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

Technology & Innovation

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

Engineering Prodrug Therapies For Infectious Disease and Cancer Therapy

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

  • Therapeutic platform targets 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
  • Platform can be engineered to extend the PK profile of small molecule drug regulators of mRNA gene circuits or cell therapeutic regulatory circuits
  • Liver-targeting of drugs has also been shown to hepatocytes to increase therapeutic index via IV or subcutaneous routes of administration
  • Tumor-associated macrophages and antigen-presenting cells can be targeted with adjuvanting immune-modulators for use in immune-therapies and vaccines
A polymeric prodrug platform has been developed for infectious disease and cancer therapeutics.  The “drugamers” can be designed to target the macrophage reservoir in organs and tumors, and with controlled and extended pharmaco-kinetic profiles. The polymeric prodrugs have been initially developed against pulmonary infections, where they have shown excellent activity against highly lethal bacterial disease models. 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.   Finally, the polymeric prodrugs are also showing promise as a new long-acting depot product for antiviral therapies, including HIV prophylaxis. Recent advances across these applications will be covered.

Biologics

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.

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

Delivery of Poorly Soluble Drugs to Pediatric Populations - Opportunities and Challenges

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

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

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

Solution Spotlight by LTS

Technology & Innovation

Solution Spotlight by Hovione

Device Development

Solution Spotlight By TBC

3:10 PM - 4:00 PM

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

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

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

QbD and Process Scale Up

  • Opportunities for continuous manufacturing of biologics
  • Current regulatory guidelines
  • Formulating drugs with manufacturing in mind

Technology & Innovation

Exploring the use of the more Advanced Approaches to DSc Analysis

• Complementary orthogonal methods
• Optimal formulations and their overall developability

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 - 5:50 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

  • TBC

Technology & Innovation

Advances in Supersaturating Drug Delivery Systems

  • What are Supersaturating Drug Delivery Systems (SDDS)?
  • Advances in developing and characterizing SDDS?
  • Complexity in in-vitro and in-silico characterization of SDDS?
  • Benefits and limitations of SDDS characterization tools and methods   

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

Solution Spotlight By TBC

10:30 AM - 11:20 AM

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

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

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

Combination Treatment of Multidrug Resistant Tumours with Stimuli-sensitive Nanocarriers Co-Loaded with siRNA and Drugs

  • Combination of siRNA and drug can be used to treat multidrug resistant tumours
  • Drug and siRNA can be loaded on the same delivery nanosystem
  • Such co-loaded nanopreparation could be made stimuli-sensitive
  • Nanopreparations can also target intracellular organelles     

Device Development

Implementing Human Factors Engineering and User-Centered Design: Practical Guidance for Combination Products

  • Introduction to human factors engineering and user-centered design
  • Suggestions for involving users throughout the development process
  • Tips for planning and implementing a human factors engineering approach optimized for combination products
  • Best practices for meeting regulatory expectations for human factors engineering

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

In What Direction is the Drug Delivery Technology Space Moving?

  • Evaluating how fast the delivery technology field has evolved until now – are there limiting factors to its continued development?
  • What is the potential of less-traditional delivery routes such as nasal and ocular delivery?
  • Discussing the future of evolving areas including polymer technologies, auto injectors, microneedles etc.
  • Looking retrospectively, has the industry done all it can until now to maintain the R&D paradigm?

Device Development

Challenges and Opportunities in Combination Product Design, Development and Use

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

Biologics

Solution Spotlight by Samsung Biologics

Technology & Innovation

Solution Spotlight by Croda Pharma

Device Development

Solution Spotlight By TBC

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

Novel CART Cell Therapy Based Tumour Treatments

  • Challenges and solutions to finding correct antigens
  • Overcoming hostile environment associated with tumours

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

Solution Spotlight by 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

Chemistry, Manufacturing and Controls (CMC) Strategies for Cell Therapies

  • Efficient and safe T-Cell Processing for CAR-T therapies
  • Maintaining stability testing standards in compressed development timeframes

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.

Biologics

CMC Considerations for Cell Therapy Formulations

  • CAR-T therapies processing best practices
  • Maintaining stability testing standards in tight timeframes

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

Solution Spotlight by Ligand

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 TBC

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

Technology & Innovation

CNS Drug Delivery and Barrier Modelling

  • Overview of modelling brain barriers methods
  • Understanding brain endothelium
  • Overcoming brain barrier

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 TBC

Technology & Innovation

Solution Spotlight by Lonza

Device Development

Solution Spotlight by TBC

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

Tumour Targeting through Novel CART Cell Therapies

  • Challenges and opportunities to finding the right antigens
  • Overcoming hostile environments found with tumours

Technology & Innovation

Injectables Formulation for Sustained Release

  • Reducing relapse rates
  • Physiochemical alteration

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