD was maintained throughout UF/DF process
    • HPβCD showed improved protein recovery and reduction in particle formation
  • Potential application of HPβCD in protein UF/DF process and formulation

Technology & Innovation

Formulation Development of Solid Oral Dosage Forms with ZoomLabTM 2.2

Martin Hofsäss, PostDoc Development Pharma Solutions, BASF SE

Learn about the latest features of BASF’s virtual formulation assistant ZoomLabTM that provide the user with immediate advice for the development process of solid oral dosage forms.

Device Development

Introducing OLAR® - A novel Oral Long Acting Release Platform

Elijahu Berkovich, Head of Emerging Science and Innovation, Clexio Biosciences

  • Continuous drug delivery to the upper GI tract
  • Orally administered, non-invasive
  • To be used for drugs in which traditional ER formulation strategies cannot support the required pharmacokinetic profile
  • Versatile platform, suitable for multiple APIs and enabling high drug loading

12:50 PM - 1:50 PM

Networking Lunch

1:50 PM - 2:25 PM - Case Studies

Small Molecules

Dry Granulation Process Development- Flexible Manufacturing

Ranjit Dhenge, Investigator R&D, GlaxoSmithKline

This presentation will cover application of Flexible Manufacturing (FM) through system modelling in dry granulation (roller compaction) process development.

Key points in presentation will be:

  • Role of Modelling in FM
  • Rational for system modelling application in OSD product and process development: Enabling FM in QbD Framework
  • Challenges in delivering FM
  • Case study examples of FM:
    • Case study 1: Clearing perception: “Modelling in FM is Expensive”
    • Case study 2: Application of System modelling in dry granulation for an immediate release oral solid tablet development
    • Case study 3- Applications of modeling approach for targeted experiments as part of QbD

Biologics

Mesoporous Silica Particles; Simple yet Powerful Tool for the Delivery of Biomolecules

Vivek Trivedi, Lecturer in Drug Delivery, Medway School of Pharmacy , University of Kent

Mesoporous silica is a versatile carrier for biomolecules due to its stability, low toxicity, and ability to be functionalized with a variety of molecules and polymers. The porous structure with hundreds of void channels can absorb/ encapsulate reasonably large amounts of biomolecules and the distinctive properties, such as high surface area, large pore volume, and tuneable pore size with a narrow distribution make them readily suitable for various controlled release applications. The adsorption of protein on solid surfaces is a common yet complicated phenomenon due to the complex behaviour of the macromolecules during this process. Hence, it is important to understand; why and how these biomolecules adsorb, the behaviour of adsorbed proteins either as individual molecules or in an ensemble, the influence of adsorption on the protein’s biological function and, if there is a general mechanistic rule for the adsorption process.

In general, the immobilisation of biomolecules onto solid surfaces is often considered irreversible, which can be addressed by the use of surface-active substances or large polymers as displacers as long as they do not affect the protein conformation during or after desorption. Displacer molecules such as surfactants encourage desorption of the macromolecules by an exchange mechanism where the attached protein is competitively substituted from the adsorbent surface in the favour of immobilisation of the smaller molecules.

This presentation discusses the possible use of mesoporous silica as a drug delivery carrier for macromolecules without compromising their biological activity by establishing the required ‘ideal parameters’ for surface immobilisation. Along with this, the optimal parameters to allow maximum desorption, especially the role of displacer will also be discussed.

Technology & Innovation

In-Vitro and In-Vivo Data Confirmation of Thermal Stability and Immune Responses Using a Wide Range of Injectable Unit Solid Dose Vaccine Constructs

David Hipkiss, Chief Executive Officer, Enesi Pharma

2:25 PM - 3:15 PM

iSolve & Networking Break

3:15 PM - 3:25 PM

Poster Presentation - From Powder to Tablet – Material Characterization of Two Mannitol Polymorphs

Lena Mareczek, PhD candidate , Merck

Poster Presentation - An Optimal Design of Experiment Approach to Produce Spray Dried Pseudomonas aeruginosa Podoviridae Bacteriophages

Emilie Tabare, PhD student, ULB

Poster Presentation - Transforming Rectal Therapy for Ulcerative Colitis: Patient Perspectives and Pilot Results of Novel Thermosensitive topical gel formulation

Ravi Pamnani, Founder/CEO, Intact Therapeutics, Inc

Poster Presentation - Analytical Tools for poly(lactide-co-glocolide) (PLGA) Implants and Microparticles: 1) Accelerated Release Testing and 2) Simultaneous Quantification of Residual Solvents

