Programme 2023

31st May – 2nd June 2023, Berlin, All Timings in CEST

7:45 AM - 8:35 AM

Registration & Refreshments

8:35 AM - 8:40 AM

8:40 AM - 9:20 AM - Panel Discussion

The Golden Age of Drug Delivery

John D. Higgins, Distinguished Scientist, MSD

Hanne Mørck Nielsen, Professor, PhD (Pharm), Center Director and Group Leader, University of Copenhagen

Philippe Lienard, Preclinical leader, Institut Pasteur

Mostafa Nakach, Global Head of Process Engineering Biologics Development/ Biologics Drug Product Development & Manufacturing, Sanofi

How are the last few years of pharmaceutical industry trends affecting the drug delivery landscape – and what opportunities should we grasp?

  • Advancements in materials science using enhanced excipients, polymers and predictive insilico science
  • Focusing on targeted delivery – less about controlling time/duration, more about site-specific release, bypassing first-pass effects, or site-specific accumulation
  • Orally delivered peptides have now made the market.  But % absorption is stuck at 2% or less.  Will we ever beat this and what will it take?
  • Interest in antibody drug conjugates is increasing.  Does the panel see any unique challenges in delivering this modality and if so how can they be mitigated?
  • Creating improved delivery options for both small and large molecules harmonizing advanced technology development and regulatory acceptance

9:25 AM - 9:55 AM - Case Studies

Small Molecules

Polymeric Prodrugs Platform For Long-Acting Injectable Depots and Parenteral Therapeutics

Professor Patrick Stayton, Distinguished Career Professor, Director, Molecular Engineering & Sciences Institute, University of Washington

  • A new class of long-acting PrEP or therapeutic drug depot platforms has been developed, achieving high drug wt % in low injection volumes, this injectable depot system is achieving zero-order release profiles and may provide key advantages in length of drug delivery, cost of goods, and stability in regions without cold-chain.
  • The therapeutic designs with this polymeric prodrug platform can target antibiotic and antiviral drugs to cellular compartments such as the lung macrophage to extend PK profiles and potentiate drug activity against bacterial and viral pulmonary pathogens after inhalation administration
  • Liver-targeting of enzyme-cleavable polymeric prodrugs has also been shown to achieve significant increases in therapeutic index via subcutaneous route of administration
  • The platform has been used to demonstrate parenteral immune therapeutics such as small molecule immune activators and adjuvants

A polymeric prodrug platform has been developed for long-acting PrEP or therapeutic drug delivery.  This platform achieves high drug wt % in low injection volumes, and has been applied to formulate drugs of widely varying lipophilicities.  The injectable depot system is achieving zero-order release profiles with HIV, contraceptive, and anti-bacterial drugs over controlled durations ranging from 3 months to 9 months at human daily dose equivalents in rat models.  This depot platform may provide key advantages in length of injectable drug delivery, cost of goods, and stability in regions without cold-chain. The “drugamers” have been developed in other parenteral applications as soluble therapeutics that again incorporate elements of poorly soluble drug formulation, as well as adding additional targeting and controlled release mechanisms.  For example, the drugamers have been designed to target the macrophage reservoir in organs such as the lung and in tumors, and focus and extend pharmacokinetic drug profiles. These polymeric prodrugs have shown excellent activity against highly lethal bacterial disease models via inhalation delivery, demonstrating key PK/PD advantages over the parent drugs. The platform has been further broadened to include other pulmonary infectious disease therapy including antiviral therapeutics against Covid. The macrophage- and APC-targeting properties also have opened new applications for immune-therapy of cancer and vaccines, in concert with cell and biologic drug combination therapy. In addition, this platform has been extended to target drugs to the liver, using GalNAc targeting of liver hepatocytes to improve PK/PD and therapeutic index.   Recent advances across these applications will be covered.


Epithelial Permeation Enhancement by Cationic Peptide Enhancers Investigated for Oral Peptide Delivery

Hanne Mørck Nielsen, Professor, PhD (Pharm), Center Director and Group Leader, University of Copenhagen

  • In addition to lipid-based permeation enhancers for oral peptide delivery, peptide excipients like penetramax display suitable permeation enhancing effects for different therapeutic peptide cargoes.
  • The talk will include data on membrane interactions of a peptide enhancer, effects on the cell morphology and tight junction proteins compared head-to-head with another enhancer.
  • The effect likely relates to reversible and stereoisomer-independent membrane interactions, potential cell uptake leading to reversible effects on the paracellular space.
  • The mode of action relates to the expression and dynamics of specific tight junction proteins in response to membrane effects and potentially intracellular signalling.
  • Overall, the present study shows that the biodegradable penetramax displayed a potent and tight junction protein specific effect that is reversible leading to permeant size-selectivity.

Biotherapeutics such as glucagon-like peptide-1 and insulin are increasingly explored for oral administration employing functional co-excipients, yet until now only with few successful examples of translation to clinical use. Permeation enhancing molecules are reported, but the success of one over the other relies on a variety of parameters, including their distinct effects on the epithelium in time and space. Here, I present robust fundamental insight into the biological effect of a relevant permeation enhancer. It is demonstrated that while end-point measures on the epithelium from a first look may show similar effects to that of an ion-chelator, real-time and recovery effects may be vastly different. Mechanistic insight is critical for selecting and designing future safe excipients with a biological functionality, and that the employed methods together advance such insight.

Technology & Innovation

Oral Macromolecule Delivery by High-Throughput GI Absorption Screening

Thomas von Erlach, Chief Scientific Officer , Vivtex

Despite decades of research, most peptide therapeutics and other macromolecule drugs require needle administration due to insufficient oral bioavailability. In this presentation, I will go over how we achieved double digit absolute oral bioavailability in dogs with a solid dosage formulation for the peptide antibiotic vancomycin.

Device Development

Device Regulatory Strategies for Drug Device Combination Products

Louise Place, Director, CMC RA Devices, GSK

  • The latest requirements for Drug Device Combination Products in EU and UK
  • Practical reflections on implementation of the EU MDR implementation
  • Important considerations for making changes to established products
  • Future requirements and next steps in regulatory guidance
Increasingly the regulatory landscape is becoming more complicated, and timelines are continuing to be constrained. Two years into the application of the new Medical Device Regulation, this presentation aims to explore some practical reflections on the implementation to date and highlight some important consequences for future changes. In addition, consideration of the latest announcements for both Medical Devices and Drug Device Combination products in the UK post-Brexit will highlight some of the challenges for harmonisation in an increasingly global market.

10:00 AM - 10:30 AM - Solution Spotlights

Small Molecules

Fully Automated and DoE-Based Development of a Oral Solid Dosage Form

Dr. Thomas Brinz, Director Engineering Pharma Services, Syntegon Technology GmbH

Goal of the presented study and data is to demonstrate the potential of a fully automated and DoE-based development of new oral solid dosage form (tablet). The combination of DoE and automated execution of each process step offer a new potential to speed up and integrate more quality-in-design in the development process.
All development steps were planned by Design of Experiment. For the granulation a automated fluid granulation module was used. All different powder granules were compressed on rotary tablet press with an integrated and automated change of 2- and 3-paddle-feeder and other compression parameters. All tablets were analyzed on an automated WHT with integrated micro-wave based weight measurement and NIR-based content uniformity measurement.

With a semi-continuous fluid bed granulation module 26 different lots of fluid-bed granules with different API-content, humidity and process parameter were prepared. The 26 lots were than automatically compressed on rotary press with different process parameter and an automated 2 and 3-paddle-feeder and wing change to get more than 200 different tablet lots (each > 500 tablets). All of the tablets were than characterized on a new high-throughput-inspection machine with weight, hardness, dimensions and content uniformity. Based on the large number of samples with different process parameter the influences and interaction of materials, recipe and formulation with the process parameter of granulation and compression were analyzed.

Based on the automation of all development steps (granulation, compression and quality analysis) a high throughput and short development time was achieved. Based on 26 formulations, more than 200 tablets lots were prepared, each of them more than 500 tablets. All tablets were tested on a new tablet analysis tool, measuring weight, dimension and API content. By the combination of DoE and automation it's possible to screen a larger parameter space, identify interaction of parameters and optimize the quality of the tablets. By the automation also the required amount of material is less than by a manual testing and change of the different modules like 2- and 3-paddle-feeder.


Application-Specific Poloxamers for Biologics

Dr. Meike Roskamp, R&D Lab Leader, BASF

Poloxamers, a family of triblock copolymers composed of polyethylene oxide (PEO) and polypropylene oxide (PPO), are used in various pharmaceutical applications. Their physicochemical properties, including surface activity and gel point, highly depend on molecular weight and PEO/PPO ratio. Most applications tolerate small variations in chemical composition and are therefore less affected by batch to batch variations of commercially available poloxamers, but some applications benefit from a more precise chemical composition to achieve reproducible performance. To address the needs of these applications, we are developing new poloxamer grades that not only address batch to batch variability but also provide an improved safety profile

Technology & Innovation

Apisolex™ and Apinovex™ Polymers: Efficient New Tools for Solubility Enhancement and Lifecycle Management

Nick DiFranco, Global Market Segment Manager for Oral Treatments, Lubrizol Life Science Health

As many as 90% of new APIs suffer from poor aqueous solubility and/or bioavailability, creating significant challenges for drug formulators. While there are excipients and techniques available to address these issues, they often have low efficiency and lead to complex manufacturing processes or undesired side effects for patients.  

Lubrizol Life Science Health’s injectable-grade Apisolex™ and oral-grade Apinovex™ polymers were designed to overcome poor solubility and unlock the future of drug delivery. Join us to learn how Apisolex and Apinovex polymers enable: 

  • New case study and in vivo safety/toxicity data for 2023 
  • Efficient parenteral and oral delivery of low solubility compounds 
  • Development of patent-protected formulations for new chemical entities (NCEs) and differentiated 505(b)(2) products 
  • Simple, scalable manufacturing processes using readily available equipment 
  • High drug loading and stability benefits versus other excipients, demonstrated by experimental data with model APIs 

Device Development

Direct-to-Organ Drug Delivery – Opportunities, Challenges, and Ideas

Harshal Shah, BioPharma Sector Head, Cambridge Consultants

The R&D pipeline across the biopharma industry is diversifying at a rapid pace with new modalities and new classes of drugs becoming a significant part of their overall research strategy. Increasingly we are observing a need for delivering drugs locally in difficult-to-access anatomy such as heart, brain, spine, and kidneys across different therapeutic areas including oncology, CNS, autoimmune and a number of rare diseases. The preference will always be to deliver these drugs with a minimally invasive approach. Our team evaluated the challenges involved in developing device technologies and new procedures for delivering such complex drugs localized to organs, tumours or tissues and developed innovative ideas and concepts that could be deployed in specific situations. This talk will focus on a few select ideas and our thought process in the design and development of such delivery platforms and procedures.

10:30 AM - 11:25 AM

1-2-1 Pre-Scheduled Meetings & Networking Break

11:25 AM - 11:55 AM - Case Studies

Small Molecules

Drug Bioavailability Challenge: How to Make New Drug Candidate Available on the Market

Philippe Lienard, Preclinical leader, Institut Pasteur

  • How to make a new drug available at the clinical/market stages considering candidate drug limitations:
  • What are options for formulation development ?
  • What is bioavailability and why it often needs to be increased to reach clinical and marketing stages for both candidates for I.V. and oral applications.


Formulation & Process Challenges in the Drug Product Development of Biopharmaceuticals

Dhananjay Jere, Director Drug Product Research & Development, Manufacturing & Supply, Fresenius Kabi

  • General CMC and regulatory aspects for biopharmaceutical development
  • Formulation development and stability considerations to different NBEs
  • Drug Product process development and manufacturing considerations
  • Considerations for product development based on the route of administrations
  • Special challenges and considerations for ATMPs
  • Special challenges and considerations for biosimilars
Rapidly diversified biopharmaceutical portfolio with innovative biological products have provided unique treatment opportunities to the patients with severe and uncurable diseases. However, these modalities also have offered significant CMC challenges, that demanded special considerations not only from the development but also from the regulatory aspects to ensure quality and consistency of products. The presentation will cover several key aspects of drug product development such as molecular formats, for example, mabs, oligos, cells, along with the routes of administration IV, SC, IVT etc. It will discuss several considerations and requirements based on the dosage forms and devices such as vial, PFS with autoinjectors, and associated manufacturing process, facility, and supply chain considerations for the holistic drug product development. Finally, presentation will cover the challenges and considerations for the development of biosimilar products to mitigate the rising cost of biopharmaceuticals with the cost-effective biosimilar alternatives.

