Programme 2023

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

7:45 AM - 8:45 AM

Registration & Refreshments

8:45 AM - 8:50 AM

Chair's Opening Remarks

8:50 AM - 9:20 AM - 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, 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.

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.

Biologics

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

Artificial Intelligence & Machine Learning Applications in Drug Formulation and Delivery

Olivier Brass, Principal Scientist– Vaccines, Sanofi

Device Development

Device Regulatory Strategies for Drug Device Combination Products

Louise Place, Director, CMC RA Devices, GSK

  • Discuss the latest requirements for Drug Device Combination Products in EU and UK
  • Practical reflections on implementation of the EU MDR for injectable products
  • Important Considerations for making changes to established products

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.

Biologics

Solution Spotlight by BASF

Technology & Innovation

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

Joey Glassco, Senior Global Market Manager for Parenteral Drug Delivery, Lubrizol Life Science Health

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

Solution Spotlight by Cambridge Consultants

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

Biologics

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

Bioavailability of Actives with a Case Study of CBD

Device Development

Topic TBC (human factors engineering in device/combo product development)

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

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

Small Molecules

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.

Biologics

Novel Developments in the Nucleic Acid-Based Drugs

Manfred Ogris, Professor for Pharmaceutical Sciences, University of Vienna

Technology & Innovation

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

Christian Hug, Senior Scientist, Novartis Institutes for Biomedical Research

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

Solution Spotlight by Quotient Sciences

Biologics

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

Solution Spotlight by Nanoform

Device Development

Solution Spotlight by EdgeOne Medical

1:05 PM - 2:05 PM

Networking Lunch

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

Biologics

Formulation Development and In Vitro Release Characterization of Oral Modified Release (MR) Formulation

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.

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

Digital Inhalers Case Study & Lessons from Aircraft Crashes!

Mark Milton-Edwards, Head of Digital Health Solutions , TEVA

For decades technology and humans have studied 'air crashes' to make the odds of catastrophic outcomes very low (1 in 70million). We can learn in terms of technology and systems thinking, as well as ambition from outside pharma. Asthma and COPD have a major burden on individuals and society. Digital technologies have a unique and significant role in society and hence potential in human health. Digital human signals can be used together with machine learning to predict attacks (aka 'air crashes').
  • Lessons can be learned from outside pharmaceuticals
  • Focus on an enduring need of patients/HCPs/Payers i.e. society
  • Ensure real-world user design focus
  • Validation and proof with RCTs is essential but also we can move forward to utilizing the 'cloud data' to give a novel real-world perspective and value

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

Small Molecules

Solution Spotlight by Adare

Biologics

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

Solution Spotlight by Hovione

Device Development

3:10 PM - 4:00 PM

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

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

Small Molecules

Formulation and Process Innovations 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.

Biologics

Development of New Methods for Characterization of Antibody Self-Association and Non-Specificity

Nikolai Lorenzen, Principal Scientist, Novo Nordisk

  • Weak interactions of antibodies with other molecules and interphases in complex mixtures (non-specific binding) and with themselves in formulation (self-association) remain to be a critical developability parameter
  • In collaboration with University of Cambridge and ETH Zürich we are working on developing new methodologies to characterize non-specific binding and self-association
  • FFF as a new methodology for screening of self-association
  • Advanced microfluidic analysis of non-specific interactions

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

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.

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

Small Molecules

Optimization of ASD Formulations for Improved Performance

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

Biologics

Formulation Development For A Human Plasma Protein – Use of DoE & 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

Formulation Environmental Decision Tool (FEDT) or 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.

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.

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 - 4:05 PM - Panel Discussion

Technology & Innovation

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

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

5:50 PM - 6:00 PM

Chair’s Closing Remarks

6:00 PM - 7:00 PM

Evening Networking Reception

8:00 AM - 8:45 AM

Registration & Refreshments

8:45 AM - 8:50 AM

Chair's Opening Remarks

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.

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.

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.

Biologics

Solution Spotlight by InnoCore Pharmaceuticals

Technology & Innovation

Solution Spotlight by Sever Pharma Solutions

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.

Biologics

Spray Drying of Lipid Nanoparticles Enables Intratracheal Delivery of mRNA

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

  • Diving into the 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

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?

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

Small Molecules

Investigation of the Impact of Coating Imperfections on Tablet Performance

Elise Vaes, Scientist OSD, Janssen

  • Effect of physicochemical properties of API and excipients
  • Implications of disproportionation on oral bioavailability
  • Alternative formulation approaches

Biologics

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

Camille Dagallier, Senior Formulation Scientist, Sanofi

Technology & Innovation

How to Achieve a Productive and Efficient R&D

Deepak Murpani, CSO, Andersen Pharma Global

  • Abstract TBC

Device Development

Usability Engineering Strategy of Developing Autoinjector with Two Different Indications

Yu-Ting Lin, Senior Usability Engineer Company: , Merck KGaA

  • Core idea of co-development
  • Usability Engineering Strategy
  • Common and individual features
  • Key challenge and learning  

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

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Biologics

Solution Spotlight by Samsung Biologics

Technology & Innovation

Solution Spotlight by Croda Pharma

Device Development

Solution Spotlight By Midas Pharma

1:00 PM - 2:00 PM

Networking Lunch

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

Small Molecules

Amorphous Solid Dispersions: Current Perspectives, Challenges and 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.