Dr Martin Körber, Director Research & Development, Pensatech Pharma GmbH

3:30 PM - 4:05 PM - Solution Spotlights

Small Molecules

Formulating Amorphous Solid Dispersions: Key Challenges and Considerations for a Streamlined Approach

Aaron Stewart, Associate Principal Scientist, Lonza

A large and increasing fraction of orally administered small molecules in pharma company pipelines have poor oral absorption due to low aqueous solubility or dissolution rate. To overcome these limitations, formulation approaches for increasing oral bioavailability are needed. Amorphous solid dispersions (ASDs) are a prevalent technology for addressing oral bioavailability challenges, given the applicability and scalability of the technology across a diverse compound physicochemical space. Developing ASDs requires an integrated approach, where bioperformance, physical and chemical stability as well as manufacturability are all considered. Combining knowledge of key drug, polymer and gastrointestinal properties together with an in vitro and in silico toolkit is critical for achieving optimal in vivo performance while reducing development timelines and drug substance requirements. This presentation covers some general strategies for developing a successful amorphous solid dispersion formulation, from formulation intermediates to final dosage forms.

Biologics

Silicone-Free Prefilled Syringe Packages for Sensitive Biologics and Ophthalmic injections

Rob Gelissen, Business Development for Drug Delivery and Packaging, W. L. Gore & Associates – PharmBIO Division

  • Injection volumes of 2 mL are becoming more common, but, USP standards limit particle counts to a quantity per injection
  • Impact of Higher concentrations biologics and concern for aggregation in presence of silicone
  • Floaters, inflammation, interocular pressure increases and silicone presence in injections
  • Impact of Aging and changing injection profiles, resulting in slower or stalled autoinjector injections

Small Molecules

Extrusion: An Enabling Technology to Create Long Acting Implants

Tony Listro, Vice President, Technology, Foster Delivery Science

  • How extrusion is uniquely positioned to create shape profiles to fill the dose requirements and body placement. 
  • Selection of polymers necessary for release and degradation parameters
  • Co-extrusions provide opportunity to create multiple API release profiles, change release profiles and can offer ways to improve safety.

Device Development

On-Body Infuser for Large Molecules up to 20 mL

Dr. Ines Rüstenberg, Director Development, Sensile Medical, a Gerresheimer company

Sensile Medical, a Gerresheimer company, is a leader in advanced micropump technology that develops a wide range of customized drug delivery platforms and dosage solutions. Now, Sensile also offers solutions for large molecules. The SenseAir technology is the core of this newly developed product group. The presentation will demonstrate the physical principle of the SenseAir technology and the technical implementation into a 10 mL on-body infusion device. It will show how the smart SenseAir technology enables a highly competitive device design in terms of robustness, reliability, cost, compactness and scalability. The concept provides an easy-to-assemble cartridge mechanism. The cartridge can be assembled into the device in the production facility where the cartridge has been filled by just one more handling step. This feature may be crucial for successful handling of sensitive drugs together with the device.

4:10 PM - 4:45 PM - Case Studies

Small Molecules

Evolving Trends in Technology and Regulatory Landscapes for Developing and Bringing Generics and Innovative Medicines to Market

Rosario LoBrutto, Head of Scientific Affairs, Sandoz

  • Developing complex generics via 505j regulatory pathway and
  • Innovative medicines following a 505b2 regulatory pathway
  • As well as an increased focus on injectables and combination products in the pharma space.

Biologics

Peptide Drug Delivery, Characterization and Product Development

Dr Ana L Gomes dos Santos, Principal Scientist, AstraZeneca

Technology & Innovation

Nanoparticles and Medicines Design

Ijeoma F. Uchegbu, Chair in Pharmaceutical Nanoscience, University College London

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Device Development

Common Pitfalls in Developing Combination Products and Medical Devices

Scott Brown, Executive Director Device Development, MSD

  • Fundamental steps in development
  • How devices and combination products fail and steps to avoid failure
  • Project and people related issues

This presentation will go over the common sources of failure in delivery device and combination product development and how to systematically avoid them while optimizing the likelihood of a successful product approval and launch.  Emphasis is placed upon design integrity, simplicity and effectively meeting the needs of patients and healthcare professionals. The impact of parts count on reliability and the importance of good human factors engineering are also covered. A useful checklist is provided to provide guidance on what should be established before investing in capital equipment for manufacturing and assembly.