Technology & Innovation

Bioavailability of Actives with a Case Study of CBD

Alexandru Zabara, Innovation Manager Pharma, dsm-firmenich

Cannabidiol (CBD) is fast emerging as an exciting ingredient across the pharmaceutical market, powered by increasing scientific research investigating its potential benefits in a number of disease states, including central nervous system (CNS) disorders, pain management, cancer and more. While there is a remarkable opportunity for the development of CBD-based therapies in the field, formulating a highly lipophilic (logP = 6.3) and poorly soluble crystalline API can be a challenging task. In addition to physical and chemical stability challenges, the oral bioavailability of crystalline CBD has been shown to be very low in humans (approx. 6%), due to incomplete absorption in the gut and significant pre-systemic elimination in the liver.

The objective of this work was to systematically explore a number of drug delivery technologies, previously claimed to possess the ability to enhance the bioavailability of poorly soluble drugs and assess their effectiveness in increasing the pharmacokinetic profile of CBD. By using a combination of characterization techniques (e.g. DSC, XRD, DLS), in-vitro assays (Caco-2 cell assays) and animal models (Sprague-Dawley rat model) we strived to balance the physical properties of the formulated API with its in-vitro bioaccessibility profile and its in-vivo pharmacokinetic performance.

Initial use of standard oils (e.g., sesame oil, corn oil), needed to solubilize the crystalline API prior to subsequent formulation, revealed not only a solubility limit (typically around 40%) which limits the CBD loading in the finished drug product, but also a tendency of the API to recrystallize after the drying process. To develop a patient-centric, stable CBD formulation (API loading >>10wt%) we explored a number of formulation approaches: encapsulation of a CBD nano-emulsion into a solid matrix, formation of amorphous dispersions by either hot melt extrusion or spray drying and encapsulation of CBD into liposomes and lipid nanoparticles.

Preliminary stability data (5°C, 25°C and 40°C) revealed that the formulated API is both chemically and physically stable for at least 6 months for all the evaluated technologies. Using in-vitro cell data, we were able to evaluate the effects of particle size, excipient choice/formulation method on the CBD bioaccessibility and implicitly select the optimal candidates for in-vivo bioavailability assessment. Finally, based on the in-vivo blood plasma levels, we were able to compare the pharmacokinetic behaviour of the CBD-containing solid dosage forms with a commercially available CBD oil and select the optimal candidates for further development and clinical testing.

The end results of our research will not only help improve the patient-centricity of CBD drug products, but also inform the pharmaceutical industry on the appropriate formulation for their intended applications, thereby enabling the industry to explore and expand upon the established therapeutic benefits of CBD more effectively.

Device Development

Human Factors in Labelling Development for Drug-Device Combination Products (DDCPs)

Florian Schauderna, Senior Manager Human Factors / Usability Engineering, Bayer Pharmaceuticals

  • Definition of labelling and grey zone materials
  • Opportunities and limits of labeling
  • Established paradigms and approaches in labelling development for pharmaceuticals challenged by applicable device regulations and standards (such as risk management).
  • Case study: Not only labelling contents are important, but also how they are presented to the user
  • Case study: Attention to details matters
It’s more than tribal knowledge that users tend to ignore labelling that comes with a product. Not only for that reason, regulators and international standards consider labelling as a weak risk control measure. Nonetheless, this presentation aims to be a plea for investing time and resources into developing labelling for DDCPs. Using case studies, the presenter will outline why involving the user and paying attention is key. If designed correctly, labelling can give reassurance to users and can be one important element of the DDCP user interface supporting safe and effective use. In some respects, established paradigms and approaches in labelling development for pharmaceuticals may have to be challenged.

12:00 PM - 12:30 PM - Case Studies

Technology & Innovation

Targeting Drugs in Vascular System

Vladimir Muzykantov, MD, PhD, Professor of Pharmacology and Medicine Vice-Chair, Department of Pharmacology Director, Center for Targeted Therapeutics & Translational Nanomedicine, University of Pennsylvania The Perelman School of Medicine

  • Circulation provides the natural route for systemic delivery of drugs
  • Coupling drugs and drug carriers to ligands of specific epitopes in the vascular and blood cells enables targeting
  • Multiple features of the targeted carriers modulate PK, binding, internalization and other parameters
  • Endothelial and red blood cells (RBCs) represent especially multifaceted and versatile targets
Endothelial cells lining the vascular lumen represent key target or/and barrier for drug delivery in many diseases. Using ligands of vascular surface determinants enables control of endothelial targeting, uptake, traffic and effects of drugs. Design parameters modulating targeting include: i) ligand nature, affinity and conjugation; ii) carrier’s size, shape and plasticity; iii) its pharmacokinetics; and, iv) configuration of multi-molecular assembly of targeted carrier (valence, ligand’s steric freedom). Biological factors modulating targeting include: i) location, accessibility, surface density and clustering of target molecules; ii) perfusion, permeability, and functional status of the target tissue; and, iii) functional consequences of anchoring to target. Permutations of design and biological factors yield multifaceted and diversified means for vascular drug delivery. Further, drugs and nanocarriers loaded on red blood cells transfer effectively to vascular cells in target organs. Vascular targeting enables effects unrivalled by untargeted agents in animal models of human diseases including acute lung injury, ischemia-reperfusion and sepsis. Current studies aim to define mechanisms and utility of “vascular nanomedicine”.


Biocompatible Polymer Systems for Efficient Systemic and Cellular Delivery of Nucleic Acid Based Drugs

Manfred Ogris, Professor for Pharmaceutical Sciences, University of Vienna

Nucleic acid-based drugs offer the unique opportunity for highly selective therapeutic intervention in areas of monogenic diseases, cancer and more. Major challenges for a successful treatment are to overcome biological barriers in the organism, to obtain selective delivery to the target site and achieve proper intracellular targeting. We have developed synthetic, polycation based and biocompatible carrier systems which address each of the aforementioned hurdles to finally unleash full biological activity of the nucleic acid delivered at the target site. Chemically stabilized oligonucleotides, small interfering RNA, mRNA and plasmid DNA can all be delivered by  biocompatible polymers and molecular conjugates. After local or systemic delivery the pharmacokinetics can be followed by noninvasive optical tomography combined with morphological imaging based on CT or MRI. On the cellular level, uptake, endosomal release and also nuclear accumulation can be followed applying assays combining biochemical and microscopy based methods. As exemplarily shown for a splice-switching antisense oligonucleotides, vectorization with a bioresponsive polymer strongly improves organ accumulation and biological activity in vivo. On the cellular level, barriers of uptake, endosomal release and nuclear uptake can be overcome. In addition, comparative studies on nucleic acid delivery in vivo, in vitro and ex vivo will be discussed.

Technology & Innovation

Automating MS-based Biotherapeutic Characterization Workflows for the Developability Assessment of Therapeutic Proteins

Christian Hug, Senior Scientist, Novartis Institutes for Biomedical Research

  • Fully automated mass spectrometry platform from request to report enabling high throughput analytics.
  • Expert review functionality implemented, allowing optimization of processing parameters and assignments in case of complex analytical questions.
  • Standardized workflows set up to ensure high quality data with high reproducibility of results across laboratories.
  • Time and effort savings allow to expand experimental conditions and thus increase the chance to select the most promising new biological entities.

Mass spectrometry (MS) is increasingly present in all stages of therapeutic protein discovery and development. The volume of produced data is consequently high and interpretation and reporting of analytical results is highly time and labor consuming. Key information must be extracted from these data and reported to assess critical quality attributes (CQA) which will eventually enable discovery, selection and developability assessment of therapeutic protein drug candidates. We have developed an IT environment and a series of workflows using Genedata Expressionist to automatically process, interpret and report protein mass spectrometry data generated on different MS platforms in various locations of our research organization. Our approach enabled reduction of data volume and CPU-intense processing and allowed for 24/7 processing of data. CQAs such as identity, integrity, and PTM modifications are determined and automatically reported using template reports. We will present strategies for bispecific antibody MS characterization and provide a set of examples describing automated determination of molecular mass and assessment of chain pairing.

Device Development

Implantable Devices for Long-Acting Drug Delivery

Eneko Larrañeta, Senior Lecturer in Pharmaceutical Sciences,  Queen’s University Belfast

Non-adherence to treatment costs European healthcare systems more €125 billion each year. Adherence is especially important when treating patients with chronic conditions that require lifetime pharmacological treatment, such as schizophrenia, Parkinsonism, HIV and Alzheimer’s disease. In addition to the economic impact, there is a direct human cost, as non-compliance significantly reduces patients’ health-related quality of life and, in many cases, is associated with early death. Considering the economic and human impact of non-adherence to treatment, there is a clear need for drug delivery systems capable of providing unattended drug administration for prolonged periods of time for these conditions. For this purpose, we have developed a wide range of long-acting drug delivery systems. These systems range from subcutaneous biodegradable implants to intranasal systems. For this purpose conventional manufacturing technologies (such as moulding or solvent casting) and novel approaches (such as 3D-printing) have been used.

12:35 PM - 1:05 PM - Solution Spotlights

Small Molecules

Leveraging Rapid Clinical Assessments to Accelerate Pediatric Formulation Development

Nazim Kanji, Executive Director, Pediatric Services Drug Development Consulting, Quotient Sciences

There are many important considerations to keep in mind when developing a pediatric dosage form and the requirements can greatly differ from drug products designed for adult use. Development scientists must consider the route of administration, safety profile, overall taste and palatability, the child’s age, weight, physiologic condition and the treatment plan’s requirements. All these key factors must be balanced appropriately in order to successfully develop a pediatric product that garners clinical, regulatory and commercial success. Arguably the greatest industry challenge however remains a lack of knowledge and guidance on how these development objectives can be successfully met.  In this talk, Nazim Kanji, Executive Director of Pediatric Services at Quotient Sciences, will share strategies and case studies illustrating how key challenges in pediatric product development can be overcome, allowing drug developers to get medicines to pediatric patients in need faster.

What you’ll learn:

  • How understanding taste issues early in development can help determine your taste masking strategy and target product profile (TPP)
  • How integrated clinical testing can be used to optimize pharmacokinetic (PK) performance of new pediatric drug products
  • How PBPK modelling and simulation can be utilized for dose extrapolation and to predict product performance
  • How adaptive clinical manufacturing and product supply strategies can get product to global pediatric trials faster


Trehalose, Sucrose and Amino Acids: Essential Components of Platform Biopharma Formulations

Christian Lotz, General Manager EMEA, Pfanstiehl GmbH

  • Commercial Biotherapeutics Stabilized with Trehalose / Sucrose
  • Essential components of a “Platform Biopharma Formulation”
  • Understanding important physicochemical properties of Trehalose and Sucrose
  • Purity, Quality, Consistency in Pfanstiehl’s Trehalose , Sucrose and Amino Acid products
  • Advantages of Trehalose over Sucrose as Stabilizer in Biopharma Formulations
  • From Liposome to m-RNA vaccines – a path, which started back in the 1965 – importance of highly purified and characterized stabilizing Excipients
  • Typical components in mRNA-LNP vaccine / Stability Enhancing Excipients in mRNA Vaccines / Technology
  • Examples for utilizations and functionalities of Sucrose and Trehalose in Covid 19 related formulations and applications
  • Amino Acid Buffers in commercial antibody formulations – Importance of utilization of highly characterized AAs as Excipients
  • Amino Acids as viscosity lowering Excipients
  • Methionine as Biopharmaceutical Stabilizer and Antioxidant
  • Pfanstiehl’s Biopharma Stabilization Portfolio (Carbohydrates and AA) with newly upcoming launches: L-Methionine and L-Glutamine
  • Conclusions

Technology & Innovation

Nanoparticle Engineering, Stabilization, and Formulation Development: A Patient and Planet Centric Approach for Formulation of Poorly Soluble Molecules

Martti Kaasalainen, Senior scientist, Nanoform Finland

  • Development of highly efficacious drug products is often hindered with poor aqueous solubility of drug substance – a well-known challenge of the pharmaceutical industry.
  • Nanoform’s patented technology of controlled expansion of supercritical solutions (CESS®) allows the production of, down to 50 nm sized, drug substance of different shape and crystallinity, increasing the dissolution rate and helping to improve dosage form design towards patient centric formulations.
  • The technology does not use organic solvents but only recycled CO2, thereby offering a greener solution compared to many other solubility enhancing technologies.
  • Nanoform has extensive experience with stabilization of nanoformed drug substances and further formulation development, thereby, offering new opportunities to reduce drug development attrition and getting innovative treatments to patients earlier.

