September 18-19, 2023; Boston, MA

For speaking opportunities please contact

7:15 AM - 8:15 AM

Registration & Refreshments

8:20 AM - 9:05 AM - Keynote

Technology & Innovation

Model for Long-Acting Injectables

Jaymin Shah, Senior Research Fellow, Pfizer

  • Recently several long acting injectables have been developed as aqueous suspensions and nanosuspensions.
  • The presentation will show using depot technology map influence of solubility and depot dose on differentiated clustering of drug by technologies
  • Development and application of PK model to predict dose and release rate combinations needed for desired PK profile
  • Integration of dissolution rate-based prediction with PK for TI to identify what is the appropriate technology for depot candidate
Long acting injectables have been developed traditionally using a variety of different approaches such as oily solution, aqueous suspensions, gels, nanosuspensions and microspheres.  However, what molecular properties, physical chemical, and PK properties of a drug, are suitable for which technology to enable success is not established.  The presentation will provide a background on depot and show a model based on PK and physical chemical properties of drug to select the appropriate technology.  Ideally with the model, one can select the most likely LAI or depot technology that will provide sustained PK profile in vivo for a candidate with examples.

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

Small Molecules

Biopharmaceutical Performance Enhancement via Nano Sized Crystalline and Amorphous Solid Dispersed Materials

Shawn Yin, Scientific Director, Bristol-Myers Squibb

  • Biopharmaceutical performance enhancement technologies
  • Nano-sized crystalline material
  • Amorphous Solid Dispersed Materials and SDD
  • Performance enhancement vs. physical stability
The potential of applying the advanced material technologies like nano and amorphous materials to pharmaceutical science and product development is fast emerging and evident.  This presentation will focus on some pharmaceutical development key issues including drug product performance and physical stability and how to use novel material technologies to mitigate the risks, illustrated by nano material and SDD product development case study examples.


A Biopharmaceutics Perspective on Oral Delivery of Peptides, Proteins and Oligonucleotides

Manuel Sanchez-Felix, Associate Director, Novartis Institute for BioMedical Research

The following topics will be discussed related to oral bioavailability of large molecules
  • Barriers to oral absorption of large molecules
  • Oral biopharmaceutic mechanisms applied in formulation approaches
  • Review of the oral formulation approaches will be discussed
  • Recent formulation advancements

Device Development

Digital Transformation: Impact on Healthcare Industry & Quality Engineering

Alie Jahangir, Director, SaMD and Medical Devices, Pfizer

  • Impact of digital transformation on traditional processes, products and organizational system within healthcare industry
  • Pillars of smart QMS and Risk Management systems
  • Best practices of digital transformation in combination products and other emerging technologies.
Pharma 4.0 represents the integration of advance technologies (i.e. AI, ML, IoT, automation) to optimize the entire pharmaceutical value chain. This is a significant paradigm shift for pharmaceutical industry which aims to improve efficiency, quality and patient outcomes by utilizing digital solutions and data driven approaches in drug discovery, development, manufacturing, supply chain management and personalized medicine. This presentation elaborates on setting an agile and smart quality management systems (QMS) that drive measurable impacts across all building blocks to improve product quality, process reliability and ultimately, efficacy as well as patient safety.

9:45 AM - 10:15 AM - Case Studies

Small Molecules

Physiologically-Based Biopharmaceutics Modelling to Inform Drug Product Quality and Clinical Decisions

Filippos Kesisoglou, Distinguished Scientist, Pharmaceutical Sciences and Clinical Supply, Merck

  • Recent developments on application of PBPK/PBBM models to inform bioequivalence decisions
  • Linking dissolution to clinically relevant specifications
  • Use of PBPK modelling to understand food effect after formulation changes
  • Emerging use of PBPK modelling to understand excipient interactions
Physiologically based biopharmaceutics models (PBBM) have been playing an increased role in development decisions as related to formulation changes, spanning both clinical development aspects and drug product quality. Especially for IR formulations, PBBM provide a means to establishing an in vitro-in vivo link addressing the low success rates of IVIVCs. The presentation will focus on three aspects of application of PBBM models: a) the concept of a bioequivalence “safe space”, established via the model, as means to inform clinically relevant specifications; b) use of the modeling to understand food effect after formulation changes and c) emerging use of models to understand impact of excipients. For specification setting and food effect predictions, the case studies will also discuss the best practices and recommended workflows for model development.


Finding Signal in the Noise: Visualizing Big Data to Design Better Drugs

Dennis  Leung, Director and Manager of the Discovery Pharmaceutics and Preformulation , Genentech

  • In recent years, high throughput technology has enabled the rapid acquisition of large amounts of data for characterizing new drug candidates. While this can significantly enhance the exploration of the drug molecule space, it can result in an overwhelming amount of data that must be carefully parsed and interpreted. While the quantity of data is important, it is critical to find the quality signal in the noise by extracting meaningful conclusions from Big Data to better influence drug discovery and development. It is important to link Big Data to tangible end points in order to better understand our work.  
  • Big Data can be used to better know what we know and making sure that we are asking the right questions. One example includes learning more about the biorelevance of our assays.
  • Data analytics can be used to better understand historical data to enable faster and improved decision making in the future.
  • Large data sets can also be used to train AI/ML models to facilitate compound optimization and predict formulation performance,
The discovery and development of new drugs has become increasingly challenging. The number of new drug candidates that encounter failure in clinical development has increased and the number of new drugs reaching the market has decreased in recent years. One factor contributing to the high attrition rate is due to unexpected behavior in the clinic, for example due to unforeseen toxicity or lack of efficacy. This has been further compounded by increasingly challenging new disease targets occupying historically “undruggable” space. The interaction between drug molecules and these undruggable targets can be difficult to predict and optimize.  As a result, it is critical to understand the properties of potential drug molecules as well as their behavior towards these targets.

In parallel with these challenges, there has been a concomitant increase in the development and application of new high throughput tools for rapidly assessing and characterizing a large number of drug molecules.  While this can significantly enhance the exploration of the drug molecule space, it can be also result in an overwhelming amount of data that must be carefully parsed and interpreted.  While the quantity of data can be useful, it is crucial to be able to find the quality “signal in the noise” and to be able to rapidly extract meaningful conclusions from Big Data in order to better influence drug discovery and development.  This presentation will focus on identifying key correlations between chemical properties obtained in high throughput approaches with meaningful study endpoints (e.g., bioperformance), resulting in improved strategies for optimizing drug molecules.

Technology & Innovation

Leveraging Melt-Spray-Congeal Microspheres as a Versatile, Patient-Centric Oral Multiparticulate Platform

Brett Kopina, Principal Scientist, Pfizer

  • Utility of multiparticulate dosage forms and survey of the technology landscape.  
  • Characterization of Melt-Spray-Congeal (MSC) using rotary atomization and the materials that enable its versatility.
  • Define formulation design space and considerations for the physicochemical properties of the suspended active ingredient.
  • Exemplify the ways in which platform versatility can deliver organizational value.
Oral multiparticulate dosage forms are widely valued for flexible dosing, tuneable release profile, GI transit consistency, and patient centricity. Melt-Spray-Congeal (MSC) using rotary atomization is one of many processes capable of producing monodisperse multiparticulate drug product, but when combined with well-studied carriers, excels as a platform technology capable of meeting ranging delivery needs for portfolios featuring small molecules with equally divergent physicochemical attributes. This presentation will define the melt spray congeal design space and exemplify its versatility as a factotum technology for the pharmaceutical industry.