4:50 PM - 5:35 PM - Keynote

Small Molecules

OcuRing-K™: The Dropless Cataract Surgery Solution

Kenneth J. Mandell, Founder and CEO, LayerBio

  • Eye drops have significant limitations with respect to compliance, safety/tolerability, ocular bioavailability, and economic costs to the healthcare system 
  • There is growing interest in “dropless” technologies that eliminate the burden of topical eye drops after cataract surgery 
  • OcuRing-K™ (ketorolac implant) is the is the only steroid-sparing dropless therapy that eliminates the need for topical anti-inflammatory eye drops in cataract surgery   

5:35 PM - 6:05 PM

6:05 PM - 6:10 PM

DDF Poster Competition Award & Chair’s Closing Remarks

8:30 AM - 9:00 AM

Registration & Refreshments

9:00 AM - 9:05 AM

Chair’s Opening Remarks

9:05 AM - 9:40 AM - Keynote

Device Development

Creating Digital Ecosystems that Improve Outcomes

Matthew Clemente, Corporate Vice President Delivery Systems Engineering, Novo Nordisk

Significant investments have been made across industry in the areas of connected devices and digital health, yet few have resulted in real world improvements to patient outcomes. While many of the value propositions are substantial and self evident, navigating the technical, clinical, regulatory and commercial landscapes have created both phenomenal success stories and dismal failures.

We will review some of these case studies and discuss strategies in navigating the hype versus hope of these promising systems. The development of highly reliable, usable, relevant and broadly adopted systems that truly improve outcomes is found at the intersection of rigorous engineering competencies and robust commercial strategies.

9:45 AM - 10:20 AM - Case Studies

Small Molecules

Formulation Development Strategies for Monoclonal and Bispecific Antibodies

Sachin Dubey, Senior Associate Director, Drug product and Analytical Development, Ichnos Sciences SA

Last couple of decades have witnessed significant growth of biopharmaceuticals; they are now playing pivotal role in the management of several diseases. Currently, they constitute around 30% of all the novel drugs approved each year; majority of biopharmaceuticals are proteins and monoclonal antibodies. Formulation development is an integral part of product development and is often used as a differentiating attribute. Multiple drug product presentations are employed during clinical development and commercial launch. Strategy of their development and timing of their launch are critical and defines product development efficiency. Increased importance of bispecifics/trispecifics have further made the formulation and drug product development   very important from strategic point of view.

Biologics

A Self-Assembly Multifunctional Platform for Therapeutic Delivery of Nucleic Acid Medicines to the Brain

Moein Moghimi, Professor of Nanomedicine, Newcastle University

A peptidic phage mimetic self-assembly (termed NanoLigand Carriers, NLCs) will be presented capable of rapidly crossing the intact blood-brain barrier (BBB) on intravenous injection in substantial quantities, and without disrupting the integrity of the BBB (Nature Communications 2019, 10:4635). The system accommodates a wide range of guest molecules, and targets two cellular receptors. On reaching the brain-parenchyma, NLCs carrying therapeutic nucleic acids target microglial cells and neurons showing unprecedented pharmacological effects and without inducing inflammation and metabolic perturbations.

Technology & Innovation

Material Properties of Amorphous Solid Dispersions – Influence of Incorporated Drugs

Karsten Fluegel, Postdoctoral Researcher | Drug Delivery & Innovation, Merck

  • Formulation development of amorphous solid dispersions (ASD) still is challenging although the technology is established and several poorly water-soluble drugs have been marketed using this technique
  • Selection of excipients for ASDs needed for tablet compression is often performed on a case-by-case basis according to the formulator’s experience and eventually trial and error
  • As the polymer is the main component in the ASD, it predominantly determines material properties and thus systematically investigating the impact of the incorporated drug on these properties (e.g. mechanical properties and wetting behavior) helps rationalizing and facilitating excipient selection

10:25 AM - 11:00 AM - Solution Spotlights

Small Molecules

Intelligent Formulation Design and Evaluation for SWIFT Product Development

Dr Fiona MacDougall, Director of Biopharmaceutics, BDD

While in vitro testing of formulations provides a good initial indication of how a product should perform, the complexities and variability in the GI tract are difficult to model and so clinical success is not always guaranteed. Intelligent formulation design, integrating biopharmaceutics and adaptive clinical testing can accelerate the successful development new products.