We are proud to launch our new formulation platform technology at DDF: Nanocrystallisation of amorphous nanoparticles for added performance and stability in formulations.

Device Development

Simulating Injection Back Pressure for Drug Delivery to Subcutaneous Tissue, Cerebral Ventricles and Beyond

Scott Ariagno, Vice President Development & Engineering Services, EdgeOne Medical Inc.

Key points:

1. Injecting into air during administration testing neglects tissue back pressure, which introduces risk into delivery device selection and qualification decisions.

2. This presentation will provide guidance on back pressure levels and identify various methods to simulate effects of back pressure.

3. Case studies from the EdgeOne Medical laboratory show how test methods can be adapted to simulate subcutaneous, ICV and other routes of administration.

Ability to achieve successful injection of drug or biologic depends on many factors including but not limited to needle diameter, drug viscosity, spring force, lubrication, plunger geometry and material properties.  One additional challenge to be considered during verification of the drug delivery system is effect of back pressure from the patient (tissue).  This talk will highlight various options and/or considerations for back pressure during feasibility and design verification testing.  Concepts are broadly applicable to pen/auto injectors, on body delivery systems, pre-filled syringes, intracerebroventricular injection catheters, and other delivery systems.

1:05 PM - 2:05 PM

Networking Lunch

2:05 PM - 2:35 PM - Case Studies

Small Molecules

Amorphous Solid Dispersions: Current Perspectives, Challenges & Opportunities

Samuel Kyeremateng, Principal Scientist , AbbVie

The terminology amorphous solid dispersion (ASD) was coined about 40 years ago to specifically describe solid pharmaceutical formulations in which the active pharmaceutical ingredient (API) is amorphously embedded in a polymer matrix. ASD as a formulation strategy, enables an increase in the apparent solubility and bioavailability of poorly-water soluble APIs, typically those in the Biopharmaceutics Classification System (BCS) II and IV. Over the last decades, significant scientific progress has been made to mechanistically understand ASDs and to address the challenges associated with their manufacturability, stability, and performance. In this presentation, an overview of recent progress made on the formulation and process modeling front to address these challenges will be discussed. Furthermore, emerging modeling techniques to address current deficiencies or gaps will be highlighted.


Formulation Development For A Human Plasma Protein – Use of DoE and Statistical Evaluation

Joris Höfinghoff, Head of Formulation Development for Plasma-derived Therapies, Takeda

  • Development of a liquid formulation for a human plasma product
  • The use of design of experiment (DoE) for formulation development
  • Long term stability prediction based on Arrhenius-equation
A case study for a formulation development project for a human plasma protein in liquid form is presented. A DoE-approach was used for the identification of potential excipients and the optimization of the final formulation composition. All developmental stages (pre-formulation-/excipient screening) are discussed. Potential formulation candidates out of the DoE-studies were subject of a stability study over six months. The stability data were use for long-term stability prediction based on the Arrhenius-equation.

Technology & Innovation

Continuous Direct Compression (CDC) Technology Implemented Using a Semi-Continuous Blending Step: a case study

Yunfei Li Song, Senior Scientist (Process Engineering and Analytics), GSK

  • Potential drivers for the use of semi-continuous blending CDC technology.
  • Review of gravimetric batch mode twin screw feeding of relevant powder materials.
  • Review of mini-blend high shear horizontal powder blending.
  • Industrialisation aspects.
A Continuous direct compression (CDC) line implemented using a semi-continuous blending step is a viable and robust platform for the manufacture of tablet formulations for a wide range of API’s and dosages. A semi-continuous high shear blending step has different operational limitations and therefore is able to attain additional operating spaces compared to more commonly employed fully-continuous blending technology.


Device Development

Connected Injection Devices - Improving Real-World Outcomes

Kasper Bayer Frøhling, R&D Programme Manager, Medical Device Development, Novo Nordisk

  • Improving real-world outcomes of our treatments will be achieved with increasing assistance from digital health solutions
  • Using our smart pen-injector NovoPen 6 for insulin treatment we demonstrated improved glycemic control and time-in-range
  • We also gained valuable user insights enabling us to target specific improvement areas
  • We continue to pursue solutions where we can bring connectivity to our portfolio of devices
Closing the gap in medical outcomes achieved in real-world settings compared to controlled clinical studies, is a major challenge for the pharmaceutical industry. One of the ambitions of digital health solutions is to facilitate better dialogues between health care professionals (HCPs) and diabetic patients, by providing data from injection devices and continuous glucose measurements (CGM). Comparing glucose levels and individual injection patterns informs HCPs on which topics to address in a patient consultation to improve long term outcomes. A pilot study in Sweden using NovoPen® 6 demonstrated significant improvements to time-in-range and fostered an increase in bolus insulin compared to baseline without increasing the risk of hypoglycaemia. According to the CORE diabetes model achieving longer periods of time-in-range will significantly reduce long term comorbidities, improve quality of life, and reduce socioeconomic burden on healthcare systems.

2:40 PM - 3:10 PM - Solution Spotlights

Small Molecules

The Perfect Match Between Patient Needs and Optimized Manufacturing: MMTS™ Minitabs

Giuseppe De Franza, Senior Manager, Pharmaceutical Development and Technical Service, Adare

  • Learn about MMTS technology and how minitablets are considered a pediatric- friendly formulation
  • Learn about an important minitablet case study: Zenpep
  • Learn about Adare’s huge expertise of manufacturing minitablets on industrial scale


More than a Theory - Successful Delivery of Biologics and Vaccines by Microarray Patch

Anna Schlüter, Head of Formulation and Process Development MAP Innovator, LTS

LTS has successfully demonstrated the capability of the dissolvable MAP to deliver a biologic in a pre-clinical setting and a vaccine in a clinical setting.

LTS conducted a pre-clinical trial with recombinant ß-Interferon, a cytokine used to treat multiple sclerosis. It was demonstrated that the MAP technology can deliver recombinant ß-Interferon comparable to IM/ID injection of the marketed product. Further, the manufacturing process for the dissolvable MAP does not impact the bioactivity of the ß-Interferon.

LTS conducted a clinical Phase 1 trial with a Hepatitis B antigen (HBsAg) VLP vaccine MAP as a boost. The application of HBsAg MAP induced a very strong immune response without the need for an adjuvant. The MAP application was able to boost the antibody titer, on average, by 116-fold with an applicator and by 14-fold when applying the MAP by hand. The MAP is superior to standard vaccination by syringe with the approved adjuvanted HBsAg VLP vaccine with respect to the protective antibody titers induced. The application was well tolerated, and no serious adverse events or adverse events were reported.

Technology & Innovation

ASD-Based Oral Drug Product Development: The Role of Biopredictive Tools

Mafalda Paiva, Senior Manager, R&D Analytical Development, Hovione

A faster and reliable early development phase is what the industry seeks, the patient expects, and the scientific community contributes to. At Hovione, we’re working to second this with high-throughput approaches for amorphous solid dispersions (ASD)-based oral drug product development with the most adequate functional excipients. This is supported by the understanding of the mechanisms that underly enhanced performance. Dissolution and permeation mechanisms of ASDs are complex and have increasingly been studied over the last years. This knowledge and the link between enhanced drug exposure and process parameters are key when formulating BCS II and IV poorly soluble compounds. One of the mechanisms identified relates to the ability of some ASDs to form drug rich colloids. These structures can, and often do, behave as a drug reservoir acting as a continuous supply of drug as it permeates. Along with the increasing demand for complex formulations comes the need for appropriate in vitro methodologies capable of predicting their corresponding in vivo performance and the mechanisms controlling the drug release which can impact on in vivo drug absorption. In this presentation we'll review formulation strategies to enhance drug exposure and the in vitro methodologies that can support effective in vitro formulation screening. Also, the data presented can shed light on the important dissolution and permeation mechanisms that govern increased drug product performance and how this knowledge - currently part of Hovione portfolio - can be a reliable partner at the early stages of development for low solubility APIs.

Technology & Innovation

Scalable Lipid Nanoparticle Formulation Using Impingement Jets Mixing Systems

Paul Pietsch, Global Engineering Solutions Manager, Knauer Wissenschaftliche Geräte GmbH

  • From formulation to GMP production: up-scaling of LNP formulation
  • Development and optimization of LNP formulations
  • Flexibility and automation of LNP formulation: automated high throughput screening and sample collection

3:10 PM - 4:00 PM

1-2-1 Pre-Scheduled Meetings & Networking Break

4:00 PM - 4:30 PM - Case Studies

Small Molecules

Formulation Environmental Decision Tool (FEDT)/How to Add Environmental Dimension in Selection of a Drug Product Formulation - Spray Drying or Hot Melt Extrusion, a Case Study Application

Emilie Belissa, Drug Product Expert Product Design & Performance (PD&P), UCB

Formulation Environmental Decision Tool (FEDT) is a tool which has been developed to compare different drug product compositions and manufacturing processes in function of their global warming potential (GWP). It take into account CO2e emission of drug substance(s) and excipients; and electrical consumption of the different equipment used during manufacturing. At UCB, it was used to help in the selection of final drug product for Phase 3 and commercialization: a film-coated tablet made out of an amorphous solid dispersion by either spray-drying, either hot melt extrusion.


Development Strategy for Ultra-High Concentrated Biotherapeutics.

Martin Huelsmeyer, Head of Science Affairs & Strategic Initiatives NBE Drug Product Development, AbbVie

In the last decade mAb-based biopharmaceutical treatments shifted more and more towards subcutaneous self-administration to improve patient convenience and compliance. The restricted dosing volume of conventional subcutaneous injections in combination with ever increasing dose requirements of many mAbs and mAb-related products pose some challenges in the development of these products, in particularly viscosity and/or stability issues. The talk will highlight AbbVie’s staged approach to define suitable formulation compositions for ultra-high concentrated products (>100 mg/ml) and introduce a novel downscale device to measure detailed concentration – viscosity profiles as a key element in formulation screening.

Technology & Innovation

Leveraging Mechanistic Models for Drug Product Development of Small Molecules

Karsten Flügel, Principal Scientist Digital Pharmaceutical Development, Merck Group

  • Mechanistic models for drug product development have the potential to accelerate process development and rationalize scale-up strategies
  • Different complementary modelling approaches are utilized depending on the aspect of interest, while being aware of their limitations
  • Most beneficial application areas for mechanistic models are scale-up, troubleshooting and process robustness testing
  • Application in several development projects have proven the benefit of mechanistic models
Utilizing mechanistic models to support process development activities has tremendous potential to reduce experimental work and to rationalize decision-making during many important development phases, not only during scale-up. At the same time, it is important to be mindful about the selection of models and to be aware of model limitations, which every model inherently has. This talk will give an overview over different complimentary modeling approaches currently in use in Merck’s pharmaceutical development and provide some case studies as application examples, where modeling was existential to elucidate a certain aspect while being supported by experimental work.

Device Development

Development of a Smart Connected Device Solution

Tonio Hoche, Device Engineer, Roche

  • Inform the audience about the user centric design process the team applied.
  • Provide insights and learnings on the UX studies conducted.
  • Describe framework and new internal collaboration models to develop the Minimal Viable Product for the connected digital device solution.

A cross functional team at F.Hoffmann-La Roche is developing a digital device solution integrated in the drugs patient support program. Starting with strong collaboration with patient and caregiver associations the team performed several UX studies to identify user needs. 

The critical success factors of these studies as well as their main outcomes are described during the presentation.

The internal & external cross-functional collaboration model is presented and the stage-gate process for the definition of the Minimal Viable Product (MVP) explained.

The Minimal Viable Product of the digital solution consists of a connected device, a smartphone app and a cloud backend. Special attention will be given to the required new internal collaboration models which are essential to define such a novel, patient centric digital solution.

4:35 PM - 5:05 PM - Case Studies

Small Molecules

In Vitro Release Characterization of Oral Modified-Release (MR) Tablet & Pellets Formulations

Yushi Sunazuka, Scientist in Analytical Science Group, Nippon Boehringer Ingelheim

  • We have experienced development of two different types of modified release (MR) formulations, swellable matrix tablet and coated pellets formulations, for the same compound.
  • Each formulation technology showed unique release characteristics against dissolution environmental changes such as agitation speed, pH, and salt concentration.
  • Characterizations were performed for understanding the release properties of each formulation.