10:20 AM - 10:50 AM - Solution Spotlights

Small Molecules

Leveraging Rapid Clinical Assessments to Accelerate Pediatric Formulation Development

Huw Jones, Executive Director, Patient Centric Medicines, Pediatric Services Drug Development Consulting, Quotient Sciences

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

What you’ll learn:

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


Innovative Gastro-Retentive Drug Delivery Technologies for Improving Therapeutic Outcomes of Challenging Compounds

Pavan Handa, Senior Vice President, Specialty Portfolio Management , Amneal Pharmaceuticals

Siva Vaka, Director, Formulation R&D, Amneal Pharmaceuticals

  • Attributes of next generation, once-daily, gastro-retentive drug delivery technologies to improve the solubility & bioavailability of BCS Class II & IV drugs.
  • Proof of concept in humans for small molecules including drugs with narrow therapeutic index along with case studies.
  • Further evaluation is ongoing to determine reduction in dose, PK variability, & AEs for selected compounds.
GRANDE® is an innovative gastro-retentive drug delivery technology that enables the drug product to retain  in the stomach for a prolonged period, up to 24 hours. This system provides extended drug release with enhanced absorption. GRANDE® technology can be applied to a wide array of weakly basic drugs with poor solubility or permeability with a narrow absorption window in the upper gastrointestinal tract. Approximately 40% of approved drugs and 75% of drugs in development are known to be weakly basic, thereby providing a wide utility for application of GRANDE®. (References: Acta Pharm Sin B. 2015 Sep; 5(5): 442–453; Perspect Medicin Chem. 2007; 1: 25–38).

Device Development

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

Tim Phillips, Principal Human Factors Engineer, Cambridge Consultants

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

10:50 AM - 11:40 AM

Networking & 1-2-1 Meetings

11:40 AM - 12:10 PM - Case Studies

Small Molecules

Predicting Powder Flow Obstruction in Solid Dosage Form Manufacture

Chen Mao, Distinguished Scientist, Small Molecule Pharmaceutical Sciences, Genentech

  • Powder flow obstruction, such as arching or ratholing, is a prevalent occurrence in the manufacture of solid dosage forms.
  • Accurate prediction of powder flow obstruction requires the knowledge of both powder flowability and stress condition of the powder in the equipment.
  • The risk of powder flow obstruction is highest under the active stress state.  Traditional Jenike methodology in hopper design assumes the passive stress state and could therefore under-predict the powder flow risks.
  • Additional insights of powder flow risks, such as stress heterogeneity and flow pattern, can be obtained via FEM analysis.
Powder flow obstruction is a prevalent issue in the manufacture of solid dosage forms.  The current industrial practice is to assess powder flowability using properties such as the flow function coefficient (ffc).  While ffc characterizes intrinsic powder cohesive strength based on the Mohr-Coulomb theory, it does not account for the external stress applied to the powder which facilitates flow.  To this end, we provided a complete methodology enabling the prediction of powder flow obstructions in bin/hopper discharge based on a complete stress analysis.  We have shown that the risk of powder flow obstruction is highest immediately upon outlet opening, at which time the stress at the bin/hopper outlet is high because the powder takes on the active stress state.  An indiscriminate use of the traditional hopper design procedure, which assumes the passive stress state, would significantly under-estimate the powder flow risks in pharmaceutical manufacturing operations.  We also showed that the FEM analysis leads to similar outcomes in powder flow prediction.  Additionally, FEM can provide additional insights such as the stress heterogeneity of the powder in the equipment, and the dynamic flow behaviour.


Drug Product Design and Biopharmaceutics Considerations for Long-Acting Injectables

Simone Alidori, Scientific Leader, Associate Fellow, GSK

  • Cross-functional approach for LAI product design and development.
  • Mechanistic understanding of LAI performance
  • Integrated biopharmaceutics concepts and opportunities
  • Enterprise vision for LAI drug product development
This presentation will cover Long-Acting Injectables (LAIs) as a novel way to design and deliver patient-centric medicines. Mechanistic understanding around LAI product performance is required to ensure efficient design and development of patient-centric and stable drug product with robust control strategy. Integrated biopharmaceutics concepts will be discussed to build mechanistic understanding and drive differentiated product design.

Device Development

Building an Autoinjector Development and Supply Ecosystem

Mark Howansky, Vice-President, Device Development and Commercialization, Viridian Therapeutics

  • QMS Requirements – FDA 21 CFR Part 4, Design Control vs Change Control
  • Device Assembly and Testing Considerations
  • Platform technologies vs customizations vs bespoke designs
  • Development of Device Packaging, Labelling, Instructions and Training Material
  • Role of Human Factors Engineering
Developing, launching and supporting a drug-delivery autoinjector requires specific systems, procedures, capabilities and skills.

12:15 PM - 12:45 PM - Solution Spotlights

Small Molecules

Role of PLGA Variability in Controlled Drug Release from Dexamethasone Intravitreal Implants

Feng Zhang, Associate Professor at the Division of Molecular Pharmaceutics and Drug Delivery at College of Pharmacy, University of Texas, Austin

Long-acting injectable formulations based on poly(lactide-co­-glycolide) (PLGA) have been commercialized for over 30 years in the form of at least 20 FDA–approved products. These dosage forms offer several advantages over comparable formulations, including reduced dosing frequency, improved patient compliance, and maintenance of therapeutic levels of drug. However, the inherent complexity of the PLGA copolymer presents many of these challenges of developing PLGA-based long-acting products. Small changes to PLGA physicochemical properties and/or the drug product manufacturing process can have a major impact on the drug release profile of these long-acting formulations.

This work seeks to better understand how variability in the physicochemical properties of similar PLGAs affects drug release from PLGA solid implants using Ozurdex (dexamethasone intravitreal implant) as the model system. The PLGAs were extensively characterized using a variety of analytical techniques prior to implant manufacture using a continuous, hot melt extrusion process. In vitro release testing of the four structurally-equivalent implants was performed in both normal saline and phosphate-buffered saline, yielding drastically different results between the two methods. In normal saline, no differences in the release profiles were observed. In PBS, drug release profiles were sensitive to small changes in residual monomer content, carboxylic acid end group content, and blockiness of the polymers. This finding further emphasizes the need for a physiologically relevant in vitro release testing method as part of a robust quality control strategy for PLGA–based solid implant formulations.


Empowering Biologics Delivery: Croda Pharma’s Comprehensive Portfolio of Biopharma Solutions

Sreejit Menon, Research and Technology Manager, Croda Pharma

Katherine McQuillan, Research Scientist, Nautilus Biosciences, a division of Croda Canada

  • Overview of Croda Pharma solutions and product offerings of Super Refined™ excipients, high purity lipids and bioprocessing aids  
  • Introduction to new Virodex™ product range: efficient and sustainable alternatives to Triton™-X-100
Triton™ X-100, a detergent often utilized in biomanufacturing, has been banned (with exemptions) in Europe effective January 2021 because of its harmful environmental impact. To address this issue, we have identified two sustainable alternatives that are compendial-grade, cGMP EXCiPACT-manufactured, REACH-compliant materials. This presentation will highlight that these alternatives exhibit equivalent performance to Triton™ X-100 for viral inactivation and cell lysis and outperform three market alternatives for viral inactivation.

