Programme 2020

December 14-15, 2020 | All times in CT

8:00 AM - 8:45 AM

Registration

8:45 AM - 8:50 AM

Chair’s Opening Remarks

8:50 AM - 9:25 AM - Keynote

Small Molecules

Bespoke Dosing for Pediatric Patients

Rick Panicucci, Senior Vice President CMC, QED Therapeutics

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

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

Small Molecules

In-Silico Excipient Screening for Pharmaceutical Formulations

Gabriele Sadowski, Professor for Thermodynamics, TU Dortmund University

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

Technology & Innovation

Dendritic Nanoparticles for Enhanced Immune Checkpoint Blockade

Seungpyo Hong, Milton J. Henrichs Endowed Chair Professor, University of Wisconsin-Madison

Dendritic polymers have drawn considerable attention to be used as a nanoscale platform for various biomedical applications over the past few decades.  Primary advantages of dendrimers include their unique properties to serve as one of the ideal mediators for multivalent binding effect that often results in binding kinetics enhancement by orders of magnitude.  This presentation will focus on our recent efforts on poly(amidoamine) (PAMAM) dendrimers to be engineered as a versatile platform for various immune checkpoint inhibitors (ICIs) to improve their immunotherapy efficacy.  In particular, we have recently prepared dendrimer-based antagonists against programmed cell death protein 1 (PD-1)/programmed death-ligand 1 (PD-L1) interactions that are known as a common pathway of tumor cells evading from immune attack. Generation 7 (G7) PAMAM dendrimers were employed as a platform to accommodate multiple antibodies against PD-L1 (aPD-L1) or PD-L1-binding peptides, followed by characterization, binding kinetics measurements, in vitro cell assays, and in vivo tests.  The three independent binding measurements using surface plasmon resonance (SPR), bio-layer interferometry (BLI), and atomic force microscopy (AFM) all revealed that the dendrimer conjugates exhibited a significantly greater binding kinetics than their free counterparts (either aPD-L1 or PD-L1-binding peptides), by up to five orders of magnitude.  Such enhancement in binding strength was likely achieved through the multivalent binding effect mediated by dendrimers and surface stabilization of the peptide structure.  The enhanced binding was directly translated into significantly improved in vitro efficiency where a significant increase in IL-2 secretion and a reduction in chemoresistance of tumor cells were observed.  Finally, our in vivo studies using mouse models indicated that the dendrimer conjugates selectively accumulated to the tumor with much longer retention than free antibodies. The enhanced biodistribution, tumor selectivity, and effective PD-1/PD-L1 blockade of the dendrimer conjugates all contributed to a significantly improved therapeutic efficacy of the nanoparticles against 4T1-xenograft Balb/c syngeneic mice.  Our results collectively provide a strong proof-of-concept indicating that the dendrimer-based system would serve as a platform for various ICIs that effectively block the immune checkpoint pathways, thereby improving efficacy of cancer immunotherapy.

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

Small Molecules

The Role of Biopharmaceutics in Early Drug Development

Chris Roe, Senior Research Fellow , Quotient Sciences

Nominated candidates entering clinical development often have sub-optimal physicochemical, biopharmaceutic or DMPK properties for oral delivery. Development teams are challenged with how to understand the properties of new drug candidates, how to design the appropriate formulation strategy and how to move quickly and successfully into early phase clinical trials. Along the way, it is important to identify developability risks and take steps to mitigate these factors, balanced carefully against time and cost investments.

This webinar discusses effective strategies being used by biotech and pharmaceutical companies to overcome biopharmaceutic challenges for small molecules in today’s drug development pipeline; and explores alternative approaches for accelerating your early development plan.

Learn about case studies relating to:

• Poor solubility molecules
• Drugs with short half-lives
• Preclinical to clinical translation

Device Development

Considerations in Primary Packaging Component Selection for Parenteral Drug Delivery

Eugene Polini, Technical Key Account Manager, Datwyler

  • Present the key considerations in selecting the appropriate parenteral packaging components.
  • Introduce solutions for manufacturing high-quality packaging components to meet the needs of the pharmaceutical industry.
  • Discuss risks that face the pharmaceutical industry today from a drug packaging perspective, and strategies to mitigate these risks.


The component selection process is a critical step in developing safe and effective primary packaging for pharmaceutical and biotech drugs. Considerations in component selection may involve evaluating a variety of elastomer compounds, assessing the need for additional protection through elastomer coatings, and establishing the best final seal, if applicable, to ensure the packaging maintains the drug product integrity.