High-Throughput Formulation Screening Approach to Develop Ultra-High Concentrated Bioproducts.

Isabelle Deimel, Scientist, Abbvie

In early stages of formulation development high throughput screenings offer the advantage to generate huge and homogenous datasets to enable comprehensive understanding of the behavior of a new drug candidate and definition of design space. With increasing demand for ultra-high concentrated (UHC) formulations there was a need to develop a screening module which combines stability and viscosity assessment. The talk introduces development and results from a new screening approach that extends our existing high throughput capabilities with focus on UHC formulations. Using a combination of pH, buffer systems and viscosity reducing excipients the formulation space is systematically evaluated for suitable compositions. The generated data set demonstrates applicability of the new screening approach to develop ultra-high concentrated bioproducts.

Technology & Innovation

Inflammation-Responsive Supramolecular Gels for Controlled Drug Delivery

Nitin Joshi, Assistant Professor Brigham and Women’s Hospital, Harvard Medical School

Multiple inflammatory diseases exhibit variable disease activity over time with exacerbations (flares) and periods of low disease activity. Previously described drug delivery systems provide sustained drug release irrespective of disease activity. This likely results in sub- or supra-therapeutic drug levels locally during periods of high or low disease activity, respectively. We have developed a supramolecular hydrogel based drug delivery platform from low molecular weight amphiphilic gelators. This platform can titrate drug release to the level of inflammation, enabling disease severity-matched drug release in inflammatory arthritis and organ transplant that are associated with periodic fluctuations in the level of inflammation. We have also tuned the surface charge of these gels to enable their electrostatic interaction-mediated selective adhesion to inflamed tissue for inflammation-targeted drug delivery in topical inflammatory diseases, including mucositis and esophagitis.  Supramolecular nature of these gels imparts them excellent self-healing property, enabling them to withstand repeated mechanical loading at levels relevant to the running human knee. We have demonstrated that due to this rapid self-healing property, these gels can be used for intra-articular delivery of disease modifying osteoarthritis (OA) drugs in physically active OA patients with early disease, without any impact on the drug release kinetics. My talk will discuss different applications of our supramolecular hydrogel platform, covering our previously published and currently ongoing work towards creating next-generation therapies for inflammatory diseases.

Device Development

Drug Device Combination Development for Large Volume Therapeutic Proteins

Claus Geiger, Global Device Leader , Sanofi

Novel approaches to the delivery of therapeutic proteins that have gradually entered the portfolios of drug makers over the last two decades are changing more and more medical treatment solutions for many patients and improving their quality of life substantially.

But there are challenges to overcome, primarily technically and clinically. Focus is provided on those challenges that revolve around the current landscape of device mediated delivery of therapeutic proteins, namely monoclonal antibodies in terms of volume ranges, device technology platforms, drug device combination development, Human Factors and Clinical Use Testing and how integrate drug and device developments.

5:05 PM - 5:35 PM - Keynote

Device Development

Recent Experiences and Challenges with Medical Device and Single Integration Combination Products for CE Mark

Mike Wallenstein, Global Head RA Medical Devices, Combination Products & Precision Medicine, Novartis Pharma AG

  • Lessons learned 1st Product getting a CE Mark under EU MDR
  • Lessons learned about a Notified Body Opinion for a Prefilled Syringe
  • Ongoing challenges
  • Outlook on MDR and Article 117


5:35 PM - 6:15 PM - Keynote

Device Development

Regulatory Strategy Lifecycle Management Considerations for Complex Drug Delivery Devices - How Safe Are My Combination Products?

James Wabby, Global Head, Regulatory Affairs (CoE), Emerging Technologies, Combination Products and Devices (Chairperson), AbbVie

  • Regulatory Landscape Understanding - Complex Products and Digital Devices
  • Product and Usability Development Key Concepts
  • Lifecycle Management Highlights
  • Regulatory Submission Aspects for Complex Products
  • Case Studies – Clinical Trial and Complaint Handing Concepts
Next generation combination products are emerging as innovative medical products due to their contribution in advancing medical care and are expected to have a major impact in the coming years.  Future technologies are most appealing to patients with ongoing medical conditions that require consistent treatment with daily injections or weekly procedures and unmet medical needs. The opportunity to work within the combination product space and to work cross functionally with medical device, software, pharmaceutical and biologic experts will be a great experience which will involve a ton of knowledge sharing across the team of experts.  While there are similarities there are also many differences in the development of various combination product therapeutic applications. Overall, the successful development of combination products will require significant collaboration within the industry to overcome regulatory, clinical, technical and lifecycle management challenges.

6:15 PM - 6:20 PM

6:20 PM - 7:10 PM

Evening Networking Reception

8:00 AM - 8:45 AM

Registration & Refreshments

8:45 AM - 8:50 AM

8:50 AM - 9:20 AM - Keynote

Device Development

Medical Devices and In-Vitro Diagnostics Used in Drug Clinical Trials: Principles of Use and Sponsor Responsibilities Under the Medical Device and IVD Regulations Requirements

Dr. Fatima Bennai-Sanfourche, Senior Director of QA & RA Compliance for Medical Devices and eHealth

  • Regulatory landscape changes
  • Overview of medical devices used in drug clinical trial
  • Sponsor responsibilities for medical devices used in the clinical trial according the requirements of MDR/IVDR
  • Combined trial and management of the dual submissions

9:25 AM - 9:55 AM - Case Studies

Small Molecules

PAT-Concept for a Semi-Continuous Fluidized-Bed Granulation Process

Dr. Frank Wolters, Lab Head, Bayer AG

Continuous manufacturing of solid dosage forms gained a lot of attention during the last years. The introduction of Process Analytical Technology (PAT) supports a right-first-time production process to ensure the correct product quality. This study shows a proof-of-concept investigation for a process control concept including process sensors and process analytical technologies (NIRS) demonstrated during long runs of the Syntegon XELUM line. The long run includes continuous manufacturing from weighing of the starting material to tableting process with a material throughput of 30 kg/h.

NIR-Sensors are implemented for moisture content determination of granules, BU and API concentration in ready-to-press blend and monitoring of tableting process. As a result, it could be shown that the process can be precisely monitored by the investigated process analytical technologies.


Concept of Drug Product Manufacturing Process Characterization

Mostafa Nakach, Global Head of Process Engineering Biologics Development/ Biologics Drug Product Development & Manufacturing, Sanofi

  • Failure mode and gap analysis
  • Application of Quality by design approach
  • Process design and its characterization for establishing a design space

Technology & Innovation

Development of a Controlled Nucleation Protocol and Transfer to a Clinical Supply Facility

Stefan Schneid, Scientific Lead Parenterals, Bayer Science Fellow, Bayer

  • Development of Controlled Nucleation Protocol
  • Assessment at Lab Scale and in Aseptic Pilot Plant
  • Transfer to Clinical Supply Line and Assessment
  • Analytical Results of Controlled and Non-Controlled Nucleated Drug Product

Abstract and take-home message:

Controlled Nucleation is recognized as versatile approach to reduce transfer risks for freeze drying processes, and offers the potential to reduce primary drying time. However, implementation in GMP processes and larger freeze dryers is so far not common. This case study describes the development of a Controlled Nucleation Protocol and its Optimization at Lab Scale and Aseptic Pilot Scale, with a subsequent technical run in a GMP facility. The analytical results including stability data from samples processed with and without controlled nucleation will be discussed.

Device Development

Device Development - Improving Patient Experience Through Digital and Combination Products

Nélio Drumond, Associate Director, Lead Process Scientist Global Manufacturing Sciences, Drug Product, Takeda

  • High incidence of handling errors during administration of inhalation therapies are still reported.
  • Development of inhalation products that do not consider patient needs will fail.
  • Inhalation devices must be developed directly with the patients from early stage to commercialization, including feedback post market launch.
  • Patient centric drug product design together with digital technology will have a key role promoting successful inhalation therapy.
Modern patients are more than ever informed about their own health and equipped with evolving technology that support continuous and accurate data tracking regarding their own wellbeing. As a result, patients are expecting a new level of high-quality medicine, making a culture of patient centric care imperative to both providers and patients. A medical environment that encourages consistent collaboration and clear communication between patients, families, and providers will benefit all parties involved. With regards to inhalation therapy, smart and digital inhalers can promote patient centric care by supporting patients to be informed about their inhalation technique and administration success. When patients are given the opportunity to have easy access to their own health data and the power to research, they can make informed decisions about their own treatment options. This results in both improved outcomes and patient experience. Healthcare providers also have an opportunity to optimize and streamline internal processes, therefore reducing costs and better allocating resources.

10:00 AM - 10:30 AM - Solution Spotlights

Small Molecules

Strategies for Reducing Nitrosamines Contamination in Drug Products

Dr. Andreas Sauer, Technical Sales Director, Shin-Etsu

  • Recent news about nitrosamines in drug products.
  • Background on formation of nitrosamines in drug products on API level, formulation level, and packaging level.
  • Case study on Metformin HCl sustained release tablets and influence of Metformin HCl and hypromellose source on the development of nitrosamines in the drug product.


Biodegradable Polymer-Based Drug Delivery Technologies for Development of Tomorrow’s Long-Acting Injectable Medicine

Rob Steendam, Chief Technology Officer, InnoCore Pharmaceuticals

  • SynBiosys microspheres for long-term sustained release of sensitive biologicals
  • Ready-to-use in situ forming depots with improved injectability for delivery of small molecule drugs
  • Learn about the unique features of our biodegradable polymeric dosage forms and how they add value to our partner’s drug molecules and improve patient’s quality of life
InnoCore Pharmaceuticals is a biopharmaceutical drug delivery company offering a versatile portfolio of biodegradable polymer-based drug delivery systems (DDS) and development services for long-acting injectable dosage forms to treat chronic and site-specific diseases. The differentiating features of our advanced DDS for extended release and site-specific delivery of small molecules, peptides and biological therapeutics will be discussed.

Technology & Innovation

Challenges Associated with In-Use Administration of Biologics

Léa L. Sorret, Principal Scientist and group head in Formulation Development, Lonza Drug Product Services

The goal of in-use testing of biotherapeutics is to ensure acceptable product quality during dose preparation and administration into patients. This testing comprises of stability and compatibility studies of the materials and diluents used in the preparation, storage, and in-use period of the drug product, tailored to its target route of administration. This ensures patient safety by identifying potential risks due to exposure of not-previously assessed contact materials or diluents.

Considering the wide range of in-use conditions and administration systems and set-ups available, we discuss some of the more challenging aspects surrounding in-use testing while reviewing potential strategies to address these issues.

Device Development

Patient-Relevant Features of an On-Body Drug Delivery Device for Large Volumes and High Viscosity Drugs

Dr. Sabine Websky, Head Medical Affairs & Applied Technologies, Medical Affairs & Applied Technologies, Gerresheimer

  • Subcutaneous self-administration of large-volume and high-viscosity medicines is becoming increasingly important. How can a drug-delivery medical device solution address such challenges?
  • Early inclusion of the patient perspective and input from the usability evaluation to identify further user needs
  • Patient loaded concept: Best addressing the supply chain requirements for patients, Healthcare Professionals and Providers. It reduces the burden of cold chain requirements of most biologic drugs since it allows device storage separately at room temperature while the drug is being maintained at cold chain temperature saving up space and energy at hospitals and throughout the entire supply chain sequences.
  • Resusable-Disposable EcoDesign concept allows for waste reduction and is hence more sustainable than a fully disposable device

10:30 AM - 11:20 AM

1-2-1 Pre-Scheduled Meetings & Networking Break

11:20 AM - 11:50 AM - Case Studies

Small Molecules

The Potential of Lipid Based Formulations for Brick Dust Molecules

Rene Holm, Prof. Drug formulation and delivery, University of Southern Denmark

  • What lipid based formulations have been investigated for brick dust molecules
  • Lipid based suspensions and their potential
  • Lipidic crystal/liquid crystals and their potential application in lipid based formulations
  • Is supersaturated lipid systems an approach that could get beyond the academical laboratories
Lipid based formulations have a number of benefits for early and late development, including low API consumption for formulation development, easy manufacturing and scale-up, a higher number of GRAS excipients and a well-established industrial infrastructure for the commercial manufacturing of the formulations. Lipid based formulations have classically found their application for lipophilic compounds, that could be solubilised in the vehicle, but as more and more compounds in the pipelines is now brick dust molecules, the formulation strategy may at first look less relevant, as these molecules do not solubilise in the lipid. This talk will present, what lipid based formulation systems that could be relevant to consider for brick dust molecules, so formulation scientists can look at all potential options, when engaging into the formulation development of a new brick dust molecule.