Biologics

Development Strategy of Ultra-High Concentrated Biopharma Products

Martin Huelsmeyer, Senior Principal Scientist, Scientific Affairs & Strategic Initiatives

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

Injectable Drug Formulation Depots with Sustained Release

  • Reducing relapse rates amongst all patients including non-complying patients
  • Achieving sustained release drugs through physiochemical alteration

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

Biologics

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.

KEY LEARNINGS:

  • 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

Solution Spotlight by Roquette

Technology & Innovation

Solution Spotlight By Catalent

3:05 PM - 3:55 PM

Networking Break

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

Small Molecules

Modelling and Control of Fluid Bed Granulation Processes

  • Fluidized bed processes are a complex equilibrium between thermodynamics, hydrodynamics and atomization.
  • A simple model based on mass and heat balances on the equipment is proposed to find the perfect alchemy to control granulation, coating and drying processes in Fluidized bed.

Biologics

Technology & Innovation

Using Nanotechnology to Better Target Therapies

  • Biological properties of nanoparticles
  • Use of nanoparticles to cross biological barriers

Device Development

Microneedle use for Vaccine Delivery

  • Microneedle design and construction
  • Vaccine drying and stabilisation
  • Future outlooks

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

Small Molecules

Trojan Horse Formulations for Tumour Drug Delivery

  • Improving stability of microparticle internalised chemotherapy
  • Potential to minimise systemic toxicity with new formulations

Biologics

Developability Assessment of Biologics and Formulation of Novel Molecules

Shahid Uddin, Director of Formulation & Stability, Immunocore

  • Abstract TBC

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.

Device Development

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

Felix Weiland, Head of Device Technology, Boehringer Ingelheim

  • Soft Mist Inhalation
  • Respimat
  • Sustainability

5:00 PM - 5:30 PM - Keynote

Technology & Innovation

Understanding the unknows in Drug Discovery and Development

Ramesh Panchagnula, Managing Director, Nektar Therapeutics

5:30 PM - 5:40 PM

Poster Award: DDF Poster Competition 2023

5:40 PM - 5:45 PM

Chair’s Closing Remarks

5:45 PM - 6:45 PM

Evening Drinks Reception

7:00 PM - 8:00 PM

Speaker's Dinner

8:30 AM - 8:55 AM

Registration & Refreshments

8:55 AM - 9:00 AM

Chair’s Opening Remarks

9:00 AM - 9:45 AM - Panel Discussion

Device Development

Reducing the Cost of Developing Medical Devices

  • Blending multiple ideas with streamlined management to innovate more effectively
  • How efficient is current device and combination products R&D
  • Accelerating product design and testing to reduce R+D cost of devices and combination products

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.

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 (www.lamproulab.com) 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 Engineer, 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.

Biologics

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 with the support of the Canadian Government, 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

Solution Spotlight By Sonceboz

10:55 AM - 11:45 AM

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

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

Small Molecules

Application of API Surrogate Approach for Continuous Manufacturing Process Development

Biologics

Device Development

Wearable Insulin Delivery Devices

  • Sweat based glucose monitoring
  • Closed loop systems for glucose measurement and dosage setting
  • Data collection and deep learning

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

Small Molecules

Solution Spotlight by WuXi STA

Biologics

Solution Spotlight by DelSiTech

Technology & Innovation

Solution Spotlight by Lonza

Device Development

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

Small Molecules

Oral Drug Delivery Strategies for Development of Poorly Water Soluble Drugs in Paediatric Patient Population

Carsten Timpe, Technical R&D Expert Scientist, Fmr. Roche

  • Challenges to develop paediatric drugs for poorly soluble drugs (technical, safety aspects)
  • Overview about different technical approaches to enhance drug solubilization
  • Promising approaches to administer poorly soluble drugs to children
  • Examples of marketed products
  • Wrap-up and outlook

 
Oral drug delivery strategies for development of poorly water soluble drugs in paediatric patient populations
Developing appropriate formulations for paediatric populations is an essential task for the formulation scientist and could represent a significant challenge for poorly water soluble drugs (PWSDs). This requires selection of the most appropriate solubility-enabling technology, the choice of the most suitable and safe excipients and further considerations about the drug administration, including addition of medical devices and specific human factor risk assessments for patients and caregivers.
Different approaches and drug delivery strategies are nowadays available to select a suitable approach and guide formulation scientists for development of such formulations in adult patient populations and recently advances have been made also for paediatric patients in that field.
The purpose of this presentation is to give participants a broad overview about different drug solubilization technologies and the opportunities, limitations and challenges of these for developing paediatric formulations.
A few marketed examples will further help to illustrate these drug delivery approaches

 

 

 

 

 

 

Biologics

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

Device Development

Development of Inhalers That Guide Patient Technique

  • Current issues with improper use
  • Medication loss
  • Programming smart inhalers to inform patients of medication adherence

2:30 PM - 3:00 PM - Keynote

Technology & Innovation

Future Opportunities for New Modalities

  • Innovation through new modalities
  • Antibody drug conjugates, Nucleic acid delivery, AAV, Cell therapy, Immune targeting Nanomedicine
  • mRNA therapeutics and delivery hurdles
  • Future outlooks

3:05 PM - 3:10 PM

Chair’s Closing Remarks