Technology & Innovation

Driving Towards Patient-Centricity with Drug-Eluting Implants

Tom Quinci, External Partnerships Leader, Celanese

Polymer-based drug-eluting implants (DEIs) offer an alternative to conventional dosage forms.  DEIs inherently address issues with patient adherence.  Accordingly, they create an opportunity to improve outcomes, offer patients a better quality of life, and improve healthcare economics across the ecosystem. These implants can be engineered to provide local routes of administration, deliver combinations of medications, enable sustained release, and may be removed to interrupt therapy. The VitalDose® Drug Delivery System is a uniquely flexible technology platform, helping formulators achieve their desired sustained-release profiles for biologics, RNA, and peptides. Using a range of formulation strategies and tunability levers, the Celanese Development & Feasibility Lab supports development of implants for a broad range of applications across oncology, ocular, central nervous system, women’s health, and rare diseases.

12:45 PM - 1:45 PM

Networking Lunch

1:45 PM - 2:15 PM - Case Studies

Small Molecules

End to End Digital Design for ER Tablet Drug Delivery Systems with Functional Coating

Alfred Berchielli, Associate Research Fellow, Drug Product Design, Pfizer

  • Selection of the drug delivery technology and modelling of the target release profile.
  • Examples of initial digital design for osmotic pump ER formulations in early phase clinical studies.
  • Enhanced control of film properties and dissolution via comprehensive process modelling and PAT for function coatings.
  • Scale-up and optimization of the functional coating process via first principles, empirical, and digital twin simulations from lab to commercial scale.
  • Eliminating or minimizing scale-up by use of the small batch or semi-continuous processes for functional coatings.

PK modelling has long been used to generate the target in-vivo release and PK profiles.  The drug delivery technology is then selected or matched with the biopharmaceutical objectives and patient medical need.  Formulation models have enabled digital design for initial prototype key attributes (e.g., functional coating weight gain) to achieve the target in-vitro dissolution profile.  More recently coating models have advanced to guide in scale-up from lab through commercial scale via first principles, empirical models and more comprehensive digital twins coupling the discrete element method (DEM) and computational fluid dynamics (CFD).  These models and new process analytical technology (PAT) are helpful to maintain consistent product performance by reducing variability in functional coating amounts or changes in coating morphology with process scale (e.g., batch size) or equipment design.  Additionally, there are new, small scale coating equipment designs that can be leveraged to reduce or avoid scale-up altogether.


Accelerating Discovery to FIH through Early Formulation Risk Assessments of Novel Biologics

Suman Luthra, Director Discovery Pharmaceutical Sciences, Merck

  • Inform candidate selection by evaluation of structural liabilities of lead candidates​ against target candidate profile
  • Develop prototype product concepts to accelerate clinical formulation development
Engineered proteins are a promising class of therapeutic agents, but they face several challenges during the CMC developability process. To match the complexity of these novel modalities and address these challenges, it is important to assess the CMC risks of engineered proteins at various stages of discovery. This includes assessing the tendency for self-interaction, aggregation, thermal stability, colloidal stability, and biochemical stability. These assessments can be performed in preformulation and prototype stability studies with representative stable pool drug substance. The data generated from these assessments can be used to rank order lead candidates, identify risks that could be mitigated, and develop formulations that meet the target product profile. This information can help to accelerate clinical development and avoid costly late-stage slowdowns or failures. By taking a comprehensive approach to CMC, it is possible to develop engineered proteins that are safe, effective, and commercially viable.

Device Development

Development of Drug Delivery and Photoactivation Devices for the Treatment of Bladder Cancer

Keith Faucher, Senior Director Device Development and Innovation, Aura Biosciences

  • Performing front end research to understand the clinical environment for administering combination product therapy.
  • Defining device development requirements based on the limitations of clinical and drug product characteristics.
  • Importance of obtaining early user feedback on device prototypes and procedures.
  • Strategies for device sourcing and testing to speed evaluation of combination products in clinical trials.
Aura Biosciences is developing a new class of oncology targeted therapies to deliver a meaningful therapeutic benefit to a range of cancer indications with high unmet need.  One of these areas of high unmet need is non muscle invasive bladder cancer, where there are approximately 420,000 new cases diagnosed globally each year.  Of these new cases, 40 % do not respond to traditional Bacillus Calmette-Guerin (BCG) treatment and might eventually require cystectomy.  Aura’s investigational therapy for bladder cancer is a drug device combination product, which is currently being evaluated in a clinical trial. Successful development of this product required an interdisciplinary approach and the integration of clinical requirements with the combination product characteristics, in addition to the incorporation of a photoactivation system.  A strategic approach for the successful development of drug-device combination products will be discussed.

2:20 PM - 2:50 PM - Case Studies

Small Molecules

Pediatric Formulation Design – From Adult to Children

Curtis (Cheuk-Yui) Leung, Ph.D, Associate Director, CMC Formulations, Agios Pharmaceuticals Inc.

  • Agios’ approach for pediatric formulation design
  • Pediatric formulation platform selection
  • Palatability and excipient selection
  • Manufacturing and packaging consideration

Supplying drug to children in the rare genetic disease area is very important since a lot of patients are being diagnosed at young ages. A medicine can really improve their lives and allow them to live more like their peers at the same age. Therefore, it is important to select an age-appropriate formulation for the pediatric population. This presentation will show case the approach of Agios in the selection of a pediatric platform with considerations include manufacturability, patient acceptance, dosage form swallowability and palatability. In addition, the choice of packaging configuration and distribution strategy of the drug is also key to a successful program due to complexity caused by various doses and age groups.


Characterization of the Hydrolytic Activity of Polysorbate Degrading Host Cell Proteins

Anthony Tomlinson, Technical Development Principal Scientist , Genentech

  • Polysorbate degradation as a topic will be introduced
  • The identification and purification of polysorbate degrading hydrolases
  • Screening of these enzymes for their degradation activity
  • Characterization of enzyme kinetics and specificity on a subset of hydrolases

 Polysorbate degradation is an industry-wide issue for biopharmaceutical drug product development. This degradation can be catalysed by the low-level presence of hydrolytic host cell proteins which co-purify with the target molecule in bioprocessing. The degradation of this key excipient can cause a variety of issues, including loss of surfactant activity and the formation of subvisible or visible products in liquid formulations. In this work, we aimed to purify and characterize several CHO cell host cell proteins with respect to their ability to degrade polysorbate.

Device Development

Addressing Challenges in Combination Product Development for SC Delivery

Ajit D'Souza, Senior Director, Combination Product Development & Manufacturing, Kiniksa Pharmaceuticals

As healthcare systems increasingly rely on medical care administered at home, the focus on ease of administration, safety, compliance and sustainability is driving the development of combination products for home use.  This presentation will discuss approaches to addressing three key challenges: i) drug-device compatibility, ii) reliability and robustness of performance and iii) usability issues in the context of high concentration mAb formulation.

2:50 PM - 3:40 PM

Networking & 1-2-1 Meetings

3:40 PM - 4:10 PM - Solution Spotlights

Small Molecules

Overcoming Challenges in Complying with a New Monograph During a Pandemic – Captisol® Success Story

Ryan Memmer, Manager, Quality Control and Development , Singota Solutions

  • Captisol®’s impact on the industry will be introduced.
  • Complexities of the EP compendial testing and worldwide issues they have caused
  • Analytical Method Challenges: Examples examined from issues to a resolution.
  • Future Captisol® methods and products discussed.

This presentation will discuss the obstacles and great efforts needed to test, approve and release Captisol® in Europe, dictated by the analytical testing requirements from the European Pharmacopeia. Captisol®, a Proprietary Sulfobutylether-β-Cyclodextrin (bonded sugar molecules) with a process specific composition is used to enable aqueous formulation for many insoluble API’s. It is applicable to many drug classes and drug product presentations including oral, ophthalmic, nasal, topical, lyophilized or liquid parenterals plus provides an alternate and safer choice compared to the use of cosolvents and surfactants.