Spray Drying of Lipid Nanoparticles Enables Intratracheal Delivery of mRNA

Kristina Friis, Associate Principal Scientist in Advanced Drug Delivery, Astrazeneca

  • Key parameters required to facilitate spray drying of LNPs
  • Enhancing LNP stability, cellular uptake, and RNA delivery to the lung
  • Potential for future applications including inhaled vaccines

Recently, RNA therapeutics have taken a giant leap forward with the first LNP-based mRNA COVID19 vaccines reaching the market. LNPs represent a powerful delivery system since they both protect the mRNA cargo from degradation and facilitate its cellular uptake and endosomal release. The stability of the liquid mRNA-LNP formulation remains a challenge, which can be addressed by changing to a dry powder formulation, which in turn allows for pulmonary administration. Spray drying is one of the most common methods for solidification of biologic formulations. We have developed and optimized a spray drying process for LNP solutions which results in functional mRNA delivery to the lung upon intratracheal administration in a rat model

Technology & Innovation

Nature-Inspired Systems for Oral Delivery of Biologics

Driton Vllasaliu, Senior Lecturer in Pharmaceutics, King’s College London

  • Oral administration is currently not possible for biologics
  • Nanomedicine-based delivery systems show potential for oral delivery of some biologics
  • Some extracellular vesicles are stable in the gut and cross the intestinal epithelium efficiently
  • Such extracellular vesicles could serve as delivery systems (or inform the design of such systems) for oral delivery of biologics
Oral administration is the most convenient and preferred way of taking drugs. However, apart from limited exceptions, oral administration is currently not an option for biologics. The use of absorption enhancers has limitations, including safety issues and its applicability to a narrow range of biologics. Different delivery strategies are therefore urgently needed. Nanomedicine-based approaches have promising potential for oral biologics, but the biological barriers in the gastrointestinal tract pose severe challenges. In this talk, I will summarise the latest findings from my lab on nanomedicine-based approaches, focusing predominantly on extracellular vesicles as nature inspired delivery systems and their potential for oral delivery of biologics such as nucleic acids.





Device Development

Liquid Inhalers – Evolution Towards a New and Diverse Technology Landscape

Dr. Gunilla Petersson, Former Science and Innovation Director Inhaled Drug Delivery, AstraZeneca

  • Why a pull for liquid inhalers although we have powder inhalers and pMDIs?
  • Liquid inhalers; current and future landscape, outline of the evolving landscape of liquid inhalers and new technology developers
  • Which are the gaps and downsides and how are they addressed?
  • What to consider selecting a device; drug, formulation, disease, patient use
  • Challenges delivering biologics in liquid inhalers and how they can be addressed
  • Paediatric lung delivery a challenging area; what is being done to improve lung delivery and patient acceptance?
Jet and vibrating mesh nebulisers have been around for a long time, and so has Respimat, the only soft mist inhaler commercialised for treatment of lung diseases. Nevertheless, powder inhalers and pressurised metered dose inhalers, pMDIs, are by far the most widely used inhalers for commercially available brands. It is therefore surprising that a significant number of relatively new device companies are investing in the development of new, modified nebulisers and softmist inhalers. The rationale for this and the challenges associated with selecting between powder and liquid for new pipeline products is reviewed. Also requirements on novel devices to be competitive and expectations and challenges when applied to new and expensive biologics are discussed. The liquid inhaler platforms are also developed to be more diverse with customisable, flexible variants, which may request an update on how we define a nebuliser vs a soft mist inhaler, is there a “hybrid” coming? Paediatric delivery is often involving a nebuliser with wellknown hurdles and draw backs, but also novel inhaler concepts are being developed for children.

11:55 AM - 12:25 PM - Case Studies

Small Molecules

Leveraging Predictive Tools for Improved Film Coating Processes

Elise Vaes, Scientist OSD, Janssen

  • Film Coating in Drug Delivery
  • Understanding tablet motion with particle simulations
  • Case study: the effect of pan load and baffle design on tablet motion
Film coating of pharmaceutical solid dosages is essential to provide the final functionality of the drug product since it gives products superior organoleptic properties, it can be used as a mean for target drug delivery and modified release and can enable patient centric medicine using coating systems containing API. The highest product quality must be strived and therefore a high focus on product quality attributes must be given, including achieving the best film quality and minimizing coating defects. An in-depth and robust process understanding of the tablet coating process, including tablet motion, spray dynamics and thermodynamics, is vital to select the most optimal processing parameters, asses the effect of equipment design, understand the effect of batch size etc. Leveraging predictive tools in the development of tablet coating processes can accelerate achieving the best process design and has the potential to increase the efficiency, flexibility and robustness of troubleshooting and technology transfer strategy.


Viscosity and pH Challenges in Development of High Concentration Injectable Protein Formulations

Camille Dagallier, Senior Formulation Scientist, Sanofi

  • Main factors of viscosity increase in protein biotherapeutics
  • Resulting challenges for process development and administration
  • Formulation and technical levers to address viscosity challenges

Technology & Innovation

The Relationship Between Liquid-Liquid Phase Separation and Protein-Protein Interactions for mAbs

Robin Curtis, Senior Lecturer in the School of Chemical Engineering and Analytical Science, University of Manchester

  • Measurements of liquid-liquid phase separation (LLPS) boundaries and the osmotic second virial coefficient b22 (a protein-protein interaction parameter) are reported for 3 different mAbs under low salt conditions at neutral pH and under salting-out conditions at high ammonium sulfate concentrations.
  • Deviations from a well-established correlation between the diffusion interaction parameter kD and b22 provide an indication of reversible self association (RSA)
  • The onset of LLPS falls in a small window of b22 values for all protein-salt mixtures except for one mAb at low salt conditions, which undergoes RSA
  • Simultaneous b22 and kD measurements can be used for predicting phase separation at higher protein concentrations.
  • The shape of the LLPS curve plotted in terms of b22 depends on the precipitation conditions: a much broader profile occurs at high salt versus low salt concentrations.  The LLPS curves provide critical insights into the form of the protein-protein interaction potential.
Being able to predict and control concentrated solution properties for solutions of monoclonal antibodies (mAbs) is critical for developing therapeutic formulations.  At higher protein concentrations, undesirable solution properties include high viscosities, opalescence, particle formation, and precipitation.   The overall aim of this work is to understand the relationship between liquid-liquid phase separation (LLPS) at high protein concentrations and commonly measured dilute solution parameters, the reduced osmotic second virial coefficient b22 and the diffusion interaction parameter kD.  This study is motivated by the observation for globular proteins (lysozyme, gammaB-crystallin) that the LLPS behaviour collapses when plotted in terms of b22 indicating phase separation is controlled by the net protein-protein interaction.  Because many mAbs undergo reversible self association (RSA), it is not known if b22 can be used as a reliable predictor for LLPS. Here, we map the LLPS curves for three mAbs at a low salt condition near the pI, where we expect strong RSA, and under salting-out conditions in concentrated ammonium sulfate solutions, which is expected to reduce RSA.  We find that the onset of phase separation occurs in a small window of b22 values, except for one mAb-salt mixture.  In addition, we show that deviations from a well-established correlation between b22 and kD provide an indication of RSA, which is used to rationalize why there is an outlier from the correlation between b22 and LLPS.  Taken together, this indicates that simultaneous measurements of b22 and kD provide an accurate indicator for phase separation at high protein concentrations.

Device Development

Are Sustainability, Reusability & Green Manufacturing major trends? - A case study of Respimat®

Felix Weiland, Head of Device Technology, Boehringer Ingelheim

  • Soft Mist Inhalation
  • Respimat
  • Sustainability
Respimat is an eco-friendly inhaler with a precise performance and a robust actuation - inhalation coordination. Current Respimat comes with 1 cartridge. With respect to environment and improvement of usability, the new re-usable Respimat was developed to be used with up to 6 cartridges.

Changes, including a dose counter attached to the cartridge, were implemented to enable the re-usable concept and to improve ease of use. Dosing principle stayed unchanged. Performance of re-usable Respimat over six cartridges was proven to be comparable to the marketed version.

At present there is an increasing interest of politics, patients and society on holistic sustainability concepts, especially in reduction and avoidance of waste and Carbondioxide. Sustainability must be considered a relevant design input for new products, thereby Reusability is a powerful option.

In conclusion, the Respimat Reusable combines high lung deposition, low carbon footprint and a good patient experience to improve patients´outcome.

12:30 PM - 1:00 PM - Solution Spotlights

Small Molecules

Nitrosamine Risk Mitigation: The Critical Role of Excipients

Dr. Alberto Berardi, Product Application Specialist, DFE Pharma

Show more


DEVELOPICK™: Rapid and Accurate Evaluation Tool for Molecular Stability

Heonchang Lim, Group Lead, Formulation Development, Samsung Biologics

As the global biopharmaceutical market continues to grow at a rapid pace, there is an increasing demand for drug development. In order to select the most suitable biologic compounds, it is essential to exclude those with poor stability and developability. This presentation introduces DEVELOPICK™, Samsung Biologics' developability assessment tool, which has been meticulously designed with test items and an evaluation process that were verified through real-life case studies.

Technology & Innovation

Availability of Vaccine Delivery Systems, Immunostimulators and Combined Adjuvant Systems in the Next Decade

Peter Tygesen, Managing Director, Adjuvant Systems , Croda Pharma

The COVID-19 pandemic shook the world, and it was a wakeup call for the vaccine industry. There are currently numerous issues faced in adjuvant technologies. These include the fact that some novel adjuvant technologies used in vaccines today are proprietary of pharma companies – so they are not available commercially for the industry to use. Furthermore, some of the industry is limited by production capacity – meaning huge strategic investments to scale production capacity is needed, or more novel adjuvants are desperately needed to initiate additional immune responses. What needs to come first to unlock novel vaccines of the future? In this presentation I will outline the Croda Pharma strategy to close this gaping gap, potentially a missing link between vaccine academic research and readily available GMP vaccine adjuvants which should be available for all.

Device Development

Avoiding Autoinjector Misfires by Bypassing the Spring Release Mechanism - is a pressurized cartridge the solution?

Daniel Primavessy, Project Manager Innovation Pharmaceuticals, Midas Pharma GmbH

A release mechanism is designed to reliably release a force. Also using it as a barrier to contain the force over a long time is a contradiction to its purpose, but mode of operation in all common spring-driven autoinjector devices. This presentation discusses the issue shortly and presents a split solution where the primary container, a cartridge, is the pressure barrier and the release mechanism of the device is separated. The presented solution to the problem is backed up with data from pressure tests regarding the primary packaging material (glass container, rubber seal and plunger). In addition, stability data for Adalimumab under pressure is presented. This set of data allows a detailed evaluation of the idea, its abilities, and boundaries. Among the latter a novel technique for the redispersion of suspensions will be presented, which additionally avoids sediment solidification in primary packaging containers.

  • Separating the spring-release mechanism from the pressure barrier
  • Pressure resilience of primary packaging materials and antibodies
  • Suspension redispersion and avoidance of sediment solidification in primary packaging containers.

1:00 PM - 2:00 PM

Networking Lunch

2:00 PM - 2:30 PM - Case Studies

Small Molecules

Accelerated Stability for Small Molecules Drug Product

Jean-Rene Authelin, Senior Scientific Advisor Global CMC  , Sanofi

Accelerated stability studies allowing to predict shelf life of drug substance or drug product or to support formulation choice or packaging choice are becoming the new paradigm. We will present 2 case studies of molecules in development where we worked in collaboration with the company Freethink and the software Asap prime ™ . Model prediction was fitting well experimental data. The use of accelerated studies was very key to design packaging and asses the relevance of specifications

Technology & Innovation

How to Achieve a Productive and Efficient R&D

Deepak Murpani, CSO & COO, Andersen Pharma Global

  • Share holder’s / stake holder’s target
  • Generic Market dynamics
  • Expectations from R&D’s in achieving the target
  • Has my Generic R&D kept pace with changing time? 
  • What are good and bad indicators – with examples
  • Short & Long term consequences of inefficient R&D?
  • How can I make my R&D more efficient & productive?