10:45 AM - 11:35 AM

iSolve & Networking Break

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

Small Molecules

Towards Building in Patient Centricity in the Design and Funnelling of Molecules for Clinical Development

Ahmad Sheikh, Senior Director, ACOS Senior Research Fellow , AbbVie

The presentation is going to focus on development and deployment of in-silico modelling tools augmented with data rich high-throughput experimentation approaches across the lead-optimization to candidate selection continuum. Both physics based and ML/QSAR type models will be discussed along with the collaborative webs necessary to facilitate efficient decision making within flexible workflows

Biologics

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

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

Show more

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

Small Molecules

Shape of Innovation: Extrusion Technologies for a Wide Range of Pharmaceutical Dosage Forms

Tony Listro, Vice President, Technology , Foster Delivery Science

Extrusion is a polymer processing technology that is used in the solubilization technology known as Hot Melt Extrusion (HME) and a manufacturing technology to produce Long Acting Implants (LAIs). HME is an attractive processing technique to enhance the solubility of poorly water soluble drugs to enhance their bioavailability after oral administration. These crystalline hydrophobic drugs can be dispersed in hydrophilic polymers using HME to manufacture amorphous solid dispersions to improve their solubility and dissolution rate. Extrusion is increasingly used to produce LAIs varying in size, shape and release kinetics. This presentation will provide an overview of extrusion technology and review case studies of both HME and LAIs.

Technology & Innovation

In Vivo-Predictive In Vitro Methods for Characterizing Amorphous Solid Dispersions (ASDs)

David K. Lyon, Senior Fellow, Global R&D, Lonza Pharma & Biotech

A significant fraction of pharma and biotech pipelines is comprised of poorly soluble and, hence, poorly bioavailable compounds.  Amorphous solid dispersions (ASDs) are a proven technology that have been commercialized in over twenty products.  Key to progressing ASDs is the use of rapid, bulk sparing methods to characterize ASD performance and identify commercially relevant formulations early and avoid re-formulation later in the project lifecycle.  This talk will focus on identification of methods for predicting ASD performance and case studies for poorly soluble molecules.

12:50 PM - 1:50 PM

Networking Lunch

Lunch & Learn Round Table with MedPharm

1:50 PM - 2:25 PM - Solution Spotlights

Device Development

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

Russ Hornung, Business Development for Drug Delivery and Packaging, W.L. Gore & Associates

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

2:30 PM - 3:05 PM - Case Studies

Small Molecules

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

Philippe Lienard, Pre-Development Science Leader, Sanofi

  • Many ASD technologies are available at the industrial scale
  • Selection of the right ASD technology is strategic for the future pre-clinical and clinical development and depends on:
  • Quantity of API available
  • Stage of development (feasibility studies, formulation/process development)
  • Formulation selection
  • CMC prerequisite (physical and chemical stability, solubility and dissolution improvement)

Device Development

Considerations with the Development of Inhaled Combination Products

David Cipolla, VP of Research, Insmed Inc.

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

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

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

3:10 PM - 3:45 PM - Solution Spotlights

Device Development

Wearable Electromechanic Injection Devices: Advantages, Challenges and Opportunities for the Future

Tom Mayer, Business Unit Manager, Sonceboz SA

This talk will take you on a journey from concept creation to preparation for clinical trial utilization of the Sonceboz Wearable Injection Device Platform. In the past 4 years Sonceboz has collaborated closely with patients and pharma companies with the goal to develop a platform of wearable injectors intended to accompany a drug product from its initial clinical phases to different evolutions in lifecycle management. This presentation will highlight current and future trends, outline how wearables can be the enabling factor when drugs are being reformulated from intravenous to subcutaneous delivery to support self-care and outline lessons learned along the way.

Small Molecules

What the Eye Doesn´t See in Molecular Interactions: a Virtual Microscope to Understand Cyclodextrins

Rebeca García Fandiño, "Ramón y Cajal" Researcher and CSO at MD.USE Innovations , MD.USE Innovations SL and Center for Research in Biological Chemistry and Molecular Materials (CiQUS) - Santiago de Compostela University

Cyclodextrins increase the solubility and stability of relatively small molecules, including many drugs, by encapsulating them in their hydrophobic cavity. The interaction mechanism leading to these host-guest complexes is often not trivial. Structural, kinetic and thermodynamic information on these systems is expected to be useful to optimize pharmaceutical formulations. 
  • MD.USE is specialized in ortogonal characterization of molecular interactions, including computational Molecular Dynamics simulations (MD) and Isothermal Titration Calorimetry (ITC). We also develop software for immersive visualization of molecules in augmented and virtual reality (AR/VR) 
  • In collaboration with Ligand Pharmaceutics, we will present our results on the interaction between Captisol® (a sulfobutyl ether derivative of β-cyclodextrin) and different drugs, including Remdesivir used against COVID-19 
  • Our studies show the dynamic movement of the molecules interacting with each other in a reliable way and with atomic resolution, as well the most stable structures, energy barriers and wells, possible aggregates and thermodynamic parameters, including affinity constants 