The USP (United States Pharmacopeia) – NF (National Formulary) monograph for Captisol® (Betadex Sulfobutylether Sodium) was established based on the validated Captisol® test methods and went into effect in 2012. The Captisol® team approached the EP (European Pharmacopeia) in 2018 to harmonize the USP methods with the methods under consideration by the EP, but were unable to establish a dialog with EP. In July 2019, the EP monograph for Sulfobutylbetadex sodium (2804) was implemented and several new test methods and materials were introduced with the EP monograph, specifically NMR for determination of degree of substitution, impurity analysis, and reducing sugars. Additionally different specification ranges were adopted with the monograph including assay, pH, degree of substitution, and impurity limits. The two analytical methods for impurity analysis and reducing sugars are examined in this presentation to convey the complexity of newly added requirements.

Captisol® was first used in an approved product in 2002 and established as a safe excipient worldwide through Pfizer’s antifungal, Vfend. Approximately 200 commercial batches have been manufactured, so why after 20 years did this excipient have to now comply with new methods and specifications when it was already deemed safe?


Scaling Up RNA LNP’s for Commercial Success

Jignesh Patel, Field Application Scientist, Precision NanoSystems

Nucleic acids (e.g., siRNA, mRNA and saRNA) can be designed and formulated to silence, express, and edit specific genes providing a flexible and powerful approach to preventing and treating diseases. The recent commercialization and widespread distribution of COVID-19 mRNA vaccines has exemplified the massive potential to rapidly develop and scale-up new genomic medicines to protect from emerging viral threats and treat a wide range of serious diseases with unmet medical needs.

As part of Danaher Life Sciences, Precision Nanosystems provides end-to-end, synergistic solutions and services to enable successful development of Genomic Medicines.

This presentation will discuss key insights and experiences that leverage modular microfluidic platform technologies to enable rapid development, testing, and scale-up of mRNA-LNP vaccines and therapeutics:

  • Insights into new platform technologies that increase efficiency to enable rapid scale-up and large-scale manufacturing of mRNA vaccines and therapeutics
  • How to improve operational flexibility by expanding single-use technology to LNPs 
  • Examples from our internal R&D work to demonstrate the versatility of the genetic medicine toolbox for the rapid development of RNA-LNP Vaccines

Technology & Innovation

Silicone-Free Option for Delivery of Sensitive Biologics and Conjugate Vaccines in Glass Pre-Filled Syringes

Michael P. Kienzle, Global Product Specialist and New Business Development Associate, W.L. Gore & Associates

Conventional plungers for pre-filled glass syringes are designed to work with lubricated barrels where silicone often acts as the lubricant between plunger and syringe during drug administration and provides a seal between plunger and syringe. However, silicone has been shown to potentially interact with sensitive biologics possibly leading to particulation and aggregation.  With the market trend of higher drug concentrations while complying with USP<788> of number of particles per injection in combination with larger syringes which contain more silicone or challenges with ophthalmic injections subject to more stringent US<789> particle level, silicone floaters or silicone induced inflammation, GORE has developed the IMPROJECTÒ plunger that eliminates all forms silicone from the barrel and the plunger of a pre-filled syringe

4:15 PM - 4:45 PM - Case Studies


Overcoming Formulation Challenges for Complex Bispecific Antibody Drug Product Development

Xiaofeng Lu, Principal Research Scientist, AbbVie

  • Overcoming Formulation Challenges for Complex Bispecific Antibody Drug Product Development
  • Challenges in formulation development for complex bispecific antibodies
  • Bispecific antibody case studies ​
  • Aggregation- a critical product quality attribute and possible causes  ​
  • Control aggregation by non-platform formulation and process parameters  
Development of complex bispecific protein drug products posed formulation challenges due to the proteins’ susceptibility to aggregation in aqueous solution. In this talk, the primary causes for protein aggregation and stabilization approaches, that were explored to develop the formulations for two complex bispecific antibody drug products will be presented.

Device Development

Formulation & Device Considerations for Subcutaneous (SC) Delivery of Biotherapeutics

Sahab Babaee, Associate Principal Scientist, Merck Research Labs – Program Lead & Device Innovation, Merck

  • Overview of subcutaneous delivery in the current market
  • Considerations when developing a target product profile for subcutaneous delivery
  • Identifying early challenges and opportunities in formulation and device development
  • Technology down selection and moving forward towards the target image
Subcutaneous (SC) delivery route has become an emergent alternative to intravenous administration across many disease areas by offering the opportunity to reduce payor costs, increase adherence, and improve the patient’s experience. High injection forces (due to high viscosity of concentrated formulation), prolonged injection time, and increase in perceived pain are major challenges of existing parenteral injection systems limiting SC delivery, especially for large volume, high viscosity biologics including monoclonal antibody and protein formulations.

In this talk I review the formulation and device considerations/challenges when developing a Target Product Profile (TPP) for a SC-delivered product. Then, I highlight early challenges and opportunities in formulation & device development space, considering a broad range of SC drug delivery devices including preloaded microneedle patches, autoinjectors, injection pens, syringes, wearable and implantable systems. Finally, a methodology for technology down-selection and final target image selection will be provided.

4:50 PM - 5:20 PM - Case Studies

Small Molecules

Beginning with the End in Mind: Risk Based Approach for Oral First in Human Formulation Development

Varsha Dhamankar, Director, Head of Formulation Development, Ribon Therapeutics

  • “Fit-for-purpose” formulations such as solutions, suspensions, or powder in capsule dosage forms are frequently preferred to accelerate the initiation of first in human studies. However, these formulations often fail to illustrate oral bioavailability limitations and can significantly impact late-stage development.
  • Early evaluation of physico-chemical properties, preclinical PK, and biopharm modeling are crucial to identify oral absorption limitations and drive formulation technology selection.
  • Using a systematic risk-based approach, an optimized and well-characterized oral solid dosage form can be introduced in the early clinical studies while still balancing material requirements and timelines.


Preformulation Assessment for Large Biotherapeutics Enabling Early Understanding of the Clinical Service Formulation

Shwetha Iyer, Principal Scientist, Novartis

  • Development of LIVI for large biotherapeutics for FiH
  • Understanding the degradation mechanism of a given therapeutic by performing accelerated stability and forced degradation studies
  • Screening and assessing excipients that enhance protein stability
  • Cross functional understanding of stability indicating assays to successfully lock a CSF
Liquid pre-formulation assessment for monoclonal antibodies and new modalities showed a significant impact in determining the success rate of developing a stable liquid formulation for biological entities. The portfolio now changing towards non-mabs like Fc silencing formats, bispecific mabs, therapeutics proteins and newer modalities such as gene therapies and AAV’s, designing stable liquid formulation is more challenging. Co-development of the clinical service formulation enables an in- depth understanding of the degradation pathways and the possibility of a stable liquid formulation. With the use of forced degradation studies and high throughput biophysical predictive tools, key developability questions can be addressed.

Device Development

Context is Key: Understanding Human Factors Validation Study Results

Alexandra Benbadis, Usability Leader, Sanofi

  • Explaining the goals of human factors validation and what data are important
  • Identifying the reasons behind human factors validation methods and how the data differ from other types of research
  • Showing the different ways non-experts can interpret results and advice for better understanding study outcomes (e.g., raw data, reports)
  • Relating human factors validation results to user needs validation

In a human factors validation study, “performance data” (use errors, close calls, and use difficulties) are the primary points of interest. Unlike many types of research, the performance data from human factors validation studies do not tell a complete story on their own. Once raw performance data from human factors validation are generated, there are several steps to understanding the results and their implications. These steps include: understanding participants’ subjective assessments of any findings, performing root cause analysis to identify why each finding occurred, and performing residual risk analysis. Only after these steps are completed can human factors experts draw conclusions on a product’s safety and effectiveness.