Device Development

Designing for Change: Behaviour Design for (Connected) Medical Devices

Soren Skov, Senior Human Factors Engineer, Roche

  • The challenge of (connected) medical device non-adherence in a home-use setting
  • Introduction to behaviour design and it’s insights from behavioural science
  • Enhancing connected device development with behaviour design principles
  • Drivers and barriers to patient adoption and engagement
Home-use of (connected) medical devices is becoming more prevalent, but adherence and engagement challenges remain a barrier to realizing their full benefit. When patients and other users on-board to a new treatment, they initiate a behaviour change process that is often not recognized during product development. Behaviour design, rooted in behavioural science and addressing the complexities of engagement and behaviour change, offers a valuable toolkit for the development of connected devices. By examining the key drivers of and barriers to short- and long-term adoption, design can be optimized to align with human behavioural tendencies.

2:35 PM - 3:05 PM - Solution Spotlights

Small Molecules

Lactide / Glycolide Polymers for Long-Acting Injectables (LAI’s): Key Chemistry and Processing Considerations for Success

Patrick Duffy, R&D Team Leader, Manufacturing Manager for Bioresorbable Polymers, Ashland

This presentation will start by introducing the benefits of lactide/glycolide based polymer chemistries that are currently utilized for controlled-release implantable and injectable drug products. The session will explore how theoretical polymer chemistry needs to be coupled with formulation knowledge and expertise to lead to commercial success, which includes formulation types such as in-situ forming depots, microspheres, nanoparticles, and pre-formed solid implants.  Ashland will also highlight how customized and novel LG polymer chemistries are used to overcome technical development challenges for respective delivery strategies.


Controlled Release of mAbs and RNA Through a new Parenteral Dosage Form

Christina Schmid, Business Development, Celanese Pharma & Medical

Long-acting controlled delivery of therapeutics continues to hold strong interest to help maximize treatment outcomes across many disease indications.  However, the sustained delivery of biologics and RNA continue to present formulation challenges.  Drug-eluting solid implants are a promising solution to these delivery challenges, providing tunable release of a wide range of drug molecules from small molecules, peptides, monoclonal antibodies to RNA.  

Celanese has developed a polymeric drug delivery implant based on ethylene-vinyl acetate (EVA) for the long-acting delivery of not only small molecules and peptides but also of biologics and RNA. High levels of tunability can be achieved based on various attributes including drug loading, implant design and polymer selection.


  • Applications for use of VitalDose EVA
  • Tunability levers to achieve sustained release
  • Formulation strategies for the release and stability of RNA and mAbs

Technology & Innovation

Overcome Capping Challenges and Increase Tableting Speed in Direct Compression

Dr. Olaf Häusler, Global Pharma Technical Application Specialist, Roquette Pharma Solutions

Tablet capping is a well-known challenge observed during formulation development and manufacturing scale up. It has been studied in detail using mannitol as an excipient model.
  • Review of key criteria leading to capping
  • Design an ultimate excipient to improve tableting productivity
  • Case study comparing commercial mannitol grades

Technology & Innovation

Improving the Odds of Success in Developing Oral Protein Degraders: A CMC Perspective

Steven Winling, Scientific Advisor, Science & Technology, Catalent

Key challenges in developing bifunctional protein degraders are due to their unconventional structures and distinctive physicochemical properties, often requiring enabling formulation technologies to achieve oral bioavailability. This presentation will discuss how developability assessment strategies for protein degraders might differ from typical small molecule development. Catalent’s tailored developability assessment for bifunctional protein degraders can help predict viable formulation and drug delivery strategies for lead drug candidates to reach their true potential. In addition, an array of specialized downstream capabilities and CMC strategy that drives success at each milestone from development to clinics will also be presented.

3:05 PM - 3:55 PM

1-2-1 Pre-Scheduled Meetings & Networking Break

3:55 PM - 4:25 PM - Case Studies

Small Molecules

Use of surrogates as API sparing approach for assessing feeding performance in continuous drug product manufacturing

Ko Cattoor, Senior Scientist, Oral Solids Development, Drug Product Development, Johnson & Johnson

  • Development and application of a surrogate approach at the material feeding stage of continuous drug product manufacturing (CM) is proposed
  • Approach includes demonstrating the surrogate effectiveness at feeder level
In this presentation, the development and application of a surrogate approach at the material feeding stage of continuous drug product manufacturing (CM) is proposed. The approach includes identification of surrogates at the material attribute level and demonstrating the surrogate effectiveness at feeder level. The approach enables to obtain a first insight in feeding performance by a reduced amount of feeding experiments with API and a faster feedback-loop to API development teams.

One of the first steps in a CM development is to characterize APIs for various material property attributes that are deemed impactful for their processing behavior. However, there may be specific failure modes that can only be captured using feeder experiments which require substantial amounts of API that may not be available at early stage.

Four API-Surrogate pairs were identified. Next, feeding experiments of these pairs were carried out on a GEA compact feeder and feeding process data were analyzed between APIs/surrogates pairs.


Protein–Protein Interactions in High-Concentration Monoclonal Antibody Solutions Probed by Quartz Crystal Microbalance?

Tim Diederichs, Post Doc, Boehringer Ingelheim

For a subcutaneous route of administration, high-concentrated monoclonal antibody formulations (>150 mg·mL-1) are required to facilitate patient centricity and patient compliance with respect to self‑administration, reduced hospitalization, and to achieve preferrable pharmacokinetics. However, these high concentrations of monoclonal antibodies (mAbs) results in exponential increases in viscosities, leading to challenges for formulation development, manufacturing, and patient self‑administration. These viscosity increases can be correlated with protein–protein interactions (PPIs), wherefore mAbs are often portrayed as “patchy colloids” with a complex interplay between attractive and repulsive interactions. For dilute mAbs solutions, dynamic and static light scattering (B22 and kD) are normally used to estimate PPIs, whereas viscosity analysis is often used to probe PPIs for concentrated mAbs solutions. Quartz crystal microbalance with dissipation monitoring (QCM‑D) can be applied to determine the viscoelasticity and viscoelastic dispersion (dependence of the complex viscosity on the frequency) of protein solutions. The QCM‑D reports shifts in resonance frequency and half-bandwidth by viscoelastic changes. Thereby, the QCM‑D turned in a viscosimeter, sensitive to relaxation processes on the nanosecond time scale. Especially for complex soft materials like mAbs, which undergo such stress relaxation not feasible for conventional viscosimetry, the QCM‑D is advantageous. Here, we studied the complex high-frequency viscosity of concentrated mAbs solutions with respect to temperature and concentration with lysozyme and bovine serum albumin (BSA) as reference. Viscoelasticity was determined for the mAb, whereas lysozyme and BSA revealed no viscoelasticity and behave like Newtonian fluids. Higher activation energies of viscous flow were derived for the mAb solutions in comparison to water or the reference materials. These differences evidence anisotropic PPIs between the mAb molecules, which do not exist in the same way for lysozyme and BSA. In general, by studying viscoelasticity and anisotropic PPIs, the potential of mAbs to self‑association and aggregation can be estimated to provide essential information for the formulation development of subcutaneous injectables.

Technology & Innovation

Novel Techniques to Manufacture Tailor-Made Pharmaceutical Microparticles

Martin Spörk, Scientific Area Leader, Research Center Pharmaceutical Engineering GmbH

  • Inkjet printing as a tool to manufacture drug particles for early-stage development
  • Processing-induced morphological changes
  • In-vial inkjet printing of proteins
  • Upscaling strategies to form tailored microparticles based on hot-melt extrusion and strand pelletization

New drugs often show poor solubility and handling characteristics. Spray drying has been used as a particle engineering platform enabling the simultaneous improvement of the bioavailability and processability of APIs. As spray drying has several drawbacks, such as inconsistent quality, yield, difficult scale up and the necessity of a large feedstock, the development of manufacturing alternatives is of importance for the pharmaceutical sector. The present talk will outline two novel techniques to manufacture tailored pharmaceutical microparticles. First, inkjet printing was investigated as a particle engineering tool for early development phases where only little material is available. By dispensing a controlled amount of liquid through piezoelectric nozzles on a substrate, consistent quality in terms of size uniformity was achieved. Even for originally highly acicular drugs, spherical particles in the vicinity of 100 µm were printed. Second, a combination of hot-melt extrusion and cold strand pelletization was investigated to continuously manufacture biodegradable microparticles in the range of 250 µm in large quantities for injectable applications. Both manufacturing techniques reveal the potential to tailor the formation of microparticles, accelerating both early development as well as large scale production.

Device Development

Optimising the Interface between Medical Devices and Soft Tissue for Enhanced Therapeutic Delivery

Eoin O’Cearbhaill, Associate Professor of Biomedical Engineering , University College Dublin

The UCD Medical Device Design Group is focused on developing platform medical device technologies, offering smart ways to deliver next-generation therapeutics through minimally invasive approaches. A key aspect to this is research is to optimise the interaction, including penetration and/or adhesion of medical devices to soft tissue. 3D printing is used as an enable technology, due to inherent design freedom that it offers.

The interface between host and implant is a key predictor of device performance. When seeking to attach or integrate medical devices with host soft tissue, current methods of fixation and integration can lead to suboptimal results. There is a reliance on (1) chemical-based adhesives, which require tissue-specific reactive chemistry and subsequent risk of an inflammatory response, or (2) mechanical methods of fixation (sutures or staples) which can induce significant local tissue damage and associated increased risk of infection.

Here, we present examples of novel minimally invasive devices being developed that offer optimal geometries for tissue adhesion and integration that can lead to improved therapeutic delivery.

4:30 PM - 5:00 PM - Case Studies

Small Molecules

Gastrointestinal drug targeting and characterization of a novel esophageal drug delivery system in humans

Werner Weitschies, Ph.D, Professor, Institute of Pharmacy, Department of Biopharmaceutics and Pharmaceutical Technology, University of Greifswald

  • Gastric retention: fact or fiction? Experiences with floating capsules and mucoadhesive mini tablets.
  • Small intestinal drug targeting using gastro-resistant devices: In vivo performance of enteric hard capsules.
  • Targeting to the esophagus: The EsoCap drug delivery system
The targeted delivery of drugs in the gastrointestinal tract remains a major challenge. An important basis for understanding the behavior of drug delivery systems in the gastrointestinal tract is provided by human imaging studies. In the lecture, experiences from imaging in humans using MRI on gastroretentive drug delivery systems and on the behavior of enteric-coated hard capsules will be presented. Furthermore, the innovative EsoCap drug delivery system for targeted drug release to the esophagus will be presented.


Developability assessment of biologics and formulation of novel molecules

Shahid Uddin, Senior Director Formulation/ Stability and Drug product design, Immunocore

  • Importance of assessing developability early in the drug development cycle
  • Close collaborations with research and CMC functions
  • Challenges of assessing stability of low concentration biologics

Technology & Innovation

The Role of Data Quality in the Computational Prediction of Stability Studies

Christos Dimitrakopoulos, Data Scientist, Roche

The stability of biologics at the intended long-term storage condition is a critical factor in their development, but obtaining real-time long-term stability data is a lengthy process. Therefore, Arrhenius-based kinetic modelling and its extensions have been proposed as an alternative for the computational prediction of the degradation rate at the intended long-term storage condition. This presentation will discuss an overview of Arrhenius-based models for predicting stability studies computationally, as well as the potential challenges in the quality of the stability data used as input in these models.

Technology & Innovation

Sudden build up of sub-visible particles over the shelf-life, an early signal of potential Polysorbate degradation issues – Case Study from the industry

Janez Jancar, Senior Expert, Novartis

  • PS degradation and particle formation
  • Understanding the PS degradation pathways
  • Technics to monitor PS degradation.
  • Case study on PS degradation issues and mitigation
Degradation of Polysorbate (PS) in biopharmaceuticals often results in formation of inherent particles over the stability studies or shelf life. During the PS degradation, no matter if the degradation mechanism is driven by oxidation or hydrolysis, free fatty acids (FFAs) are released into the drug product, sooner or later resulting in formation of particles.

Constant monitoring of sub-visible particle count and in-depth understanding of PS degradation mechanisms and solubility behaviour of PS degradation products in complex DP environment can significantly reduce sudden pop up of inherent particles.