Technology & Innovation

Performance Enhancement Strategies for Low Solubility APIs – do more with less

Mafalda Paiva, Group Leader, R&D Analytical Development, Hovione

João Henriques, Group Leader, R&D Drug Product Development, Hovione

This presentation will address the main challenges in the formulation of ASDs, and highlighting the importance of integrated development and the synergies between pre-formulation and commercial manufacturing expertise. The science-based development platform for ASDs will be presented. This platform relies on the integration between formulation and analytical development, from API to drug product, coupled with the use of computational models, formulation databases and miniaturized high-throughput in vitro techniques.

Here we will also present how the use of state-of-the-art, automated, spray drying equipment, coupled with in-silico formulation tools enable a high-throughput screening (HTS) approach for the development of ASDs.

Technology & Innovation

3D Powder Bed Printing with Lactose: a showcase

Korinde van den Heuvel, Senior Product Developer , DFE pharma

3D powder bed printing offers huge potential benefits in pharmaceutical manufacturing, but limited information is available on excipient selection in relationship to functionality in the dosage form in literature.

In this presentation we investigate the benefits of powder based 3D printing by utilizing a base formulation of lactose monohydrate and pregelatinized starch. Both powder characterization as well as the impact of print settings and formulations on tablet properties will be discussed.

11:00 AM - 11:50 AM

Networking Break

11:50 AM - 12:25 PM - Case Studies

Small Molecules

Continuous Manufacturing of Pharmaceutical Nanoparticles (CM-NANO)

Luis Padrela, Lecturer SSPC, Bernal Institute, University of Limerick

Poor solubility of new chemical entities (NCEs) is a major bottleneck in the pharmaceutical industry, which typically leads to poor drug bioavailability and efficacy. Nanotechnologies offer an interesting route to improve the apparent solubility and dissolution rate of pharmaceutical drugs. The work presented herein demonstrates a novel production and isolation technology for drug nanoparticles using batch and continuous CO2-assisted spraying processes. Nanoparticles of three BCS class II Active Pharmaceutical Ingredients (APIs), namely carbamazepine (CBZ), ketoprofen (KET) and risperidone (RIS), were produced and successfully coated onto micron-sized microcrystalline cellulose (MCC) particles. The API nanoformulations exhibit enhanced dissolution and improved rheological properties. This nanotechnology is able to control the solid state form of the APIs through a controlled balance between antisolvent supersaturation in the nozzle versus spray drying atomization, and combining that with an isolation/coating step. [1]

 [1] V. Verma; K. M. Ryan; L. Padrela, Int. J. Pharm., 592, 2020, 120032.

Biologics

Oral Delivery of Peptides Using a Core-Shell Silica Construct

David Brayden, Full Professor (Advanced Drug Delivery), University College Dublin

We have designed a nanoparticle construct for oral delivery of peptides and other macromolecules based on a core-shell principle.  A peptide candidate is combined with the excipients, L-arginine and zinc chloride, to produce a nm core, over which silica is coated.  The basis of the technology is that the excipients provide stability to the peptide and can also act as intestinal permeation enhancers when co-released in the small intestine close to the epithelium. It is therefore designed to promote epithelial uptake of the released peptide and not the nanoparticle.  Silica is used because it is biocompatible and dissolves at the small intestinal pH but not gastric pH.  Proof-of-concept studies in rodents using insulin and exenatide show that the process is not damaging to these peptides and, in the case of insulin, significant plasma glucose lowering was achieved in rats following duodenal instillations. The synthetic process is a simple four step one, which can be scaled. Next steps will examine the concept when the peptide-entrapped nanoparticles are gavaged to rats and then as a solid dose formulation in a large animal model.

Technology & Innovation

Peptide Nanomaterials: The Future of Biomaterials and Drug Delivery

Dr Garry Laverty, Senior Lecturer in Pharmaceutical Sciences, Queen’s University Belfast

Peptides represent a promising class of molecules for healthcare applications. Their chemical versatility (i.e. wide availability of functional groups) mean they can be designed, using a bottom up approach, to provide very specific functional and biological properties. Our research has shown very short peptide sequences (<10 amino acids) can be modified/synthesised to form fibrous (hydrogel) and tubular (nanotubes) nanostructures in response to a variety of physiological stimuli (pH change, enzymes). These peptide materials have the potential to be harnessed for future use as: antimicrobial/anti-biofilm agents (hydrogels, nanotubes); medical device coatings (hydrogels); wound healing (hydrogels); drug delivery platforms to cross biological barriers (nanotubes) and injectable in situ forming implants for sustained drug delivery (hydrogels).