This presentation will provide non-human factors experts with a deeper understanding of human factors validation goals and how to interpret human factors validation results. The objective of this presentation is to help stakeholders shift their focus away from the raw data and understand that the complete context of the performance data is key to drawing conclusions from a human factors validation study.

5:20 PM - 5:50 PM - Case Studies

Technology & Innovation

Tuning LNP Formulation to Enable Extrahepatic Delivery

Nathan Westcott, Director, Delivery Platform, Beam Therapeutics

  • Extrahepatic delivery of LNPs represents the next challenge in the field.
  • LNPs may need to be formulated differently to overcome this challenge.
  • Finding novel ionizable lipids will help enable delivery to extrahepatic tissues.
Lipid nanoparticles (LNPs) containing nucleic acids have delivered life-saving vaccines to billions and accelerated the adoption of a new therapeutic modality. At Beam, we aim to deliver base editing gene therapies using lipid nanoparticles containing mRNA expressing a base editor and a guide RNA. Our base editors do not induce double strand breaks and instead safely and efficiently rewrite the genetic code one base at a time which results in life-long treatments. The mRNA is directly and transiently expressed, eliminating any liabilities associated with genomic insertion. Additionally, we have shown that LNPs can be dosed multiple times without adverse events allowing us to titrate the appropriate level of editing when necessary. Finally, LNPs promise to deliver these therapies directly to the patient as an intravenous injection. The key challenge is determining the proper formulation to selectively drive uptake in the tissues of interest. To overcome the challenge of delivering these complex therapies, we rely on our large lipid library to generate potent LNPs capable of delivering our base editing therapies to the liver and tissues beyond.


The Challenges and Opportunities for Subcutaneous Delivery of High Dose/Volume Biologics

Deep Bhattacharya Ph.D, Senior Scientist, Drug Product Design and Development, Pfizer

  • The potential to apply novel subcutaneous technologies to several therapeutic areas through collaborations across industry
  • Summarizing key patient and HCP perceptions of high dose/volume subcutaneous delivery
  • Insights into developing subcutaneous technologies that could further facilitate high dose/volume subcutaneous administration

Device Development

Patient-Centric Product Design & Digital Technology as Successful Inhalation Therapy Enablers

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

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

5:55 PM - 6:25 PM - Keynote


Selecting and Developing High Quality Antibody Theranostics

Sathya Venkataramani, Director Biological Research, AbbVie

The exploding growth of antibody therapies is predicted to reach ~$300 billion of market in 2026. Over 600 bispecifics are currently being investigated at various stages and >80% of this in clinical trials are developed to treat broad range of cancers. Being a genomic disease, each cancer needs early and precise diagnosis followed by personalized treatment. Antibody theranostics are currently used for both diagnosis and treatment. This presentation is tailored to highlight critical factors and prudent choice of profiling assays from early to late stage for successful development of antibody based theranostics for cancer treatment.

6:25 PM - 6:35 PM

American DDF Poster Presentation Award & Chair's Closing Remarks

Rosario LoBrutto, Head of Pharmaceutical Sciences and Global Expansion, Radius Health

6:35 PM - 7:20 PM

Drinks Reception

8:00 AM - 8:30 AM

Registration & Refreshments

8:35 AM - 9:05 AM - Case Studies

Technology & Innovation

Mesoporous Silicon and Silica Nanomaterials for Drug Delivery Applications

Prof. Michael Sailor, Distinguished Professor of Chemistry and Biochemistry, University of California, San Diego

  • The oxide chemistry of mesoporous silicon nanoparticles allows dynamic restructuring of the mesopores to sequester protein therapeutics
  • The chemistry provides a means to protect biologics against proteolytic, nucleolytic, or hydrolytic degradation
  • Peptide targeting groups provide a means to home nanoparticles to selected tissues while minimizing immune system responses.
  • Multiple copies of a peptide targeting group on a single nanoparticle enhances binding via multivalent interactions
Porous silicon nanoparticles possess an oxide chemistry that is well suited for loading and delivery of biologics (antibodies, enzymes, and nucleic acids). The material can be tuned for either slow release or for triggered release, depending on the specific chemistries and coatings used. Attachment of peptide targeting groups provides a means to direct delivery of therapeutic payloads to selected tissues.  One area with an unmet therapeutic need is in the treatment of antibiotic-resistant bacterial infections, and the deployment of porous silicon nanoparticles to meet this challenge will be described. The intrinsic photoluminescence that derives from quantum confinement in the silicon skeleton provides a built-in luminescent probe that can be used for in vivo and in vitro tracking and self-reporting drug delivery in these systems.

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

Small Molecules

Impact of Crystallinity on Performance of Amorphous Solid Dispersions

James DiNunzio, Director, Merck

  • Origins of crystallinity in amorphous solid dispersions
  • Detection approach and control strategies to minimize risk
  • Assessing impact of crystallinity on in vitro and in vivo performance of amorphous solid dispersions.
Amorphous solid dispersions have been established as a robust approach to improve oral bioavailability of solid dosage forms.  Commonly manufactured by hot-melt extrusion and spray drying, formulations using these platforms enable supersaturation but must also be designed to prevent crystallization which can result in reduced performance of the product.  This talk provides a background on the origins of crystallization and considerations pharmaceutical scientists must keep in mind when developing such systems.  It also reviews control strategy approaches to identify critical water activity of drug products and ways to implement an end-2-end control strategy.  Presented in this talk is recent data from a PK study assessing the impact of spiked crystalline content on oral bioavailability, demonstrating that it may not be necessary to have zero crystallinity to achieve acceptable product performance.


Formulation & Process Development Considerations for Non-Viral Gene Delivery

Dimitrios Pelekoudas, Scientist , Sanofi

Comirnaty® and Spikevax® along with siRNA-LNP based Onpattro® have demonstrated the manufacturability and clinical viability of LNP based nanocarriers. In development of LNP based drug product, the choice of each lipid component and the manufacturing technology is one of the key factors for success. The choice of lipid matrix can be modulated to maneuver and change the biodistribution of LNPs depending on the organ of interest and type of nucleic acid. The manufacturing technology can have significant impact on the biophysical properties, structural characteristics, colloidal stability, and efficacy of the LNP. This study describes the key considerations for designing LNP formulations and impact of different manufacturing parameters and the scale up consideration to enable successful LNP drug product manufacturing.

Device Development

Developing Wearable Injection Devices for Large Volume Drug Delivery

Anthony F. Coston, Technical Lead Device Design and Process Development, Biogen

  • Strategies establishing optimal volume range for an on-body
  • device
  • Assessing the impact of viscosity on injection force and best
  • driving mechanisms for use
  • Maximising therapeutic effect, patient tolerance and user
  • acceptability
  • Case studies on wearable device development
  • Insight into developing and commercialising of a portfolio of
  • device and combination products

9:45 AM - 10:15 AM - Solution Spotlights

Small Molecules

A Patient and Planet Centric Approach to Enabling Bioavalability

Sergie Letser, Deputy CCO, Senior VP Business Development, Nanoform

Introducing Nanoform’s new formulation platform technology: Nanocrystallisation of amorphous nanoparticles for added performance and stability in formulations.  Presenting the benefits of nano particles across many types of formulations.