3:45 PM - 4:35 PM

iSolve & Networking Break

4:35 PM - 5:10 PM - Case Studies

Device Development

Biologic Drug Product Challenges with Closed System Transfer Devices

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

Closed System Transfer Devices (CSTDs) are meant to offer protection to healthcare professionals against harmful exposure of hazardous drugs during compounding and administration. Although there has been much focus on CSTDs in the context of drug contamination and safety, there has not been much discussion around the compatibility of CSTDs with biological products and patient safety. This talk will address the major challenges with the use of CSTDs including drug product incompatibility and hold up volume to name a few. These aspects are crucial given the impending USP <800>, which will mandate healthcare professional to use CSTD for compounding and administration of all biologics as per the National Institute of Occupational Safety and Health (NIOSH) list of antineoplastic and hazardous drugs.

Small Molecules

Impact of Bile Salts on Solubilization and Membrane Transport of Supersaturated Drug Formulations

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

  • Supersaturating formulations have been developed to address the issues of inadequate aqueous solubility of new chemical entities and improve oral absorption
  • Bile salts, naturally present in the gastrointestinal tract, have been shown to impact the crystallization kinetics of supersaturated formulations
  • The effect of bile salt on thermodynamic properties of supersaturated drug solutions was evaluated. The interplay between solubilization and membrane transport was studied
  • The findings indicate that the impact of bile salts on solution thermodynamics is of relevance for improved understanding of in vivo performance of supersaturating dosage forms

6:00 PM - 6:05 PM

Chair’s Closing Remarks

8:00 AM - 8:45 AM

Registration

8:45 AM - 8:50 AM

Chair’s Opening Remarks

8:50 AM - 9:25 AM - Keynote

Technology & Innovation

Developing a QbD based Control Strategy for Continuous Manufacturing

  • Identifying QbD elements suitable for continuous manufacturing
  • Putting control strategies into practice for small and large molecule situations 

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

Technology & Innovation

Reformulation Strategies for Improved Patient Compliance

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

Drug delivery technologies are one of the product life cycle extension strategies to combat generic competition and harness value of the product to the maximum. Generally, it is based on the principles of improved usage and compliance, safety and efficacy and indication extension. Some of the successful examples from Sun Pharma are Xelpros, Bevetex, Cequa, Bromsite etc. The presentation shall cover few successful case studies from SPARC that has endured all the developmental, clinical and regulatory challenges to eventually enter into the market.

Small Molecules

Multivariate Analysis in the Pharmaceutical Industry

Mike Tobyn, Research Fellow, Bristol-Myers Squibb

  • Big data
  • Multivariate analysis
  • Validated techniques

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

Technology & Innovation

Small is powerful: Delivering nanoparticle solutions to BIG formulation challenges

Dr Satu Lakio, Pharmaceutical Development Manager, Nanoform

The presentation will cover, Nanoform, our CESS® small molecule nanoparticle technology and showcase two case studies in solubility and bioavailability enhancement. The case studies will highlight use of CESS® nanoparticles in oral drug delivery formats, corresponding PK studies and a comparison of CESS® technology dissolution data with a variety of other commonly used techniques such as spray dried amorphous dispersion, milling, micronization, hot melt extrusion, salts and co-crystals.

Device Development

Solution Spotlight Topic TBC (Sensile Medical)

10:45 AM - 11:35 AM

iSolve & Networking Break

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

Technology & Innovation

Nanoparticles and Medicines Design

Ijeoma F. Uchegbu, Professor of Pharmaceutical Nanoscience , University College London

Show more

Technology & Innovation

Enhancing Vaccine Stability and Ease of Administration Using Microneedle Skin Patches

Anne Moore, Professor, University College Cork

The endgame to the COVID-19 pandemic requires the identification and manufacture of a safe and effective vaccine and a subsequent global immunization campaign. However, the vaccine supply chain is complicated by the requirement for cold chain distribution logistics and sufficiently trained vaccinators to administer the vaccine by injection. This complicated supply chain adds to the cost and delays full global roll-out of a pandemic vaccine resulting in delayed and inequitable vaccine coverage.