Technology & Innovation

Bespoke Dosing for Pediatric Patients

Rick Panicucci, Senior Vice President CMC, QED Therapeutics

  • Current clinical manufacturing utilize a “one size fits all” approach
  • For very young patients this is far from ideal
  • Personalized dosing based on weight of subject
  • One patient per batch 

12:30 PM - 1:05 PM - Solution Spotlights

Small Molecules

Development and Continuous Manufacturing of SMART Nano-Particles, Meeting Bottleneck Challenges

Andrea Cusack, Chief Executive Officer, LEON-NANODRUGS GMBH

  • Increasing process efficiency, possibility of fewer steps and providing for smaller modular plant footprints delivers increased facility options for industry
  • Leon technology for continuous manufacturing via parametric process control, continued process verification and management systems.
  • Scalability in reach allowing for fast and easy integration into existing production facilities

Technology & Innovation

BEPO® - a Clinically Advanced Long Acting Injectable Technology that Enables Controlled, Systemic or Local Delivery to Address a Broad Range of Therapeutic Needs

Christophe Roberge, Senior Technology Advisor, MedinCell

We will introduce BEPO®, a clinically advanced long-acting injectable proprietary technology with outstanding versatility. BEPO® is an in situ forming depot technology whereby a solvent-exchange process elicits the precipitation of the polymer upon injection, leading to the formation of a fully biodegradable drug releasing matrix in situ. Several BEPO® products have now reached the clinical stage targeting different indications and routes of administration. We have communicated in January 2021 the positive results of our PIII trial for mdc-IRM, our most advanced product in development.
 
Following the introduction of the scientific elements highlighting the tunability and maturity of our drug delivery platform, we will also present case studies to exemplify why and how BEPO® brings the flexibility to formulate a wide variety of APIs, from small molecule drugs to more complex biomacromolecules, and addresses various unmet medical needs for both emerging and developed countries alike.

Technology & Innovation

Maximizing Formulator’s Tools to Enhance Bioavailability of Poorly Soluble Oral Drugs

Stephen Tindal, Director of Science & Technology, Catalent

Taking advantage of coupling different technologies can reduce drug development timelines and drive greater long-term success in preclinical and clinical studies for poorly soluble oral drugs. In this talk, learn how to classify new therapeutic drug candidates and identify issues relating to oral product development, anticipate and address challenges early using PBPK modelling, preformulation and formulation tools, as well as how to plan for success in preclinical and early-stage clinical studies.   
  • An overview of the types of tools available and how coupling different technology solutions could improve issues with API form and oral bioavailability of poorly soluble compounds.
  • Utilising technology as an enabler to deliver enhanced bioavailable drug to rescue failing projects
  • Insights to physiologically based pharmacokineitc modeling (PBPK) by discussing the way a company ought to approach drug absorption challenges.
  • Understanding how to seamlessly integrate candidate selection, optimal form assessment, bioavailability enhancement, and cGMP clinical materials delivery for less re-work and better solutions.

1:05 PM - 2:05 PM

Networking Lunch

2:05 PM - 2:40 PM - Case Studies

Small Molecules

Amorphous Solid Dispersion: Strategies in Early Stage Development and Technology Choice

Philippe Lienard, Pre-Development Science Leader, Sanofi

  • Many ASD technologies are available at the industrial scale
  • Selection of the right technology is strategic for the future development pre-clinical and clinical such as
    • Quantity of API available for early development studies
    • Stage of development (feasibility studies, formulation/process development)
    • Formulation selection
    • CMC prerequisite (physical and chemical stability, solubility and dissolution improvement)
    • Biological performance enhancements.
This talk will describe how to make early pre-formulation appropriated strategic choices for these complex ASD formulations with tangible case studies.

Biologics

Understanding and Overcoming of LER (low endotoxin recovery) in Pharmaceutical Formulations.

Klaus Brandenburg, CSO of Brandenburg Antiinfektiva, , Forschungszentrum Borstel

  • Understanding and overcoming of LER (low endotoxin recovery) in pharmaceutical formulations‘ by Klaus Brandenburg
  • LPS (endotoxin) contamination of pharmaceutical drug products according to FDA and  others must be excluded due to the strong ability of LPS to induce inflammation in humans
  • LPS spiking of the formulations frequently shows a loss of measurable LPS (´masking`)  in particular by the Limulus assay.
  • The reason for this observation can be assigned to a conformational change of the LPS aggregates
  • Possibilities are presented with respect to a prevention of the LER by changing the composition of the formulations.