Technology & Innovation

Rapid and Material-Sparing Feasibility Screening for Hot Melt Extrusion

Amanda Pluntze, Scientist III, Research & Development, Lonza

Amorphous solid dispersions (ASDs) offer a bioavailability enhancement drug delivery option for poorly soluble crystalline APIs. Spray drying and hot melt extrusion (HME) are the two most common methods for manufacturing ASDs. HME is a higher throughput process and more environmentally friendly, yet it often results in lower drug loadings, and the risk of degrading the API is higher. To determine whether HME is appropriate for a given API, it is important to understand the loading that can be achieved without risking degradation. HME screening methods can give misleading results if they do not consider the kinetic aspects of API dissolution and degradation. There is, therefore, a need for an approach more representative of the extrusion process. This presentation will demonstrate a screening process to quickly assess HME feasibility. Compared to traditional approaches, this protocol yields more accurate predictions for both API loading and degradation, while saving material and time.


Key Learning Objectives:
  • Know the key differences between spray drying and HME, and why different feasibility screening approaches are needed
  • Understand the limitations of current small-scale HME screening techniques
  • See how a newly developed HME screening process works for a variety of model systems

Technology & Innovation

Formulation Strategies to Improve the Bioavailability of APIs with BCS Classification II and IV

Dave Seghers, Head of Sales , Ardena

  • The poor solubility and low bioavailability of active pharmaceutical ingredients (APIs) present significant challenges in drug development, especially for compounds falling into BCS class II or IV categories. To address these issues, this presentation explores advanced formulation approaches that aim to enhance the solubility and dissolution rate of poorly water-soluble APIs.
  • The presentation covers several techniques and formulation types, including amorphous solid dispersion via spray drying, nanosuspensions, and lipid formulations.
  • By attending this session, participants will gain a comprehensive understanding of the challenges related to solubility and bioavailability of poorly water-soluble drugs. The presentation provides valuable insights into the latest formulation strategies used to enhance drug efficacy and bioavailability, featuring compelling case studies of successful implementation.
Formulation development is currently playing a critical role in improving compound solubility or the compound dissolution rate and thereby increasing bioavailability of poorly soluble compounds. Amorphous solid dispersion via spray drying, nanosuspensions and lipid formulations are considered the most popular options. In his presentation Dave Seghers, Business Development Director at Ardena will describe how the first technique, the crystalline structure of the API is modified into a less stable but more soluble, amorphous form. In a nanosuspension formulation, the particle size of the API is reduced by wet milling techniques to the nano range, increasing the surface area of the particles and hence, the dissolution rate.

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

Small Molecules

Enabling or Not? A FIH Oral Solid Dosage Form Development Journey for a Poorly Water-Soluble Deuterated Weakly Basic Candidate

Yan  He, Head of Early Formulation Development, Sanofi

  • Bioperformance driven and physico-chemical properties-based formulation development
  • Spring effect in conventional formulation - Evaluation and selection of acidic excipients to provide a micro-environment to offer higher solubility in gastric fluid
  • Parachute effect in conventional formulation - Evaluation and selection of polymers to sustain the dissolved molecules in intestine fluid
  • Prototype formulation development using pentagastrin treated dog exposures to help select the best performer
  • Enabling formulation, Amorphous solid dispersion formulation, development to be a backup option
  • Stability evaluation using ASAPprime® for shelf-life determination and packaging configuration selection


Formulation and Analytical Challenges in Development of Fixed-Dose Combination Products for Biologics

Zhi Chen, Associate Scientific Director, Bristol Myers Squibb

  • Definition and Potential Benefits of Fixed-Dose Combination (FDC) Biologics
  • Formulation Challenges: Stability and Interactions
  • Analytical Challenges: Specifications, Method Development, Specificity
Fixed-Dose Combination (FDC) products combine two or more active ingredients in a single dosage form, offering the convenience and simplification of multiple therapeutic benefits. While these products can include both small molecule drugs and biologics, the focus of this presentation will be on the challenges and strategies involved in the coformulation development of biologics.

FDC biologics present unique advantages, such as reducing the number and volume of injections, reducing infusion time, minimizing dosing errors, and enhancing patient compliance. However, their development poses significant challenges owing to the inherent complexity and sensitivity of biological substances.

Key challenges include maintaining the stability and activity of all components within the same formulation, predicting, and analyzing the potential interactions between different components, and ensuring the efficacy and safety of the product. Further complexities arise when attempting to separate individual components for analysis, especially if they share similar physical or chemical properties. This can complicate the determination of each component's concentration in the product, thereby affecting the assessment of its quality and consistency.

The development of analytical methods for biologics, due to their size, complexity, and diversity, also poses significant hurdles. These methods must reliably measure the identity, purity, potency, and stability of each component in the FDC.

In this presentation, we will delve into these challenges, exploring potential solutions and strategies for the successful development of FDC biologics.

Technology & Innovation

Simplifying Design: Empowering Future Drug Delivery Innovations

Nitin Joshi, Assistant Professor , Harvard Medical School

Our laboratory's mission is to pioneer translatable drug delivery technologies aimed at addressing unmet clinical needs spanning various diseases, such as arthritis, HIV, respiratory infections, lung inflammation, and brain-related disorders. A consistent thread weaving through our diverse technological endeavors is the concept of 'Design Simplicity'. This guiding principle has propelled us toward swift progress in multiple technology development pathways, all with a clear trajectory toward clinical translation.

During this presentation, I will spotlight select instances from our research, showcasing how we've harnessed the power of design simplicity to tackle pressing medical challenges and expedite the creation of solutions primed for rapid clinical translation.

10:50 AM - 11:40 AM

Networking & 1-2-1 Meetings

11:40 AM - 12:10 PM - Case Studies

Small Molecules

Patient Centric Drug Development

Twinkle Christian, Principal Scientist, Amgen

Patient-centric drug development describes the systematic approach to incorporating the patient’s perspectives and preferences into the design, assessment, and production of a therapeutic product. While a patient centric approach can be applied at any stage of the drug development lifecycle, an integrated end-to-end strategy is often most effective to create an optimized product for the patient at the earliest possible timepoint. The importance of patient centricity is well recognized by health authorities and biopharmaceutical organizations which have established toolsets, guidances, and methodologies for incorporating patient input during the clinical stage of development. However, in addition to clinical research, there are other significant aspects of product development that profoundly impact the patient experience. Specifically, chemistry, manufacturing, and control (CMC) and device aspects must also be acknowledged and addressed as part of a cohesive patient-centric development strategy. This review explores current applications and regulatory considerations for patient-centric approaches across the product lifecycle, including R&D, early product development, clinical development, device and combination product development, and post-approval change management. Specific topics of discussion include the contributions of product modality, formulation, and devices to the patient experience; usage of the Quality Target Product Profile (QTPP) as a patient-centered design tool; and post-approval product optimization. Future advancements in regulatory data management and information exchange are also explored as potential enablers of patient engagement which support enhanced communication and interconnectivity between stakeholders. Multidisciplinary collaboration between patients, health authorities, health care providers, and the biopharmaceutical industry is ultimately necessary for ensuring that medicinal products, and their corresponding
regulatory processes, take on a patient-first mindset that prioritizes patient needs, values, and preferences.