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

Device Development

Connected Care: Overcoming Roadblocks and Identifying Opportunities

Kasper Bayer Frøhling, R&D Programme Manager, Medical Device Development, Novo Nordisk

Nélio Drumond, Associate Director, Lead Process Scientist Global Manufacturing Sciences, Drug Product, Takeda

Tonio Hoche, Device Engineer, Roche

Soren Skov, Senior Human Factors Engineer, Roche

Harshal Shah, BioPharma Sector Head, Cambridge Consultants

  • Evolving nature of the medical device due to software, data and connectivity, and how it is changing approaches to device development
  • Patent considerations and responsibilities in data collection and sharing
  • How to define overall responsibility for connected ecosystems when multiple legal manufacturers are involved?
  • How connected medical products can integrate in the wider health management ecosystem
  • How are organisations organized to allow for digital health solutions containing smart devices: Challenges around it.
  • Experiences around data exchange between companies and the care network (patients, suppliers etc.)
  • Future opportunities in designing sensor and biomarker driven closed loop drug delivery systems to enable fully automated dosing decisions

5:45 PM - 5:55 PM

Poster Award: DDF Poster Competition 2023

5:55 PM - 6:00 PM

6:00 PM - 7:00 PM

Evening Drinks Reception

8:00 AM - 8:35 AM

Registration & Refreshments

8:35 AM - 8:40 AM

8:40 AM - 9:10 AM - Keynote

Technology & Innovation

In Silico and Risk Based Approaches to Choice of Formulation- What Works and What Does Not

Kishore Ravuri, Chapter Leader, Drug Product Pharmaceutical Development, Roche

With a surge of candidates in the development portfolio, it becomes increasingly important to take measured approaches to cutshort development timelines to ensure speed to development of new molecular entities.An often considered aspect in this regard is the use of in silico methods to enhance understanding and support decision making. Is it possible to completely do away with formulation development based on in silico approaches? The current presentation evaluates this aspect based on examples and case studies.

9:15 AM - 9:45 AM - Keynote


Advances in Strategies Towards Improving Oral Bioavailability of Peptides and Proteins

Stephen Buckley, Director and Head of Oral Delivery Technology, Novo Nordisk

Peptides such as insulin and glucagon-like peptide-1 (GLP-1) receptor agonists are used in the treatment of type 2 diabetes. The inherent physicochemical properties of these peptides (high molecular weight, enzymatically labile, hydrophilicity, and low permeability) have hampered attempts to deliver peptides via the oral route and necessitated that they be administered by injection. Recently, oral semaglutide, a GLP-1 analog, coformulated with the absorption enhancer sodium N-[8-(2-hydroxybenzoyl) aminocaprylate] (SNAC) in a tablet was approved in the US, EU and Japan, representing the world’s first GLP-1 peptide in a tablet. This coformulation provides unique, site-directed release and absorption in the stomach and effectively surmounts inherent challenges relating to solubility, molecular size, and proteolytic lability to achieve therapeutically relevant plasma exposure of semaglutide. Even more recently, another breakthrough technology exploiting the stomach as a delivery site has emerged – an ingestible self-orienting millimeter-scale applicator (SOMA) that autonomously positions itself to engage with gastric tissue and deploys milliposts fabricated from peptides or proteins directly through the stomach mucosa, which result in plasma levels comparable to those achieved with subcutaneous millipost administration in pre-clinical animal models. This presentation will provide a review of the aforementioned scientific advancements within oral peptide delivery and reflect upon the current and future prospects in this field.

9:50 AM - 10:20 AM - Case Studies

Small Molecules

Glaucoma Medication Dilemma - Turquoise OD, Yellow BID and Purple TID regimen. Why not all OD?

Ajay J Khopade, Vice President R&D Formulations (Non-Oral), Sun Pharmaceutical Industries Ltd.

When glaucoma patients shift from first line to second or third line or a combination therapy, the treatment also increases from one drop to two and three drops per day and mix of these regimens. This creates a turquoise one drop, yellow 2 drops and purple 3 drops dillema. How easy or difficult it is to remember or how it affects patients psyche about his/her disease progression may be debated but it is always helpful if we can make one regimen fit all. Thus, we have a chance to disrupt and change the market every day in order to increase patient and physician satisfaction. We accomplish this through creating and commercialising ophthalmic pharmaceutical candidates to address serious diseases that require novel therapeutic approaches. Recently, our partner announced the filing of a New Drug Application to the FDA for the lead product candidate, PDP-716, which may be the first once-daily brimonidine to be sold to treat glaucoma. A large unmet need for glaucoma patients will, in our opinion, be addressed by the proprietary TearAct delivery system, which offers slow, constant, and sustained release for IOP control throughout the day. Developmental, preclinical and clinical data indicating IOP reduction for ocular hypertension shall be discussed.


Oxidative Degradation of Polysorbate

Johanna Weber, Pharmacist, Martin-Luther-University in Halle

  • What is PS? What does it look like, what is it used for?
  • Where are the problems?
  • What kind of excipients promote oxidation?
  • What can you do to prevent oxidation?
  • PS20 vs. PS80 - who is more prone to oxidation?

Technology & Innovation

3D & 4D Printing in Drug Delivery and Tissue Engineering: From Bench to Patients

Prof Dimitrios Lamprou, Chair of Biofabrication & Advanced Manufacturing, Queen’s University Belfast

The field of additive manufacturing (AM) encompasses a wide range of processes, using various printing technologies, hundreds of materials, and various resolutions and speeds. Techniques such as 3D and 4D printing allow for a patient-center approach, which can be used to create complex drug delivery devices and offer more personalized medical treatments. Innovations in pharmaceutical development and an interdisciplinary approach can lead to new and more effective drug-delivery systems (DDSs), ushering a new era of treatments to various diseases. The introduction of the fourth dimension (4D) has led to an increase in the degree of complexity and customization possibilities. This talk will present new developments in the area by providing examples from our research lab ( in the manufacturing of DDSs and medical devices using innovative AM technologies. The implantable medical devices that manufactured / designed in our lab, have been tested for the delivery of a variety of molecules for the treatment of diversity of diseases, such Cardiovascular, Cancer, Diabetes, Gynaecological, Ocular, and Parkinson’s, among many others. The studies include in-house prepared Bio-inks using natural or synthetic polymers, preparation of drug-loaded filaments by hot-melt extrusion (HME), physicochemical characterisation using state-of-the-art techniques, computational modeling, in vitro, and in vivo evaluation.

Device Development

Special Requirements and Challenges during Ophthalmic Pre-Fillable Syringe Development

Petrick Schneider, Senior Pre-Fillable Syringe Device Engineer, F. Hoffmann-La Roche

  • How do PFS for intravitreal injections differentiate from hypodermic PFS?
  • Specific ophthalmic related requirements that must be considered during PFS development
  • User related requirements for intravitreal injections
  • Challenges and learnings during PFS development

The number of patients that need to be treated due to ophthalmic diseases is increasing continuously. At the same time, the development of a pre-fillable syringe for intravitreal injection brings several specialties and ophthalmic related requirements with it. Besides ensuring particle free injections and an aseptic handling, also the user – in the case of intravitreal injections a health care professional (HCP) – sets certain requirements for those specific syringes. On top of that, the supply chain is more complex than for e.g. subcutaneous products, resulting in longer lead and manufacturing times than for other products.

As there are only few pre-fillable syringes for intravitreal injections commercialized as of today, sharing learnings and experiences is essential to shorten development times in the future.

10:25 AM - 10:55 AM - Solution Spotlights

Small Molecules

Furosemide- How to Make a Leading Drug Better and Its Use Wider

Pieter Muntendam, President & CEO, SQ Innovation Inc.

  • Introducing subcutaneous infusion of a novel formulation of Furosemide
  • A new chapter for one of the most important cardiovascular drugs of our time
  • Reducing the volume of administration for an improved patient experience and lower cost.

Novel formulations are often required for subcutaneous delivery.  Furosemide is poorly soluble at neutral pH. Furosemide Injection USP, the product which has been used for over 50 years for intravenous administration, has a pH of around 9.0.  This is too alkaline for subcutaneous delivery and may case a stinging/burning sensation and skin irritation.  A novel higher concentration formulation with a neutral pH has been developed by SQ Innovation  The higher concentration allows for use of standard 3 mL cartridges combined with a smaller patch pump originally developed for insulin delivery.

The idea may not be new, but our goal is to make it convenient, comfortable, affordable, and ready for widespread adoption.


Scale Up and Manufacturing of Self-Amplifying RNA-LNPs for a COVID-19 Vaccine using the NanoAssemblr® GMP System

Pierrot Harvie, Clinical Manufacturing Manager, Precision NanoSystems

  • A SARS-CoV-2 self-amplifying RNA (saRNA) vaccine was developed using Precision NanoSystems proprietary ionizable lipid and proprietary saRNA.
  • PNI’s lead vaccine composition led to the production of neutralizing and SARS-CoV-2 specific IgG in mice and in Non-human primates.
  • İn a GLP tox study the PNI vaccine candidate was well tolerated with no adverse PNI-vaccine candiate -related effects observed
  • PNI’s Lyophilized saRNA-LNP vaccine candidate retains activity
The recent SARS-CoV-2 pandemic has shown the importance of developing RNA vaccines as well as the importance of cost-effective and timely manner vaccine production to be able to deliver a quick response to the disease outbreaks. Having an effective RNA-based medicine requires a delivery vehicle to bring the nucleic acid into the cytoplasm and protect it from degradation, however, obtaining an efficient delivery to achieve the full potency of RNA therapeutics remains a key challenge. Therefore, Precision NanoSystems has generated a full vaccine development workflow, the Genomic Medicine Toolkit, including self-amplifying mRNA (saRNA) production, a lipid delivery platform where one can use an off-the-shelf ionizable LNP mix or leverage a library of novel ionizable lipids, together with utilizing a scalable manufacturing platform thanks to NxGen™ microfluidic technology. 

SaRNA has emerged as an alternative to traditional mRNA vaccines as its self-replicating feature allows substantially lower doses for effective immune responses.  Additionally, our experts, who are developing a self-amplifying RNA vaccine, are available through our Biopharma Services to support and supplement each step in your development process. Here, we provide examples from our R&D to demonstrate the versatility of the toolbox for the rapid development of vaccines, gene therapies and cell therapies from idea to approved drug product.

Technology & Innovation

Molecular Modelling to Assist Drug Formulation for Small Molecule and Biologic Drugs

John Shelley, Fellow, Schrodinger

  • Complementary use of machine learning and physics-based modeling contribute to the drug development and formulation process
  • Polymorph prediction for small molecule drugs
  • API and excipient physical and chemical property prediction
  • Molecular modelling provides a basic understanding of the structure and behaviour of drugs as formulated that compliments experimental data and informs decision making in drug formulation
Selecting and combining the right ingredients in the right manner to obtain optimal properties are essential for successful drug formulation given the inherent challenges and a competitive market. With advances in modern machine learning, physics-based simulation techniques and computer hardware, 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 Suite for modeling either small-molecule drugs or biologics for the development process.  For small-molecule drugs workflows have been created for characterizing crystal polymorphs, crystal morphology and degradation risks as well as calculating elastic constants (bulk modulus, shear modulus, etc.), powder diffraction patterns, glass transition temperatures (Tg), diffusion constants, pKa values, melting points, water adsorption and solubility. For biologics our toolset supports homology modeling, and the calculation of aggregation propensity, titration curves, isoelectric points and viscosity among other things.  Complex and evolving structures, often in fluid states, play a crucial role in the pharmaceutical industry.   For both small-molecule and biologics formulations powerful simulation tools using atomistic or 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).

Device Development

Key Considerations for the Selection of a Truly Flexible Large Volume Delivery Device Platform

Thomas Mayer, Business Unit Manager, Sonceboz SA

  • Delivery pathway updates from IV to SC are in increasing trend, fueled by the need for decentralized care
  • Such updates come with challenges such as high delivery volumes and primary packaging considerations
  • Especially during early phases, the final dose is unknown which requires built-in device flexibility
  • >90% of large volume biologic drugs are filled in vials, shouldn’t the device be compatible?
  • Self-Injection will be increasingly important in the future, do you have the right toolkit?

10:55 AM - 11:45 AM

1-2-1 Pre-Scheduled Meetings & Networking Break

11:50 AM - 12:20 PM - Case Studies

Small Molecules

Looking Through the Magnifying Glass – Advanced Characterization of ASD Tablets

Tatiana Marcozzi, Drug Product Development Scientist - Oral Solid Dosage , Janssen

  • Innovative imaging techniques combined with machine learning are proposed as a tool to increase intermediates and final drug product understanding.
  • A case study is presented in which MFV and XRM are applied to characterize tablet internal structure: tablet porosity, pore size, pore distribution, and tablet tortuosity  
Tablets are the most widespread pharmaceutical dosage forms. In the framework of quality by design and increasing intermediates and drug product understanding, imaging techniques such as Mosaic Field of View (MFV) and X-Ray Microscopy (XRM) in combination with advanced machine learning allows for a more in-depth tablet characterization.