Technology & Innovation

Nano-structured Films for Ocular Drug Delivery

Murty Vyakarnam, Co-founder & Chief Executive Officer , Oculinea Inc

Oculinea’s core technology developed at UCSF is a nano-engineered polymer thin film encapsulating drug payload in various configurations for ocular drug delivery of both small molecules and biologic therapeutics. The bio-resorbable drug delivery implant allows transport of drug molecules from a reservoir payload through a micro- and nano-structured porous polymer layer providing zero-order drug delivery for a few months to a year. This sustained drug delivery technology provides an ideal solution to address compliance issues with patient administered daily eye drops in treating chronic ocular conditions like glaucoma. The presentation will provide results from the progress made towards clinical translation of this novel technology.

Biologics

Delivery Technologies for mRNA Nanotherapeutics

Heinrich Haas, Vice President, Formulation, BioNTech

  • mRNA technology offers various new opportunities for therapeutic intervention  in fields including cancer infectious disease or protein substitution therapy
  • Delivery plays a paramount role for successful development of clinical product on this basis
  • Tailored delivery systems  are required for  different RNA formats and application routes
  • Thorough understanding of molecular coherencies inside these systems is helpful for rational development of clinical candidates
  • Meanwhile several mRNA based products have been successfully developed up to clinical stage

2:45 PM - 3:20 PM - Case Studies

Small Molecules

Recent Advances in Modified and Controlled Release Formulations

Arul Joseph, Senior Director, Pharmaceutical Development and Clinical Supply Chain, Avanir Pharmaceuticals

Discussion of new formulation technologies and trends in modified and controlled release formulations

Biologics

Development, Manufacturing and Supply of Ebola Vaccine

Lynne A. Isopi, Principal Scientist, Vaccine Drug Product Development, MSD

A historic Ebola outbreak occurred in March 2014 in three Western African countries, Guinea, Liberia, and Sierra Leone.  Over 28,000 cases were reported and led to more than 11,000 deaths, more than ten times the amount of cases compared to all past outbreaks combined.  On August 8, 2014, the World Health Organization declared a Public Health Emergency of International Concern (PHEIC).  MSD, partnering with NewLink Genetics, entered into an exclusive worldwide licensing agreement to research, develop, manufacture, and distribute an investigational Ebola vaccine candidate based on recombinant Vesicular Stomatitis Virus (rVSV) technology. Working with multiple partners, MSD has brought forward an efficacious vaccine candidate from Phase I trials in October 2014 to Phase III consistency studies by August 2015.  This presentation will provide background into MSD’s strategy to bring the vaccine to licensure, product development activities to scale-up the process from clinical to commercial, and the challenges faced during product development.

Technology & Innovation

The Confluence of Innovation in Therapeutics, Drug Delivery and Regulation

Twinkle Christian, Process Development Scientist, Drug Product Technologies Group, Amgen

The field of human therapeutics has evolved from small molecules to complex biologics with tremendous acceleration in innovation of new modalities and drug delivery technologies. These innovations have led to corresponding therapies seeking regulatory approval globally. The accelerated growth in innovative therapies represent challenges for sponsor and regulatory agencies. This talk will briefly cover the innovative modalities and drug delivery, current CMC guidance for mAb like therapies and propose recommendations from an industry perspective to make sure innovation and regulation are not out of sync. It is critical for industry and health authorities to work together towards a confluence of innovation and regulation to accelerate access of life-saving therapeutics to patients globally.

Device Development

Considerations with the Development of Inhaled Combination Products

David Cipolla, Vice President of Research, Insmed Inc.

The development of any pharmaceutical product is a challenge, but the development and regulatory hurdles are even greater for an inhaled product.

Furthermore, most inhaled products are also combination products and that adds another layer of complexity, both from a technical, and a regulatory perspective, and thus there is increased risk of failure. This presentation will address the following:

  • What are the regulatory considerations for inhaled combination products?
  • What analytical tests are required for QC release and stability testing of inhaled combination products?
  • When can characterization studies be conducted during development to replace routine QC testing?

4:10 PM - 4:15 PM

Chair’s Closing Remarks