Small Molecules

Insights on Developing Amorphous Solid Dispersion Formulations for Early Clinical Studies

Wei Zhang, Principal Scientist, Small Molecule Pharmaceutics , Genentech

Amorphous formulation becomes an attractive strategy to deliver poorly water soluble drugs owing to its higher solubility. This presentation will cover three major topics for amorphous drug product development to support clinical studies – (1) theoretical solubility calculation for amorphous drugs; (2) effect of ASD composition on the disintegration and drug release of ASD tablets; (3) a case study of developing an ASD formulation to improve bioavailability and mitigate mechanical instability risk of crystalline form for early phase clinical studies. The first topic will discuss different methods of calculating amorphous drug solubility and the experimental evaluation of these equations. Based on the evaluation, one calculation equation was recommended based on the overall accuracy and complexity. The validated amorphous solubility calculation provides a guidance for ASD composition screening and developing ASD formulation. To successfully apply ASD formulation in clinics, final dosage form (e.g. ASD tablets) is usually needed. Owing to great amount of polymers in the ASD composition, the disintegration and drug release usually become an issue for ASD tablets especially when ASD loading increases. The second topic will discuss how polymer type, ASD loading in tablet and polymer-drug ratio affect the disintegration and drug release of ASD tablets. At last, the third topic will present a case study of developing an ASD tablet, guided by amorphous solubility calculation, to improve the drug bioavailability and mitigate mechanical instability risk of the selected crystalline form. To summarize, the overall goal of this presentation is to provide an industrial perspective for the design and formulation of amorphous drug products based on fundamental understanding of amorphous materials.

Technology & Innovation

Development and Clinical Translation of Neuroinflammation-Targeted Systemic Dendrimer Nanomedicines

Kannan Rangaramanujam, Arnall Patz Distiniguished Professor of Ophthalmology/Co-director, Center for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine

  • Inflammation plays a central role in the pathogenesis of many disorders – selective targeting of inflammatory cells may provide a powerful tool to improve therapeutic window in humans
  • Hydroxyl dendrimers enable systemic targeting of reactive inflammatory cells in the brain, eye and tumors, without a need for ligand or antibodies.
  • Cell-targeted dendrimer therapies produce significant efficacies in multiple preclinical models of neuroinflammatory and ocular disorders.
  • Early Phase 1 and Phase 2 trials of dendrimer-drug conjugates showed that lead candidates are safe in humans and produce significant benefits in patients with severe COVID-19, with Phase 2 trials for systemic wet AMD treatments ongoing.

12:15 PM - 12:45 PM - Solution Spotlights

Small Molecules

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

Sheng Hu, Director, CMC Project Management, WuXi STA

Technology & Innovation

Controlled and Sustained Drug Delivery - How Epidel™ is Changing Long-Acting Drug Development

Márcio Temtem, Senior Director, Strategic Business Management Products, Hovione

Ian Parrag, Vice President of R&D, Ripple Therapeutics

Polymeric drug carriers are widely used for providing temporal and/or spatial control of drug delivery, with corticosteroids being one class of drugs that have benefitted from their use for the treatment of inflammatory-mediated conditions. However, these polymer-based systems often have limited drug-loading capacity, suboptimal release kinetics, and/or promote adverse inflammatory responses. In this presentation we’ll present EpidelTM a controlled release platform. This platform is based on a discovery that low molecular weight corticosteroid dimers can be processed into drug delivery materials using a broad range of established fabrication methods, without the use of polymers or excipients. These implants undergo surface erosion, achieving tightly controlled and reproducible drug release kinetics in vitro. Successful examples will show how Epidel™ is changing long-acting drug development.

Don’t miss the chance to speak with our experts – Table #13 – and learn how a simplified pro-drug synthetic approach is being used to fine-tune the delivery of drugs with higher drug loads, precise dosing, and prolonged therapeutic effect. Schedule your meeting -

Device Development

Facilitating Subcutaneous Self-Administration of Biopharmaceuticals for Enhanced Patient Experience

Dr. Reza Abedian, Senior Medical Affairs Manager, Gerresheimer

The pharmaceutical industry is showing a significant growth trend in the clinical development of biologicals, most specifically monoclonal antibodies, with seven of the top ten selling drugs globally being biopharmaceuticals. High costs and resources required for intravenous administration for these medications is  pointing to subcutaneous self-administration as a suitable alternative (Jones, G.B. et al. Science translational medicine (2017)).

The majority of mAbs are sensitive, highly viscose and often need to be administrated in high volume. Devices for subcutaneous self-administration therefore need to be developed that respond to these features of biopharmaceuticals and, at the same time, facilitate patient home usage.

The FDA recognizes that the voices and perspectives of patients are critical to understanding the impact of medical devices (Patient Preference Information (PPI) in Medical Device Decision-Making. (2020, 10 27)). Hence, it is crucial to implement a user-centric approach in the development of drug delivery devices.

This presentation will outline how early-stage user insight studies, together with human factor evaluations, ensure that voices of patients are implemented in the development of medical devices for subcutaneous self-administration of biopharmaceuticals. Moreover, there will be an overview of how delivery devices designed by Gerresheimer address unmet needs of patients and healthcare professionals in order to further improve patient experience. Finally, the challenges and solutions associated with  home administration of biologic drugs will be discussed including, but not limited to, cold chain requirements and sustainability considerations.


Keywords: patient-centred care; sustainability; subcutaneous self-administration; Patient Preference Information (PPI)

12:45 PM - 1:45 PM

Networking Lunch

1:45 PM - 2:15 PM - Case Studies

Technology & Innovation

Formation and Inhibition of Nitrosamines in Drug Product

Kausik Nanda, Principal Scientist , Merck

Nitrosamines, in the absence of toxicological data, are regarded as potential mutagens, and ICH M7 considers N-nitrosamines as part of the highly mutagenic cohort of concern. Recently, several drug withdrawals have been resulted from the contamination of drugs with nitrosamines. To ensure patient safety and access to life saving drugs, it is important to understand the phenomenon of the formation of nitrosamines in drug products and how to inhibit the formation of nitrosamines in drug products. We will present data, primarily, on the formation and inhibition of formation of nitrosamines in solid oral dosage form.


Strategy for Intestinal Delivery of a GIP/GLP1 Co-Agonist Peptide

Huyen Tran, Ph.D., Director in Formulation and Delivery, Eli Lilly and Company

The presentation addresses 2 key questions:
  • What are effects of C10 on intestinal absorption of peptides with different physicochemical properties?
  • What is interaction of C10 with the intestinal barriers and mechanism of action (MoA) of C10 for improving intestinal absorption of a Lilly’s peptide in vivo?
In this study, we evaluated effects of sodium caprate (C10) on intestinal absorption in rats with a GIP-GLP1 dual agonist peptide (LY) and semaglutide with different physicochemical properties as well as the oral exposure of the LY peptide in minipigs. Furthermore, we investigated the mechanism of action of C10 for improving intestinal absorption of the LY peptide in vivo via live imaging of the rat intestinal epithelium and tissue distribution of the LY peptide in minipigs. The LY peptide with higher proteolytic stability in pancreatin, monomer in solution, and higher in vitro permeability in the minipig intestinal organoid monolayer showed higher intestinal absorption in rats compared to semaglutide and achieved ~2% relative bioavailability upon ID administration in minipigs. Using confocal live imaging, we observed that C10 enabled rapid oral absorption of FD4 in the rat intestine. In the duodenum tissues of minipigs, C10 facilitated the GIP-GLP1 peptide absorption into circulation via both paracellular and transcellular pathways by reducing tight junction protein level allowing peptide uptake to the intestinal cells. These data indicated that the LY’s favorable physicochemical properties combined with the effects of C10 enabled intestinal delivery of the peptide.

Technology & Innovation

3D Tablet Design Software as Patient Centric Approach to Promote Swallowability of Solid Oral Dosage Forms

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

  • Real-time 3D engineering platform that allows personalized design for solid oral dosage form (SODF).
  • Intelligence-guided design process, including live scores for tablet manufacturability and swallowability index.
  • Simultaneous cross-functional & centric collaboration between stakeholders for faster decision-making process.
  • Supply chain workflow steered to expedite SODF development and accelerate market access to patients worldwide.