A case study is presented in which MFV and XRM were applied to support screening of different ASD tablet concepts. Results show that these techniques can be useful to evaluate tablet homogeneity, tablet porosity and pore structure, as well as tablet tortuosity. Moreover, a good correlation between tablet disintegration time and tablet porosity quantified via tablet imaging is proven.


Nucleotide Delivery through Novel Biodegradable and Bioresponsive Polymers (biodynamers)

Dr. Sangeun Lee, Head of Department Drug Delivery and Biological Barriers, HIPS Helmholtz Institute for Pharmaceutical Research Saarland

Novel polymers called proteoid biodynamers have been developed as transfection agents. This pH-responsive biopolymer is composed of amino acid derivatives that are connected by dynamic covalent bonds. Under acidic aqueous conditions, the biodynamers undergo dynamic polymerization due to the pH-responsive bonds on their backbone. This biodynamer series possesses several advantageous characteristics for biological applications; pH-responsive degradation, the ability to self-fold into a nanorod structure with an ethyleneglycol shell, biocompatibility, and easy tunability through changes in amino acid composition. Taking their advantages, we have successfully demonstrated their application in delivering mRNA and siRNA. Specifically, positively charged biodynamers such as Lys-, His-, Arg-, and KHR-biodynamers were utilized to form nano-sized polyplexes with mRNA or siRNA, referred to as dynaplexes (DPs). The formed DPs proved to be highly effective in transfecting mRNA/siRNA, exhibiting significantly greater efficacy (2.7-fold increase for mRNA and 2.2-fold increase for siRNA in cell population) compared to commercially available transfection agents. These results highlight the promising potential of biodynamers as safe and pH-responsive vectors for gene delivery.

Technology & Innovation

3D Printing Technologies at the Point of Care

Dennis Douroumis, Professor in Pharmaceutical Technology and Process Engineering, University of Greenwich

We are living in a new era where technological advances in as 3D printing (3DP), have now become reality and create new opportunities to improve patient’s life and ensure the quality and safety of treatments. The concept of producing customised medicines at the point of care (PoC) is expected to revolutionised pharmaceutical manufacturing and supply chain but most importantly the patient’s wellbeing. There is a range of suitable technologies that can be implemented at the PoC but there are several hurdles to overcome including processing, quality assurance and regulatory matters

Device Development

Usability Engineering Strategy of developing autoinjector with two different indications

Yu-Ting Lin, Senior Usability Engineer, Merck KGaA

  • Core idea of co-development of the device hardware  
  • Common features and distinct user interface development and associated evaluation strategy
  • Development and design control phase arrangement
  • Key challenge and learning  
Product development takes years from design ideation to product launch. The co-development of a core device to be applied with two different therapeutic areas enable us to shorten the development timeline and utilize resources efficiently. In consideration of distinct user group and their needs, two distinct firmware and User Interface were developed to fit into two indications. In this session, the topics of core idea of co-development, the challenges, and strategies of user interface design, the QMS considerations will be presented.

12:25 PM - 12:55 PM - Solution Spotlights

Small Molecules

Accelerate First-in-Human Studies through Integrated CMC Platform and Solid State Development

Dr. Santipharp Panmai, Vice President and Head of Early Phase Formulation R&D, WuXi STA

This presentation focuses on acceleration of early phase formulation development through the use of integrated CMC platform as well as solid state development. The success of any new drug development starts in its early phase. The probability of success of a molecule increases dramatically after it passes first-in-human (FIH) and proof-of-concept (POC) studies. Building a comprehensive understanding of the molecule is crucial to successful early phase development. API salt form and formulation selections are also key factors to successful phase I studies. Effective CMC strategies can significantly enhance the speed of early phase development, and ultimately chance for success in late-stage development. Solid state studies not only provide fundamental understanding of a molecule, but also serve as a platform that leverages the knowledge from API development and manufacturing, which can further enhance the speed of early-phase development. With more and more new chemical entities being poorly soluble, having a comprehensive bioavailability enhancement toolbox and strategies is critical to success in the early phase. Case studies covering solid state development and early-stage formulations will be provided.


Small Molecules to Large Biological Entities: Biodegradable DelSiTech Silica Matrix Technology for Controlled and Long-Acting Release for Ophthalmic and Sub-Cutaneous Administration

Frederic Dargelas, Head of Business Development and Alliance Management , DelSiTech Ltd.

DelSiTech Silica Matrix is highly versatile delivery platform. The technology is not only suitable for any molecules from small molecule to large biological drugs but applicable for multiple administration routes as well. Besides subcutaneous administration DelSiTech Silica Matrix can remarkably be utilized for both, topical ophthalmic and intravitreal delivery.

Technology & Innovation

From Powder to Particles: Engineering Particles for Inhalation via Jet Milling

Beatriz Fernandes, Senior Principal Scientist, R&D, Lonza

Jet milling is a well-established particle engineering technology for producing micronized powders with controlled particle size distribution and increased surface area, making them suitable for drug delivery via inhalation. However, successful implementation of jet milling requires a robust Quality by Design (QbD) approach to ensure the critical quality attributes of the micronized particles are maintained throughout process development.

This presentation will cover the principles and intricacies of the jet milling process. It will also highlight challenges when micronizing for inhalation delivery, and their possible solutions. A case study will demonstrate a reliable process development strategy for micronizing nilotinib, a drug with potential in severe chronic asthma. It will showcase the benefits of implementing a QbD approach.

  • This presentation aims to provide an in-depth understanding of the jet milling process, and the significance of implementing a Quality by Design approach.
  • It will address the intricacies of developing micronization processes as a particle engineering technology for inhalation.
  • A case study will be presented to illustrate a reliable process development strategy that ensures all quality-critical attributes are maintained.

Technology & Innovation

The High Drug Load Delivery System Enabling the Next Generation Long-Acting Injectables Through Atomic Layer Precision

Joel Hellrup, Head of Pharmaceutical R&D, Nanexa AB

Sustained drug release can improve the efficacy, reduce side effects, and improve patient compliance of many drugs. PharmaShell® offers a versatile drug delivery system with a high drug load leading to convenient administration that reduces burden for patients and health care providers. PharmaShell® has an innovative mechanism of action that allows for subcutaneous or local controlled release of small molecules, peptides, and proteins. The release profile can be tailored using atomic layer precision to apply a controlled release coating onto microparticles of the active pharmaceutical ingredient. The PharmaShell® technology is currently explored in phase I clinical trials. In this talk, examples of what can be achieved with the technology will be presented varying from coated monoclonal antibodies and peptides to clinical data on the highly soluble azacitidine.

12:55 PM - 1:55 PM

Networking Lunch

1:55 PM - 2:25 PM - Case Studies

Technology & Innovation

Current Status Upon Polysorbate Degradation

Tamara Phan, Post Doc at Pharmaceutical Development Biologicals, Boehringer Ingelheim

Polysorbates (PS) are the most applied non-ionic surfactants in biopharmaceutical formulation due to their non-toxic character and high efficiency in protein stabilization. However, PSs undergo chemical and enzyme-mediated hydrolysis and oxidation.

Hydrolysis of PSs is characterized by the cleavage of the ester bond between the fatty acid moiety and the polyoxyethylene (POE) chain, releasing POE units, as well as free fatty acids (FFAs) as major degradation products. While the chemical hydrolysis of PS is catalysed by acidic or alkaline pHs and is almost negligible under relevant pharmaceutical conditions, the hydrolysis induced by the host cell protein (HCP) traces is more difficult to avoid. Despite the best efforts for their complete removal, HCPs often linger in very small amounts in the purified therapeutic protein and thus, detection and control of residual HCPs are highly challenging.

In contrast to hydrolysis, exposure to air, light and transition metals are known factors to cause oxidation at the PS ethylene oxide moieties and unsaturated fatty acids resulting in peroxides, alkenes, aldehydes, ketones, FFAs. PS degradation products mediate the formation of sub-visible and visible particles or may oxidize the therapeutic protein under long term storage and thus, threatening the formulation integrity.

Hence, analytical approaches that monitor PS content, PS subspecies and PS degradants has been established, e.g. fluorescence micelle assay (FMA) and reverse-phase high performance liquid chromatography (RP-HPLC) coupled with a single quadruple mass (QDa) detector to understand the causes and mechanisms of PS degradation. Additionally, pharmaceutical industries use various PS degradation mitigation strategies e.g. adding antioxidants, changing storage conditions, or testing alternative surfactants.

This talk will give an overview of the current understanding, monitoring, prediction, and mitigation of PS degradation pathways


Oleogels for the Nose-to-Brain Delivery of Oligonucleotides

Dr Vivek Trivedi, Senior Lecturer in Drug Delivery, University of Kent

Nasal administration of therapeutics is advantageous due to its ability to provide rapid absorption of active pharmaceutical ingredients from the nasal mucosa to blood circulation for local and systemic therapeutic effects. This route of drug delivery also avoids first-pass metabolism, resulting in higher bioavailability and direct delivery of active molecules to the brain via olfactory and trigeminal nerve pathways. The brain is majorly sheltered by the cerebrospinal fluid (CSF), the CSF-blood barrier, and the blood-brain barrier (BBB). The non-fenestrated capillaries in the endothelial cells of the BBB form tight junctions which becomes an extensive hindrance for molecules like ASO and other neuro-therapeutics as the paracellular pathway gets hard to cross. This work entails developing a mucoadhesive oleogel capable of potentially delivering the drug to the brain by avoiding BBB. The cell uptake study using Calu-3 cell lines suggested that ASO uptake from the oleogel was time-dependent and increased over 72 hours, demonstrating a slowed/protected release when compared to naked ASO over the same period.

Technology & Innovation

Innovative Carrier Design and Engineering for Dry Powder Inhalation

Amrit Paudel, Associate Professor, Graz University of Technology

  • Engineering of lactose and mannitol as carriers for improved drug dose delivery via carrier-based dry powder inhalation (DPI)
  • Innovative particle engineering approaches (eg. spray drying, spray congealing, ink-jet printing, and wet sieving) for the DPI carrier design and fine-tuning
  • Interrelation between DPI carrier properties and their downstream processability as well as aerosolization performance


Surface Patches Control Antibody Specificity

Hannes Ausserwöger, Doctoral Student, University of Cambridge

  • Outline of current understanding of non-specificity of antibodies in literature
  • Highlighting the role of surface patches in driving non-specificity
  • Surface patch libraries specifically alter non-specificity
  • Correlating surface patch properties with non-specificity affinities and phase separation

The susceptibility of antibodies to bind unwanted off-targets is a key limitation in the development of therapeutic antibodies. Although the mechanisms have yet to be resolved, such unwanted interactions are linked to aberrant assembly processes, which can impact storage and administration as well as the potency of antibodies. In order to better understand this problem, we highlight the role of surface patches  — clusters of surface-exposed amino acid residues with similar physicochemical properties — as inducers of nonspecific interactions. We quantify these nonspecific interactions across a designer library to correlate them with the protein surface properties, and link nonspecific off-target interactions to the propensity of antibodies to undergo heteromolecular phase separation. We show that both phenomena are governed by the nature and size of surface patches and demonstrate that modulations in surface patches can vastly change nonspecific binding as well as macroscopic behavior as manifested by phase separation. Hence, surface patches collectively drive unwanted non-specific interactions and assembly processes limiting the specificity and developmental potential of these highly potent molecules.

2:30 PM - 3:15 PM - Panel Discussion

Technology & Innovation

How will the FDA PRIME Pilot Program Support Development of “new” Excipients

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

Heinrich Haas, Vice President Formulation & Drug Delivery Formulation & Drug Delivery, BioNTech

Janez Jancar, Senior Expert, Novartis

Martin Huelsmeyer, Head of Science Affairs & Strategic Initiatives NBE Drug Product Development, AbbVie

Nick DiFranco, Global Market Segment Manager for Oral Treatments, Lubrizol Life Science Health

Verena Geiselhart, Pharmacist, BASF

  • The need for excipient innovation and the challenges for the adoption of novel excipients in drug development
  • Expected uses of the PRIME program, once finalized and it’s potential impact on innovation in drug and novel excipient development
  • The need to globalize and harmonize this type of approach to novel excipients to maximize benefits
  • Why is the pharmaceutical industry is sticking to Polysorbates (PS), even though we know Polysorbates are a complex molecule and keen to degradation

3:15 PM - 3:20 PM