We have been addressing this problem by creating an easy-to-administer skin patch that thermostabilizes vaccines. As vaccine administration only requires placing the patch on the skin, we have removed the need for needles, syringes, biohazardous waste and training of healthcare workers. Ease-of-administration is critical to ensure cost-effective, high vaccine coverage and disease eradication, as evidenced by the success of the oral polio vaccine.  The patch format stabilizes the vaccine out of cold chain conditions, thereby simplifying vaccine distribution and logistics.  Skin delivery of thermostabilized vaccines therefore presents several advantages compared to intramuscular delivery, including increased compliance due to simplicity of vaccination, broadening the profile of the immune response and the potential for dose sparing.

Here, we will discuss  our previous work on developing these skin patches, which use dissolvable microneedles to stabilize vaccine in the solid state and deliver it into the body.  We will discuss our previous work on incorporating different vaccine platform technologies into these skin patches, including adenovirus virus vectors, lipid-based, nucleic acid-based and subunit vaccines, which are the leading platforms used in successful COVID-19 vaccines.

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

Technology & Innovation

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

Christophe Roberge, Senior Technology Advisor, MedinCell

We will introduce BEPO®, a clinically advanced long-acting injectable proprietary technology with outstanding versatility. BEPO® is an in situ forming depot technology whereby a solvent-exchange process elicits the precipitation of the polymer upon injection, leading to the formation of a fully biodegradable drug releasing matrix in situ. Several BEPO® products have now reached the clinical stage targeting different indications and routes of administration.

Following the introduction of the scientific elements highlighting the tunability and maturity of our drug delivery platform, we will also present case studies to exemplify why and how BEPO® brings the flexibility to formulate a wide variety of APIs, from small molecule drugs to more complex biomacromolecules, and addresses various unmet medical needs for both emerging and developed countries alike.

Technology & Innovation

Discriminative Dissolution Testing of Nanoparticle Formulations with NanoDis

Emre Türeli, CSO, MyBiotech

Dissolution is one of the first methods applied in formulation development to gain information on the expected in-vivo performance of the pharmaceuticals. Nanoparticle development process suffers currently from the limited capability of dissolution methods especially on the separation of nanoparticles from the dissolution media for the quantification of dissolved drug. Conventional methods such as filtration, centrifugation, dialysis membrane have all different limitations in separation of nanoparticles from dissolution media where none of these methods can supply sufficient information on the in-vivo performance of nanoparticle formulations. This information is of crucial importance for the scientists developing nanoparticle formulations to be able to select the lead formulations with the best expected in-vivo profile. In this presentation the drawbacks of current dissolution methods for nanoparticles are described together with proposals for the improvement of the performance of dissolution methods for nanoparticles introducing an online sytem for the separation of the nanoparticles from dissolution media.

12:50 PM - 1:50 PM

Networking Lunch

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

Device Development

How to use Smart Devices Intelligently to Deliver Outcomes

Jace Blackburn, Smart Device Engineer, Genentech

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

Biologics

CMC Optimisation of a Monoclonal Antibody Using Predictive in Silico Approaches

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

2:30 PM - 3:05 PM - Case Studies

Biologics

Challenges and Opportunities in Cell Therapy Formulation and Delivery

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

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

Technology & Innovation

V-Smart®: Non-Invasive, Dual-Targeted BBB Delivery Solution for Non-Brain Penetrant CNS Therapeutic Agents

Susan Rosenbaum, Founder, Chairman, CEO , Lauren Sciences

Learn from Award Winning Biotech how Innovative V-Smart® Nanovesicle Platform for Non-Invasive, Dual-Targeted Delivery of Non-BBB Penetrants (Ab, siRNA, biologic, sm. mol.) is used to develop Transformative V-Smart® Nanomedicines Pipeline for Unmet CNS Medical Needs (ALS, PD, GBM, AD, Pain, Neuro-HIV)

3:05 PM - 3:55 PM

Networking Break

Round table by David K. Lyon, Senior Fellow, Global R&D, Lonza Pharma & Biotech

In vitro test methodologies for characterizing bioavailability enhancing formulations

3:55 PM - 4:30 PM - Case Studies

Biologics

Biologics

Liquid Preformulation Assessment of Biological Candidates (1)

Shwetha Iyer, Senior Scientist- Formulation and Characterization , Novartis

  • Abstract TBC
     

4:35 PM - 5:20 PM - Panel Discussion

Technology & Innovation

What could be future of medicine : Small molecule, Biologics or Cell and Gene Therapy

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

5:20 PM - 5:25 PM

Poster Presentation Award

5:25 PM - 5:35 PM

Chair’s Closing Remarks