2:20 PM - 2:50 PM - Solution Spotlights

Small Molecules

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

Nick DiFranco, Global Market Manager, Oral Drug Delivery , 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 


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

Sudhakar Voruganti, Director, Business Development, Pfanstiehl

Applications, related Functionalities and Performance in Biologic Drug Product Formulations and m-RNA Technologies

Learning objectives:
  • Learn about Formulation development and challenges
  • Novel excipients
  • High concentration formulations

Technology & Innovation

Lipid Manufacturing via CTDMO Services – A Process Chemistry Perspective

Christian Kuzniewski, Senior Manager Process Development, MilliporeSigma

  • Insight into our Lipid manufacturing capabilities as part of our fully integrated CTDMO offering for mRNA therapeutics
  • Overcome process challenges in lipid manufacturing and analytics
  • Case studies: how to develop and optimize lipid processes for a smooth transition from pre-clinical development to cGMP manufacturing
  • Experience sharing: Key considerations for lipid excipients used in LNPs
Lipid molecules are crucial in drug delivery, and the strong process development branch for lipids at MilliporeSigma serves two important purposes: conserving and extending lipid synthetic expertise to provide efficient access to various types of high-quality lipids. As part of our globally integrated mRNA-LNP CTDMO capabilities, our Lipid R&D Center of Excellence in Schaffhausen is one of the leading suppliers for both customized and portfolio lipids, supporting our CTDMO clients' various drug delivery programs from pre-clinical to commercial scale.

Challenges in process development frequently led to greatly optimized lipid syntheses and better scalability of newly developed lipid structures. During our talk, we will highlight some interesting examples of process development in action.

2:50 PM - 3:30 PM

Networking & 1-2-1 Meetings

3:30 PM - 4:00 PM - Case Studies

Small Molecules

Small Molecule Crystallographic and Modeling Strategies to Improve Solubility

Tina Xiong, Associate Principal Scientist, Merck

  • Molecular design of high solubility compounds that balances potency and selectivity imposes a challenge in drug discovery.
  • Here we present a case study to explore the process of applying single crystal X-ray diffraction and identification of key intramolecular and intermolecular interactions to drug design.
  • This work aims to build upon well-established Discovery methods for enriching for solubility and integrate a pharmaceutical strategy that leverages crystallographic determination to enable rational design of soluble preclinical candidates.
Solubility remains a foremost challenge in small molecule drug design and development. Molecular design of high solubility compounds that balances potency and selectivity imposes a challenge in drug discovery. Recent publications provide insight into insolubility in part driven by the interactions involved in stable crystal packing of solid API. Methods for elucidating key interactions in small molecule crystal formation is thus important for the determination of the origin of insolubility and potency, where disruption of crystal packing could be an alternative method for improving aqueous solubility. Crystallographic approaches, however, are often time-intensive, making implementation in drug discovery a challenge. Here we present a case study from Merck aimed to explore the process of applying single crystal X-ray diffraction and identification of key intramolecular and intermolecular interactions to drug design. Crystal structure of the protein-ligand complex was used to determine key interactions driving potency. Solubility and melting point analysis were carried out to understand the origin of poor solubility and molecular match pair (MMP) analysis was used to guide understanding of chemical modifications that might broadly serve as methods for disrupting otherwise strong crystal packing. Further, crystal packing energy and hydration energy were evaluated using a simplified crystal structure prediction workflow. Altogether this work suggests that insight into molecular interactions might guide chemical modifications that favor high solubility and enable rapid generation of a lead molecule.


Current Challenges During CMC Development and Delivery of Novel Modalities

Cesar Calero, Senior Scientist Biologics Drug Product Development & Manufacturing, Sanofi

The expansion of biopharmaceuticals into so called “novel modalities” have encountered new challenges and opportunities during CMC development and clinical administration/drug delivery. This presentation will cover some of the pain points for novel modalities and highly potent (low dose) biotherapeutics, considerations for Drug Product design, formulation, manufacturing, and compatibility in-use studies, and case studies for early clinical studies

Technology & Innovation

Release Mechanisms of Amorphous Solid Dispersions: Role of Drug-Polymer Phase Separation and Morphology

Hitesh S. Purohit, Principal Research Scientist, AbbVie

  • Formulating poorly soluble molecules as amorphous solid dispersions (ASDs) is an effective strategy to improve drug release. However, drug release rate and extent tend to rapidly diminish with increasing drug loading (DL).
  • In this work, release profiles of ASDs formulated with ritonavir (RTV) and polyvinylpyrrolidone/vinyl acetate (PVPVA) at different DLs were determined using surface normalized dissolution.
  • Surface morphologies of partially dissolved ASD compacts were evaluated with confocal fluorescence microscopy.
  • ASD phase behavior during hydration and release of components were also visualized in real time using a newly developed in situ confocal fluorescence microscopy method.
  • The domain size and interconnectivity of phase separated drug-rich domains appear to be critical factors impacting drug release from RTV-PVPVPA ASDs.
Formulating poorly soluble molecules as amorphous solid dispersions (ASDs) is an effective strategy to improve drug release. However, drug release rate and extent tend to rapidly diminish with increasing drug loading (DL). The poor release at high DLs has been postulated to be linked to the process of amorphous-amorphous phase separation (AAPS), although the exact connection between phase separation and release properties remains somewhat unclear. Herein, release profiles of ASDs formulated with ritonavir (RTV) and polyvinylpyrrolidone/vinyl acetate (PVPVA) at different DLs were determined using surface normalized dissolution. Surface morphologies of partially dissolved ASD compacts were evaluated with confocal fluorescence microscopy, using Nile red and Alexa Fluor 488 as fluorescence markers to track the hydrophobic and hydrophilic phases respectively. ASD phase behavior during hydration and release of components were also visualized in real time using a newly developed in situ confocal fluorescence microscopy method. RTV-PVPVA ASDs showed complete and rapid drug release below 30% DL, partial drug release at 30% DL and no drug release above 30% DL. It was observed that formation of discrete drug-rich droplets at lower DLs led to rapid and congruent release of both drug and polymer, whereas formation of continuous drug-rich phase at the ASD matrix-solution interface was the cause of poor release above certain DLs. Thus, the domain size and interconnectivity of phase separated drug-rich domains appear to be critical factors impacting drug release from RTV-PVPVPA ASDs.

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

Technology & Innovation

Global Alliance Management and Partnership Success

Rosario LoBrutto, Head of Pharmaceutical Sciences and Global Expansion, Radius Health

Business alliances are critical within the biotech/pharma industry.  Companies joining forces via partnerships offer complementary strengths to their partners such as research acumen, development expertise, regulatory expertise,  manufacturing facilities, distribution channels, commercial footprint, or marketing prowess. Without proper nurturing of an alliance, collaborations can be fraught with difficulties such as poor coordination, insufficient communication, and culture clashes that can undermine an otherwise promising partnership.

The culture of enhancing alliance value and successful partnerships can be built with a focus on social physics, partnering mindset, and partnering skill sets. Emotional intelligence (EQ) lies at the heart of this “partnering pyramid” which cultivates high-performance alliance management relationships. In summary, implementing elements of the “partnering pyramid” can enhance trust and collaboration, and design alliances that are more innovative, efficient, and productive while successfully managing and negotiating alliance conflict. Opportunities for rebooting “alliance value” as an alliance architect and builder will be presented.